27th Annual EuroMeeting

13-15 April 2015 Palais des Congrès

Paris

Final Programme

27th Annual EuroMeetingDevelopment, Innovation, Access and Patient Safety

DIA EuroMeeting 2015Development, Innovation, Access and Patient Safety

13-15 April, Palais des Congrès, Paris

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TABLE OF CONTENTS

Welcome Form DIA Chair, DIA Board of Directors and DIA Global Chief Executive .........................................................................2

Welcome from the EuroMeeting 2015 Co-Chairs & Programme Advisors ................................................................................3

EuroMeeting 2015 Theme Leaders ...................................................................4

Schedule at a glance .............................................................................................5

General Information A-Z ..................................................................................6-7

Award Winners .......................................................................................................9

Professional Posters ............................................................................................ 10

Student Posters / Patient Advocates ............................................................. 11

EuroMeeting Pre-Conference Optional Tutorials ..................................16-18

Clinical Forum Pre-Conference Optional Tutorials .................................... 19

HAS Satellite Symposium ................................................................................. 20

50th Anniversary of the EU Parmaceutical Legislation .......................... 20

50 Years of EDQM .............................................................................................. 20

Opening Plenary .................................................................................................. 21

Theme 1 | New R&D models and innovative clinical trial methodologies. ..................................................................................... 22-23

Theme 2 | Medical Devices and Combination Products .................... 24-25

Theme 3 | Advanced Therapies, Novel Treatments for Rare Diseases .................................................................................. 25-27

Theme 4 | Early Dialogue with Regulators and HTA Bodies on Innovative Medicines .............................................. 27-28

Theme 5 | Collaborative Consortia, IMI and the Way to Horizon 2020 ...................................................................29-30

Theme 6 | Implementing Innovation – Globally Conducive Regulatory Policies .......................................................... 31-32

Theme 7 | Special Development Pathways in Paediatrics, the Elderly and in Pregnancy ............................................................33-34

Theme 8 | Translational Medicine and Regulatory Science .............. 35-37

Theme 9 | Big Data, Mobile Health .......................................................... 37-39

Theme 10 | Innovation in Vaccine Development ..................................39-41

Theme 11 | Availability of Medicinal Products: Drug shortages ........41-42

Theme 12 | Pharmacovigilance in 2015 - Poised for convergence of innovation .................................................................43-45

Hot Topics and Stand-Alone Sessions .......................................................... 46

Focus Sessions .................................................................................................... 47

Speaker Index ................................................................................................48-53

Networking Events ............................................................................................ 54

EuroMeeting Session Plan ..........................................................................56-58

Dear Colleagues, It is our pleasure to welcome you to Paris, the hub of European biotech IPOs (international public offerings), and a city that boasts not only centuries of science, but also the highest density of publically traded biotech companies in Europe. Paris is a city that actively engages in translating research into tangible economic realities with a robust regulatory framework. Thank you for joining us at DIA’s 27th Annual EuroMeeting. As we come together to share our collective knowledge to address significant challenges in human health, we will reflect on our collective accomplishments in Advancing Innovation to Combat the Global Burden of Disease, the theme of the Opening Plenary. Our two keynote speakers, Olivier Charmeil, Executive Vice President, Sanofi Vaccines, and Andrew Morris, Professor, University of Edinburgh, and of the Farr Institute, will address this subject in view of the need for pharmaceutical innovation. Their focus will be centered on making innovation more efficient and relevant through the use of big data. This EuroMeeting brings together innovators and leaders from all parts of the health care ecosystem. Our scientific offerings are organised into themes and led by experts who will cover a wide range of interest areas, such as access to innovative treatments, advanced therapies, special development pathways, and pharmacovigilance, as well as glocal outlook, collaborative consortia, and dialogue between regulators and HTA bodies to advance translational medicine. Also this DIA 27th EuroMeeting will celebrate the 50th anniversary of the European Pharmaceutical Legislation as well as the 50th anniversary of the EDQM (European Directorate for the Quality of Medicines and Health Care). We will highlight the achievements of these landmark organisations and provide a perspective into what can be expected in the future for health care product development and regulation. Together with DIA’s recent 50th anniversary milestone, this EuroMeeting marks an important milestone in our efforts to foster innovation by providing forums for industry, regulators, patients, and other stakeholders to exchange information and discuss health products, technologies, services, and related issues in order to advance our collective mission of developing and delivering effective therapies to patients worldwide. On behalf of the DIA Board of Directors and all of our EuroMeeting programme volunteers, exhibitors, poster presenters, and staff, thank you very much for attending our celebratory DIA 2015 27th EuroMeeting. Enjoy the programme!

Per SpindlerChair, DIA Board of Directors

Barbara Lopez KunzGlobal Chief Executive

DIA EuroMeeting 2015Development, Innovation, Access and Patient Safety13-15 April, Palais des Congrès, Paris

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WELCOME FROM THE EUROMEETING 2015 CO-CHAIRS

Dear Colleagues,

We are delighted that the 27th Annual DIA EuroMeeting is held in Paris this year.

Our meeting comes at an exciting and challenging time for healthcare innovation. The demand for new therapies and new solutions has never been higher, and the pace of innovation is increasing to meet these needs. This makes Paris an ideal choice for the venue. The city has been at the forefront of scientific developments since the foundation of the “Académie des Sciences” in 1666. Since then, Paris-based scientists have brought us stethoscopes and hypodermic needles, antibiotics and antipsychotics, vaccines against TB and rabies and have successfully identified HIV. Now, in 2014, Paris remains pivotal in healthcare research, as the hub for Biotech IPOs in Europe.

If European companies are to remain competitive, they need a regulatory environment that supports their investment. The current rate of innovation, with new approaches and innovative therapies are testing the limits of the existing system, presents its own challenges for regulators and policymakers. Addressing these will require a collaborative approach between all stakeholders to create a streamlined, harmonised approach to regulation. A future regulatory system should be capable of anticipating potential hurdles and barriers, allowing the fruits of research to reach those people who need them more quickly. This will offer an impetus to companies to continue to invest in research. In addition, it will add momentum to the kind of public-private partnership – such as the Innovative Medicines Initiative – capable of accelerating effective drug development.

Adaptive pathways approvals, also named Adaptive Licensing or Medicine Adaptive Pathways to Patients, as prospectively planned adaptive approach the regulation of drugs, is without any doubt one of the relevant method to accelerate access to innovative medicines. While it is particularly relevant for medicines to treat serious conditions with unmet medical needs, it should also help developing innovative regulatory strategies for all new drugs and vaccines.

The Annual DIA EuroMeeting provides the ideal opportunity to build the type of collaboration that will underpin future innovation in Europe. An intense 3-day programme will present high quality information and discussion, along with opportunities for face-to-face meetings and professional networking between all the main stakeholders. Participants can exchange knowledge and build the kind of partnerships that will support innovation in drug development and address today’s health challenges.

The DIA EuroMeeting 2015 should be the "go to" meeting for all professionals in drug discovery, drug development and regulation. We will be able to add a little more to the reputation of Paris as a city of health innovation. We look forward to seeing you here.

Lidia Retkowska-Mika and Olivier Charmeil

PROGRAMME ADVISORS

Martin Harvey AllchurchHead of Communication, European Medicines Agency, EU

Jan GeisslerDirector, EUPATI, Belgium

Angelika JoosExecutive Director, Global Regulatory Policy, MSD (Europe) Inc., Belgium

Yves JuilletSenior Vice-President, Industrie Santé, France

Spiros VamvakasEuropean Medicines Agency, EU

Christa Wirthumer-HocheHead, Austrian Medicines and Medical Devices Agency (AGES), Austria

Lidia Retkowska-MikaDirector, Legal Department, Office for Registration of Medicinal Products, Medical Devices and Biocidal Products, Poland

Olivier CharmeilExecutive Vice President Vaccines, Sanofi, France

PROGRAMME CO-CHAIRS



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EUROMEETING 2015 THEME LEADERS

Sabine AtzorHead of EU Regulatory Policies,

F. Hoffmann-La Roche,Switzerland

Salah-Dine ChiboutGlobal Head Discovery &

Investigative Safety, Global Head Preclinical Safety Therapeutic Areas,

Novartis Pharma, Novartis

Anna CieślikDirector Department of

Documentation Assessment, Office for Registration of Medicinal

Products, Poland

Isabelle ClamouRegulatory Affairs Director – EU Policy, Amgen, Belgium

Susanna Del SignoreAssociate Vice-President Global

Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D,

France

Belén Escribano RomeroHead of Department,

Pharmaceutical Inspection and Enforcement Department, Spanish Medicines Agency (AEMPS), Spain

Zaide FriasHead of Regulatory Affairs and Best Evidence, Head of Human Medicines

Research and Development Support (ad interim),

European Medicines Agency, EU

Sabina Hoekstra-van den BoschGlobal Regulations and Standards, Philips Healthcare, the Netherlands

Paul JansenGlobal Head Medical Devices US,

Sanofi, USA

Jun KitaharaInternational Liaison Officer

stationed in Swissmedic, Pharmaceuticals and Medical

Devices Agency (PMDA), Japan

Maarten LagendijkPharmacovigilance Coordinator,

Medicines Evaluation Board (MEB), the Netherlands

Jordi Llinares GarciaHead of Product Development Scientific Support, European

Medicines Agency, EU

Henrik Kim NielsenCorporate Vice President,

Regulatory Affairs GLP-1 and Obesity, Novo Nordisk, Denmark

Detlef NieseConsultant, Dr. Niese Health Science

and Policy, Germany

Luca PaniDirector General, Italian Medicines

Agency (AIFA), Italy

Richard PilsudskiVice President Global Regulatory

Affairs, Sanofi Pasteur, France

Dorthe PoulsenChief Legal Adviser, EU-Coordinator,

Danish Health and Medicines Authority (DKMA), Denmark

Fatiha SadallahPrincipal Scientific Manager,

Innovative Medicines Initiative (IMI), Belgium

Paula SalmikangasChair CAT, Senior Researcher,

Finnish Medicines Agency (FIMEA), Finland

Duane SchulthessManaging Director, Vital Tranformation, Belgium

Henk SchuringGroup Vice President Regulatory

Affairs - Europe, Genzyme Europe, the Netherlands

Pär TellnerEFPIA ICH Coordinator, Director

Regulatory Affairs, EFPIA, Belgium

Jan Willem van der LaanDepartment of Pharmacology, Toxicology and Biotechnology

(FTBB), Medicines Evaluation Board, the Netherlands

Maria Grazia ZurloQPPV,

Pfizer, Italy


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SCHEDULE AT-A-GLANCE Sunday, 12 April 2015

Registration Hours:14:00-19:30 Exhibitor Registration and Set-up

14:00-18:00 Attendee and Speaker Registration*

17:30-18:30 Students and Young Professionals Welcome

Reception

* Avoid the rush on Monday by picking up your badge and conference

material on Sunday afternoon

Monday, 13 April 2015

Registration Hours:07:30-11:00 Exhibitor Registration and Set-up

08:00-18:00 Attendee, Speaker and Exhibitor Registration

Schedule:09:00-12:30 Pre-Conference Tutorials*

10:30-11:00 Pre-Conference Tutorials Coffee Break

11:00-12:30 HAS Satellite Symposium

12:00-18:00 Conference and Exhibition Open

12:00-14:00 Lunch & Innovation Theatre Presentation in the

Exhibition Hall

13:30-15:00 Regulatory Town Hall Meeting

14:00-17:30 Optional Clinical Forum Tutorials*

15:00-16:00 Extended Refreshment Break in the Exhibition Hall

16:00-17:45 Opening Plenary Session

18:00-20:30 “Bienvenue à Paris” Reception

*Space is limited for Pre-Conference Tutorials, therefore pre-registration is strongly recommended. Availability for onsite registration is not guaranteed

Tuesday, 14 April 2015

Registration Hours:08:00-18:30 Attendee, Speaker and Exhibitor Registration

Schedule:08:00-09:00 Welcome Coffee in the Exhibition Hall

08:00-18:30 Exhibition Hall Open

09:00-10:30 Parallel Scientific Sessions - Session 1

Choose from Parallel Sessions

10:15-11:00 Coffee Break in the Exhibition Hall




Exhibition Hall

12:30-13:00 Speed Networking

13:00-14:00 DIA Communities - Meet and Eat



14:00-15:30 Exhibition Guest Passes




17:30-18:30 Networking Reception in the Exhibition Hall

17:45-18:15 Student Poster Award Ceremony at the DIA Booth

Wednesday, 15 April 2015

Registration Hours:08:00-17:30 Attendee, Speaker and Exhibitor Registration

Schedule:08:00-09:00 Welcome Coffee

08:00-16:00 Exhibition Hall Open






11:00-12:30 50 Years of EDQM Session


Exhibition Hall






17:30 End of Conference



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The 27th Annual EuroMeeting takes place at:Le Palais des Congrès de Paris2, Place de la Porte Maillot75017 Paris – France

Registration will be located on Level 3. The conference and exhibition will take place on levels 2 and 3.

For the first time ever, the EuroMeeting is organised in parallel to another DIA flagship meeting in Europe, the 8th Annual Clinical Forum. This will afford delegates unparalleled opportunities to interact and network with qualified professionals from throughout Europe and the world, and to meet a wealth of exhibiting companies, all under one roof.

AppDownload the Free DIA Global App Today.

The DIA Global App is designed to enhance your meeting experience and provide valuable information in one place. Create your session agenda, network with attendees and exhibitors, and connect to DIA resources, social media channels, member communities, and more.

To download, search for “DIA Global” in your app store.Access the EuroMeeting 2015 App:• Sign in with the email address you registered for the EuroMeeting 2015• Click on the Events Icon• Select 27th Annual DIA EuroMeeting 2015

You can find assistance at the DIA Booth #3.D17 on Level 3

Banking/ATMCash dispensers are available on levels -1 and 0.

Business CentrePlease note that there is no business centre at the Palais des Congrès de Paris. There is a copy/printing shop within 5 minutes walking distance from the Palais des Congrès, located at 62, Avenue de la grande Armée. www.copytop.com/agence-grande-armee-maillot

Certificate of AttendanceA Certificate of Attendance can be printed at the Scan-and-Go desks in Hall Bordeaux on Level 3 on Wednesday, 15 April 2015 from 10:30.

Cloakroom/BaggageThe cloakroom is in the Entrance Hall in Hall Bordeaux on Level 3. There is a charge of € 2 per coat/jacket and € 3 per piece of luggage.

Monday 08:00 - 21:00Tuesday 08:00 - 19:00Wednesday 08:00 - 18:00

Conference BagsAll attendees with a full meeting registration can collect a conference bag from the Conference Bag Distribution Point in Hall Bordeaux on Level 3. Please bring the bag voucher you received when collecting your badge.

Credits DIA meetings are accredited by the SwAPP (Swiss Association of Pharmaceutical Professionals) Commission for Professional Development (CPD) and SGPM (Swiss Society of Pharmaceutical Medicine).

The 27th Annual EuroMeeting has been awarded 13.75 CPD credits from the Faculty of Pharmaceutical Medicine (FPM) of the Royal College of Physicians (RCP) of the UK. Medical practitioners who are eligible for credits can click on http://www.fpm.org.uk/cpd/registration for more information. If you are already a CPD member, please go directly to http://cpd.fpm.org.uk to claim your credits.

DIA BoothFind out more about all DIA can offer you, how membership can advance your career, how to join a DIA Community, submit an article for publication and lots more, at the DIA Booth # 3.D17. Stop by at the DIA Booth in the Havane Exhibition Hall on Level 3. See “Exhibition” for opening hours.

DIA Patient BoothDIA actively promotes the involvement of patient representatives in the EuroMeeting. Since 2006, more than 200 patient representatives have been involved as participants, speakers, session chairs, and also in the Programme Committee. The Patient Fellowship Booth #3.D06 is located on Level 3 and acts as a focal point for patient fellows and other stakeholders to meet and network.

ExhibitionVisit the joint EuroMeeting and Clinical Forum Exhibition, with 140+ companies and service providers in a single venue. With many new companies exhibiting this year, the exhibition offers more opportunities than ever to connect with participants.

Monday 12:00 - 18:00Tuesday 08:00 - 18:30 | Public Access 14:00 - 15:30Wednesday 08:00 - 16:00

Please see the exhibition floor plan and list of exhibiting companies in the Exhibition Guide, or use the interactive floor plan in the “DIA Global" mobile app.

Exhibitor ServicesThe Exhibitor Services Desks are located in Hall Maillot on Level 2. See Exhibition for opening hours.

First Aid A pharmacy located in the shopping center on Level 0 is open daily from 09:00 to 20:00. For medical assistance during the conference hours, please visit the DIA Onsite Registration Desk. Alternatively, any DIA host/hostess will be more than happy to be of assistance.

Hotel Accommodation InformationIf you have any queries about hotel accommodation, please visit the Hotel Information counter at the DIA Registration Desk located in Hall Bordeaux on Level 3.

Information DesksIf you have any questions about the Clinical Forum or the EuroMeeting, from finding session rooms to networking activities, stop by the DIA Information Desks located on Level 2 and Level 3. Alternatively, any staff member or DIA host/hostess will be more than happy to be of assistance.


GENERAL INFORMATION A-Z


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Lost and FoundAll items will be stored at the DIA Registration Desk in Hall Bordeaux on Level 3 until the end of the conference.

Members LoungeV.I.P. Feeling for DIA MembersDIA Members can relax in the Members-only Lounge in the Exhibition Hall on level 3 during breaks, or whenever a few minutes in a quiet setting is needed. Coffee and drinks are served throughout exhibition opening hours and charging stations for your electronic devices are available. Make sure you bring your membership card to identify yourself as a DIA Member at the desk. Access exclusively for DIA Members.

Messaging ServicesDownload the “DIA Global” mobile app and use the messaging function to set appointments or send messages to other attendees.

PostersStudent, professional and patient representative posters will be displayed in the Bordeaux/Havane Hallway on Level 3. Come and talk to our student poster presenters during lunchtime on Tuesday between 13:00 and 14:00. A selected group of professional poster presenters will share their research results on various topics.

Join us at the DIA Booth #3.D17 on Level 3 on the Exhibition Floor for the Student Poster Award Ceremony on Tuesday, 14 April 2015 at 17:45.

Name BadgeName badges must be worn at all times in the Conference Center. Participants will incur a € 25 fee for badge reprints. If you have misplaced your badge, you will be required to have a badge reprinted. Please visit Attendee Onsite Registration located in Hall Bordeaux on Level 3. Identification will be required. Please note, allowing exhibitors to scan the barcode on the front of your badge will provide them with your contact information. No children under the age of 18 years will be allowed in the Exhibition Halls due to liability issues.

PresentationsPresentations will be available to full conference attendees on the DIA web site from 9 April until 15 Ocotber 2015. Presentations are made available to full conference attendees only. To access presentations, visit diahome.org/EM2015 and follow the links for the EuroMeeting presentations.

Press LoungeThe Press Lounge is located in Room 204 on Level 3.DIA welcomes qualified representatives of news organisations for the purpose of reporting and publishing and broadcasting articles and stories. All media must present a copy of their press credentials upon arrival at the DIA Registration Desk. For more information, please contact Jacqueline Bowman on [email protected]


Refreshments/LunchesRefreshments and Lunches will be served each day on the Exhibition Floors on Levels 2 and 3. Enjoy extended refreshment and lunch hours to visit more than 140 exhibiting companies.

Monday12:00 - 14:00 Lunch15:00 - 16:00 Afternoon tea/coffee with snack

Tuesday10:15 - 11:00 Morning tea/coffee with snack12:00 - 14:30 Lunch15:15 - 16:00 Afternoon tea/coffee with snack

Wednesday10:15 - 11:00 Morning tea/coffee with snack12:00 - 14:00 Lunch15:15 - 16:00 Afternoon tea/coffee with snack

Water dispensers are located around the Exhibition Hall and session room hallways.

RegistrationRegistration is located in Hall Bordeaux on Level 3 and will be open as follows:

Sunday 14:00 - 18:00Monday 08:00 - 18:00Tuesday 08:00 - 18:00Wednesday 08:00 - 17:30

SecurityWe take the safety of our participants very seriously. Please help us by cooperating fully with the security personnel on duty and wear your badge at all times. Only participants with a valid conference badge will be allowed into the conference center.

Services for the DisabledAll the rooms at the congress centre are fully accessible to participants with disabilities.

Shopping centreThe Palais des Congrès de Paris has a shopping center with 70 shops: Palais Boutiques (www.lesboutiquesdupalais.com).

Speaker Resource CenterAll speakers are required to visit the Speaker Resource Center located in room 341 on Level 3 and re-check their slides at least 2 hours before the start of their session(s).

Sunday 14:00 - 18:00Monday 08:00 - 18:00Tuesday 08:00 - 18:00Wednesday 08:00 - 16:00

Student CornerA dedicated area is located in Booth #3.A07 in the Exhibition Hall on Level 3 for students to network, plan their day, and meet for lunch.

TwitterTweet about the EuroMeeting using #Euro and @DIA_Europe

WiFi Wireless internet access is available throughout the conference center and is powered by Oracle Health Sciences.

Network: DIA 2015 Password: PARIS2015

GENERAL INFORMATION A-Z



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Thanks to our Media Partners!

International Pharmaceutical Industry Supporting the industry through communication

JOURNAL FOR

Your Resource for Multisite Studies & Emerging MarketsCLINICAL STUDIESU

from diagnosis to cure

DIA would like to express its sincere thanks to the following exhibitors for their contributions and support of the 27th Annual EuroMeeting & 8th Annual Clinical Forum

Oracle Health Sciences Booth 2.C09

WIFI and Exhibit Hall Signage

Accenture Life ServicesBooth 2.E18

Recharging Stations

APCER PHARMABooth 2.D12

Welcome to Paris Reception

Thanks to our Contributors!


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DIA AWARD WINNERS

Outstanding Contribution to Health Award Dr June Raine Director of Vigilance and Risk Management of Medicines Medicines and Healthcare products Regulatory Agency (MHRA)

Dr June Raine trained in general medicine in Oxford after completing a Master degree by research in Pharmacology. Her interest in drug safety led to a career in medicines regulation which has spanned a number of roles in assessment, management and strategic development within the UK national authority. Appointed in 1999 to head Pharmacovigilance in the Medicines Control Agency (now Medicines and Healthcare products Regulatory Agency), she was elected in 2005 to chair the CHMP’s Pharmacovigilance Working Party and in 2012 as the first chair of the Pharmacovigilance Risk Assessment Committee. She is also a member of the WHO Advisory Committee on Safety of Medicinal Products. Her special interests are in monitoring the outcomes of regulatory action, risk communication and patient involvement in the regulatory process. She has made outstanding contributions towards delivering a world-class European Union pharmacovigilance system. She has served as DIA EuroMeeting Programme co-Chair and has been a welcomed Speaker at DIA.

Excellence in Service Award Dr Birka Lehmann Head of Executive Department EU and International Affairs of the Federal Institute for Drugs and Medical Devices (BfArM)

Dr Birka Lehmann studied Medicines at the Free University Berlin and trained at the Kinderklinik Norderney. Her working experience includes 9 years preclinical assessment in the division ‘Pharmacology and Toxicology’ of BfArM and she served as head of unit ‘Decentralised Procedure’ (1996-2002) and as deputy head of EU Division (2000-2002). From 2002 – 2006 she joined the European Commission, Directorate-General Enterprise and Industry as expert on secondment to in the unit ‘Pharmaceuticals’ responsible for inter alia Marketing Authorisation and implementation of Clinical Trials Directive. From September 2006 till October 2011 she was head of the division 3 Marketing Authorisation procedure at the BfArM. Since 2007 she is member of the Paediatric Committee at the European Medicines Agency.

She contributed continuously since 1994 to the DIA EuroMeetings, DIA Annual Meetings and several conferences, training courses and workshops.In 2004 she co-chaired with Yves Juillet the DIA EuroMeeting in Prague. Since 2013 she is chair of the Content Committee for DIA Europe, Middle East and Africa and a member of the DIA Advisory Committee for Europe, Middle East & Africa.

Excellence in Service Award Rolf Banholzer, PhDGlobal Head e-Clinical QA, Novartis Development, Basel

Rolf Banholzer has a PhD in Molecular Biology from the University of Basel.His focus on Computer Systems to be understood as an integral part of efficient and compliant clinical processes and must enable end to end data integrity in the clinical research and development process.

