A Technical - Europa · 2016-05-26 · d. Support to capacity‐building activities of patient...

OPERATING GRANT FOR RARE DISEASE ASSOCIATIONS EURORDIS FY2011

Con t ra c t 20103205 F i n a l Re p o r t

A – Techn i ca l Imp l emen ta t i o n Repo r t

EURORDIS OPERATING GRANT EURORDIS_FY2011 N°2010 32 05

FINAL REPORT

A. TECHNICAL IMPLEMENTATION REPORT

TABLE OF CONTENTS

A. TECHNICAL IMPLEMENTATION REPORT

1. Executive Summary

2. “Qualitative evaluation of the execution of all tasks mentioned in Annex I of Grant

Agreement 2010 32 05”:

2.1. Introduction to Activity Tables 2.1.a. Table of Correspondence

2.2. Achievement of Strategic Objectives and List of Activities for 2011 (Activity Tables)

A. Communication to patients organisations, stakeholders and the public

a. EURORDIS website and electronic newsletter: 1. EURORDIS website 2. Electronic Newsletter

b. Preparation of ECRD 2012 c. Update of reference documents on RD, reports, brochure; translations d. RDD 2011 and media monitoring service. Preparation of RDD 2012 e. Awareness raising through patient stories, video & photo contest f. Maintenance of EURORDIS RD community databases g. International dialogue h. Promoting RD as an international priority

B. Capacity building for patient representatives: 1. Health policy, POs networking and empowerment

a. & b. Outreach to POs and members, dissemination of information, consultation of members, focus on new Member States incl. Fellowships

c. Support to CNA; 2 Workshops; involvement in public awareness (RDD) and empowerment on RD national strategies

d. Support CEF through dissemination and sharing good practices; 1 two‐day Workshop; awareness and empowerment on EU policies

e. Support to volunteers representing EURORDIS in EU committees f. Fact Sheets for capacity‐building purposes on RD policy aspects

2. Research and Therapeutics Development

a. Patient involvement in EMEA activities 1. Support to the participation of patient representatives in the EMA Committees (COMP,

PDCO, CAT, PCWP) and in Protocol Assistance 2. Support to the participation of patient representatives in the EMA committees (PCWP)

and implementation of the new legislation on Pharmacovigilance 3. Supporting the EURORDIS Therapeutic Advisory Group (TAG) 4. Monthly reports compiling feedback from each Committee

b. Review and validation of public information on RD therapies disseminated by EMA (PSOs, EPARs, PLs)

c. Supporting the involvement of more RD patient representatives in the OD, PD, ATMP policies and in drug development through:

1. Support to EURORDIS Task Forces on Orphan Drugs, Paediatrics, Drug Information, Transparency & Access (DITA)

2. Summer School 2011 3. e‐Learning on specific and advanced aspects of drug development, clinical trials and

regulatory affairs d. Support to capacity‐building activities of patient advocates in HTA e. Supporting good practice relations between POs & sponsors on RD clinical trials based on

EURORDIS Charter on Clinical Trials

2.3. Deliverables (outputs) 2.4. Indicators (metrics) 2.5. List of seminars, conferences, etc. indicating the organiser, the number of participants, etc.,

and the travel and subsistence costs associated in connection with the implementation of

the programme of activities

1. EXECUTIVE SUMMARY

The Operating Grant “EURORDIS FY2011 – n°2010 32 05” was granted to the European Organisation for

Rare Diseases (EURORDIS) for the third time in 2011. Throughout the year, the Operating Grant

significantly contributed to the implementation of its Strategy 2010-2015 and the achievement of its

Work Plan 2011 as approved by its members at its annual General Assembly meetings. The Operating

Grant of EURORDIS covers its recurrent core activities with the exceptions of those in the area of

governance and advocacy activities, as in previous years. In addition, recurrent activities have not been

included due to the lack of funding from Operating Grant FY2011. These activities are: Rare Disease Help

Lines, online patient communities (“RareConnect”), respite care services and therapeutic recreational

programmes, described in our 2009 and 2010 Final Reports in the section called “Support and

information services to patients”.

The following Report on the technical implementation of the beneficiary’s Work Programme and the

Financial Statements referred to Art. I.4 and I.5 of the Grant Agreement1 aim to show how the

activities have been performed and resources allocated. All the activities listed in the Description of

Work (page 26, Annex I of Grant Agreement, “Summary of the organisation’s work programme for

2011”, as amended following EURORDIS request sent on October 21st, 2011) and all the Deliverables

(page 27 of the Grant Agreement) have been achieved in due time and according to the contract and its

amendment.