He acts as a translator between IT and Business to obtain optimal user requirements as the basis for successful implementation of IT systems. He promotes clear and transparent communication as well as striving for simplicity, imperative to manage the ever increasing complexity of computer systems.

He works for Novartis in Basel since 17 years in roles with increasing responsibility. He started as a Safety System Administrator and then moved to a Business Analyst role in the Pharmacovigilance area before he took over a global team of QA IT Systems and Process Experts responsible for all IT systems in Novartis Pharma Development. Since almost 2 years, he is also providing QA oversight over clinical data management and statistics processes with his team.

He was co-leading the DIA/ISPE CSV workshop recently held in Basel, is actively supporting the organization committee of the DIA EU Clinical Forum and he is a member of the DIA Advisory Committee for Europe, Middle East & Africa.

Excellence in Service Award Breffni MartinDirector, Regintel, Ireland

Breffni Martin was educated at St Micheal’s College Dublin and Cistercian College Roscrea. At University College Dublin he received his BSc specialising in biochemistry in 1983. Thereafter he worked for a number of major pharmaceutical companies in the area of late stage drug development, in both a scientific, regulatory and information technology context, as well as in environmental management. These include GlaxoSmithKline, Bristol-Myers Squibb, Laboratoire Guerbet and Fermenta (a Pharmacia spin-off). In 1997 he joined IMS Health (IDRAC, a regulatory intelligence company) where he developed regulatory content covering North America, Japan, Australia and elsewhere, including a dedicated FDA Advisory Committee intelligence business. He subsequently set up and operated regintel and CanReg (Europe), regulatory intelligence and services companies.

He is a long-standing member of the DIA a regular programme chair, speaker and contributor to DIAs meetings and publications. He was co-leading the DIA/ISPE CSV workshop recently held in Basel and he served on the DIA Advisory Committee for Europe from 2004 – 2009.



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PROFESSIONAL POSTERS

PROFESSIONAL POSTER AUTHORS

A selected group of professional poster presenters will share theirresearch results in various topics. Visit the hallway of Hall Bordeaux on Level 3 to view the Professional Posters.

P1 Tatyana Benichiva, Professor Drug Regulatory Affairs, Faculty of Public Health, Medical University Sofia, Bulgaria

HTA Methodologies of Novel Treatments (T.Benisheva, G.Petrova, A.Stoimenova, A.Savova, D.Sidgimova,

P.Trendafilova, V.Petkova)

P2 Romuald Braun, CEO, Uanotau, Switzerland How IDMP Can Help Structure Product Information/ Labelling

P3 Magadalena Bujar, Research Analyst, Centre for Innovation in Regulatory Science (CIRS), UK

Expediting the Availability of New Medicines: What Role Do Priority Pathways and Special Designations PlayiIn ICH Countries

P4 Daniele De Martini, Academic Professor, Università Di Milano – Biocca, Italy

Evaluations When Adaptation by Design Is Applied

P5 Claudia Hernandez, Associate Director Regulatory Affairs, Sunovion Pharmaceuticals Inc., USA

Clinical Trial Application Approval Rates for Global Paediatric Placebo Controlled Trials

P6 Sherri Hubby, Director, US Quality Assurance, Premier Research Group, USA

A Compare and Contrast of Significant Changes in Clinical Trial Regulation of FDA, EMA and PDMA and Inspectional Readiness

P7 Tatsuya Ito, Visiting Fellow in Research Integrity, Research and Enterprise Development, University of Bristol, UK

Comparison of the Research Governance Frameworks for Research Involving Humans, Their Tissue and/or Data in the UK and Japan

P8 Christa Janko, Project Manager, Medical University of Vienna, Austria

Stay Ahead in Biomedical Sciences – Catch the LifeTrain

P9 Heinrich Klech, Medical University of Vienna, Austria The Specialist in Medicines Development (SMD) – A Global

Certification Programme by PharmaTrain Federation

P10 George Mathis, CEO, Appletree CI Group AG, Switzerland Clinical Development of Medical Devices in Europe and the USA –

Differences and Similarities

P11 Magdalena Matusiak, Manager, Clinical Development, KCR SA, Poland

Safety Monitoring and Reporting in Clinical Trials – Ensuring Quality and Effectiveness

P12 Stella Mokiou, Research Scientist, UBC: An Express Scripts Company, UK

Use of Patient-Reported Outcomes in Post-Authorisation Safety Studies: A Targeted Review

P13 Marie Lou Munson, Safety Science Leader, Genentech, USA Application of the DILI Expert Working Group Criteria in the

Assessment of Hepatotoxicity

P14 Toshinori Murayama, Professor, Kanazawa University Hospital, Japan

Toward The Advancement for Investigator- Initiated Clinical Trials – A Challenge From Kanazawa/Hokuriku in Japan

P15 Nermina Nakas, Inventiv Health Clinical, USA Pharmacovigilance and Safety: Pediatric Research Challenges

P16 Sue Rees, QPPV, Executive Director Global Safety, Amgen Ltd, UK Pharmacovigilance Process Redesign – Changing the Wheels Whilst

the Car Is Moving

P17 Joerg Seebeck, Chief Medical Officer, PrimeVigilance Ltd, UK QT-Prolonging Effects of Biological Drugs in Humans: A Systematic

Review of a Literature and Public Adverse Event Database

P18 Iris Tam, Director, Managed Care Medical Communications, Genentech, USA

A Review of the World Health Organization (WHO) Essential Medicines List (EML) Decision Making Process

P19 Marie Trad, Quintiles, France Epidemiological Data: An Untapped Resource for Developing an

Operational Strategy for Clinical Trials in Rare Diseases

P20 Jasper Van Berkel, National Institute of Public Health and the Environment, the Netherlands

Empowerment of Patients in Online Discussions about Medicine Use

P21 Linda Van Kerkof, National Institute of Public Health and the Environment, the Netherlands

Characterisation of Apps and Other E-Tools for Medication Use

P22 Angela Van der Salm, Director Pharmacovigilance, Dada Consultancy B.V., the Netherlands

Potential for Medication Errors or Off-label Use – Practical Challenges

P23 Judy Walker, Drug Safety and Product Advisory Services, Quintiles, USA

Benefit-Risk Assessments (BRAs) of an Established Products’ Portfolio (M. Mease and J. Walker)

P24 Janine Collins, Senior Director, European Risk Management, United Biosource Corporation, Switzerland

Failure Modes and Effects Analysis – strategy to minimise and mitigate risks associated with home administration of drug


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STUDENT POSTERS | PATIENTS ADVOCATES

STUDENT POSTER AUTHORS

Student abstracts selected by the review committee, addressing similar topics to those in the programme, will be on display in the hallway of Hall Bordeaix on Level 3.

Presenters will be able to discuss their work during the coffee and lunch breaks on Tuesday, 14 April 2015. An awards ceremony will be held on Tuesday, 14 April 2015 at 17:45 at the DIA Booth in the exhibition hall to award the first and second place student poster winners.

Poster Selection Committee:Gaby Danan, Pharmacovigilance Expert, FranceAngelika Joos, Executive Director, Global Regulatory Policy, MSD (Europe) Inc., BelgiumBirka Lehmann, Head of Executive Department EU and International Affairs of the Federal Institute for Drugs and Medical Devices (BfArM), Germany

SP1 – Nadia Amaouche, University of Paris-Sud, France European Paediatric Regulation launched in 2007: Feedback since

its implementation and perspective for 2017 and beyond

SP2 – Liban Ashkir, University of East Anglia, UK The ECG effects of Moxifloxacin in thorough QT Trials: Systematic

review exploring the impact of design features

SP3 – Amandine Beras, Claude Bernard Lyon 1, France The new version of international electronic transmission of adverse

drug reactions: E2B (R3)

SP4 – Emeline Blanc, Claude Bernard Lyon 1, France Counterfeit drugs: means and technologies to fight against this peril

in ICH countries

SP5 – Ioana-Laura Bogdan, Carol Davila University of Medicine and Pharmacy, Romania

Pharmacy-ethics and economic constraints

SP6 – Marija Simaitienė, Lithuanian University of Health Sciences, Lithuania

Prevalence, risk factors and self-management strategies of primary dysmenorrhea among Lithuanian women

SP7 – Solène Viera de Araujo, Claude Bernard Lyon 1, France What could be publicly retrieved from adverse drug reaction

databases? An overview of 10 pharmacovigilance databases

PATIENT ADVOCATES AT THE EUROMEETING

• Vesna Aleksovska, Association of citizens for rare diseases - Life with

Challenges, Republic of Macedonia

• Dimitris Athanasiou, Parents Project MDA Hellas, Greece

• Claudia Crocione, HHT ONLCUS - Italian Association for Hereditary

Hemorrhagic Telandiectasia, Itlay

• Ivana Dragoievic, HRONOS, Serbia

• Esraa El-Sherbiny, CanSurvive, Egypt

• Elisabetta Gecchele, Acondroplasia Insieme Per Crescere - ONLUS, Italy

• Tess Harris, Polycystic Kidney Disease Charity, UK

• Alexander Kosenko, Russian Union of Patients and Patients Organisations

of Rare Diseases, Russia

• Boris Machavariani, The Alliance of CMI Patients of Georgia, Georgia

• Ananda Plate, Myeloma Patients Europe (MPE) AISBL, Belgium

• Ieva Plume, PHA Latvia, Latvia

• Jill Prawer, LPLD on RareConnect, UK

• Ana Sofia, Sá Cardoso, APCL, Portugal

• Antonella Esposito, Angeli Noonan Onlus, Italy

• Juan Fuertes-Guillen, Pulmonary Hypertension Association (PHA) Europe,

Spain

• Anne Hugon, AFG French Glycogen Storage Disease Association, France

• Mary Lynne Van PoelGeest-Pomfret, World Federation For Incontinent

Patients (WFIP), the Netherlands

• Nicola Bedlington, EPF, Belgium

• Rob Camp, EURORDIS, France

• Jan Geissler, EUPATI, Belgium

• David Haerry, European AIDS Treatment Goup, EU

• Francois Houyez, EURORDIS, France

• Alistair Kent, Genetic Alliance, UK

• Alison Lightbourne, IAPO, UK

• Badri Rengarajan, International Pemphigus and Pemphigoid Foundation,

United States

• Bettina Ryll, Melanoma Patient Network Europe, Belgium

• Christoph Thalheim, European Multiple Sclerosis Platform, Belgium

• Barbro Westerholm, AGE Platform, Sweden

• Phil Willan, Healthcare Quality Improvement Partnership (HQIP), UK

• Yann Le Cam, Eurordis, France

• Durhane Wong-Reider, Canadian Organization for Rare Disorders, Canada

PATIENT WELCOME LUNCHMonday, 13 April 2015 | 12:30 - 14:00 | Room Arlequin Level 3Reserved for patient representatives and patient speakers only

Overview of the EuroMeeting scientific programme and an opportunity for patient representatives to meet and network with each other prior to the conference.

DIA Communities: Get Engaged. Develop Your Horizons.

Together We Can Do So MuchDIA’s Communities provide our members with an opportunity to build relationships across disciplines and around the world… and at the same time contribute to improving the process of health care product development.

DIA Communities provide a way for members across the globe to interact with their peers and to form cross-disciplinary teams. It is here that members share information, raise concerns, mentor one another, and find answers together—accomplishing more as a group than any one person could accomplish alone. DIA members are encouraged to join those Communities that match their interest areas.

DIA Communities Meet & Eat Tuesday, 14 April 2015 | 13:00-14:00Room Arlequin, Level 3

Are you active in a Community? Come attend a relaxed, informal and rare opportunity to meet your fellow Community members face-to-face. Enjoy your lunch and share your experiences with those who are curious about DIA Communities.

New to DIA? Not part of a Community? You’re invited too! This is an opportunity to ask questions and learn more about this member benefit. Look for DIA Community members who will be wearing a special button.

Scan this QR code to learn more about DIA Communities

To learn more about DIA Communities visit the DIA Booth #3.D17 or stop by the Membership Lounge located on Level 3 or scan the QR code below.

DIA Membership

DIA and You: Driving Ideas to Action

Join DIA. Visit DIAGlobal.org/Membership

To learn more about DIA Membership visit the DIA Booth # 3.D17 or stop by the Members Lounge located on Level 3 or scan the QR code below.

DIA Members Lounge Hall Bordeaux Level 3

Are you looking for a space to relax or connect with other DIA members? Stop by the Members Lounge located in the Hall Bordeaux on Level 3.

This exclusive lounge offers members a place to take an important call, get online, charge devices or just relax.

Coffee and drinks are served throughout exhibition opening hours and charging stations for your electronic devices are available. Make sure you bring your membership card to identify yourself as a DIA Member at the desk. Access exclusively for DIA Members.

Note: if you have not yet received your new membership card, have it printed onsite in the Members Lounge.

Scan this QR code to learn more about

DIA Membership

It’s all about connections With DIA, people and ideas come together on a global scale to accelerate innovation and identify solutions. DIA provides invaluable forums to network with your peers, build new relationships, share ideas, and gain knowledge that can improve health care around the world.

As a DIA member, you will:

• Be a part of a global network, working to advance health care product development

• Work closely with like-minded professionals to solve real issues through DIA Communities and Scientific Task Forces

• Enjoy discounts on more than 200 educational opportunities offered worldwide each year

• Keep informed of news as it happens with our daily enewsletter

• Learn the latest on health care products and regulatory science through our bi-monthly news magazine

• Read about recent discoveries—and submit your own papers—through our peer-reviewed scientific journal

The more you engage, the more you gain.

Together we can create a world where improved health is a reality for everyone

The goal of DIA is simple: To help you gain the knowledge you need to improve health and wellbeing worldwide.



16

EM Tutorial 1 | Monday 13 April 2015, 09:00-12:30

Room 252B Level 2

HOT TOPICS IN PHARMACOVIGILANCE AND ADVERSE REACTION REPORTINGSabine Brosch, Principle Scientific Administrator, European Medicines Agency, EUGaby Danan, Pharmacovigilance Expert, France

Taking into account the implementation experience with GVP module VI, which came into force on 2 July 2012, this tutorial will focus on frequently asked questions from stakeholders with regards to the day-to-day operational aspects.

In addition, practical considerations on the update of GVP module VI focusing on a simplification of the reporting of suspected adverse reactions from non-interventional post-authorisation studies, compassionate use and patient support programmes will be addressed.

At the start of 2015, the European Medicines Agency will launch a new process of monitoring medical literature for selected substances and selected medical literature in line with the provisions set out in Article 27 of Regulation 726/2004. The tutorial will provide the opportunity to discuss first experiences of marketing authorisation holders (MAHs) and to address specific implementation questions.

Article 24 of Regulation 726/2004 also outlines a new approach for marketing authorisation holders to access EU adverse reaction reports directly in EudraVigilance, following the successful outcome of an audit of the European pharmacovigilance database. In preparation of these changes, the EudraVigilance Access Policy will be updated to define the data elements of Individual Case Safety Reports (ICSR) for which access can be provided in compliance with EU personal data protection legislation. The tutorial will provide an opportunity to discuss those data elements in support of the marketing authorisation holders’ pharmacovigilance obligations.

The tutorial will conclude with a detailed discussion of the reporting principles in line with the new ICH Individual Case Safety Report E2B (3) guideline thereby highlighting new concepts such as causality assessment at event level, drug reaction relatedness, amendment reports and reporting of special situations (e.g. counterfeit medicines, product defects, medication errors).

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to:• Address FAQs on GVP Module VI “Management and reporting of adverse

reactions to medicinal products” and recent updates• Discuss the new process for monitoring of medical literature by the EMA

and the potential impact on MAHs• Describe the principles of access to EudraVigilance based on revised

policy• Discuss reporting principles based on the new ICH ICSR E2B(R3)

guideline

Target AudienceThis tutorial is designed for Qualified Persons Responsible for Pharmacovigilance (QPPVs) and individuals involved in Pharmacovigilance, clinical development, information management, and safety databases.


Room 253 Level 2

INTERACTIONS BETWEEN REGULATORY AND LEGAL IN INTELLECTUAL PROPERTY, PRODUCT LIABILITY AND COMPETITIONGeneviève Michaux, Counsel, Hunton & Williams, Belgium Karina Hellbert, Partner, FIEBINGER POLAK LEON RECHTSANWÄLTE GmbH, Austria

Interactions between the regulatory regime and intellectual property (for example, SPC, data exclusivity), product liability (e.g., off label information in summary of product characteristics (SmPC), competition (for example, public procurement) and privacy (e.g., clinical trials) rules are increasing. As a result, those matters can no longer be approached in isolation, and a more comprehensive perspective is required when addressing regulatory issues.

Learning ObjectivesAt the conclusion of this tutorial, participants will be able to:• Explain and discuss the basics of intellectual property, product liability

and competition rules applicable to medicinal products• Identify and better address the regulatory issues that present an

intellectual property, product liability or competition aspect

Target AudienceThe tutorial is designed for non lawyers with good regulatory experience and for regulatory lawyers who want to learn the basics of intellectual property, competition, and product liability rules as they apply to the pharmaceutical sector.


Room 251 Level 2

ANALYSIS OF SAFETY DATA FROM CLINICAL TRIALSJürgen Kübler, Global Head, Clinical Design, Analysis and Reporting, CSL Behring AG, GermanyChristine Wichems, Global Head of Medical Writing and Disclosure, CSL Behring GmbH, Germany

This tutorial is a combination of theory, guidelines, practical considerations, and real-life solutions for those working in the clinical development environment (pharmaceutical, biotech industry, or CRO). The aim of this tutorial is to provide a basic understanding of the underlying methodology and the current guidelines on safety data. Aspects of the planning of clinical trials as well as the problems and pitfalls during the analysis of safety data will be presented. Opportunities for prospective planning of safety analysis at the project level will be discussed. The presentations will also include case studies.

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALS

MONDAY, 13 APRIL 2015 | 09:00-12:30


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Learning ObjectivesAt the conclusion of this tutorial, participants should be able to:• Examine relevant guidelines and regulatory requirements for clinical

trials• Recognise how to contribute to safety analysis plans• Assess statistical safety analysis and identify pitfalls in safety analysis• Recognise the impact of benefit/risk assessment in safety data

Target AudienceThis tutorial is designed for biostatisticians, medical writers, clinical researchers, drug safety specialists, project managers, and investigators.


Room 252A Level 2

PRIVACY AND PERSONAL DATA PROTECTION IN DRUG DEVELOPMENTPierre-Yves Lastic, Associate Vice President, Chief Privacy Officer, Sanofi, France

This tutorial addresses issues in data privacy today:• Why is personal data protection important?• Principles of personal data protection, based on the European

regulations• Overview of worldwide regulations and the differences between them• Specific regulations for biomedical research and pharmacovigilance

impacting drug development• How to comply?• Information & consent• Communication & training• IT security & validation• Legal instruments (contracts, Safe Harbor, BCRs)• Data Privacy organisation

Learning ObjectivesAt the conclusion of this tutorial, participants should be able to:• Understand the principles of personal data protection in the European

Union• Understand the differences between European, US and Asian data

privacy regulations• Have a basic knowledge on how to comply with regulations in the

specific field of drug development

Target AudienceThis tutorial is designed for: • All individuals involved in the organisation and management of clinical

trials and pharmacovigilance, or handling data collected to perform these activities

• Professionals working with health data, clinical data, genetic data, tissue samples, medical imaging, mobile health apps, and any kind of personal data in the field of drug development


Room 241 Level 2

MOVING FROM RISK MANAGEMENT TO BENEFIT/RISK MANAGEMENT EMBEDDING PHARMACOVIGILANCE PRINCIPLES INTO THE PRODUCT LIFE CYCLEShelley Gandhi, Director Pharmacovigilance and Drug Safety, NDA Group, UKWilliam Richardson, Medical Advisor, NDA Group, UK

Pharmacovigilance, or the activity of monitoring the safety of medicines in clinical use and taking appropriate action to minimise risk, is governed by a range of new EU legislation, a new Pharmacovigilance Risk Assessment Committee and guidance. The value that can be gained from adopting a benefit/risk management system not only addresses known and potential risks to support the current regulatory status of product but also will feed into the further development of a product as regard new indications and potentially moving from prescription only to over the counter.

This tutorial will discuss how access to robust evidence on emerging risk in post-authorisation phase, good data on how a medicine is used in clinical practice and data on background rates in the exposed population – gathering evidence throughout the product lifecycle will help move companies to a benefit/risk system. The ultimate challenge – is working towards an integrated regulatory system so you can query across all information within a company, designing safety studies, monitoring the effectiveness of the risk management systems and gather robust evidence from clinical practice.

The lessons learned and our experiences so far with post-authorisation commitments (e.g. BRMPs, PASS, PSURs) will be reviewed as will whether these commitments really do support an acceptable benefit/risk profile. This will include the novel approaches to managing benefit/risk to meet the needs of licensing medicines in biotechnology such as advanced therapies. Communicating benefit/risk will also be discussed as the new legislation will push for greater patient involvement within a benefit/risk system. Better methodologies and tools are required to support this integrated approach and adoption of a quality management system across global enterprise could achieve this.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to:• Learn about what are effective strategies and the current thinking on risk mitigation in the context of benefit throughout the product lifecycle. Access to robust evidence on emerging risk is critical• Discover what the principles are for proportionate risk based assessment • Find out about hurdles which get in the way to a systematic approach and how these might be tackled

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALSMONDAY, 13 APRIL 2015 | 09:00-12:30



18

Target AudienceProfessionals in companies or regulatory authorities who are involved in pharmacovigilance operations and with responsibilities for post marketing clinical safety including those who are involved in:

• Pharmacovigilance• Regulatory• Clinical research• Risk management• Medical product safety assessment• Data analysis• Epidemiology• Labelling• Quality assurance and compliance


Room 242A Level 2

MODELLING AND SIMULATION (M&S) / MODEL INFORMED DRUG DIS-COVERY AND DEVELOPMENT (MID3)Amy Cheung, Clinical Pharmacometrician, AstraZeneca R&D, UKFlora Musuamba Tshinanu, Research Associate, University of London, UK

The tutorial will be an opportunity to understand better the value of Modelling and Simulation (M&S) / Model Informed Drug Discovery and Development (MID3) in the development of new medicinal products, and the impact of MID3 on regulatory decision making and product labelling.

The tutorial will be divided in three parts.

First, the basic concepts and principles of modelling and simulation will be recalled and the added value over two stage approach will be discussed using examples.

The second part will be dedicated to the use of M&S and MID3 in the context of drug development and the impact it can have in key decision making: study design optimization, data analysis, dose selection and extrapolation will be discussed.

The last part will be dedicated to model evaluation given the purpose. For each of the application of M&S/MID3 in drug development, the relevant criteria for model evaluation will be presented and discussed. Focus will be made on data needed, model assumptions and uncertainties and fitting performances and examples will be provided for good and bad practices. Challenges in current practices (given the current regulatory requirements) will also be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to:• Recall the basic concepts of Modelling and Simulation and its advantages

over other approaches for Pharmacokinetics (PK) / Pharmacodynamcis (PD) description and predictions

• Discuss/explain the added-value and importance of Modelling and Simulation Model Informed Drug Discovery and Development in the context of drug development

• Outline/discuss the criteria for model evaluation given the purpose in drug development setting

Target AudienceModeller, biostatisticians, medical writers, clinical researchers, drug safety specialists, project managers, investigators, clinical practitioners, physicians, regulators, with basic experience/awareness of Modelling and Simulation (M&S)/Model Informed Drug Discovery and Development (MID3).

OPTIONAL EUROMEETING PRE-CONFERENCE TUTORIALS

MONDAY, 13 APRIL 2015 | 09:00-12:30

21-23 SEP VIENNA, AUSTRIA | #15543Quality by Design - New concepts for chemical and biotech product development and optimisation

Participants from pharmaceutical, biotech and generic industry as well as regulators will learn how to use QRM, Process Characterisation, DoE, Development of a Design Space and Control Strategy, as well as the tools of Knowledge Management (KM).

EUROPE, MIDDLE EAST & AFRICA

2015 TRAINING COURSES

CHEMISTRY, MANUFACTURING & CONTROLS

CF Tutorial 1 | Monday 13 April 2015, 14:00-17:30

Room 242A Level 2

A LEGAL UPDATE: RECENT AND CURRENT DEVELOPMENTS IN EUROPEAN PHARMACEUTICAL LAWJohn Lisman, Lawyer, Lisman Legal Life Sciences, NetherlandsKoosje van Lessen Kloeke, Life Sciences Lawyer – Partner, Leijnse Artz advocaten, Netherlands

Pharmaceutical law is at the basis of daily business in the pharmaceutical and biotech industry. In the last few years many developments have occurred in the field of pharmaceutical law, as well as in the marketing authorisation practice.

This tutorial brings you up to date in respect to marketing authorisation for Biosimilars - Paediatric Regulation - Advanced Therapy Medicinal Products Regulation - Clinical Trials Regulation - Early Access to promising new medicines - Adaptive licensing - Transparency. For each of these topics the most relevant highlights will be presented in an interactive manner. Furthermore, recent ECJ case law relevant for the audience will be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to:• Apply pharmaceutical legislation in their daily practice. • Explain the changes in pharmaceutical legislation in their companies. • Participate in an interactive way in the DIA Clinical Forum sessions

dealing with new legislation, e.g. Clinical Trial Regulation.• Recognise recent case law of the Court of Justice and the General Court.

Target AudienceNon-lawyers who work in a regulated (clinical or medical) environment from industry, government agencies and competent authorities. Lawyers from industry, government agencies and competent authorities who have recently started in this position.

CF Tutorial 2 | Monday 13 April 2015, 14:00-17:30

Room 242B Level 2

ENSURING DATA QUALITY AND DETECTING POTENTIAL FRAUD/GCP MISCONDUCTMarc Buyse, Chairman, IDDI (International Drug Development Institute) Inc., USAStephen George, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, USA

A large number of options are available to operationalise the recommendations outlined in the FDA Guidance on Risk-Based Monitoring (RBM) and the EMA Reflection Paper on Quality by Design. TransCelerate’s position paper provides a detailed view of some of the tools and processes that can be used for a successful implementation of RBM. This tutorial session will review the landscape of current practices with a focus on data quality and integrity. The session will demonstrate how analytical tools can support an RBM strategy by analysing clinical data to identify risks in some sites arising from lack of training, misunderstanding, carelessness, negligence or even fraud.

The session will cover the tenets of CSM (Central Statistical Monitoring) and KRI (Key Risk Indicators). The types of findings that can be detected by these two approaches will be discussed and illustrated with concrete examples. Importance will be given on how the cause of the findings can be identified and classified by seriousness, from poor protocol understanding to intent to cheat. The operational implications of the findings in terms of corrective actions to take will also be discussed.

Learning ObjectivesAt the conclusion of this tutorial, attendees will be able to:• Implement the recommendations outlined in the FDA Guidance on Risk-

Based Monitoring• Demonstrate how the use of objective, analytical tools can be used to

identify sites at risk and detect potential fraud• Interpret findings from KRIs and CSM to drive operational corrective

actions

Target AudienceProfessionals involved in the management of clinical trials: Data Managers, Statisticians, Medical Reviewers, Study Physicians, Project Managers, Study Coordinators, Quality Managers and Senior Management.

SAVE TIME AND TRAVEL COSTS with IN-HOUSE TRAININGAll DIA Training Courses can be held on your premises and tailored to your needs.

Get the best value and train your whole team!

OPTIONAL CLINICAL FORUMPRE-CONFERENCE TUTORIALSMONDAY, 13 APRIL 2015 | 14:00-17:30

The 8th Annual Clinical Forum will take place in parallel of the 27th Annual EuroMeeting.Participants have the opportunity to register for optional tutorials for both conferences.



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EU REGULATORY TOWN HALL MEETING

50th Anniversary of the EU Pharmaceutical Legislation

MONDAY, 13 APRIL | 13:30-15:00ROOM BORDEAUX LEVEL 3

Moderator: Fernand Sauer, French Academy of Pharmacy, Former Executive Director of the European Medicines, France

The Regulatory Town Hall will celebrate this unique occasion with a panel discussion on how we can build on past achievements to address the challenges ahead.

The European Commission will make an opening address followed by the panel discussion covering these subjects:• Building the European Medicines Regulatory Network. How

is the house looking today and in the future?• What can regulatory authorities do to support innovation?• Implementing new legislation - what are the challenges,

what are the solutions?

Panellists:Xavier De Cuyper, Chief Executive Officer, Federal Agency for Medicines and Health Products, BelgiumViola Macolić Šarinić, Head of Agency, Agency for Medicinal Products and Medical Devices (HALMED), Croatia Guido Rasi, Principal Adviser, European Medicines Agency, EUAndrzej Rys, Director of Health Systems and Products, European CommissionChristoph Thalheim, Director External Affairs, European MS Platform, BelgiumChrista Wirthumer-Hoche, Head, Austrian Medicines and Medical Devices Agency (AGES), Austria

50 YEARS OF EDQM Preparing for the Future to Continuously Ensure the Quality of Medicines

WEDNESDAY, 15 APRIL | 11:00-12:30ROOM MAILLOT LEVEL 2Session Chair: Susanne Keitel, Director, EDQM/Council of Europe

The European Directorate for the Quality of Medicines and HealthCare (EDQM) turned 50 in 2014. While EDQM and its 37 Member States are proud of the tremendous achievements over the past 50 years, globalisation and technical and scientific developments in all fields pose constant challenges.

See page 46 for details.

HAS SATELLITE SYMPOSIUM

MONDAY, 13 APRIL | 11:00-12:30 ROOM MAILLOT LEVEL 2

Introduction of Innovative Drugs and Devices in France- Recent Advancements in HTA, Pricing and Reimbursement

Moderator: François Meyer, Advisor to the President, International Affairs, Haute Authorité de Santé (HAS), France

How does France, one of Europe’s largest countries, deal with the assessment and introduction of Innovative Drugs and Devices? The session will provide an overview by HAS with regard to its first-year experience in health economic assessments of new health products, examine the current situation and challenges of pricing for innovative drugs and devices and describe the evaluation and decision-making process at the level of hospital committees.

Introduction of Health Economics in the HTA Process for New Drugs and Medical Devices in France: First-year experienceJean-Luc Harousseau, President, Haute Autorité de Santé (HAS)

Price Determination for New Drugs and Medical Devices in France: Current situation and perspectives Dominique Giorgi, President, Comité Economique des Produits de Santé (CEPS)

Role of Institutional Committees for Admission of New Medicines and Innovative Medical Devices in French Hospitals (COMEDIMS)Philippe Lechat, French Society of Pharmacologie and Therapeutics (SFPT)

Experience of Early Dialogue Pilots Conducted within SEED and EUnetHTA: Coordinator’s perspective on the feasibility of implementing a permanent model in Europe and the promise of improving clinical data collection prior to market approvalMira Pavlovic, Deputy Director DEMESP, Haute Autorité de Santé (HAS)

The session will conclude with HAS’s experience in coordinating the early dialogue/scientific advice activity in Europe through the EUnetHTA and SEED projects, highlighting the benefits of a collaborative approach between HTA agencies, regulatory and health product developers.


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OPENING PLENARYMONDAY, 13 APRIL 2015 | 16:00 - 17:45 | BLUE ROOM LEVEL 2

ADVANCING INNOVATION TO COMBAT THE GLOBAL BURDEN OF DISEASE: A CALL TO ACTION

A panel of respected leaders from academia, industry, health agencies, patient advocacy, and the payer

community will share perspectives on the dichotomy between evolving diseases, well-known conditions,

and neglected and rare diseases, contrasted with the challenges that exist in understanding the physiology

of these conditions and developing therapies to treat them.

The challenges include topics such as the efficiency of R&D, access to health care, ethics and transparency

issues related to the clinical trials, and the increasing challenges related to investments required to bring

safe, effective, economically-viable, and accessible products to patients worldwide.

All stakeholders see the challenges, but each from a different perspectives: health authorities in terms of

the public health preservation, industry in terms of ROI, academics in terms of generating new knowledge,

payers in terms of risk management, patients in terms of managing their disease.

During this provocative and stimulating discussion, the panellists will work to converge on a shared vision

and call to action.

Keynote Speakers:

• Olivier Charmeil, Executive Vice President Vaccines, Sanofi, France

• Andrew Morris, Professor, University of Edinburgh, Chairman and Centre Director, Farr Institute, UK

Panellists:

• David Haerry, Board of Directors, European AIDS Treatment Group, EU

• Hugo Hurts, Executive Director, Medicines Evaluation Board (MEB), the Netherlands

• Guido Rasi, Principal Adviser, European Medicines Agency, EU

Moderator:

Maureen Kenny

Editor, Scrip Regulatory Affairs, Informa Business Intelligence, UK



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EM0101 | Tuesday, 14 April, 09:00-10:30 | Blue Room Level 2

THE ROLE OF THE NEW CLINICAL TRIALS LEGISLATIONSession Chair:Lidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

The objective of the session is to present and discuss major changes in the overall approach, assessment, approval and conduct of clinical trials in the EU introduced in the new legislation. New definitions, new procedures for sponsors and Member States, the role of ethics committees, new IT tools introduced in the legislation will be presented with the view to inspire discussion on the forthcoming challenges and the use of new legal instruments to make the EU a more attractive area for the conduct of clinical trials.

Key Content of the New Clinical Trials Legislation- Setting the sceneLidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

EMA Preparedness for the Implementation of the New LegislationAna Rodriguez, Head of Clinical and Non-Clinical Compliance, European Medicines Agency, EU

Challenges in the Ethical Review of Clinical Trial ApplicationWojciech Maselbas, Vice President, Association for Good Clinical Practice, Poland


CLEARING THE PATH FOR INNOVATIONS IN DRUG DEVELOPMENTSession Chair:John Lisman, Lawyer, Lisman Legal Life Science, the Netherlands

This session will focus on the activities stakeholders perform to prepare and implement the novelties in the Clinical Trials Regulation. What will Member States do to lift their new responsibilities? How will industry adapt to the new regulatory set-up and where do patients foresee strengths and challenges?

Pathways for Early Access to Medicines - How can the use of existing tools be improved from an industry perspective? Sabine Atzor, Head of EU Regulatory Policies, F. Hoffmann-La Roche, Switzerland

Member States’ Activities for Implementing Innovative Initiatives in Respect of Clinical TrialsChristian Schneider, Senior Medical Officer, Danish Health and Medicines Authority (DKMA), Denmark

Patients’ Expectations in Respect of the Development of New MedicinesBettina Ryll, Founder, Melanoma Patient Network Europe, Sweden


PRIMARY FACTORS FOR TRIAL LOCATIONSession Chair:Dorthe Poulsen, Chief Legal Adviser, EU-Coordinator, Danish Health and Medicines Authority, Denmark

Which factors are decisive for the decision to conduct a clinical trial in a certain part of the world? There is no doubt that the EU competes with other parts of the world such as the BRIC countries. In this session, we’ll have a look at the EU in a more global context.

Country Allocation in Global PerspectiveHenrik Troelsen, Director, Global Trial Allocation, Novo Nordisk, Denmark

A Brazilian Perspective Speaker invited

The Swedish Way to Foster InnovationChrister Backman, EU Coordinator & Senior Expert, Medical Products Agency (MPA), Sweden

Theme 1 Access to Innovative Treatments: New R&D models and innovative clinical trial methodologies. The role of the new clinical trials legislation

Susanna Del Signore, Associate Vice-President Global Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D, FranceDorthe Poulsen, Chief Legal Adviser, EU-Coordinator, Danish Health and Medicines Authority (DKMA), Denmark

Researching and developing new medicines demands high levels of innovation. This in turn delivers insights that benefits both society and patients. This research is dependent on clinical trials; without these, there would be no new medicines, no improvement of existing medicines and no evidence-based improvement in how we use current treatments.

Despite this, the number of clinical trials undertaken in Europe is decreasing. Clearly, if we wish to stimulate healthcare R&D, we need a robust, operative and cost-effective EU framework for clinical trials.

This theme will examine how we can use the new legal framework for clinical trials to create this environment. How can we make the best use of available tools whilst safeguarding the rights and safety of the clinical trial subject? What are the considerations for clinical trial design, such as location and disclosure requirements? Practically, how can we have a regulatory system that lets us respond quickly to the demands of an ageing population and a rapid rise in chronic conditions?


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EM0105 | Wednesday, 15 April, 09:00-10:30 | Room 251 Level 2

IMPACT OF THE CLINICAL TRIAL REGULATION ON CMC FILING AND RELEVANT GMP REQUIREMENTSChair invited

The new Clinical Trial Regulation will be a key step towards supporting medical research in Europe, covering multiple aspects of the clinical trial, from the organisation, through implementation and the reporting process. The quality of CT material needs to be properly designed and appropriately manufactured. Which quality system should be applied? What are the expectations from regulators at this stage? What consequences for the industry?

This session will be a good opportunity, with appropriate timing, to go through it and clarify the legal and regulatory impact (legislation and delegated act) as well as identifying the challenges and opportunities for the pharmaceutical industry.

How CTR May Change IMP Supply and How Ready Are We? Anthony Moult, Director Clinical Supplies Operations, Daiichi Sankyo Development, UK

Impact of the CTR on Investigational Product Supply - Potential Challenges for the Industry Sabine Pfister, Senior Compliance Professional, Quality Compliance & Auditing, Novartis, Switzerland

EM0106 | Wednesday, 15 April, 11:00-12:30 | Blue Room Level 2

MEDICINES ADAPTIVE PATHWAYS TO PATIENTS (MAPPS) - HOLDING HANDS ACROSS THE LIFECYCLESession Chair:James Anderson, Head of Corporate Government Affairs, GlaxoSmithKline, UK

MAPPs refer to flexible development and access pathways within the current regulatory framework that balance early patient access, public health and societal benefits. To afford patients faster access to innovative medicines a fundamental and co-ordinated change is now required across all elements of the bioscience sector, with collaboration and partnership becoming a core capability for success. The requirements and expectations for sustainable change will be discussed from various perspectives.

Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency, EU

Gigi Hirsch, Executive Director, CBI and Program Director, NEWDIGS, MIT, USA

Yann Le Cam, CEO, EURORDIS, France EM0107 | Wednesday, 15 April, 14:00-15:30 | Blue Room Level 2

MAPPS: FOSTERING COLLABORATIVE MODELS FOR CLINICAL DEVELOPMENT Susanna Del Signore, Associate Vice-President Global Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D, France

Chris Chinn, VP and Head of Health Investment Evidence, GSK; Project Coordinator IMI GETREAL

Alastair Kent, Director General, Genetic Alliance UK

Nathalie Seigneuret, Senior Scientific Project Manager, Innovative Medicines Initative (IMI), Belgium


DISCLOSURE OF INFORMATION - WHAT WILL BE DISCLOSED ACCORDING TO THE NEW SCHEME IN THE CLINICAL TRIALS LEGISLATION? Alexander Roediger, Director European Union Affairs, MSD (Europe) Inc., Belgium

The Clinical Trials Regulation requires that the EU database shall be publicly accessible unless, for all or part of the data and information contained therein, confidentiality is justified for certain reasons. Some factors that may justify confidentiality is the protection of commercially confidential information, the protection of confidential communication between Member States in relation to the preparation of the assessment report and the protection of personal data.

This session will take a closer look at the circumstances under which “confidentiality is justified” as listed in Art. 81(4), in particular commercially confidential information. It will also address the questions what constitutes an “overriding public interest in disclosure” and which legitimate factors from various stakeholders need to be taken into account? Finally, it will put these requirements into the broader context, in particular regarding EMA’s Policy 70 “On publication of clinical data for medicinal products for human use”.

The presentations/discussion will shed light on controversial issues and a panel debate will follow.

The EMA Proposal – The Agency’s Attempt to Strike a Balance between Disclosure and Protection of Clinical Trials InformationAna Rodriguez, Head of Clinical and Non-Clinical Compliance, European Medicines Agency, EU

Disclosing Information about Clinical Trials- Where are the limits? The Industry Research PerspectiveFabien Peuvrelle, Director, Clinical Trials and Labelling Translations Group, Regulatory Affairs Europe, Celgene R&D, Switzerland

Public Access to Clinical Trials Data: The consumer perspectiveIlaria Passarani, Health Policy Officer, European Consumer’s Organisation (BEUC), Belgium

To What Extent Will the European Citizen Benefit from the Clinical Trial Information? Experiences from Empirical ResearchFrederic Bouder, Associate Professor, Maastricht University, the Netherlands



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EM0201 | Tuesday, 14 April, 09:00-10:30 | Room 241 Level 2

NEW MEDICAL DEVICE REGULATIONS IN THE EUSession Chair:Sabina Hoekstra-van den Bosch, Global Regulations and Standards, Philips Healthcare, the Netherlands.

The Strengthening of the EU Medical Devices Regulatory SystemErik Hansson, Deputy Head of Unit, Health Technology and Cosmetics, European Commission, EU

New Legislation on Medical Devices – Update on progress and future prospectsGraeme Tunbridge, Head of Medical Devices EU Policy, Medicines and Healthcare Products Regulatory Agency (MHRA), UK

A Notified Body’s Perspective on the Impact on Stakeholders of the “Immediate Action Plan” and the Proposed Medical Device Regulation Neil Adams, Director Operations and Delivery, Medical Devices, BSI, UK


IMPACT OF THE REGULATIONS IN PRACTICE ACROSS REGIONSSession Chair:Michelle O’Connor, Director Regulatory Consulting, Icon Clinical Research, Ireland

Medical Devices Regulatory Pathways across the EU and US regions Michelle O’Connor, Director Regulatory Consulting, Icon Clinical Research, Ireland

Ning Li, Vice President and Head, Asia/China Regulatory Affairs, Sanofi

Medical Device Requirements in India Daniel Verstappen, Vice President Quality & Regulatory Affairs Europe - EAGM – India, GE Healthcare, Belgium


IMPACT OF THE REGULATIONS ON SUBSTANCE-BASED MEDICAL DEVICESSession Chair:Miranda Moussa, Manager for Medical Devices, AESGP, Belgium

In light of the growing interest in substance-based medical devices across Europe, the session will look into their particularities and consider ways to ensure that safe, effective and innovative substance-based medical devices continue to reach citizens. The latest European developments on the proposed regulation on medical devices and their potential impact on this category will be discussed.

A Notified Body’s Perspective on the Impact of the Proposed Medical Device Regulation on Substance-based Medical DevicesNeil Adams, Director Operations and Delivery, Medical Devices, BSI, UK

Adjusting the Medical Device Legislation to Substance-based Products- The perspective of one competent authority Judite Neves, Head of Medical Devices, Infarmed, Portugal

George Jessen, Member of the AESGP Committee on Medical Devices; Regulatory Affairs Manager, Procter & Gamble, UK

EM0205 | Wednesday, 15 April 09:00-10:30 | Room 242 Level 2

NEW IVD REGULATIONS IN THE EUSession Chair:Jesús Rueda Rodríguez, Regulatory Affairs Director, European Diagnostic Manufacturers Association

MSdialog: A Novel System for Engaging Patients with Multiple Sclerosis in Routine Health Outcomes Reporting and MonitoringMark Thristan, Associate Director, IS - R&D - Embedded IT, EMD Serono, USA

View from a Notified BodyCatherine Holzmann, IVD department Manager, GMED Certification Division

Theme 2 Medical Devices and Combination Products

Paul Jansen, Global Head Medical Devices US, Sanofi, USASabina Hoekstra-van den Bosch, Global Regulations and Standards, Philips Healthcare, the Netherlands.

The healthcare industry is increasingly embracing the opportunities offered by medical devices, in vitro diagnostics combination products and health information technology.

This theme examines the trends in this rapidly evolving field. It will examine the regulatory challenges posed by these novel combination therapies, particularly the use of the biologics used in drug / device combinations. It will also consider the regulatory aspects of how information technology and health apps collect and use the data generated by modern medical devices. How can we ensure we that have a regulatory environment capable of reacting quickly to the needs of this dynamic sector and maximizing its benefits for the patient? The theme will also provide the latest news on the upcoming European Medical Device Regulation and In Vitro Diagnostic Regulation, highlight the latest trends in international medical device regulation and analyse consequences of the future European legislative changes for substance-based medical devices.


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Theme 3 Advanced Therapies, Novel Treatments for Rare Diseases

Paula Salmikangas, Chair CAT, Senior Researcher, Finnish Medicines Agency (FIMEA), FinlandHenk Schuring, Group Vice President Regulatory Affairs - Europe, Genzyme Europe B.V., the Netherlands

We now have five years of experience of the Advanced Therapy Medicinal Products (ATMP) legislation. It is time to review whether this legislation has met expectations. Has it produced the numbers – and effectiveness - of treatments expected? Although some products have already reached patients, the success rate has been low and there have been several issues over effectiveness.

This session will examine what we have learned from the ATMP legislation and whether it now requires revision. Will the new regulatory pathways developed for ATMP, such as adaptive licensing, change how we approach drug development? What lessons have the work with rare diseases and orphan drugs taught us, and how do we leverage those insights more widely?


APPROVAL PROCESS AND EXPERIENCE TO DATE/CHALLENGES AND SUCCESSFUL OUTCOMES OF ATMP DEVELOPMENT Session Chair: Beatriz Silva Lima, Advisory Board, NDA, UK

The progress on gene and cell therapies from regulation implementation will be addressed from European perspective. The rapid knowledge evolution will be illustrated with examples of products history from their research until their current stage.

An Overview to MAA Process for ATMPs and CAT Experience from Five First YearsPatrick Celis, Scientific Administrator, Committee for Advanced Therapies (CAT) Secretariat, European Medicines Agency, EU

Commercially Viable Options for ATMP ManufactureKlaus Maleck, CEO, TETEC AG, Germany

International Regulatory Co-Operation for ATMPsLiz Anne Gillham-Eisen, A/Director, Health Products and Food Branch, Health Canada, Canada

Anticipation of the Coming Changes in the EU Regulatory Framework for Companion DiagnosticsSylvie Le Gledic, Director, IVD/CDx, Voisin Consulting Life Sciences, France


HEALTH APPS: DEFINITION, REGULATION & USESession Chair: Erik Vollebregt, Partner, AXON Lawyers, the Netherlands

Erik Vollebregt, Partner, AXON Lawyers, the Netherlands

Brad Thompson, Member of the Firm, Epstein, Becker & Green

Pierre Leurent, CEO, Voluntis, France


CLINICAL, QUALITY & REGISTRATION CONSIDERATIONS FOR BIOLOGICS COMBINATION PRODUCTS DEVELOPMENT Session Chair: Paul Jansen, Global Head Medical Devices US, Sanofi, USA

There are a growing number of Biologics in active development, many of them in late stage development. This session will explore some of the key considerations and unique challenges of gaining approval of Biologics combination products.

EU/US Registration Dossier Challenges for Biologics/Device CombinationsMarielle Fournier, Director, Combination and Borderline Products, Voisin Consulting Life Sciences, France

Risk-Based Quality and Compliance Management in Clinical Trials with Combination ProductsMarina Malikova, Executive Director, Surgical Translational Research Operations and Compliance, Boston University School of Medicine, USA

The EU Framework for Combination Products – A Regulatory ViewChrista Wirthumer-Hoche, Head, Austrian Medicines and Medical Devices Agency, Austria



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QUALITY ASPECTS FOR AN ATMP (M3 OF MAA)Session Chair: Valerie Pimpaneau, Voisin Consulting Life Sciences, France

The development of ATMP comes with a complex set of CMC challenges that developers need to overcome in order to design an adapted control strategy. The complex nature of cell and gene therapy products combined with mode of actions that may not always be fully understood and often involve multiple pathways lead to quality assessment approaches likely to be unique to each product. This session will provide an overview of major quality issues encountered during the development of cell based medicinal products, discuss the difficulties encountered in developing potency assays and the value of using risk assessment to support development choices and quality assessment strategies.