In 2011, the Operating Grant allowed for the development of areas which consolidated and expanded

since 2009, when the first Operating Grant was awarded. This growth in recurrent core activities stems

from EURORDIS’ progressively increasing outreach to patient associations and communities, successful

public awareness raising, as well as a growing number of technical activities in which to involve patient

representatives so to turn the new EU and national rare disease policy frameworks into reality. This is

the case of the communication activities revolving around the Rare Disease Day 2011; the outreach to

patients and patient organisations and their empowerment; the support to the activities in the

therapeutic area; support to the involvement of volunteers in EU and national policy working groups;

the support to international activities.

1 Grant Agreement 20103205 between the European Organisation for Rare Diseases (EURORDIS) and the Executive

Agency for Health and Consumers (EAHC).

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 1. Executive Summary 2011 - Page 1

Notwithstanding, in 2011 EURORDIS continued to restrain its activities in other sectors to take account

the reduction of the Operating Grant awarded in 2011 and confirmed in 2012 with a further slight

decrease. This reduction affected primarily two areas: the research policy activities (the planned hire of

a Research Policy Manager is still on hold until after 2011) and the support & information services for

patients, including Respite Care Services, Therapeutic Recreational Programmes and the European

Network of Help Lines (following the departure of the Health Policy Project Coordinator, Shane Lynam,

in charge of the development of these services, no replacement could be made because of the

Operating Grant reduction of last year.

Altogether, this confirmed the great importance of the Operating Grant in support of the recurring core

activities of EURORDIS and therefore its direct impact on the operations of the organisation. Overall, the

Operating Grant proved to be once again instrumental to fulfill the Mission and to reach the General

Objectives of EURORDIS, as described in the Grant Agreement (respectively pages 24 and 25); and it

allowed the organisation to continue to play its pivotal role in the definition and implementation of the

EU strategy on rare diseases (European Commission Communication on Rare Diseases, December 2008;

Council Recommendations for actions in the area of rare diseases, June 2009; the recommendations of

the EU Committee of Experts on Rare Diseases; the Commission Roadmap for the implementation of the

Communication and Recommendation on Rare Diseases).

The qualitative evaluation of the execution of all tasks mentioned in Annex I of the Grant Agreement

is reported in the Section A.2 of this Report. In each of the tables describing therein the performed

activities in detail, the analysis of the results achieved is highlighted in a specific box.

For the Strategic Objective “Communication and information to patient organisations, stakeholders

and the public”, EURORDIS website, revamped in 2010, has been developed further in 2011 and

achieved a greater turnover of information. New sections were created and/or tailored to the needs of

Rare Diseases Policy in Europe, such as, for example, a new section on National Plans, the establishment

of online training and e-learning resources for patient advocates or the translation of the orphan drug

section in 6 languages. These improvements translated into a steady average of over 18 000 visitors per

month and a rapidly growing use of related social media.

The organisation of the Rare Disease Day 2011 “Rare but Equal” was another EURORDIS key activity,

which brought rare disease stakeholders together to discuss the future of rare disease research as well

as bringing closer researchers, patient groups and policy makers. It involved 55 participating countries

(25% more than in 2010); the campaign website had over 49 000 hits from 150 countries and the media

outreach was strong, with hundreds of news articles collected through a media monitoring service;

remarkably, around 1000 events were organised all over the world. A European Symposium “Rare but

Equal - Addressing Health Inequalities for Rare Disease Patients in Europe” was held in Brussels and

gathered nearly 100 attendants. The successful video and photo contests have been organised again

this year around the Rare Disease Day, as two additional tools to raise public awareness.

10 issues of the electronic newsletter were published in 6 languages (English, French, German, Spanish,

Italian and Portuguese) throughout the year. In November, a 7th language, Russian, was added, while a


landing page in Russian has been added to the EURORDIS website. The subscription rate went up 4%,

with an increased number of subscribers up to 8189, still gathering our main audience of patient

organisations. The presentation brochure, revamped in 2010 in English and French, has been published

in 5 additional languages, including Russian. Moreover, a “Become a Member” booklet was produced in

7 languages.

The database of EURORDIS RD community has reached 5405 contacts in 4146 organisations, including

1656 patient organisations, including 492 members of EURORDIS, 23 projects and 60 events. The

remainder are mostly members of either National Alliances or European Federations, and some still

being isolated groups. This database is updated every week and the list of members is interfaced with

EURORDIS website.