Major Quality Issues for CBMPsMargarida Menezes-Ferreira, Senior Assessor and National Scientific Advice Coordinator, INFARMED, Portugal

The Challenges of Potency Assay Development of Cell and Gene Therapy ProductValerie Pimpaneau, Voisin Consulting Life Sciences, France

New Developments for GTMPsBastien Calmels, Quality Control Senior Manager, Transgene, France EM0303 | Tuesday, 14 April, 14:00-15:30 | Room 243 Level 2

ADAPTIVE LICENSING FOR ATMPS Session Chair: Hans Ovelgönne, Member SAWP, CAT; Medicines Evaluation Board, the Netherlands

In March 2014 the EMA launched a pilot project into adaptive pathways. The aim was to investigate a better use of real-world data in the licensing of medicinal products, in conjunction with HTAs. Several companies submitted products into this pilot, including a larger than expected proportion of ATMPs. What is special about ATMPs? ATMPs and the EMA Pilot Project on Adaptive PathwaysHans Ovelgönne, Member SAWP, CAT; Medicines Evaluation Board, the Netherlands

How to Meet Post-Marketing Obligations?Maria Pascual, Vice President Regulatory Affairs and Corporate Quality, Tigenix, Spain


ATMP REGULATIONS - ONGOING REVIEWSession Chair:Paula Salmikangas, Chair CAT, Senior Researcher, Finnish Medicines Agency (FIMEA), Finland

With Regulation 1394/2007/EC the legal framework is now built for ATMPs in EU. It has provided certainty and predictability for the development of

ATMPs and set the requirements for their authorisation. However, many aspects of the legislation have been found problematic, as can be seen from the results of the European Commission consultation concerning the ATMP regulation and the subsequent EC report, published in April 1st, 2014. The comments received and conclusions of the EC report call for revision of the legislation, but what should be changed and how?

Need for Revision of the ATMP legislation? Rocio Salvador-Roldán, Policy Officer, European Commission, EU

Bottlenecks for ATMP DevelopmentAlex Bloom, Manager, Global Regulatory Affairs, Biotechnology & Advanced Therapies, Chiesi Farmaceutici, Italy

Boundaries between ATMPs and Transplant/Transfusion Products, Revision of Guidance on ATMP ClassificationMarit Hystad, CAT member, Head of Section, Norwegian Medicines Agency, Norway EM0306 | Wednesday, 15 April, 11:00-12:30 | Room 253 Level 2

ATMPS - CLINICAL TRIAL DESIGN CHALLENGES FOR ATMPS AND MEDICINAL PRODUCTS FOR RARE DISEASESSession Chair: Gopalan Narayanan, Biologics & Advanced Therapies Expert, NDA Group, UK

Many advanced therapy products are being developed to fulfil an unmet medical need, particularly in the area of orphan diseases. This combination of new technology and rarity of the disease(s) concerned can significantly raise the challenges developers face in establishing positive clinical benefit. However, the regulations, recognising these difficulties, offer sufficient flexibility to enable successful development.

Regulatory Requirements for ATMP Clinical TrialsGopalan Narayanan, Biologics & Advanced Therapies Expert, NDA Group, UK

Clinical Study Designs and Possible Challenges for ATMPsEtienne Sokal, Founder and CEO, Promethera Biosciences, Belgium

Statistical ConsiderationsTomasz Burzykowski, Vice President, IDDI, Belgium


CHALLENGES FOR DEVELOPMENT OF RARE DISEASESSession Chair: Peter Bogaert, Advocaat, Convington & Burling LLP, Belgium

The orphan medicines regime in the EU is almost 15 years old and has been a success. There remains, however, a need to develop new medicines and other therapies that target rare diseases. The session will review the challenges for effective development programmes and explore possible routes for improvement.


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Strategic View of a Regulator on Effective Development of Therapies for Rare DiseasesDiederick Slijkerman, Head of Policy, Governance & Regulatory Affairs, Medicines Evaluation Board (MEB), the Netherlands

Registries for Rare DiseasesRengarajan Badri, President, Board of Directors, International Pemphigus and Pemphigoid Foundation, USA

Experiences from Orphan Exclusivity in the EUPeter Bogaert, Advocaat, Convington & Burling LLP, Belgium

Theme 4 Early Dialogue with Regulators and HTA Bodies on Innovative Medicines

Isabelle Clamou, Regulatory Affairs Director – EU Policy, Amgen, BelgiumZaide Frias, Head of Regulatory Affairs and Best Evidence, Head of Human Medicines Research and Development Support (ad interim), European Medicines Agency, EU

If innovative treatments are to reach patients quickly, manufacturers need clarity and consistency in what to expect from regulatory processes and Health Technology Assessments (HTAs). How can these bodies cooperate and coordinate regularly?

This theme will examine the impact of recent activities by regulatory and HTA bodies, and how it will affect approvals of new treatments. What will be the consequences of new approval pathways on the industry, and can we build on these to streamline processes? How the delivery of new initiatives and both European and national level change the regulatory environment?

It will also examine how to create constructive interactions between stakeholders on core issues such as evidence standards, adaptive licensing, lifecycle advice, patient involvement and EU collaboration on HTAs.

EM0402 | Tuesday, 14 April, 11:00-12:30 | Room Havane Level 3

REGULATORY EVIDENCE STANDARDS: ARE WE MAXIMISING VALUE GENERATION?Session Chair:Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency, EU

Good pharmaceutical regulation creates value, poor regulation destroys value. This basic rule goes for public health value as well as for economic value. The current business model of the bio-pharmaceutical industry is predicated on robust regulation. Without regulation, there would be no drug development as we know it, and sponsors would not be able to

claim public health value for their innovative products. Yet, regulation that produces no or only limited net health benefit may stifle innovation, disincentivise investors in R&D, and, most importantly, delay or deny patients access to beneficial treatments. Focussing on diabetes drugs as an illustrative example, this session will address how we can best find a level of evidence standards that maximises both public health and economic value.

Panellists:André Broekmans, Director, Escher (TI Pharma platform for regulatory innovation), the Netherlands

Jean-Luc Harousseau, President, Haute Autorité de Santé (HAS), France

Yann Le Cam, CEO, EURORDIS, France

Tomas Salmonson, Chair CHMP, Senior Scientific Advisor, Medical Products Agency, Sweden


SHAPING EUROPEAN EARLY DIALOGUES: THE SEED PROJECTSession Chair: François Meyer, Advisor to the President, International Affairs, Haute Authorité de Santé (HAS), France

SEED is a project funded by the European Commission involving 14 HTA bodies from 10 countries and coordinated by HAS (France). The main objectives are: 1) the conduct of 10 early dialogues (ED) between companies and HTA bodies on the development of new pharmaceuticals and medical devices and 2) the proposal for a permanent model for ED in Europe. The session will provide an update on the advancement of the project and preliminary results, with discussion on the benefits and potential challenges for the future of this activity.

Early Dialogues Involving Multiple HTA Bodies: Lessons learnt and future challengesFrançois Meyer, Advisor to the President, International Affairs, Haute Authorité de Santé (HAS), France

Early Dialogue and SEED Project: The perspective from a pharmaceutical companyLuk Maes, Executive Director, Regulatory Scientific Policies – Europe, Bristol-Myers Squibb, Belgium

The European Commission PerspectiveJérôme Boehm, Administrator / Policy Officer, DG Health- eHealth, Health Technology Assessment, European Commission, EU


LIFECYCLE OF REGULATORY/HTA ADVICESession Chair: Spiros Vamvakas, Head of Scientific Advice, European Medicines Agency, EU

This session will focus on the experience of the new approaches of interaction among regulators, HTAs and companies during drug development throughout the life-cycle of medicinal products. Furthermore, the session will address new concepts currently under exploration of approaching the lifecycle of medicinal products such as adaptive licensing and post-authorisation efficacy studies.



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State of the EMA Adaptive Pathways Pilot ProjectHans Ovelgönne, Member SAWP, CAT; Medicines Evaluation Board, the NetherlandsEMA-HTA Scientific AdviceJudith Creba, Head EU Liaison and Policy, Novartis, Switzerland

HTA Perspective on Early Engagement in New models of Scientific AdviceLeeza Osipenko, Associate Direct, National Institute for Health and Care Excellence (NICE), UK

EM0405 | Wednesday, 15 April 09:00-10:30 | Room Bordeaux Level 3

PATIENT INVOLVEMENT IN THE DEVELOPMENT DIALOGUE WITH REGULATORY AUTHORITIES AND HTAS Session Chair: Isabelle Stöckert, Head Global Regulatory Affairs Europe/Canada, Bayer Pharma AG, Germany

Patient involvement in innovative drug development and access considerations are hot topics; methodological work is rapidly evolving. The session provides insight into latest research of EMA and IMI PROTECT on patient value elicitation methodologies.

How Can Regulatory Authorities and HTAs Build on Patient Input - The European Medicines Agency perspective Francesco Pignatti, Head of Oncology, Haematology and Diagnostics, European Medicines Agency, EU

The Patient Perspective in Drug DevelopmentFrançois Houyez, Treatment Information and Access Director, Health Policy Advisor, EURORDIS, France

Patient and Public Involvement in Innovative Drug Development and Benefit/Risk Decision Making – What is state of the art after PROTECT WP5?Kimberley Hockley, Research Associate, Team Leader of the Patient and Public Involvement Working Group in WP5 PROTECT), Imperial College, UK

EM0406 | Wednesday, 15 April, 11:00-12:30 | Room Havane Level 3

ONE-STEP APPROACH ON HTA ASSESSMENT, APPRAISAL AND PRICING REIMBURSEMENTSession Chair: Jérôme Boehm, Administrator / Policy Officer, DG Health- eHealth, Health Technology Assessment, European Commission, EU

State of Play EUnetHTASpeaker invited

Industry PerspectiveEdith Frénoy, Director, Market Access/HTA, EFPIA, Belgium

Mapping of Existing SchemeNeil McAuslane, Director, Centre for Innovation in Regulatory Science (CIRS), UK


HTA AND ADAPTIVE PATHWAYSSession Chair: Sarah Garner, Associate Director R&D, NICE, UK

EMA Pilot on Adaptive Pathways: State of play and case studyFrancesca Cerreta, Scientific Administrator, European Medicines Agency, EU

Adaptive Approaches to Licensing HTA and the Use of TechnologySpeaker invited

Please see EuroMeeting Extra for updates


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Theme 5 Collaborative Consortia, IMI and the Way to Horizon 2020

Salah-Dine Chibout, Global Head Discovery & Investigative Safety, Global Head Preclinical Safety Therapeutic Areas, Novartis Pharma, Novartis Institutes for BioMedical Research, SwitzerlandFatiha Sadallah, Principal Scientific Manager, Innovative Medicines Initiative (IMI), Belgium

Evolutions in drug discovery are encouraging research-based companies to be less introspective and explore new approaches to drug development. Increasingly, they seek partnerships with multiple stakeholders, including academia, small and medium-sized enterprises and others, sharing knowledge and diversifying risk.

These collaborations may differ in their structure and approach. However, all are based on the recognition that combining resources will deliver outcomes exceeding those they could achieve individually.

This theme will examine how major stakeholders perceive these collaborations, and will examine the future potential for new public-private partnerships (PPPs).


STRENGTHENING COLLABORATIVE INNOVATION AND MAXIMISING MUTUAL BENEFIT: PHARMACEUTICAL INDUSTRY PERSPECTIVESSession Co-Chairs: Salah-Dine Chibout, Global Head Discovery & Investigative Safety, Global Head Preclinical Safety Therapeutic Areas, Novartis Pharma, Novartis Institutes for BioMedical Research, Switzerland

Will PPPs revive breakthrough innovation at the pharmaceutical industry? Cross-private and public partners’ innovation entails distinctive innovation opportunities and challenges because of the heterogeneity between the collaborating parties. Such opportunities and risks will be discussed using the example of IMI with strong and mature PPP networks in key areas of R&D.

The U-BIOPRED (Unbiased BIOmarkers in PREDiction of Respiratory Disease Outcomes) Severe Asthma Consortium - An Industry PerspectiveChris Compton, VP, Medicines Development Leader, GlaxoSmithKline, UK

Experiences from IMIMatthias Gottwald, Head R&D Policy and Networking, Bayer, Germany

Excellence in RWD: GetReal and its benefit to industryMelvin Olson, Health Economics Manager, Novartis, Switzerland


MINIMISING COSTS, HURDLES, AND DUPLICATION: HOW REGULATORS, PAYERS AND POLICY MAKERS MAY PROFIT FROM PPPSSession Chair: Hans-Georg Eichler, Senior Medical Officer, European Medicines Agency, EU

Though PPPs might create a powerful mechanism for addressing difficult problems by leveraging on the strengths of different partners, they also package complex ethical and process-related challenges. Participation of regulators, payers and policy makers warrants that topics addressed by PPPs will be set within a comprehensive policy and operational framework. The session will highlight interest in and potential legal/ethical conflicts with PPPs of regulators, payers and policy makers.

Policy Maker Perspectives from the IMI GetReal ProjectSarah Garner, Associate Director R&D, NICE, UK

Collaborative Learning from the Regulatory System of Medicines: Recent results from the Escher ProjectAndré Broekmans, Director, Escher (TI Pharma platform for regulatory innovation), the Netherlands

PPP: Beyond Publication - From scientific collaboration to regulatory and medical practice and new business modelsMagda Chlebus, Director, Team Leader, EFPIA, Belgium


IN BETWEEN: SMEs PERSPECTIVE ON PPPsSession Chair: Ruxandra Draghia-Akli, Director, Directorate Health, Directorate-General for Research, European Commission

‘If I have seen further it is by standing on the shoulders of giants’ (Isaac Newton, letter to Robert Hooke 15 Feb 1676). The relevance of PPPs for the economic viability of SMEs will be discussed in this session.

SMEs as Success Factors for PPPs and Technology Transfer on the Ex-ample of the European Lead Factory Dimitrios Tzalis, Founder and CEO, Taros Chemicals, Germany

SMEs in IMI Projects: A Management & Governance Perspective - Ex-pected positive outcomes for consortia and SMEs (SAFE-T, MIP-DILI, DDMoRe) Nicolas Creff, Project Officer, Interface Europe, Belgium



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DEVELOPING BEST PRACTICE GUIDANCE ON PATIENT ENGAGEMENT IN MEDICINES R&D Session Chair: David Haerry, EUPATI Co-Leader on Sustainability (WP7), European AIDS Treatment Group, Switzerland

EUPATI IMI is developing best practice guidance on patient engagement with all relevant stakeholders (industry, ethics committees, health technology assessment bodies and regulatory agencies). This session aims to inform about the current status of the deliverable and to collect further feedback from the audience.

Short introductory statements and Q&A on the present status of patient involvement in the context of:• Industry-led R&D• Ethics Committees• HTA

Patient Involvement in Industry-Led R&DMatthias Gottwald, Head R&D Policy and Networking, Bayer Pharma AG, Germany

Patient Involvement in HTAKaren Facey, Patient Involvement Methods and Impact Working Group Coordinator, Health Technology Assessment International, UK

Patient Involvement in Ethics CommitteesAndrea Heckenberg, Senior Staff Member, Ethics Committee Office, Vienna Medical University, Austria

Patient Involvement in Regulatory AgenciesIsabelle Moulon, Head of Patients and Healthcare Professionals, European Medicines Agency (EMA), EU

EM0506 | Wednesday, 15 April 11-12:30 | Room 243 Level 2

COLLABORATIVE INNOVATION IN BIOMEDICINE SERVING INDUSTRY, ACADEMY AND SOCIETYSession Chair: Julio Celis, Professor and Associate Scientific Director, Danish Cancer Society Research Center, Denmark

With the international recognition of the importance of innovation, technology transfer, and entrepreneurship for sustained economic revival and the decline of industrial research laboratories, research universities often play a central role in collaborative discoveries, innovations, and inventions. This session will discuss how the innovation ecologies at and around universities are meeting this societal challenge.

Cancer Core Europe: Sustainable Capacity Building to Address the Cancer Care-Research ContinuumAlexander Eggermont, Director General, Gustave Roussy, France

IMI - driven advances in diabetesBeatriz Silva Lima, IMI SC Chair, University of Lisbon, Portugal

Pre-Competitive Gains from Data Sharing across Sponsors: The power of big data and collaborationJonathan Rabinowitz, Bar-Ilan University, Israel


A PARTICIPATORY SOCIETY FOR A BETTER HEALTH: PATIENT, CITIZEN, AND CONSUMERS VOICE IN PPPSSession Chair: Jan Geissler, Director, EUPATI, Belgium

The medical landscape is changing at a very fast pace. Particularly in areas with strong unmet medical needs, new collaboration models between all key healthcare players are essential to address these. In order to create better health, researchers, industry, policy makers and regulators increasingly understand the need to involve patients and citizens to make research and healthcare provisioning more effective. This session will provide an overview of the level of patient involvement across Public-Private Partnerships in the Innovative Medicines Initiative, and will provide practical examples of projects where patient involvement has become reality.

Empowering Patients in Medicines R&D for Better Health: The EUPATI IMI projectNicola Bedlington, Executive Director, European Patient's Forum, Belgium

Patient Involvement in IMI Public-Private Partnership: Strategy and current statusMadga Gunn, Scientific Project Manager, IMI, Belgium

"How it Works" - 18 years of experience by the European Community Advisory Board (ECAB)David Haerry, European AIDS Treatment Group, Switzerland


PANEL DISCUSSION WITH PUBLIC-PRIVATE PARTNERSHIP STAKEHOLDERSSession Chair: Fatiha Sadallah, Principal Scientific Manager, Innovative Medicines Initiative (IMI), Belgium

PPPs – a Trojan Horse of neoliberal development or an innovation opportunity for all stakeholders? In this session we invite to a lively panel discussion with all stakeholders to appreciate the mutual benefits and drawbacks of PPPs.

Matthias Gottwald, Head R&D Policy and Networking, Bayer, Germany

Jan Geissler, Director, EUPATI, Belgium

Ruxandra Draghia-Akli, Director, Directorate Health, Directorate-General for Research, European Commission, EU

Magda Chlebus, Director, Team Leader, EFPIA, Belgium

Isabelle Moulon, Head of Patients and Healthcare Professionals, European Medicines Agency (EMA), EU


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Theme 6 Implementing Innovation – Globally Conducive Regulatory Policies

Jun Kitahara, International Liaison Officer stationed in Swissmedic, Pharmaceuticals and Medical Devices Agency (PMDA), JapanHenrik Kim Nielsen, Corporate Vice President, Regulatory Affairs GLP-1 and Obesity, Novo Nordisk, Denmark

This theme examines developments in harmonising rules internationally and how they can stimulate innovation by simplifying global requirements. Do the concepts of regulatory ‘convergence’, ‘harmonisation’ and ‘co-operation’ represent the reality? Is there a role for the ICH and other harmonisation initiatives? What are the developments in regulatory harmonisation in major Asian markets such as Japan and China? It will also explore how increased harmonisation can reduce costs and complexities in gaining approvals, speeding the benefits of innovation to the patient globally.

EM0601 | Tuesday, 14 April, 09:00-10:30 | Room Maillot Level 2

ICH, IPRF, ICMRA: HOW ARE THESE INITIATIVES ADVANCING REGULATORY HARMONISATION AND CONVERGENCE? Session Chair: Petra Dörr, Head of Communication and Networking, Deputy Director, Swissmedic, Switzerland

ICH, IPRF, ICMRA: How do all these initiatives “play” together? Can duplication of efforts be avoided? What are the accomplishments so far with regard to advancing regulatory harmonisation and convergence?

Reform of ICHLenita Lindström Gommers, EU Member ICH Steering Committee, Senior Policy Officer, Unit 5D, European Commission, EU

A New Initiative among Regulators - The International Coalition of Medicines Regulatory Authorities (ICMRA)Nobumasa Nakashima, International Planning Director, for Pharmaceutical Affairs, Minister’s Secretariat, Ministry of Health, Labour and Welfare

Industry View on Different Regulatory Harmonisation InitiativesPär Tellner, EFPIA ICH Coordinator, Director, Team Leader, Regulatory Affairs, EFPIA, Belgium


ASIA: CREATING POSSIBILITIES FOR CLINICAL DEVELOPMENT AND COLLABORATIONSession Chair: Toshiyoshi Tominaga, Associate Executive Director for International Programs, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Asia is increasing its responsibility in worldwide pharmaceutical market, because it is expanding its presence not only as a key market, but also as a pharmaceutical manufacturing hub. Therefore, many Asian regulators make strong efforts to accelerate the dealing with urgent issues such as capacity building for their product review and safety monitoring, development of guidelines for development of pharmaceuticals, etc.

This session contains the current situation of regulatory environment as well as the recent achievements and challenges of Asian regulators, by focusing on multi-regional clinical trials in Asia, followed by Q&A with the audience to offer the opportunity to interact directly with Asian representatives to consider how to facilitate regulatory development of innovative products.

Regulatory Perspective to Review MRCT Data for Drug Approval: Focusing on ethnic factor evaluation Yoshiaki Uyama, Director, Division of Epidemiology, Office of Safety I, Pharmaceuticals and Medical Devices Agency, Japan

Efforts of MFDS for Facilitating Access to Medicines Ho-Jeong Kim, Drug Review Management Division, Drug Evaluation Department, National Institute of Food and Drug Safety Evaluation, MFDS, Korea

Regional Heterogeneity in Multi-Regional Clinical Trials - Experiences with regional health authoritiesJane Xu, Vice President, Data Science, Sunovion Pharmaceuticals, USA


CHINESE REGULATORY SESSION: UPDATE FROM THE CFDASession Chair: Ning Li, Vice President Regulatory and Medical Policy, Asia Region, Sanofi, China

Updates from two reports will be presented by the officials of Drug & Cosmetics Registration Dept. and the Center for Drug Evaluation of the China and Food Administration (CFDA). The progress of the ongoing Reform of Drug Review and Approval System and the encouragement of innovation, as well as the 2014 annual report of the drug technical review will be discussed.

CFDA’s Perspective on Clinical Trial Review for Drug Registration: Update from CDE/CFDAWang Tao, Division Director, Center for Drug Evaluation, China Food and Drug Administration, China

Issues and General Considerations of IND/CTA Applications in ChinaWang Haixue, Chief Pharmacist, Center for Drug Evaluation, China Food and Drug Administration, China



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CHINA: OPPORTUNITIES AND CHALLENGES FOR INNOVATIONSession Chair: Joseph Scheeren, Head of Global Regulatory Affairs Pharma and Consumer Care, Bayer Consumer Healthcare, Switzerland

China has very complicated and dynamic regulatory policies in driving the development of innovative new drugs. The session will elaborate on these key regulatory policies and share experience on the regulatory aspirations, barriers and opportunities that will impact global drug development and availability of new medicines in China. How to optimize implementing innovations within such an environment requires strategic considerations, which this session aims to enlighten.

Understanding Regulatory Laws and Policies in ChinaShaoyu Chen, Partner, Managing Director, Covington & Burling LLP, China

Quantifying the Chinese Regulatory Environment: Challenges and OpportunitiesNeil McAuslane, Director, Centre for Innovation in Regulatory Science (CIRS), UK

An Overview of the Drug Development Landscape in ChinaAmar Kureishi, Vice President and Regional Chief Medical Officer, Head of Drug Development Asia, Quintiles, Singapore EM0605 | Wednesday, 15 April, 09:00-10:30 | Room Maillot Level 2

PMDA UPDATES: DISCUSSION OF LATEST PLANS AND NEW DIRECTIONS IN REGULATION – TOWN HALL Session Chair: Toshiyoshi Tominaga, Associate Executive Director for Office of International Programs, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

In spring 2014, the Pharmaceuticals and Medical Devices Agency (PMDA) developed the Third Mid-term Plan based on the results of the Second Mid-term Plan, in which we achieved the shortened review time of new drug approval, established a risk management plan, shortening the review period of relief service, and established the structures for the promotion of regulatory sciences. In the new plan, we will accelerate drug development, especially for innovative ones, by improving the scientific quality of review, consultation, and post- marketing safety measure through the regulatory sciences such as the science board, advanced review/consultation system, and other approaches.

This session contains the report for recent and future challenges of PMDA, followed by Q&A with the audience to offer the opportunity to interact directly with PMDA senior level representatives regarding almost all services rendered by PMDA, including international programmes.

New Regulation in Japan and Future Direction of PMDATaisuke Hojo, Senior Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

Science-Based Initiatives of PMDATetsuo Nagano, Executive Director, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

New Streams of Risk ManagementTomiko Tawaragi, Chief Safety Officer, Pharmaceuticals and Medical Devices Agency (PMDA), Japan

EM0607 | Wednesday, 15 April, 14:00-15:30 | Room Maillot Level 2

FROM THE SHOP FLOOR: PRACTICAL ADVICE ON BRINGING NEW MEDICINES TO PATIENTSSession Chair: Henrik Kim Nielsen, Corporate Vice President, Regulatory Affairs GLP-1 and Obesity, Novo Nordisk, Denmark

Those working in industry present proposals from their daily experience for improving and expediting safe and effective medicines to patients globally. Strategies are presented for effective submission management processes for global dossiers, in particular for emerging markets; possibilities for trading molecules that have been shelved during development; and creation of a Regulatory Compliance Matrix to simplify GMP submissions.

Submission Project Management Strategy: Coordinating Simultaneous Global Submissions & Re-using Content for Future Emerging Markets Submissions with a Focus on Latin America Sophia Kourliouros, Senior Manager, Regulatory Submissions, Esai, USA

Collaborate to Innovate: Strategic alternatives towards developing a seconds marketDoris Pereira, Assistant Manager, International Operations Department, Torrent Pharmaceuticals, India

How to Meet Regulatory Challenges when Sourcing Biologicals from Multiple SitesSpeaker invited


EMERGING MARKETS UPDATE: WHAT ON EARTH IS GOING ON WITH REGULATOR CONVERGENCE?Session Chair: David Verbraska, Vice President, Worldwide Public Affairs & Policy, Pfizer, USA

Identify successes and challenges in the rapidly evolving area of regulatory harmonisation, particularly in the emerging markets. Understand the role of WHO, public-private partnerships and the bio-pharmaceutical companies, current initiatives and challenges.

Jaime Oliveira, Director Chairman, ANVISA, Brazil

Durhane Wong-Rieger, President and Chief Executive Officer, Canadian Organization for Rare Disorders (CORD), Canada

Successes and Challenges of Regulatory Convergence: WHO’s viewpoint Lembit Rägo, Head, Regulation of Medicines and other Health Technologies, World Health Organization (WHO), Switzerland


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Theme 7 Special Development Pathways in Paediatrics, the Elderly and in Pregnancy

Sabine Atzor, Head of EU Regulatory Policies, F. Hoffmann-La Roche, SwitzerlandJordi Llinares Garcia, Head of Product Development Scientific Support, European Medicines Agency, EU

Children, pregnant women and the elderly have pose specific treatment challenges. This is a highly charged and emotional interface between science and society. Meeting these requires a dedicated approach to drug development. This theme examines how we can leverage scientific developments and define specific development pathways to address these needs.

We will consider what we have learned since the Paediatric Regulation was introduced ten years ago. How do we leverage the advances in science since then to make further improvements?

We will also examine the health demands of an ageing population, where new treatments are struggling to keep pace with Europe’s demographic change. How can we bridge to their unmet needs more quickly?

Medicine use during pregnancy remains a scientific, ethical and regulatory challenge for researchers. How do we address this specific need, and can broader, multi-stakeholder approaches make a difference?


WHAT WILL THE FUTURE BRING – INNOVATIVE PAEDIATRIC DEVELOPMENTSession Chair: Nathalie Schultze, Regulatory Affairs Team Leader, F. Hoffmann-La Roche, Switzerland

Despite obvious phenotypic differences, increasing evidence demonstrates that adult and paediatric diseases may share similar molecular determinants of disease occurrence and progression. Pursuing a disease-centred, rather than drug-centred approach, allows a change in emphasis from adult indications with paediatric counterparts to diseases in children with greatest unmet need based on the mechanism-of-action and therapeutic target of a molecule. While the current era of targeted therapies and personalised medicine has created the opportunity to change the paradigm of paediatric drug development, clinical trials in rare paediatric conditions remains a major challenge. Accurate projection of recruitment rates and successful accrual of patients in paediatric trials is often difficult due to the limited number of cases, the absence of historical data of paediatric patients participating in clinical trials, and logistical challenges

of conducting a global clinical trial in rare paediatric conditions. Leveraging academic/industry partnerships and clinical trial networks are an efficient way to share scientific and clinical information across geographic regions, conduct comprehensive feasibilities to inform initial enrolment projections, and provide ongoing feasibility to mitigate clinical trial conduct challenges.

New Mechanisms of Action - Changing the paradigm in paediatric drug development

What are Innovative Trends? An industry perspectiveNathalie Schultze, Regulatory Affairs Team Leader, F. Hoffmann-La Roche, Switzerland

Regulators’ Approach to Enable InnovationKoenraad Norga, Head of Clinic Paediatric Oncology, PDCO member, Belgium

Clinical Trial Networks as Enablers to Overcome Challenges with Paediatric Clinical TrialsIrmgard Eichler, Professor of Paediatrics, Scientific Administrator, European Medicines Agency, EU

Panel Discussion: What navigation is needed by industry and regulators to ensure children’s early access to innovative medicines?


HOW WILL THE GLOBAL NEEDS OF PAEDIATRIC DEVELOPMENT BE ADDRESSED?Session Chair: Dirk Mentzer, Chair of PDCO; Head of Pharmacovigilance Unit, Paul-Erhlich-Institut (PEI), Germany

Eight years after the implementation of the Regulation EU 1901/2006 = Paediatric Regulation, this piece of legislation has drastically changed the way the Pharmaceutical Industry is considering the development of new candidate medicinal products for children. Pharmaceutical industry, investigators and patients are aware that newly developed medicinal products are formulated and tested in children and it is expected to increase in the coming years.Of course the needs of the paediatric population is not restricted to the Europe. In addition, a considerable number of medicinal products are developed and manufactured by multi regional acting pharmaceutical industry. Therefore, it is important to harmonise the paediatric study program in each region to limit the number of paediatric clinical trial needed to bring the medicinal product to the market. At the same time guidance documents should facilitate the framing of medicinal product development across the regions.In this respect, Europe, US and Japan and FDA have commenced activities for updating the ICH guideline E11 with the support of all regions involved. This is to support the equal opportunities for child health in all regions.

The Next Era of ICH Paediatric GuidelinesDirk Mentzer, Chair of PDCO; Head of Pharmacovigilance Unit, Paul-Erhlich-Institut (PEI), Germany



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Optimising EMA Advice on Development and Putting the Patient FirstFrancesca Cerreta, Scientific Administrator, European Medicines Agency (EMA), EU

How Does Industry Address the Needs?Susanna Del Signore, Associate Vice-President Global Regulatory Affairs, Head of Global Regulatory Policy, Sanofi R&D, France

Are Patients Convinced their Expectations are Met?Barbro Westerholm, AGE Platform Europe, Belgium


HOW TO OVERCOME CHALLENGES IN THE DEVELOPMENT OF MEDI-CINES TO TREAT DEMENTIASession Chair: Karl Broich, President, BfArM, Germany

Can We Overcome Development with Set-Backs? Outcome of EMA Alzheimer WorkshopKarl Broich, President, BfArM, Germany

Considering Bioethical Aspects in DevelopmentXavier Carne, Head of Clinical Pharmacology Department, Hospital Clinic, University of Barcelona School of Medicine, Spain

Industry’ s Learning Curve - answering to challenges Luc Truyen, Vice President, Clinical Research & Development, Johnson & Johnson, USA

Panel discussion


50 YEARS AFTER THALIDOMIDE – DEVELOPING DRUGS IN PREGNANCYSession Chair: Jordi Llinares Garcia, Head of Product Development Scientific Support, European Medicines Agency, EU

Despite the urgent need, the development of products for use in pregnant women is one of the most challenging situations clinicians, patients, developers and regulators can encounter. Nevertheless the daily reality is that in most cases pregnant women have to be treated without a clear understanding of the effects on the drugs on the woman and the fetus. The session will offer the opportunity to discuss the needs for regulatory and development progress in this area and an overview of current initiatives.

Defining the Boundaries of the Current Regulatory FrameworkJordi Llinares Garcia, Head of Product Development Scientific Support, European Medicines Agency, EU

How Does Industry Translate Experiences into a Way Forward?Lode Dewulf, Vice President, Chief Patient Affairs Officer at UCB, Belgium

Clinical, Ethical and Socioeconomic Considerations – An analysis in epilepsy, rheumatoid arthritis and schizophreniaVictoria Tzouma, Market Access Manager, MSD, Greece

How is Convergence on Timing between EU-US Possible?Irmgard Eichler, Professor of Paediatrics, Scientific Administrator, European Medicines Agency, EU

Which Other Areas Would Benefit from Convergence – how successful have we been to date? Jane Møll Pedersen, Global Regulatory Director, Novo Nordisk, Denmark

Panel discussion with: Sabine Fuerst-Recktenwald, Senior Medical Director Pediatric Oncology, F. Hoffmann-La Roche, Switzerland - Solange Rohou, Director Global Regulatory Affairs, AstraZeneca, France - Stephen Spielberg Editor-in-Chief, Therapeutic Innovation & Regulatory Science, DIA, USA


MODELLING AND SIMULATION IN PAEDIATRICS: THE NEXT STEP- KEEPING THE MOMENTUMSession Chair: Solange Rohou, Director, Global Regulatory Affairs, AstraZeneca, France

The 2011 EFPIA/EMA workshop was a unique event bringing together scientists from academia, industry and regulatory agencies from Europe and beyond to take an in depth look at the role of Modelling & Simulation (M&S) in drug development. Since then, both regulators and industry representatives have kept the momentum. This session will be the opportunity to understand the status of this discussion, the work done so far, and share some learnings. Case examples will be also presented for discussion to show how extrapolation/M&S can be used to support drug development in children and labelling claim.

The EMA Modelling and Simulation Working Group – A strategic enabler to further support PIP developmentEfthymios Manolis, Scientific Officer, European Medicines Agency, EU

Good Practices in Model Informed Drug Discovery & Development (MID3) Marylore Chenel, Director of Clinical PK and PMx, Institut de Recherches Internationales Servier, France (on behalf of the EFPIA M&S group)

From Theory to Practice - Case examplesAmy Cheung, Senior Clinical Pharmacometrician, AstraZeneca, UK (on behalf of the EFPIA M&S group)

Panel discussion


THE SILVER GENERATION - HOW DOES DEVELOPMENT ADDRESS THEIR NEEDS?Session Chair: Francesca Cerreta, Scientific Administrator, European Medicines Agency (EMA), EU

The European population is rapidly aging, with over 80s being the fastest growing population group. This session will provide an update on the EMA latest regulatory activities with respect to the assessment of the Benefit/Risk balance in the older population, including the progress on the Frailty Points to Consider and the guideline on packaging and formulations, together with the perspective of industry and patients on addressing the needs of the older population.


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Theme 8 Translational Medicine and Regulatory Science

Anna Cieślik, Director Department of Documentation Assessment, Office for Registration of Medicinal Products, PolandDetlef Niese, Consultant, Dr. Niese Health Science and Policy, Germany

Regulatory science is essential in bringing the benefits of therapeutic innovation to patients. It systematically analyses existing regulatory frameworks, ensuring they evolve to keep pace with scientific advances. This provides a dual benefit of promoting innovation whilst protecting public health.

However, regulatory science faces a new challenge. An understanding of how diseases operate a molecular or cellular level is replacing our traditional disease definition based on phenotypes and symptom descriptions.

This theme explores how existing regulations will need to adapt to recognise these new definitions. It also examines the consequences of these new approaches, particularly in clinical trials and the role of clinical end points versus molecular end points. How will regulatory science help drive this change? We harness the views of scientists, regulators and patients in looking at the implications, implementation and likely benefits of this change.


THE ROLE OF TRANSLATIONAL MEDICINE IN THE FUTURE OF LIFE SCIENCESession Chair: Dariusz Śladowski, Associated Professor, Medical University Warsaw, Committee for Advanced Therapies (CAT) Representative, Poland

Translational medicine is a discipline bridging biomedical and public health research to improve and speed up the process of practical implementation of novel scientific achievements. The session will bring together the issues of “knowledge network” which seems crucial for the better use of clinical trials data which are about to be released by the EMA, the prospects of innovative biomarkers and a problem of new technologies assessment from the point of view of a regulator. It will focus on the future of university, enterprise and regulatory scientists integration, in the translation process of a new ideas into practical medicine.

Precision Medicine and the Evolving Role of Clinical Research and DevelopmentClaudio Garutti, Senior Consultant, Oracle Health Sciences, the Netherlands

Precision Medicine and the Evolving Role of Clinical Research and DevelopmentJoel Haspel, Senior Marketing Strategist, Oracle, UK

Diagnostic and Pharmacodynamic Biomarkers and Mass Spectrometry Assays Thereof: Current status and perspectivesHarald Mischak, Institute of Cardiovascular and Medical Sciences, University of Glasgow, UK

CAT View of the Role of Translational Medicine in the Future of Life ScienceDariusz Śladowski, Associated Professor, Medical University Warsaw, Committee for Advanced Therapies (CAT) Representative, Poland


IS THE CLASSIFICATION OF DISEASE ON PAR WITH THERAPEUTIC INNOVATION?Session Chair: Detlef Niese, Consultant, Dr. Niese Health Science and Policy, Germany

This session will address the role of ATC/DDD system for drug utilisation research to improve the harmonisation of regulatory decisions.

A Mechanism-Based Disease Taxonomy: Need or hype?Detlef Niese, Consultant, Dr. Niese Health Science and Policy, Germany

A New Disease Taxonony: A view from academiaRolf Bass, University of Basel, Switzerland


THE RISK-BASED APPROACH FOR NEW MEDICINES DEVELOPMENT: FROM ATMPS TO "CONVENTIONAL" DRUGSSession Chair: Beatriz Silva Lima, Professor, University of Lisbon, Portugal

The risk-based approach (RBA) for the safety assessment of ATMPs consists on product-based risk anticipation followed by confirmation, rather than risk observation followed by mechanistic understanding. Will predictive science allow using the RBA to study conventional drugs safety?

Quality AspectsTobias Ostler, Senior Manager Scientific Affairs Biopharmaceuticals/Immunologist, Dr. Regenold GmbH, Germany

Clinical AspectsAndrea Laslop, Head of Scienific Office, AGES, Austria

Non-Clinical AspectsBeatriz Silva Lima, Professor, University of Lisbon, Portugal



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TRANSLATING REGULATORY APPROVAL INTO HEALTHCARE UPTAKE: THE NEXT CHALLENGE FOR BIOSIMILARSSession Chair: Virginia Acha, Executive Director – Research, Medical and Innovation, Association of the British Pharmaceutical Industry, UK

This session will explore the gap that now remains is the translation from regulatory assessment and approval to incorporation of biosimilars within our healthcare systems in Europe. Presenters will offer new insights on how to bridge this gap.

Mind the Gap: Setting out the translational challenges for biosimilars in healthcare settingsVirginia Acha, Executive Director – Research, Medical and Innovation, Association of the British Pharmaceutical Industry, UK

Translating from Approval to Uptake – The experiences of the first biosimilar mAb in EuropePaul K. Audhya, Vice President, Medical Affairs EMEA, Hospira, UK

Bridging the Gap for Hospital PharmacistArnold Vulto, Hospital pharmacist, University Hospital Rotterdam, the Netherlands

Bridging the Gap for Hospital PharmacistRichard Sullivan, Director of the Institute of Cancer Policy and KHP Integrated Cancer Centre Global Health Work, Kings College London

Bridging the Gap for PatientsAlison Lightbourne, Policy Manager, International Alliance of Patients (IAPO), UK

Incorporating the Patient Perspective into Clinical Decision Making in Type 2 DiabetesMatthew Reaney, Senior Scientist, ERT, UK


DIAGNOSTIC AND PHARMACODYNAMIC BIOMARKERS AND MASS SPECTROMETRY ASSAYS THEREOF: CURRENT STATUS AND PERSPECTIVESSession Chair: Pierre Marquet, Professor of Clinical Pharmacology, University of Limoges, France

Proteomics, peptidomics and metabolomics are considered to be sources of potential biomarkers of disease or drug response in many clinical areas, including organ transplantation and such biomarkers are already well advanced towards clinical practice. In this session we will present examples of protein, lipid and metabolite biomarkers, together with the mass spectrometry assays involved; the regulatory paths for approval of biomarkers, as well as mass spectrometry assays in Europe.

Protein and Metabolite Biomarkers of Graft Injuries in Renal TransplantationPierre Marquet, Professor of Clinical Pharmacology, University of Limoges, France

Mass Spectrometry as a Routine Clinical Diagnostic TechniqueOlof Beck, Adjunct Professor, Division of Clinical Pharmacology, Department of Laboratory Medicine, Karolinska Institute, Sweden

Regulatory Issues in Clinical Biomarkers and Mass Spectrometry Assays in EuropeHarald Mischak, Institute of Cardiovascular and Medical Sciences, University of Glasgow, UK


CLASSIFICATION OF MEDICINAL PRODUCTS - FACTORS AFFECTING REGULATORS DECISIONS, IS HARMONISATION POSSIBLE AND NECESSARY?Session Chair: Hubertus Cranz, Director General, AESGP, Belgium

The session will look into the change of classification status from prescription to non-prescription by paying particular attention to the challenges for a successful application in the centralised procedure. Recent experiences will be reviewed and proposals for future improvements will be discussed.

Moving Emergency Contraception to Non-Prescription StatusHelen Guillard, Director, Self-Care Medicine, HRA Pharma, France

How to Make the Centralised System Work Better for Non-Prescription MedicinesMark Griffiths, Senior Director, Pfizer Consumer Healthcare, UK

Panel Discussion with all speakers, Viola Macolić Šarinić, Head of Agency, Agency for Medicinal Products and Medical Devices (HALMED), Croatia, and Tomas Salmonson, Chair CHMP, Senior Scientific Advisor, Medical Products Agency, Sweden


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Theme 9 Big Data, Mobile Health

Luca Pani, Director General, Italian Medicines Agency (AIFA), ItalyDuane Schulthess, Managing Director, Vital Tranformation, Belgium

Harnessing Big Data to make better, quicker medical decisions is a logical development. However, this demands a delicate balance between individual patient rights and the wider public good. This theme explores how to reconcile these issues to maximise the benefits offered by harnessing available health data. How can Big Data improve patient outcomes and accelerate clinical development? What are the implications of data ownership, and what will be the role of social media in healthcare?

EM0901 | Tuesday, 14 April | 09:00-10:30 | Room 242 Level 2

eHEALTH: PUTTING INFORMATION TO WORKSession Chair:Jillian Oderkirk, Senior Analyst, Health Division, Organisation for Economic Cooperation and Development (OECD), France

There is a wide agreement that system wide Electronic Health Records and Electronic Heath Management systems have the potential to fundamentally change the way we currently evaluate new therapies and manage treatment pathways. However, many institutions and governments are leery of proceeding, aware of the many failed and expensive EHR system implementations that have gone wrong and not delivered their expected results. This session will feature and demonstrate several successful 'best in breed' eHealth platforms that focus on divergent parts of the healthcare value chain to harness medical data for improved decision making, efficiency gains, and better patient outcomes. A Road Map for New Care Models in the 21st CenturyBrian Rothman, Medical Director Informatics, Vanderbilt University Medical Center, USA Using National Health Records to Speed DevelopmentTim Williams, Head of Research, Clinical Practice Research Datalink (CPRD), MHRA, UK

eHealth UKGeorgina Evans, Industry Manager, Farr Institute of Health Informatics Research, UK


REGULATORY SCIENCE- NEW PARTNERSHIPSSession Chair: Per Spindler, Director, Biopeople, University of Copenhagen, Denmark

Regulatory Science in relation to medicines development has been a field of growing interest for regulators and the pharmaceutical industry for a number of years. The shaping and implementation of legislation and guidelines are recognised as having a significant impact on public health as well as R&D of medicinal products. Current concepts of benefit/risk assessment, patient involvement and the regulatory pathways are being challenged and new ideas are being launched by the pharmaceutical industry, patient organisations and regulatory authorities. Universities are increasingly contributing with research projects, educational projects, and collaborations to make regulatory science a research-driven contribute to regulatory decision-making. The universities possess expertise not only within the traditional disciplines of medicines research and development but also with other disciplines such as law and pharmacoeconomics. New approaches from universities might indeed open doors for new perspectives to be explored and new partnerships to be formed. Examples are plenty such as transparency, patient involvement, definition of unmet medical needs, methodologies in clinical research, licensing and health economics. This session will show that research and educational initiatives in universities offer a plethora of opportunities for new cross-sectorial partnerships to be created – and it will headlight the important role of academia in setting the trends.

Patients’ PerspectiveNicola Bedlington, Executive Director, European Patient's Forum, Belgium

Industry PerspectiveMerete Schmiegelow, Novo Nordisk A/S, Denmark

Academic PerspectiveKarin Friis Bach, Research Assistant, Faculty of Health and Medical Sciences, University of Copenhagen, Denmark

Authority PerspectiveHans-Georg Eichler, Senior Medical Officer, European Medicines Agency, EU

Panel discussion with all speakers and moderators Karin Friis Bach and Per Spindler



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GOING MOBILE: DISRUPTIVE TECHNOLOGY AT YOUR FINGERTIPSSession Chair:Sten Stovall, The Pink Sheet, European Bureau Chief, Informa Business Intelligence, UK

Bakul Patel, who heads the FDA’s initiatives on Mobile Health (mHealth) strategy, describes their potential as "taking care of patients 100% of the time”. While many governments and healthcare systems are focused on large-scale national and regional health IT projects, mHeatlh is a catch-as-catch-can of Angry Birds disruptive innovation that can fundamentally change the way manage patient care and outcomes. This session will investigate how companies, governments and industry are addressing the challenges and opportunities of mHealth, and are integrating it into strategy and policy.

mHealth for Patient Recruitment in Global Clinical TrialsMatthew Stumm, Corporate Promotion and Lead Generation, BBK Worldwide, USA Integrating mHealth Into National Healthcare Decision MakingJohn Eaglesham, CEO, Advanced, Digital Health Institute, UK Connected, Continuous & Coordinated: How mobile delivers connected therapies that improve outcomes & powers business modelsGene Dantsker, Director of Business Development, Qualcomm Life, USA


BIG DATA: MOVING BEYOND BUZZ WORDS TO RESULTSSession Chair:Luca Pani, Director General, Italian Medicines Agency (AIFA)

Big Data world is invading the field of life sciences. Research organisations, pharma companies as well as policy makers should be prepared to manage this enormous amount of information and all the knowledge changes that will be derived from it at a global level. This approach can lead to a better and more accurate definition of several diseases and to the development of monitoring activities and targeted responses.

"From the world to a water pump”: this is the story of John Snow who mapped all the cases of typhus fever in the 19th century using the London city map. He could find that the center of the epidemic was indeed in a water pump and was able to stop it. Since then Big Data analysis became more sophisticated and as people are sharing more and more information future scenarios are even now hard to imagine...

Simplifying Clinical Research and Cost-Effective Trial in the Era of Big DataCandida Fratazzi, President, Boston Biotech Clinical Research, USA Metadata Management at the Core of “Big Data Management”Isabelle de Zegher, Worldwide Director, Clinical Data Standards, PAREXEL informatics, Belgium

Big Data Analytics Revolutionising Real Life Evidence Journey across the Pharma Value Chain - challenges and opportunitiesDinesh Singh, Senior Manager- Business Consulting, Cognizant Technology Solutions, UK EM0906 | Wednesday, 15 April, 11:00-12:30 | Room 241 Level 2

SOCIAL MEDIA, PATIENTS AND PUBLIC POLICYSession Chair:Alastair Kent, Director General, Genetic Alliance UK

As information becomes increasingly democratised, the barriers between confidential data and public information are becoming increasingly blurred. There are many examples of patients sharing information on clinical research, promising new therapies, and locations of trials, but where does this cross a line into impacting the impartiality of the research and regulatory process? How can policy makers, patients, practitioners and researchers harness social media tools for better stakeholder engagement, while not impacting the need for some health information to remain confidential as part of the research process? Using Social Media Tools to Change the Nature of ResearchIfty Ahmed, Founder and CEO, Pow Health, UK The EPF's View of Social MediaNicola Bedlington, Executive Director, European Patient's Forum, Belgium

Perspectives on Stakeholder Engagement in Regulatory Intelligence ActivitiesCarolyn Hynes, Head of Global Regulatory Intelligence, GlaxoSmithKline, UK


WHO OWNS MY HEALTH DATA? Session Chair:Duane Schulthess, Managing Director, Vital Tranformation, Belgium

Who has the right to use patient data, and who gets to decide how it's used? The European Parliament in March of 2014 overwhelmingly voted for a data protection policy that could limit the use of existing health databases without an opt-in by patients, but the US has an approach to data that allows institutions to harness their captured medical information as long as that usage falls within established confidentiality guidelines. What are the implications if health data is not allowed to be harnessed as easily in Europe as it is in the US and increasingly, Asia? What is the correct path forward to make sure data is harnessed to provide the best patient outcomes, but confidentiality is preserved?

This session will include four 15-minute presentations on patient data and an open discussion of the impact of data usage and policy


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Harnessing Patient Data to Develop New TherapiesJohn Parkinson, National Institute for Cardiovascular Outcomes Research, UK

Who Owns My Data? Data Protection When Faced with Terminal IllnessBettina Ryll, Founder, Melanoma Patient Network Europe, Uppsala University, Sweden EU Health Data and ResearchNathalie Kayadjanian, Senior Scientific Officer, Science Europe

Balancing the Need for Data Protection and Securing the Public GoodRuxandra Draghia-Akli, Director, Directorate Health, Directorate-General for Research, European Commission


REAL WORLD EVIDENCE FOR MEDICINES ADAPTIVE PATHWAYS TO PATIENTS (MAPPS)Session Chair:Chris Chinn, VP and Head of Health Investment Evidence, GSK; Project Coordinator IMI GETREAL

After nearly a decade of discussion, analysis and development, Medicine’s Adaptive Pathways to Patients (MAPPs) is beginning to see acceptance from regulators, industry and payers with the first live adaptive pilot project under the guidance of the European Medicine’s Agency (EMA) in 2014. A key plank of adaptive licensing is the ability to harness data in real time to measure and capture an expanding evidence base. What are the current frontiers and best practices of real world evidence, and how it incorporate into new clinical pathways and accelerated decision making while insuring data quality and security? Harnessing Real World EvidenceJonathan Cave, Departement of Economics, University of Warwick, UK

Optimising Patient- Centricity through Multilingual Social IntelligenceLiesl Leary, Director, Social Intelligence & Solutions, SDL, UK

Opt-in, Opt-out, & Patient Led Databases. Better Patient Outcomes, Faster and CheaperDuane Schulthess, Managing Director, Vital Tranformation, Belgium

Theme 10 Innovation in Vaccine Development

Richard Pilsudski, Vice President Global Regulatory Affairs, Sanofi Pasteur, France Jan Willem van der Laan, Department of Pharmacology, Toxicology and Biotechnology (FTBB), Medicines Evaluation Board, the Netherlands

Vaccines play an increasingly important role in delivering global public health. They offer a rapid and highly cost-effective way to reduce the global disease burden, with an untapped potential for many more benefits.

This theme explores current and future trends in vaccine development. How do we balance the needs of supplying essential vaccines to the developing world whilst maintaining innovation? What regulatory pathways will support these differing needs? How do we harmonise regulation to speed access where it is most needed?


ADAPTIVE PATHWAYS AND PATIENT ACCESS MODELS TO INNOVATIVE TREATMENTS: IS IT APPLICABLE TO THE DEVELOPMENT OF VACCINES?Session Chair:Michel Stoffel, Vice-Chair Vaccines Europe, Belgium

Current considerations on how adaptive pathways (also referred as Medicine Adaptive Pathways to Patients) could streamline the research, licensing and market access processes have mainly focused on drugs and patients. Vaccines Europe Regulatory Affairs Working Group initiated a reflection to test the applicability of such adaptive pathways to vaccines, and would like to open up a debate with main stakeholders in this field such as regulators, payers/providers.

Adaptive Pathways and Patient Access Models to Innovative Vaccines: A view from the industrySteve Lockhart, Vice President, Development, Sanofi Pasteur-MSD, France

Adaptive Pathways and Patient Access Models to Innovative Vaccines: A view from the regulatorMarco Cavaleri, Head of Anti-infectives and Vaccines, European Medicines Agency, EU

Viral Challenge Studies: An innovative way to speed up the vaccine development: An influenza case studyBruno Speder, Head Clinical Regulatory Affairs & Document Management, SGS Life Science Services, Belgium



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VACCINE DEVELOPMENT ACROSS REGIONSSession Chair:Françoise Rey, Regulatory Policy and Intelligence, Sanofi Pasteur, France

The vaccine development is a long and very complex process that could last up to 20 years before risk benefit of a new vaccine could be confirmed. It involves a combination of investigators, volunteers, health authorities, scientific experts and international organisations across all regions of the world. The recent development of vaccines against Polio disease will illustrate the development of new ways of working and interacting between Sponsors, National Health Authorities, World Health Organization and Public Private Partnership such as GAVI Alliance. The theme will explore whether it is possible to further harmonize global development across regions.

Global Regulatory Collaboration in the Framework of Polio EradicationFotula Fegaras, , Global Regulatory Affairs Franchise Head, Sanofi Pasteur, Canada

The Role of IPV in a Post-Polio WorldSona Bari, Senior Communications Officer, WHO, Switzerland

Polio Endgame: Opportunities for collaborationKeith Chirgwin, Deputy Director, Regulatory Affairs, Bill & Melinda Gates Foundation, USA

Registration, Regulation and Practice in ChinaLi Maozhong, Deputy Director, Department of Drug and Cosmetics Registration, China Food and Drug Administration, China EM1004 | Tuesday, 14 April, 16:00-17:30 | Room 243 Level 2

NEW AVENUE FOR NON-CLINICAL REGULATORY SCIENCE Session Chair:Béhazine Combadière, Director of Research, Head of Immunity and Vaccination team, INSERM, Center for Immunology and Microbial Infection, France

On February 2010, the FDA launched its Advance Regulatory Science (ARS) Initiative on groundbreaking efforts to transform the way medical products are developed, evaluated, and manufactured on the basis of development of new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of drugs and vaccines. The example of new technologies allowing to forecast different vaccine responses before clinical trials are conducted and to provide predictive in vitro immunological models and assays will be presented. Acceptance of such innovative technologies by European regulatory agencies will be discussed.

Innovation in Methods of Vaccine Administration and Immune EfficacyBéhazine Combadière, Director of Research, Head of Immunity and Vaccination team, INSERM, Center for Immunology and Microbial Infection, France

Bioengineering in vitro Models of Human Immunity: Science Fiction or Science Fact?William Warren, Vice President, Head of VaxDesign Campus, USA

Advancing Vaccine Non-Clinical Testing by Harnessing New Technologies- A regulator’s viewYuansheng Sun, Senior Reviewer, Viral Vaccine Section, Paul-Ehrlich-Institut (PEI), Germany


ADJUVANTS IN VACCINESSession Chair:Jan Willem van der Laan, Department of Pharmacology, Toxicology and Biotechnology (FTBB), Medicines Evaluation Board, the Netherlands

The last decade several vaccines have reached the market with new adjuvants. Issues have been raised about their safety, e.g., in relation to autoimmunity. The WHO took the initiative of writing a guideline on the non-clinical evaluation of vaccine adjuvants and adjuvanted vaccines. With this experience and new insights on the impact of Toll-like receptors on the immune responses, an update of the regulatory testing was needed, preferably from a global perspective. New adjuvants are still under development.

Safety of Vaccine Adjuvants with Focus on Autoimmunity: HESI workshop reportHeather Davis, Vaccines Research, Pfizer, Canada

WHO Guideline on Adjuvanted Vaccines Jan Willem van der Laan, Department of Pharmacology, Toxicology and Biotechnology (FTBB), Medicines Evaluation Board, Netherlands

Regulatory Strategy for Non-Clinical Safety Assessment to Support Malaria CandidateMicaela Damsten, Manager, Nonclinical Toxicology, Global Regulatory Affairs, GSK Vaccines, Belgium


BIOVACSAFE (IMI PROJECT)Session Chair:David JM Lewis, Imperial College London, UK

BIOVACSAFE is a 30M€, 5 year Innovative Medicines Initiative project with 20 partners including industry (GSK, Sanofi Pasteur, Novartis Vaccines, deCODE), academia and SMEs. It integrates human clinical experimental medicine studies with animal models and large cohorts of adults and children to identify biomarkers associated with vaccine reactogenicity and immunosafety. A systems biology, bioinformatics and cross-species approach is pursued, together with careful clinical characterisation of adverse reactions following immunisation.

BIOVACSAFE: Translational Medicine and Systems Biology to Identify Biomarkers of Vaccine ReactogenicityDavid JM Lewis, Imperial College London, UK

Questing for Early Markers of Safety: A circular approachGiuseppe Del Giudice, Global Head Translational Medicine, Novartis Vaccines, Italy


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NEW DEVELOPMENTS, EBOLA VACCINESSession Co-Chairs:Richard Pilsudski, Vice President Global Regulatory Affairs, Sanofi Pasteur, France Jan Willem van der Laan, Department of Pharmacology, Toxicology and Biotechnology (FTBB), Medicines Evaluation Board, the Netherlands

The Ebola epidemic in African countries put a huge challenge to public health authorities how to cope with it. Existing programs developing vaccines are enhanced to shorten the time to reach clinical stages. New vaccine strategies have to reach maturity in a short time frame.

Accelerated Development of a Heterologous Prime Boost Approach Combining Ad26.ZEBOV and MVA-BN-FILO Benoit Callendret, Compound Development Team Leader, Janssen (Crucell), the Netherlands

rVSV-ZEBOV-GP Vaccine: Development Update and Regulatory ConsiderationsGeert Preuveneers, Merck Sharp & Dohme, Belgium

Regulatory Activities Related to Ebola Vaccines – EMA perspectiveMarco Cavaleri, Head of Anti-infectives and Vaccines, European Medicines Agency, EU

Theme 11 Availability of Medicinal Products: Drug shortages

Belén Escribano Romero, Head of Department, Pharmaceutical Inspection and Enforcement Department, Spanish Medicines Agency (AEMPS), SpainPär Tellner, Team Leader, Regulatory Affairs, EFPIA, Belgium

Shortages of medicinal products are an increasingly problem globally. Although there are many causes of shortages – issues of manufacturing, supply, quality and commercial viability – the consequences are inevitably on patients.

This theme examines ways to address this problem. It will set out why preventing drug shortages needs to be a priority and explores the steps that regulators can take to protect patients. How do we encourage the broad cooperation between regulatory authorities inside and outside Europe that can help reduce shortages and limit their impact when they do occur?


SOME BASICS ABOUT SHORTAGES OF MEDICINAL PRODUCTS: CONCEPTS, CAUSES, SITUATION AND TRENDSSession Chair:Belén Escribano Romero, Head of Department, Pharmaceutical Inspection and Enforcement Department, Spanish Medicines Agency (AEMPS), Spain

Shortages are increasing and have a negative impact mainly for patients but also for the industry and the regulatory authorities. In this session we will try to establish the basic concepts about shortages, taking into account different perspectives of the problem. Shortages concept, classification and their root causes as well as trends will be presented. These basic discussions will help us to progress towards the other sessions of this theme: shortages consequences, solutions and future scenario.

Basics about Shortages, Supply Chains and QualityDoménico Di Giorgio, Director of the Counterfeit Prevention Unit, Italian Medicines Agency, (AIFA), Italy

EFPIA Good Practices for Drug Product ShortagesBirgitte Holst, Quality Intelligence Director, Novo Nordisk, Denmark

Medicines Shortages, Supply Chains and QualityStephen Spielberg Editor-in-Chief, Therapeutic Innovation & Regulatory Science, DIA, USA



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SHORTAGE CONSEQUENCES: THEIR IMPACT oN PATIENTS, HEALTH CARE PROFESSIONALS AND REGULATORSSession Chair:Pär Tellner, Team Leader, Regulatory Affairs, EFPIA, Belgium

At this session we are focusing on consequences of shortages. Representatives for healthcare professionals and patients are - based on their experience - describing what could happen when it is not possible to deliver the medicinal products. A media representative is also giving their view on the level of transparency regarding shortages in Europe and the challenges to inform the public about this serious issue.

Consequences of shortages – HCP perspectiveRoberto Frontini, President, European Association of Hospital Pharmacists, Healthcare Professionals Working Group, Germany

Consequences of Shortages – Patient perspectiveFrançois Houyez, Health Policy Officer, EURORDIS, France

Consequence of Shortages – Press perspectiveSten Stovall, The Pink Sheet, European Bureau Chief, Informa Business Intelligence, UK


SOLVING AND PREVENTING SHORTAGES / COMMUNICATION BETWEEN DIFFERENT STAKEHOLDERS Session Chair:Lidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

Shortages of Medicinal Products as a Result of Quality Defects or GMP Non-ComplianceBrendan Cuddy, Scientific Administrator, Manufacturing and Quality Compliance European Medicines Agency, EU

Recommendations to Address ShortagesFrançois Houyez, Health Policy Officer, EURORDIS, France

Industry Communication Principles to AuthoritiesJulie Marechal, Senior Manager Quality & Regulatory Affairs, European Generics Association (EGA), Belgium

EM1104 | Tuesday, 14 April, 16:00-17:30 | Room 352B Level 3

LOOKING AT THE FUTURE OF SHORTAGES OF MEDICINAL PRODUCTSSession Chair:Brendan Cuddy, Scientific Administrator, Manufacturing and Quality Compliance European Medicines Agency, EU

NCA PerspectiveLidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland

Proposals to Decrease the Problems with Shortages in the FutureFrançois Bouvy, Director Market Access, EFPIA (European Federation of Pharmaceutical Industries and Associations), Belgium

Panel discussion with speakers and Belén Escribano Romero, Roberto Frontini, François Houyez and Sten Stovall


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Theme 12Pharmacovigilance in 2015 - Poised for convergence of innovation

Maarten Lagendijk, Pharmacovigilance Coordinator, Medicines Evaluation Board (MEB), the NetherlandsMaria-Grazia Zurlo, QPPV, Pfizer, Italy

The 2010 European Pharmacovigilance Legislation has converted many of the concepts and approaches of pharmacovigilance into practical applications. Whilst refinement of this process continues, it already provides a system that promotes patient safety whilst remaining proportionate.

This theme reviews the important learnings in pharmacovigilance to date, and what role it plays in stimulating innovation. It will examine which principles and technologies are driving change. How should these new insights and new approaches affect the way we consider risk – benefit? Should risk – benefit be applied in the same way for new and established treatments?

We will also look at PRAC (Pharmacovigilance Risk Assessment Committee) and its impact on approaches to safety. How is participation between bodies changing, nationally and internationally and how do we improve this process? We also explore the role of patients in pharmacovigilance; how has their role changed, and are we giving them the right information to participate fully?

EM1201 | Tuesday, 14 April | 09:00-10:30 | Room Bordeaux Level 3

PHARMACOVIGILANCE DEBATE: BRAVE NEW WORLD OF DECISION SUPPORTSession Chair: Paul Eisenberg, Senior Vice President of Global Medical and Chief Medical Officer, Amgen, USA

Evidence-based drug safety decisions must be made as early as possible, often with incomplete, missing, or misleading data. ICSRs historically have had a major role in this process and there have been increased efforts to gather more detailed data for ICSRs, although the impact that would have on patient safety is unclear. The digital age has ushered in new possibilities for additional data streams. This session will consist of a debate centered around pragmatic approaches to quality decision making in pharmacovigilance: Should the focus be on gathering more detailed data for ICSRs? Should intelligent signaling systems be developed to analyse electronic health records and social media data? Can intelligent signaling applied to digital data be used to augment traditional ICSR-based system to more rapidly reach acceptable decisions in pharmacovigilance? Where should we apply future efforts and investment?

Post-approval Safety Surveillance: FDA PerspectiveGerald Dal Pan, Director, Office of Surveillance and Epidemiology, Food and Drug Administration (FDA), USA

The Good, the Bad and the Ugly of Patient Safety Data SourcesSue Rees, Executive Director and EU QPPV, Global Patient Safety, Amgen, UK

Harnessing the Power of Emerging Data and Technologies: Balancing qualitative and quantitative needs in safety surveillanceAndrew Bate, Senior Director, Epidemiology Group Lead, analytics, Worldwide R&D, Pfizer Ltd., UK

Will Social Media Aid Decision Making in Pharmacovigilance? Look Before You TweetDavid J. Lewis, Global Head of Pharmacovigilance, Novartis Pharma, Switzerland

Signal Detection in an EMR Healthcare Environment: Danish experienceMona Vestergaard Laursen, Senior Adviser, Danish Health and Medicines Authority, Denmark

EM1202 | Tuesday, 14 April, 11:00-12:30 | Room Bordeaux Level 3

PHARMACOVIGILANCE: HOW WELL ARE PATIENTS INFORMED AND HOW CAN THEY CONTRIBUTE?Session Chair:Vicki Edwards, QPPV and Head of Affiliate Vigilance Excellence, Abbvie, UK

Current EU legislation and guidance encourage patients to engage the pharmacovigilance system by reporting safety concerns. Are they doing so? Regulated communication of patient-centric safety information must be conveyed in lay terms, which should give consideration to individual understanding, translatability, and cultural and other communication issues. Given the communication channels currently available to patients, a portion of this session will explore whether the new legislation has had an impact on the value of this information to patients, i.e., is there evidence of improved patient comprehension, retention, and behavior regarding the safe and proper use of medicinal products? Another segment of this session will be devoted to patient engagement in safety and whether this has begun to contribute to the evolving body of safety information in EudraVigilance? Are there additional opportunities for two-way communication with patients, particularly for citizens who have difficulty understanding and using health information?

Pharmacovigilance and Patient Information - What's new?Theo Raynor, Professor of Pharmacy Practice, University of Leeds, UK

The Patient Information GapPhil Willan, Patient Representative, Healthcare Quality Improvement Partnership (HQIP), Member of the Outcomes Publication Advisory Group, UK

Using Patient Insights to Improve Safety ManagementMichael Richardson, VP International GPV&E and EU QPPV, Bristol-Myers Squibb, UK



44


INTERNATIONAL COLLABORATIONS IN PHARMACOVIGILANCE: COMMON OBJECTIVES, COMMON CHALLENGESSession Chair:Peter Arlett, Head of Pharmacovigilance, European Medicines Agency, EU

Pharmacovigilance is a global enterprise and regulatory science is constantly evolving globally to meet new scientific challenges, particularly those with common objectives. As a result, a patchwork of agreements amongst regulatory authorities has evolved that permit confidential data exchange across jurisdiction and the ability to share perspectives on common objectives and challenges. This session will cover current collaboration between EMA and FDA in pharmacovigilance. Focus will be on drug-specific scientific evaluations, new pharmacovigilance tools, and how regulators address common challenges in the context of legislative constraints.

Peter Arlett, Head of Pharmacovigilance, European Medicines Agency, EU

Gerald Dal Pan, Director, Office of Surveillance and Epidemiology, CDER, FDA, USA

Val Simmons, EU QPPV, Executive, Global Patient Safety, Eli Lilly and Company Ltd., UK


BENEFIT/RISK - PRAGMATIC LIFECYCLE MANAGEMENTSession Co-Chairs:June Raine, Vigilance and Risk Management of Medicines Division, MHRA, UKValerie Simmons, EU QPPV, Executive, Global Patient Safety, Eli Lilly & Co., Ltd, UK

The benefit/risk profile of a medicinal product must be favorable to gain and maintain marketing authorisation. From a pragmatic view, however, different approaches and proportionality are needed to assess and manage the benefit-risk balance across a product’s lifecycle in a resource-appropriate manner. This session will address practical aspects of benefit-risk management from First-In-Human studies through late-cycle product maturation. Different lines of evidence, challenges in their evaluation, and triggers for PASS and PAES will be discussed in a pragmatic context, considering both patient safety and the changing realities of data across a product’s lifecycle.

How is the Regulatory Framework Helping?Peter Arlett, European Medicines Agency, EU

Will the New PROTECT Tools Make a Difference?Shahrul Mt-Isa, Imperial College UK

Assessment of Benefit/Risk in the Lifecycle of Biologics Isma Benattia, Vice President Global Safety, Amgen, USA

Panel Discussion with all speakers and Tomas Salmonson, Chair CHMP, Senior Scientific Advisor, Medical Products Agency, Sweden


IS IT OFF-LABEL USE OR MEDICATION ERROR – INTO THE UNKNOWN?Session Co-Chairs:June Raine, Vigilance and Risk Management of Medicines Division, MHRA, UK Carla Schoonderbeek, Partner, Hoyng Monegier, the Netherlands

GVP Annex I defines ADRs, off-label use, medication errors, and misuse. Specific MedDRA terms are available to code these medical concepts, but, based on available information, term selection for coding may not always be obvious. This session will address regulatory expectations and challenges in applying these definitions for ICSRs and implications for PSURs. Focus will be on practical and legal aspects of categorising, monitoring, and assessing medication errors, misuse, and off-label use and how to identify and implement opportunities to prevent them.

The Challenges and Pitfalls of Off-Label and Other Unlicensed Use of MedicinesAlexander Meier, Global Head Legal Regulatory & Development, Novartis Pharma AG, Switzterland

How the Expanded Definition of AE is Helping PharmacovigilanceGeorgy Genov, Head of Signal Management, Division Inspections and Human Medicines Pharmacovigilance, European Medicines Agency (EMA), EU

Making the Pharmacy an Even Safer Place to Get MedicinesJohn Chave, Secretary General EU pharmacist organization PGEU

Panel discussion with Rob Camp, EURORDIS, France

EM1206 | Wednesday, 15 April, 11:00-12:30 | Room Bordeaux Level 3

MANAGING THE RISK OR MANAGING THE PLAN?Session Chair:Maarten Lagendijk, Pharmacovigilance Coordinator, Medicines Evaluation Board (MEB), the Netherlands

Harmonisation of RMPs (Risk Management Plans) across different regulatory jurisdictions, including triggers for RMP updates, is challenging due to discordant perceptions of true risk and the availability, burden, and effectiveness of various minimisation interventions. The RMP is a living document; as more and more information on a product becomes available during its lifecycle risks can change. However, removing risks can especially be a challenge and in this session a possible method is discussed. Preparing an RMP for mature products gives rise to challenges, and several practical issues to effectively manage RMPs for generics, such as multiple divergent assessments, developing a RMP when the reference products has none, etc. exist as well. In the session plans to streamline the process will be discussed.

RMPs for GenericsKora Doorduijn, CMDh member/Policy adviser, Policy, Governance and Regulatory Affairs Department, Medicines Evaluation Board (MEB), the Netherlands


45

RMPs for Mature ProductsWendy Booth, Director, Global Clinical Safety & Pharmacovigilance, GlaxoSmithKline, UK

Are Risks Written in Stone? Strategies to Successfully Remove Risks from Risk Management PlansSamuel Ramsden, Safety Surveillance Risk Management Specialist, Novo Nordisk, Denmark


SIGNAL DETECTION AND MANAGEMENT: CAN YOU WHISTLE?Session Chair: Phil Tregunno, Signal Management Unit Manager, MHRA, UK

The session will present signal detection recommendations from the collaborative Innovative Medicines Initiative (IMI) PROTECT Project and discuss the key principles of signal management in the EU. Over the course of the past 5 years, IMI PROTECT has conducted research across multiple partners to explore various aspects of statistical signal detection. Its results are already beginning to shape changes in the approach to signal detection within the EU Network. This session will provide a whistle-stop tour of those recommendations, and key principles for signal management in the EU.

Introduction to PROTECT Research and Recommendations for Statistical Signal DetectionAntoni Wisniewski, Analytics Systems Lead, AstraZeneca, UK

Choice of Granularity for Statistical Signal Detection: When and how to group adverse events Niklas Norén, Chief Science Officer, Head of Research, Uppsala Monitoring Centre, Sweden

Statistical Signal Detection for Spontaneous Reports: Which method to use?Suzie Seabroke, Senior Epidemiologist, Medicines and Healthcare Products Regulatory Agency (MHRA), UK

The Signal Management Process in the European Pharmacovigilance Network - key principles, transparency, process optimisation, and interface with stakeholders Georgy Genov, Head of Signal Management, European Medicines Agency, EU


PRAC: HIGHLIGHTS, CHALLENGES AND REFERRALSSession Co-Chairs:Sarah Montagne, Head of EU Regulatory Policy, Bayer, UKSabine Straus, Head of Pharmacovigilance, Medicines Evaluation Board, the Netherlands

More than two years after the inauguration of PRAC, PV legislation remains high on everyone’s agenda and has fundamentally changed the way safety related activities are conducted in the EU.

There has been a major shift from a predominately reactive system to a proactive lifecycle benefit/risk management approach using risk management plans (RMPs), post-authorisation safety studies and real-time signal detection. Whilst managing this shift, PRAC has devoted a significant part of its time conducting safety referrals, often on well-established products.

The consequence of a proactive risk management system in the post-marketing environment has seen increased levels of activity and complexity not previously experienced within the EU.A panel discussion including Industry and regulators will consider some of the key challenges and latest hot topics in this area. Three short presentations will be made to set the scene: PRAC highlights, impact of PASS, and lessons learn on safety referrals for well-established products.

PRAC: Highlights from a busy agendaSabine Straus, Head of Pharmacovigilance, Medicines Evaluation Board, the Netherlands

The Impact of Imposed PASS and the Challenges of Making Multi-MAH Studies WorkKatarina Nedog, Safety and Regulatory Manager, European Generic Medicines Association (EGA), Belgium

Union Safety Referrals: Shared experiences of the referral journey for well-established products and the importance of effective communication Emmanuelle Pines, Head of Corporate Pharmacovigilance Division, Pierre Fabre Medicament, France, on behalf of AESGP

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46

EM1302 | Tuesday, 14 April | 11:00-12:30 | Blue Room Level 2

ESSENTIAL BUSINESS CHANGE MANAGEMENT IN PHARMACOVIGI-LANCE: SYSTEMS, SERVICES AND SIMPLIFICATIONSSession Chair:Peter Arlett, Head of Pharmacovigilance, European Medicines Agency, EU

The coming months will see the launch of the EMA literature monitoring service for the industry, piloting of the PSUR repository for centralised document submission, the Article 57 database of products and substances in full maintenance mode and significant progress made in the development of new functionalities for the EudraVigilance system for collecting and analysing reports of suspected adverse drug reactions. All these services and systems will impact industry and regulators and informed business change management will allow the improved data and information to be utilised while realising efficiency gains for the industry. The session will set out the scope of the imminent change and explore the impact on different industry stakeholders.

Scope and Benefits of Business Change in PharmacovigilancePeter Arlett, Head of Pharmacovigilance, European Medicines Agency, EU

Impact of New Systems and Services on Innovative Industry SectorMargaret Walters, Director & Deputy EU QPPV, MSD, UK

Impact of New Systems and Services on Generics SectorWendy Huisman, EU QPPV, Teva Pharmaceuticals Europe., the Netherlands

Discussion on Impact on IndustryElmar Kroth, Managing Director, German Medicines Manufacturers’ Association (BAH), Germany

EM1305 | Wednesday, 15 April 09:00-10:30 | Room Havane Level 3

PHARMA MEDICAL AFFAIRS: VISION THROUGH 2020Session Chair:Gustavo Kesselring, President IFAPP; DIA Advisory Council of Latin America; Executive Director Latin America, ViS Research, Brazil

Medical affairs organisations have emerged over the past half century in response to federal regulations around the separation of medical and commercial activities within drug companies. Many companies also chose to focus R&D resources on developing new products and moved post-launch activities, such as finding new indications for existing drugs, into the medical affairs function. Continued pressure from regulatory agencies and public sentiment have pushed more and more activities into Medical affairs organisations. Today, these organisations commonly involve the following medical activities: Medical education, medical field teams, post-launch clinical trials, medical-information services, medical communications, medical strategic activities and health economics and outcome research activities. The vision from medical departments of three global pharmaceutical companies will be addressed and balanced with the vision of the Standing Committee of the European Doctors (CPME).

Michael Devoy, Head, Global Medical Affairs & Pharmacovigilance; Member of the Executive Committee and Chief Medical Officer, Bayer HealthCare AG, Germany

Jacques de Haller, Vice President, Standing Committee of European Doctors (CPME), Switzerland

Michael Rosenblatt, Executive Vice President and Chief Medical Officer, Merck & Co., Inc, USA

Tamas Suto, Chief Medical Officer, Sanofi Asia, China


50 YEARS OF EDQM: PREPARING FOR THE FUTURE TO CONTINUOUSLY ENSURE THE QUALITY OF MEDICINESSession Chair: Susanne Keitel, Director, EDQM/Council of Europe

The European Directorate for the Quality of Medicines and HealthCare (EDQM) turned 50 in 2014. While EDQM and its 37 Member States are proud of the tremendous achievements over the past 50 years, globalisation and technical and scientific developments in all fields pose constant challenges.

The session will provide an insight to EDQM’s activities, and focus on current hot topics such as the introduction of finished product monographs in the European Pharmacopoeia, the impact of Quality by Design (QbD), recent developments in pharmacopoeial harmonisation, the holistic strategy in combating counterfeit medicines and the establishment of a harmonised European paediatric formulary of unlicensed medicines.

Finished Product Monographs and Current “Hot Topics” of the European PharmacopoeiaCathie Vielle, Head of the European Pharmacopoeia Department, EDQM/Council of Europe

QbD and the PharmacopoeiaGraham Cook, Sr. Director, Global Quality Intelligence and Compendial Affairs, Pfizer

An Update on Pharmacopoeial HarmonisationSusanne Keitel, Director, EDQM/Council of Europe

The EDQM’s Holistic Anti-Counterfeiting StrategyFrançois-Xavier Lery, Head of Section, EDQM/Council of Europe

“Paedform” – A Harmonised European Paediatric FormularyDirk Leutner, EDQM/Council of Europe

HOT TOPICS/STAND ALONE SESSIONS


47

Focus on Quality

Session 1101 | Tuesday, 14 April, 09:00-10:30

SOME BASICS ABOUT SHORTAGES OF MEDICINAL PRODUCTS: CONCEPTS, CAUSES, SITUATION AND TRENDSSession Chair: Belén Escribano Romero, Head of Department, Pharmaceutical Inspection and Enforcement Department, Spanish Medicines Agency (AEMPS), Spain


QUALITY ASPECTS FOR AN ATMP (M3 OF MAA)Session Chair: Valerie Pimpaneau, Voisin Consulting Life Sciences, France


SOLVING AND PREVENTING SHORTAGES/ COMMUNICATION BETWEEN DIFFERENT STAKEHOLDERS Session Chair: Lidia Retkowska-Mika, Director, Legal Department, Office for Registration of Medicinal Products, Poland


NEW AVENUE FOR NON-CLINICAL REGULATORY SCIENCE Session Chair: Béhazine Combadière, Director of Research, Head of Immunity and Vaccination team, INSERM, Center for Immunology and Microbial Infection, France

Session 0105 | Wednesday, 15 April, 09:00-10:30

IMPACT OF THE CLINICAL TRIAL REGULATION ON CMC FILING AND RELEVANT GMP REQUIREMENTSSession Chair: Georges France, Global LOC Quality lead, GlaxoSmithKline, UK


50 YEARS OF EDQM: PREPARING FOR THE FUTURE TO CONTINUOUSLY ENSURE THE QUALITY OF MEDICINESSession Chair: Susanne Keitel, Director, EDQM/Council of Europe


FROM THE SHOP FLOOR: PRACTICAL ADVICE ON BRINGING NEW MEDI-CINES TO PATIENTSSession Chair: Henrik Kim Nielsen, Corporate Vice President, Regulatory Affairs GLP-1 and Obesity, Novo Nordisk, Denmark


CLINICAL, QUALITY & REGISTRATION CONSIDERATIONS FOR BIOLOG-ICS COMBINATION PRODUCTS DEVELOPMENT Session Chair: Paul Jansen, Global Head Medical Devices US, Sanofi, USA

Focus on Adaptive Pathways


MEDICINES ADAPTIVE PATHWAYS TO PATIENTS (MAPPS) - HOLDING HANDS ACROSS THE LIFECYCLESession Chair: James Anderson, Head of Corporate Government Affairs, GlaxoSmithKline, UK


HTA AND ADAPTIVE PATHWAYSSession Chair: Sarah Garner, Associate Director R&D, NICE, UK


REAL WORLD EVIDENCE FOR MEDICINES ADAPTIVE PATHWAYS TO PATIENTS (MAPPS)Session Chair: Chris Chinn, VP and Head of Health Investment Evidence, GSK; Project Coordinator IMI GETREAL

Focus on Patient Involvement

Monday, 13 April, 13:30-15:00

EU REGULATORY TOWN HALL MEETING50th Anniversary of the EU Pharmaceutical LegislationModerator: Fernand Sauer, French Academy of Pharmacy, Former Executive Director of the European Medicines, France

Session 0405 | Wednesday, 15 April 09:00-10:30

PATIENT INVOLVEMENT IN THE DEVELOPMENT DIALOGUE WITH REGULATORY AUTHORITIES AND HTAS Session Chair: Isabelle Stöckert, Head Global Regulatory Affairs Europe/Canada, Bayer Pharma AG, Germany


DEVELOPING BEST PRACTICE GUIDANCE ON PATIENT ENGAGEMENT IN MEDICINES R&D Session Chair: David Haerry, EUPATI Co-Leader on Sustainability (WP7), European AIDS Treatment Group, Switzerland


SOCIAL MEDIA, PATIENTS AND PUBLIC POLICYSession Chair: Alastair Kent, Director General, Genetic Alliance UK

Session 0507 | Wednesday, 15 April 14:00-15:30

A PARTICIPATORY SOCIETY FOR A BETTER HEALTH: PATIENT, CITIZEN, AND CONSUMERS VOICE IN PPPSSession Chair: Jan Geissler, Director, EUPATI, Belgium

FOCUS SESSIONS



48

Acha Virginia EM0807 36

Adams Neil EM0201, EM0204 24

Ahmed Iftakhar (Ifty) EM0906 38

Anderson James EM 0106 23

Arlett Peter EM1203, EM1204, EM1302 44, 46

Atzor Sabine EM0103, Theme 7 22, 33

Audhya Paul EM0807 36

Backman Christer EM0104 22

Bari Sona EM1002 40

Bate Andrew EM1201 43

Beck Olof EM0805 36

Bedlington Nicola EM0507, EM0808, EM0906 30, 36, 38

Benattia Isma EM1204 44

Bloom Alexander EM0305 26

Boehm Jérôme EM0403, EM0406 27, 28

Bogaert Peter EM0308 26

Booth Wendy EM1206 44

Bouder Frederic EM0108 23

Bouvy François EM1104 42

Broekmans André EM0503 29

Broich Karl EM0706 34

Brosch Sabine EM Tutorial 1 16

Burzykowski Tomasz EM0306 26

Callendret Benoit EM1008 41

Calmels Bastien EM0302 26

Camp Rob EM1205 44

Cavaleri Marco EM1001, EM1008 39, 41

Cave Jonathan EM0908 39

Celis Patrick EM0301 25

Celis Julio EM0506 30

Cerreta Francesca EM0407, EM0705 28, 34

Charmeil Olivier Opening Plenary 21

Chave John EM1205 44

Chen Shaoyu EM0604 32

Chenel Marylore EM0703 34

Cheung Amy EM Tutorial 6, EM0703 18, 34

Chibout Salah-Dine Theme 5, EM0501 29

Chinn Chris EM0908 39

Chirgwin Keith EM1002 40

Chlebus Magda EM0505, EM0508 29, 30

Clamou Isabelle Theme 4 27

Combadière Béhazine EM1004 40

Compton Chris EM0501 29

Cook Graham EM1306 46

Cranz Hubertus EM0806 36

Creba Judith EM0404 28

Creff Nicolas EM0504 29

Cuddy Brendan EM1103, EM1104 42

Last Name First Name Session Page Number

SPEAKER INDEX


49

Dal Pan Gerald EM1201, EM1203 43, 44

Damsten Micaela EM1006 40

Danan Gaby EM Tutorial 1 16

Dantsker Gene EM0903 38

Davis Heather EM1006 40

De Cuyper Xavier EU Town Hall Meeting 20

De Haller Jacques EM1305 46

de Zegher Isabelle EM0904 38

Del Giudice Giuseppe EM1007 40

Del Signore Susanna Theme 1, EM0107, EM0705 22,23,34

Devoy Michael EM1305 46

Dewulf Lode EM0708 34

Di Giorgio Domenica EM1101 41

Doorduijn Kora EM1206 44

Dörr Petra EM0601 31

Draghia-Akli Ruxandra EM0504, EM0508, EM0907 29, 30, 38

Eaglesham John EM0903 38

Edwards Vicki EM1202 43

Eggermont Alexander EM0506 30

Eichler Hans-Georg EM0106, EM0402, EM0503 23, 27, 29

Eichler Irmgard EM0701, EM0702 33, 34

Eisenberg Paul EM1201 43

Escribano Romero Belén Theme 11, EM1101, EM1104 41, 42

Evans Georgina EM0901 37

Facey Karen EM0505 30

Fegaras Fotula EM1002 40

Fournier Marielle EM0208 25

Fratazzi Candida EM0904 38

Frénoy Edith EM0406 28

Friis Bach Karen EM0808 36

Frontini Roberto EM1102, EM1104 42

Gandhi Shelley EM Tutorial 5 17

Garner Sarah EM0407, EM0503 28, 29

Garutti Claudio EM0802 35

Geissler Jan EM0507, EM0508 30

Genov Georgy EM1205, EM1207 44, 45

Gillham-Eisen Liz Anne EM0301 25

Giorgi Dominique Satellite Symposium 20

Gottwald Matthias EM0501, EM0505, EM0508 29, 30

Griffiths Mark EM0806 36

Guillard Helen EM0806 36

Gunn Magda EM0507 30

Haerry David Opening Plenary, EM0505, EM0507 21, 30

Hansson Erik EM0201 24

Harousseau Jean-Luc Satellite Symposium, EM0402 20, 27

Heckenberg Andrea EM0505 30

Hellbert Karina EM Tutorial 2 16

Hirsch Gigi EM0106 23


SPEAKER INDEX



50

Hockley Kimberly EM0405 28

Hoekstra-van den Bosch Sabina Theme 2, EM0201 24

Hojo Taisuke EM0605 32

Holst Birgitte EM1101 41

Holzmann Catherine EM0205 24

Houyez François EM0405, EM1102, EM1103, EM1104 28, 42

Huisman Wendy EM1302 46

Hurts Hugo Opening Plenary 21

Hynes Carolyn EM0906 38

Hystad Marit EM0305 26

Jansen Paul Theme 2, EM0208 4, 24, 25

Jessen George EM0204 24

Kayadjanian Nathalie EM0907 39

Keitel Susanne EM1306 46

Kenny Maureen Opening Plenary 20

Kent Alistair EM0906 38

Kesselring Gustavo EM1305 46

Kim Ho-Jeong EM0602 31

Kitahara Jun Theme 6 31

Kourliouros Sophia EM0607 32

Kroth Elmar EM1302 46

Kübler Jürgen EM Tutorial 3 16

Kureishi Amar EM0604 32

Lagendijk Maarten Theme 12, EM1206 43, 44

Laslop Andrea EM0804 35

Lastic Pierre-Yves EM Tutorial 4 17

Laursen Mona EM1201 43

Le Cam Yann EM0106, EM0402 23, 27

Le Gledic Sylvie EM0205 25

Leary Liesl EM0908 39

Lechat Philippe Satellite Symposium 20

Lery François-Xavier EM1306 46

Leurent Pierre EM0207 25

Leutner Dirk EM1306 46

Lewis David J EM1201 43

Lewis David JM EM1007 40

Li Maozhong EM1002 40

Li Ning EM0202, EM0603 24, 31

Lightbourne Alison EM0807 36

Lindström Gommers Lenita EM0601 31

Lisman John EM0103 22

Llinares Garcia Jordi Theme 7, EM0708 4, 33, 34

Lockhart Steve EM1001 39

Macolić Šarinić Viola EU Town Hall Meeting 20

Maes Luk EM0403 27

Maleck Klaus EM0301 25

Malikova Marina EM0208 25

Manolis Efthymios EM0703 34


SPEAKER INDEX


51

Mantua Valentina EM0904 38

Marechal-Jamil Julie EM1103 42

Marquet Pierre EM0805 36

Maselbas Wojciech EM1101 22

McAuslane Neil EM0406, EM0604 28, 32

Meier Alexander EM1205 44

Menezes-Ferreira Margarida EM0302 26

Mentzer Dirk EM0702 33

Meyer François Satellite Symposium, EM0403 20, 27

Michaux Geneviève EM Tutorial 2 16

Mischak Harald EM0802, EM0805 35, 36

Montagne Sarah EM1208 45

Morris Andrew Opening Plenary 21

Moulon Isabelle EM0505, EM0508 30

Moult Anthony EM0105 23

Moussa Miranda EM0204 24

Mt-Isa Shahrul EM1204 44

Nagano Tetsuo EM0605 32

Nakashima Nobumasa EM0601 31

Narayanan Gopalan EM0306 26

Nedog Katarina EM1208 45

Neves Judite EM0204 24

Nielsen Henrik Kim Theme 6, EM0607 4, 31, 32

Niese Detlef Theme 8, EM0803 4, 35

Noren Niklas EM1207 45

Norga Koenraad EM0701 33

O'Connor Michelle EM0202 24

Oderkirk Jill EM0801 37

Olson Melvin EM0501 29

Osipenko Leeza EM0404 28

Ostler Tobias EM0804 35

Ovelgönne Hans EM0303, EM0404 26, 27

Pani Luca Theme 9, EM0904 4, 37, 38

Parkinson John EM0907 39

Pascual Maria EM0303 26

Passarani Ilaria EM0108 23

Pavlovic Mira Satellite Symposium 20

Pedersen Jane Møll EM0702 34

Pereira Doris EM0607 32

Peuvrelle Fabien EM0108 23

Pfister Sabine EM0105 23

Pignatti Francesco EM0405 28

Pilsudski Richard Theme 10, EM1008 4, 39, 41

Pimpaneau Valerie EM0302 26

Pines Emmanuelle EM1208 45

Poulsen Dorthe Theme 1, EM0104 4, 22

Preuveneers Geert EM1008 41

Rabinowitz Jonathan EM0506 30


SPEAKER INDEX



52

Rägo Lembit EM0608 32

Raine June EM1204, EM1205 44

Ramsden Samuel EM1206 45

Rasi Guido EU Town Hall Meeting, Opening Plenary 20, 21

Raynor Theo EM1202 43

Reany Matthew EM0807 36

Rees Sue EM1201 43

Rengarajan Badri EM0308 27

Retkowska-Mika Lidia EM1103, EM1104 3, 42

Rey Françoise EM1002 40

Richardson Michael EM1202 43

Richardson William EM Tutorial 5 17

Rodriguez Ana EM0101, EM0108 22, 23

Roediger Alexander EM0108 23

Rohou Solange EM0703 23

Rosenblatt Michael EM1305 46

Rothman Brian EM0901 37

Rueda Rodriguez Jesús EM0205 24

Ryll Bettina EM0103, EM0907 22, 39

Rys Andrzej EU Town Hall Meeting 20

Sadallah Fatiha Theme 5, EM0508 4, 29, 30

Salmikangas Paula Theme 3, EM0305 4, 25, 26

Salmonson Tomas EM0402, EM0806, EM1204 27, 36, 44

Salvador-Roldán Rocio EM0305 26

Sauer Fernand EU Town Hall Meeting 20

Scheeren Joseph EM0604 32

Schmiegelow Merete EM0808 37

Schneider Christian EM0103 22

Schoonderbeek Carla EM1205 44

Schulthess Duane Theme 9, EM0907, EM0908 4, 37, 39

Schultze Nathalie EM0701 33

Schuring Henk Theme 3, EM0305 4, 25

Seabroke Suzie EM1207 45

Silva Lima Beatriz EM0301, EM0506, EM0805 25, 30, 35

Simmons Valerie EM1203 44

Singh Dinesh EM0904 38

Sladowski Dariusz EM0802 35

Slijkerman Diederick EM0308 27

Sokal Etienne EM0306 26

Speder Bruno EM1001 39

Spielberg Stephen EM0702, EM1101 34, 41

Spindler Per EM0808 36

Stöckert Isabelle EM0405 28

Stoffel Michel EM1001 39

Stovall Sten EM0903, EM1102, EM1104 38, 42

Straus Sabine EM1208 45

Stumm Matthew EM0903 38

Sullivan Richard EM0807 36


SPEAKER INDEX


53

Sun Yuansheng EM1004 40

Suto Tamas EM1305 46

Tawaragi Tomiko EM0605 32

Tellner Pär EM0601, Theme 11, EM1102 4, 31, 41, 42

Thalheim Christoph EU Town Hall Meeting 20

Thompson Brad EM0207 25

Thristan Mark EM0205 24

Tominaga Toshiyoshi EM0602, EM0605 31, 32

Tregunno Phil EM1207 45

Troelsen Henrik EM0104 22

Truyen Luc EM0706 34

Tshinanu Flora EM Tutorial 6 18

Tunbridge Graeme EM0201 24

Tzalis Dimitrios EM0504 29

Tzouma Victoria EM0708 34

Uyama Yoshiaki EM0602 31

Vamvakas Spiros EM0404 3, 27

Van der Laan Jan Willem Theme 10, EM1006, EM1008 39, 40, 41

Verbraska David EM0608 32

Verstappen Daniel EM0202 24

Vielle Cathie EM1306 46

Vollebregt Erik EM0207 25

Vulto Arnold EM0807 36

Walters Margaret EM1302 46

Wang Haixue EM0603 31

Wang Tao EM0603 31

Warren William EM1004 40

Westerholm Barbro EM0705 34

Wichems Christine EM Tutorial 3 16

Willan Phil EM1202 43

Williams Tim EM0901 43

Wirthumer-Hoche Christa EU Town Hall Meeting, EM0208 20,25

Wisniewski Antoni EM1207 45

Wong-Rieger Durhane EM0608 32

Xu Jane (Jing) EM0602 31

Zurlo Maria Grazia Theme 12 4, 43


SPEAKER INDEX



54

NETWORKING EVENTS

EuroMeeting “Bienvenue à Paris” Opening Reception Monday, 13 April 2015 | 18:00-20:30 | Hall Passy Level 1

Join us at the the best-attended networking event of the conference for an excellent opportunity to renew your existing contacts and to make new ones.

The reception will take place in a unique setting. Take the exceptional opportunity to join colleagues from the EuroMeeting and Clinical Forum for an inspirational evening of networking.

Patient Welcome LunchMonday, 13 april 2015 | 12:30-14:00 | Room Arlequin Level 3Reserved for patient representatives and patient speakers only. Overview of the EuroMeeting scientific programme and an opportunity for patient

representatives to meet and network with each other prior to the conference.

Speed Networking SessionsMonday, 13 April 2015 – 13:00-13:30 | Tuesday, 26 March 12:30-13:00 in the Exhibition Area 3B on Level 3

The EuroMeeting is used as a networking opportunity by all participants. However, it is not easy to walk right up to someone, introduce yourself and have a

conversation. The EuroMeeting Speed Networking session aims to make this process a lot easier.

DIA Communities – Meet and EatTuesday, 14 April 2015 | 13:00-14:00 Room Arlequin Level 3

An opportunity for all Community members – and those interested in joining one – to get together for networking at lunchtime.

Tuesday Networking Cheese & Wine ReceptionTuesday, 14 April 2015| 17:30-18:30 | Exhibition Halls Level 2 and 3

Tuesday’s networking reception takes place in the Exhibition Halls on both floors. Drinks and snacks are included. It is open to all registered attendees.

Student Poster Award CeremonyTuesday, 14 April 2015 | 17:45-18:15

Students from around Europe will be showcasing their research in this year’s Student Poster Session. Posters will be located in the Bordeaux Hallway on

Level 3. In addition, join us at 17:45 in the DIA Booth 3.D17 as we present the awards for the first and second place student poster winners.

Patient Feedback LunchWednesday, 15 April 2015 | 12:45-13:45 | Room Arlequin Level 3Reserved for patient representatives and patient speakers only.

Young Professional Mentoring LunchWednesday, 15 April 2015 | 12:45-13:45 | Exhibition Area 3B on Level 3

Network on the Exhibition FloorsAll refreshments and lunches will be served in the Exhibition Halls, making it the ideal place to meet the people you want to meet.

Networking is an integral part of the EuroMeeting. Past attendees tell us that the networking opportunities presented by the EuroMeeting are one of the

key reasons for attending. Each year, the EuroMeeting offers numerous opportunities to catch up with existing contacts and to make new ones in a relaxing

setting. All networking events at the EuroMeeting are included in the registration fee.

201527-28 APR DAKAR, SENEGAL | #151054th African Regulatory Conference and Exhibition - Moving towards regulatory harmonisation to enhance access to medicines in Africa

This conference offers the opportunity for key stakeholders active in the region to exchange progressive views on key topics of interest and identify focus areas for ongoing efforts aimed to increase patient access to new and improved medicines.

18-19 MAY BASEL, SWITZERLAND | #15113Oncology Drug-induced Cardiotoxicity Conference From bench to bedside

As survival rates in oncology patients further increase, early detection and early development of diagnostic and mitigation strategies are needed to facilitate optimal outcomes in the treatment of oncology patients.

24-25 JUN DAR ES SALAAM, TANZANIA | #151074th Global Animal Health Conference Regulatory Convergence

The International Federation for Animal Health (IFAH) is organising the 4th in the series of Global Animal Health Conferences with the support of an Organising Committee and funded by the Bill and Melinda Gates Foundation.

Leading figures from government, academia, industry, and international organisations will share over 1.5 days challenges and solutions for improving regulatory convergence for veterinary product registration. The focus will be on developing countries and on how to enable the widest availability of high quality, safe and efficacious products in these markets, with an emphasis on Africa, and East Africa in particular.

30 JUN BRUSSELS, BELGIUM | #15117DIA/TOPRA Workshop on Adaptive Pathways

Adjusting the route to the market is vital to support innovation and timely approvals for products that could cover unmet or high medical needs. The workshop aims to look into both the regulatory hurdles as well as discuss improvements in market access based on evidence from recent years and the situation in European countries. Insights from experts from both regulatory as well as market access (HTA) side are brought to the table, connecting all stakeholders. The outcomes will be compiled into white paper.

22 SEP VIENNA, AUSTRIA | #15118The New Medical Device Regulation Information Day

You will get an overview of the EU device legislative system updates. This Information Day provides an insight of the essential changes in various fields, such as the role of notified bodies and all requirements in clinical or post-market requirements.

1-2 OCT LONDON, UK I #15109Annual joint DIA / EFGCP / EMA Better Medicines for Children Conference

This annual conference on Medicines for Children is a unique opportunity to get the update of the latest developments on the paediatric medicines and the regulatory challenges on the area. On the conference there will be presentations and discussions on the Pathfinder initiative and Adaptive pathways and Clinical Trials design/modelling and simulation in psychiatry and neurology. The voices of patients and Health Care Professionals and the perspectives of regulatory authorities, industry and CROs is an essential part of this conference.

13-14 OCT LONDON, UK | #15104(Pre-Conference on 12 OCT) 9th Annual QPPV Forum

This is the only forum designed for QPPVs by QPPVs, now in its 9th year and ever growing. Over time, one of the key successes of the Forum has been the ability to secure continuing support and involvement of key regulators. Sessions have been open and interactive with attendees appreciating opportunities to raise challenging issues in an informal environment. This 9th QPPV Forum aims to continue to attract such key speakers and encourage open debate.

26-27 OCT LONDON, UK I #15111Clinical Trials Workshop

The new EU Clinical Trials Regulation is expected to become applicable in 2016. The new legislation will have implications on clinical trial sponsors preparing and submitting clinical trial applications. Member States will have to adapt their procedures and the new Regulation will impact how the conduct of a clinical trial is managed after approval has been granted. New provisions for public access to an EU Clinical Trials Database will enforce disclosure of clinical trials data and information. This 2-day workshop will provide a forum for information exchange and discussion on conceptual and practical questions through lectures and panel discussions.

4-5 NOV LONDON, UK | #15115Biosimilars Conference – European view on biosimilars with a global outlook

This 2-day conference is the third of its kind. It will provide an update on the current status for biosimilars in EU and internationally with focus on both regulatory and scientific challenges as well as market access and experiences. Patients and physicians approach to use of biosimilars will be part of the conference scope including a discussion on biosimilars adoption into current treatment guidelines in EU. The conference will consist of plenary lectures followed by interactive panel discussions providing participants an opportunity

NOVEMBER/DECEMBER I #1511016th Conference on Electronic Document Management

Join to find out about the latest trends in document management, content and information management, electronic submissions and a lot more.

This conference is currently in development and more information will be available shortly.

10-11 NOV LONDON, UK | #15106 9th European Medical Information and Communications Conference

This is a unique conference organised by medical information professionals for medical information professionals. The speakers share hands-on experience of dealing with current challenges as well as successes. It provides opportunities to showcase success stories or stories to learn by, in the popular Putting Theory into Practice session and to explore the impact of new technologies on information delivery and customer interactions. A dedicated poster session will also provide an opportunity to broaden the topics at the conference to other areas.

Call for Abstracts is open and can be submitted online at www.diahome.org/Get-Involved/Submit-Abstracts

17-18 NOV MIDDLE EAST LOCATION I #1510211th Middle East Regulatory Conference (MERC)

This is the 11th DIA Middle East Regulatory Conference (MERC) in partnership with the Middle East Regulatory Network (MERN). This Conference marks an important milestone – it will celebrate the 20 year anniversary of MERC and will reflect on the significant progress made over that period. It provides a forum for all participants to contribute to active discussion and identify actions to expedite access of valued innovative medicines to Middle Eastern patients. This year’s conference will explore advances in regulatory science and discuss the benefits of regulator collaboration & convergence. It will ask how this can be put into practice to meet the challenges of assessing innovative medicines.

20166-8 APR HAMBURG, GERMANY | #1610128th Annual EuroMeeting

Solutions are found through exchange of knowledge between professionals involved in therapeutic innovation and regulatory science. Attend the DIA EuroMeeting 2016 and join us in advancing the future of world health! DIA’s European flagship conference is the place to meet and learn what will be hot in 2016. Choose from 12 themes, 100 sessions and tutorials, and visit one of the largest exhibition halls in Europe, where you will find innovation on every corner, and benefit from countless opportunities to network with thought leaders from industry, academia, and regulatory agencies.

26-28 MAY EDINBURGH, UK I #161068th European Conference on Rare Disease & Orphan Products (ECRD 2016) – “Game changers in rare diseases”

This biennial conference is the unique forum across all rare diseases, across all European countries, bringing together all stakeholders.ECRD covers research, development of new treatments, healthcare, social care, information, public health and support at European, national and regional levels. ECRD provides the state of the art of the rare disease environment, monitoring and benchmarking initiatives.


CONFERENCES & WORKSHOPS

Monday, 13 April 2015 Monday, 13 April 2015 Monday, 13 April 201509:00-12:30 Pre-conference Tutorials 09:00-12:30 Pre-conference Tutorials 09:00-12:30 Pre-conference Tutorials

11:00-12:30 HAS Satellite Session | 13:30-15:00 Regulatory Town Hall Meeting | 15:00-16:00 Refreshment Break in the Exhibition Halls | 16:00-17:45 Plenary Session 11:00-12:30 HAS Satellite Session | 13:30-15:00 Regulatory Town Hall Meeting | 15:00-16:00 Refreshment Break in the Exhibition Halls | 16:00-17:45 Plenary Session 11:00-12:30 HAS Satellite Session | 13:30-15:00 Regulatory Town Hall Meeting | 15:00-16:00 Refreshment Break in the Exhibition Halls | 16:00-17:45 Plenary Session

18:00-20:30 EuroMeeting Welcome to Paris Opening Reception on Level 1 18:00-20:30 EuroMeeting Welcome to Paris Opening Reception on Level 1 18:00-20:30 EuroMeeting Welcome to Paris Opening Reception on Level 1

Theme 1 Theme 2 Theme 3 Theme 4 Theme 5 Theme 6 Theme 7 Theme 8 Theme 9 Theme 10 Theme 11 Theme 12 Theme 13

Access to Innovative TreatmentsMedical Devices and

Combination ProductsAdvanced Therapies, Novel

Treatments for Rare Diseases

Early Dialogue with Regulators and HTA Bodies

on Innovative Medicines

Collaborative Consortia, IMI and the Way to Horizon 2020

Implementing Innovation – Globally Conducive Regulatory Policiess

Special Development Pathways in Paediatrics, the Elderly and in

Pregnancy

Translational Medicine and Regulatory Science

Big Data, Mobile Health Innovation in Vaccines

DevelopmentAvailability of Medicinal

Products

Pharmacovigilance in 2015 - Poised for convergence of

innovation

Hot Topics/Stand Alone Sessions

Tuesday, 14 April 2015 Tuesday, 14 April 2015 Tuesday, 14 April 2015

Session 109:00-10:30

Session 0101The Role of the New Clinical

Trial Legislation

Session 0201New Medical Device

Regulations in the EU

Session 0301Approval Process and Experience

to Date/Challenges and Successful Outcomes of ATMP Development

Session 0501Strengthening Collaborative Innovation and

Maximising Mutual Benefit

Session 0601ICH, IPRF, ICMRA: How are these initiatives advancing regulatory harmonisation and

convergence?

Session 0701What Will the Future Bring – Innovative

Paediatric Development

Session 0901eHealth: Putting information to work

Session 1001Adaptive Pathways and Patient Access Models to Innovative Treatments: Is it

applicable to the development of vaccines?

Session 1101Some Basics about Shortages of

Medicinal Products: Concepts, causes, situation and trend

Session 1201Pharmacovigilance Debate: Brave

new world of decision support

Blue Room Level 2 Room 241 Level 2 Room 251 Level 2 Room Havane Level 3 Room Maillot Level 2 Room 252 Level 2 Room 242 Level 2 Room 243 Level 2 Room 253 Level 2 Room Bordeaux Level 3

Session 211:00-12:30

Session 0202Impact of the Regulations in Practice

across Regions

Session 0302Quality Aspects for an ATMP

(m3 of MAA)

Session 0402Regulatory Evidence Standards: Are

we maximising value generation?

Session 0602Asia: Creating possibilities for clinical

development and collaboration

Session 0702How Will the Global Needs of Paediatric

Development be Addressed?

Session 0802The Role of Translational Medicine in the

Future of Life Science

Session 1002Vaccine Development across

Regions

Session 1102Shortages Consequences: Their impact in patients, health care

professional and regulators

Session 1202Pharmacovigilance: How well are

patients informed and how can they contribute?

Session 1302Essential Business Change

Management in Pharmacovigilance

Room 241 Level 2 Room 242 Level 2 Room Havane Level 3 Room Maillot Level 2 Room 252 Level 2 Room 251 Level 2 Room 243 Level 2 Room 253 Level 2 Room Bordeaux Level 3 Blue Room Level 2

Session 314:00-15:30

Session 0103Clearing the Path for Innovations

in Drug Development Session 0303

Adaptive Licensing for ATMPs

Session 0403Shaping European Early Dialogues:

The SEED project

Session 0503Minimising Costs, Hurdles, and Duplication

Session 0603China Regulatoy Session: Updates from the CFDA

Session 0703Modelling and Simulation in Paediatrics: The

next Step- keeping the momentum

Session 0803Is the Classification of Disease on Par with

Therapeutic Innovation?

Session 0903Going Mobile: Disruptive technology

at your fingertips

Session 1103Solving and Preventing Shortages/ Communication between Different

Stakeholders

Session 1203International Collaborations in Pharmacovigilance: Common

objectives, common challenges

Blue Room Level 2 Room 243 Level 2 Room Havane Level 3 Room 241 Level 2 Room Maillot Level 2 Room 252 Level 2 Room 251 Level 2 Room 242 Level 2 Room 253 Level 2 Room Bordeaux Level 3

Session 416:00-17:30

Session 0104Primary Factors for Trial Location

Session 0204Impact of the Regulations on Substance-

Based Medical Devices

Session 0404Lifecycle of Regulatory/HTA Advice

Session 0504In Between: SMEs Perspective on PPPs

Session 0604China: Opportunities and challenges for

innovation

Session 0804The Risk-Based Approach for New Medicines Development: From ATMPs to "conventional"

drugs

Session 0904Big Data: Moving beyond buzz

words to results

Session 1004New Avenue for Non-Clinical

Regulatory Science

Session 1104Looking at the Future of Shortages

of Medicinal Products

Session 1204Benefit/Risk- pragmatic lifecycle management

Blue Room Level 2 Room 241 Level 2 Room Havane Level 3 Room 252 Level 2 Room Maillot Level 2 Room 251 Level 2 Room 242 Level 2 Room 243 Level 2 Room 352B Level 3 Room Bordeaux Level 3

Wednesday, 15 April 2015 Wednesday, 15 April 2015 Wednesday, 15 April 2015Theme 1 Theme 2 Theme 3 Theme 4 Theme 11 Theme 6 Theme 7 Theme 8 Theme 9 Theme 10 Theme 12 Theme 13









Pregnancy



Development


innovation


Session 509:00-10:30

Session 0105Impact of The Clinical Trial Regulation

on CMC Filing and Relevant GMP Requirements

Session 0205New IVD Regulations in the EU

Session 0305ATMP Regulations - ongoing review

Session 0405Patient Involvement in the

Development Dialogue with Regulatory Authorities and HTAs

Session 0505Developing Best Practice Guidance on Patient

Engagement in Medicines R&D

Session 0605PMDA Updates: Discussion of latest plans and

new directions in regulation – Town Hall

Session 0705The Silver Generation- how does development

address their needs?

Session 0805Diagnostic and Pharmacodynamic Biomarkers

and Mass Spectrometry Assays Thereof: Current status and perspectives

Session 1205Is it Off-Label Use or

Medication Error?

Session 1305Pharma Medical Affairs:

Vision though 2020

Room 251 Level 2 Room 242 Level 2 Room 253 Level 2 Room Bordeaux Level 3 Room 241 Level 2 Room Maillot Level 2 Room 252 Level 2 Room 243 Level 2 Blue Room Level 2 Room Havane Level 3

Session 611:00-12:30

Session 0106MAPPs- Holding hands across

the lifecycle

Session 0306Moving from Risk Management to

Benefit/Risk Management Planning

Session 0406One-Step Approach in Pricing, HTA

Appraisal and Reimbursement

Session 0506Collaborative Innovation in Biomedicine Serving Industry, Academy and Society

Session 0706How to Overcome Challenges in the

Development of Medicines to Treat Dementia

Session 0806Classification of Medicinal Products -

factors affecting regulators decisions, is harmonisation possible and necessary?

Session 0906Social Media, Patients and Public

Policy

Session 1006Adjuvants in Vaccines

Session 1206Managing the Risk or Managing the Plan?

Session 130650 Years of EDQM

Blue Room Level 2 Room 253 Level 2 Room Havane Level 3 Room 243 Level 2 Room 252 Level 2 Room 251 Level 2 Room 241 Level 2 Room 242 Level 2 Room Bordeaux Level 3 Room Maillot Level 2

Session 714:00-15:30

Session 0107MAPPS: Fostering Collaborative Models

for Clinical Development

Session 0207Health Apps: Definition, regulation & use

Session 0407HTA and Adaptive Pathways

Session 0507A Participatory Society for Better Health

Session 0607From the Shop Floor: Practical advice on

bringing new medicines to patientss

Session 0807Translating Regulatory Approval into

Healthcare Uptake: The next challenge for biosimilars

Session 0907Who Owns My Health Data?

Session 1007BIOVACSAFE (IMI Project)

Session 1207Signal Detection and Management:

Can you whistle?

Blue Room Level 2 Room 242 Level 2 Room Havane Level 3 Room 253 Level 2 Room Maillot Level 2 Room 251 Level 2 Room 241 Level 2 Room 243 Level 2 Room Bordeaux Level 3

Session 816:00-17:30

Session 0108Disclosure of information

Session 0208Clinical, Quality & Registration Considerations for Biologics

Combination Products Development

Session 0308Challenges for Development

of Rare Diseases

Session 0508Panel Discussion with Public-Private

Partnership Stakeholders

Session 0608Emerging Markets Update: What on Earth is

going on with regulator convergence?

Session 070850 Years after Thalidomide –

developing drugs in pregnancy

Session 0808Regulatory Science- new parnerships

Session 0908Real-World Evidence for Medicines

Adaptive Pathways to Patients (MAPPS)

Session 1008New Developments, Ebola Vaccines

Session 1208PRAC: Highlights, challenges

and Referrals
















Pregnancy




Products


innovation



Session 109:00-10:30


Trial Legislation








convergence?











Session 211:00-12:30


across Regions


(m3 of MAA)










Regions








Session 314:00-15:30





The SEED project








at your fingertips


Stakeholders




Session 416:00-17:30







innovation


drugs


words to results


Regulatory Science














Pregnancy



Development


innovation


Session 509:00-10:30
















Medication Error?


Vision though 2020


Session 611:00-12:30


the lifecycle











Policy





Session 714:00-15:30













Can you whistle?


Session 816:00-17:30





of Rare Diseases












and Referrals
















Pregnancy




Products


innovation



Session 109:00-10:30


Trial Legislation








convergence?











Session 211:00-12:30


across Regions


(m3 of MAA)










Regions








Session 314:00-15:30





The SEED project








at your fingertips


Stakeholders




Session 416:00-17:30







innovation


drugs


words to results


Regulatory Science














Pregnancy



Development


innovation


Session 509:00-10:30
















Medication Error?


Vision though 2020


Session 611:00-12:30


the lifecycle











Policy





Session 714:00-15:30













Can you whistle?


Session 816:00-17:30





of Rare Diseases












and Referrals




17-18 SEP LONDON, UK | #15552Paediatric Investigation Plans (PIP)This course will provide a full introduction to PIPs and the EU Paediatric Regulation.

21-23 SEP VIENNA, AUSTRIA | #15536Medical Devices: Regulation and lifecycle managementAll you need to know about medical devices, if you are not working with devices on a daily basis or your need an overview.

14-15 OCT LONDON, UK | #15546How to Prepare for your Meeting with Health AuthoritiesThis course covers Health Authority (HA) meetings and other interactions in the EU and the US. You will learn by performing role plays yourself and by many case studies.

25-27 NOV VIENNA, AUSTRIA | #15545Biopharmaceuticals, Biosimilars and Advanced Therapies in Europe - Development and Regulatory Framework in EuropeParticipants will learn about the legislative and regulatory framework for biopharmaceuticals in Europe and the roles of the European Medicines Agency and National Competent Authorities in market access.

NEXT OCCURANCE TO BE ANNOUNCEDComprehensive Training on European Regulatory Affairs including Different Registration Procedures and Variations: Expert OverviewThis is a complete comprehensive in-depth training on European regulatory affairs, exploring all aspects of medicinal product lifecycle management.

NEXT OCCURANCE TO BE ANNOUNCEDApproval of Generic Medicines in the EU. Focus on CMC requirements and bioequivalenceThe overall requirements for the approval of generics will be detailed including problems in relation to generic substitution and falsified medicines.

Safety and Pharmacovigilance

28-29 APR BASEL, SWITZERLAND | #15550How to Prepare for Pharmacovigilance Audits and InspectionsThe course will teach you how to prepare for an inspection from the announcement (or of the arrival of the inspectors at your doorstep) to the conclusion.

27-28 APR BASEL, SWITZERLAND | #15534 5-6 NOV PARIS, FRANCE | #15549Signal Management in PharmacovigilanceThe course will teach basic concepts of signal detection and signal management and how to apply them within the participants’ functions. 3-4 NOV PARIS, FRANCE | #15547Benefit/Risk ManagementThis intensive course explores current opportunities made possible by the new legislation, advances in information technology and a new scientific methodology to enhance and modernise the approaches in the product lifecycle management.

NEXT OCCURANCE TO BE ANNOUNCEDExcellence in Pharmacovigilance: Clinical trials and post-marketing By attending this course you will gain a firm grounding in key aspects of safety and pharmacovigilance from a European and global standpoint.

Chemistry, Manufacturing and Controls21-23 SEP VIENNA, AUSTRIA | #15543Quality by Design - New concepts for chemical and biotech product development and optimisationParticipants from pharmaceutical, biotech and generic industry as well as regulators will learn how to use QRM, Process Characterisation, DoE, Development of a Design Space and Control Strategy, as well as the tools of Knowledge Management (KM).

Clinical Research

18-20 MAY LONDON, UK| #15557Essentials of Clinical Study ManagementAfter successful completion of the training course, partici-pants will be able to plan, execute and manage a clinical study from protocol to final report.

16-18 SEP LONDON, UK | #15542Clinical Project Management – Part IThis course provides a comprehensive foundation in clinical project management. Using the Project Management Body of Knowledge (PMBOK®) as a guide, participants will be taught how to apply project management strategies, tools and techniques to their clinical trial projects.

16-18 NOV LONDON, UK | #15555Clinical Project Management – Part IIThis is the second part of the 2-part course on Clinical Project Management, concentrating on specific concepts such as project quality risk management.

14-16 OCT LONDON, UK | #15531 Practical GCP Compliance Auditing of Trials and SystemsThis course provides practical training for industry auditors and regulatory authority inspectors, who are faced with the task of auditing clinical trials and related systems. It will also be of interest to those with managerial responsibilities.

15-16 OCT LONDON, UK | #15532Clinical Statistics for Non-StatisticiansThis course is an introduction of basic statistical concepts fundamental to clinical research, for professionals who have regular exposure to statistics.

Regulatory Affairs

4-5 NOV PARIS, FRANCE | #15556Essentials of European Regulatory Affairs This is the must-attend training course for anyone needing to learn the essentials of European Regulatory Affairs.

16-18 NOV LONDON, UK | #15554US Regulatory Affairs: A comprehensive review of regulatory procedures for INDs and NDAs in the US This course is designed for persons with a background in pre-clinical research, clinical research, quality assurance or academia, who need knowledge of the US regulatory processes.

9-10 NOV DUBAI, UNITED ARAB EMIRATES | #15562Building the eCTD - Practical Approaches to compiling Electronic SubmissionsParticipants will learn about the Electronic Common Technical Document (eCTD), its components and history, preparing submission ready documents, and how to change your busi-ness processes in preparation for moving towards electronic submissions.

6-7 MAY AMSTERDAM, THE NETHERLANDS | #15551HTA for Non-HTA userA foundation course designed especially for non-HTA experts interested in gaining a better insight into the role of HTA in decision making processes in the health care sector.

TRAINING COURSES

Discover new opportunities at diahome.org. Call DIA offices at +41 61 225 51 51 or [email protected]


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IN-HOUSE TRAINING

All DIA Training Courses can be held on your premises and tailored to your needs.

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2 0 1 5

26 APR DAKAR, SENEGAL | #15559ICH Endorsed PharmacovigilanceParticipants will gain solid knowledge and a clear understanding of international approaches to drug safety pharmacovigilance, as well as the best practices for successful local and global regulatory applications.

18-19 MAY LONDON, UK | #15539Pre-Marketing Clinical SafetyParticipants will be guided through all the regulations and guidelines pertinent to pre-marketing safety in the EU. The course offers an overview of all the current major methodological approaches and hands-on solutions for day-to-day challenges.

10-11 JUN PARIS, FRANCE | #15540Medical Approach in Diagnosis and Management of ADRs How to use medical knowledge in the diagnosis and management of selected Adverse Drug Reactions (ADRs).

11-12 JUN PARIS, FRANCE | #15544Diagnosis and Management of Drug-Induced Liver Injury (DILI)This course will provide tools, explanations, examples and several exercises for a better understanding of DILI and how best to apply that knowledge in day to day work.

21-22 SEP BERLIN, GERMANY | #15535Post-Authorisation Safety Studies (PASS)This course delivers insight in (pharmaco-)epidemiological methodology for non-interventional studies, and the concept of multi-departmental collaboration for the development and conduct of a PASS.

YEAR-ROUND EMA HEADQUARTERS, LONDON AND SELECTED EUROPEAN CITIES

EudraVigilance Courses and EMA Information Days

EudraVigilance — Electronic Reporting of ICSRs in the EEA

eXtended EudraVigilance Medicinal Product Dictionary 28 APR LONDON, UK | #15522Information Day on New Services and Systems in Pharmacovigilance: Preparing for Business Change 29 APR LONDON, UK | #15521ICSR Information Day 1 JUN LONDON, UK | #15523Risk Management Plan Information Day

5 JUN LONDON, UK | #15524Information Day on Post-Authorisation Studies (PAS)

23 JUN LONDON, UK | #15525ISO IDMP Information Day

Combo Pricing*

Combo Pricing*

28th AnnualEuroMeeting

CCH Congress Center Hamburg

6-8 April 2016

TRAINING COURSES

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27th Annual EuroMeeting

Documents