The organisation put in place to support EURORDIS’ volunteers has been strengthened. This concerns

volunteers representing EURORDIS in key European Medicines Agency (EMA) and European Commission

(EC) Committees, internal EURORDIS committees and task forces, as well as volunteers and staff

involved in the implementation of the EU pharmaceutical legislations, the EC Communication and

Council Recommendation on Rare Diseases, the EUCERD recommendations and their translation into

national policies. A similar organisation is also in place to support staff and volunteers in fulfilling

obligations arising from partnerships with 6 European and international not-for-profit organisations

working on transversal issues relevant for rare disease patients (NORD, CORD, EPPOSI, ICORD, DIA, EPF).

Of particular note, EURORDIS provided advice on how to organise a US conference on rare diseases and

orphan drugs, based on the model of the European conference on rare diseases and orphan products

(ECRD). The conference eventually took place on 11-13 October 2011.

Finally, at the international level, EURORDIS resumed the work towards the drafting of a Joint

Declaration on Rare Diseases as a Public Health Priority. Extensive consultations took place with experts

and stakeholders from all continents. As a result, an advanced draft of the Declaration was ready by the

end of 2011.

In order to reach the Strategic Objective “Capacity-building for patient representatives”, two main

Objectives have been identified:

“Health policy, POs networking and empowerment”. In 2011, EURORDIS strengthened its membership

with 50 new members, thus reaching 492 total member organisations. It also strengthened its outreach

to patient organisations and members, by consolidating its activities which link up European and

national policies and providing support to members in implementing national policies on rare diseases.

Last year, approximately 1000 person/days from EURORDIS membership were mobilised to participate

to events, conferences, trainings and workshops, thus building their capacity and acquiring skills. The

European Network of 25 Rare Diseases National Alliances has been consolidated and two European

Workshops of the Council of National Alliances were organised, mostly focused on the organisation of

Rare Disease Days 2011 and 2012, on the development of National Plans for Rare Diseases, the analysis

of the outcomes of the 15 National Conferences on Rare Diseases in the framework of the EUROPLAN

project and the preparation of a second series of EUROPLAN conferences in 2012-2014 onwards under


the EUCERD Joint Action on RD. Last year, EURORDIS also supported new patients groups in Russia,

Armenia and Georgia to organise international rare diseases awareness-raising conferences in their

respective countries.

The European Network of 35 Rare Disease European Federations or Informal Networks held one

European Workshop of its Council of European Federations, focused on the new EU legislation on cross-

border healthcare, the Online Patient Communities developed by EURORDIS and NORD, the

presentation of the new EU clinical trials online register and the organisation of the Rare Disease Day

2011. The meeting was followed by a one-day capacity-building session on clinical trials. EURORDIS

continued its pilot “Grant Programme for European Federations” funding with it 7 meetings of European

Rare Disease Federations; the pilot programme is still greatly appreciated by European Federations as an

effective and flexible instrument to support and leverage their activities. One additional Policy Fact

Sheet on rare disease related topics was developed in 2011, whereas the whole set of existing Fact

Sheets was widely distributed to members and at relevant events. Fact Sheets are aimed to provide

member organisations and their representatives with advocacy tools to encourage them to implement

the key recommendations of EU rare disease policy and facilitate their transposition into national plans

on rare diseases.

For the Objective “Research and Therapeutic Development”, EURORDIS continued its support to the

participation of patient representatives in the Committees (COMP, PDCO, CAT, PCWP) of the European

Medicines Agency (EMA), cumulating 142 days of meeting and 377 scientific dossier examined; the

Therapeutic Action Group (TAG), composed of all EURORDIS representatives in these Committees and

support staff, held monthly conference calls and organised a face-to-face meeting in November, right

after the Board of Directors meeting. 11 monthly Therapeutic Activity Reports were produced on the

activities of the EMA Committees and EURORDIS’ patient representatives in these Committees; they

were disseminated to all TAG members, Task Forces, EURORDIS Board and participants to EURORDIS

Summer School.

Another major activity was the coordination of the three Task Forces involving 34 patient

representatives and volunteers, from different rare diseases and different EU Member States, trained

and active in issues concerning Orphan Drugs, Paediatric Drugs and Drug Information & Transparency &

Access (DITA). The DITA Task Force held two Workshops in 2011, whereas the Paediatric and Orphan

Drugs TF only had conference calls and email exchanges, in preparation of 2012 when they will be

turned into groups of volunteers for consultation. The DITA TF held discussion with and provided advice

to the EMA on request of the Agency on topics such as its new policy on conflict of interest, its

pharmacovigilance database (Eudravigilance) and the EU clinical trials register.

EURORDIS is also extensively involved in the EMA’s activities related to the provision of information to

patients and the public about medicines authorised via the centralised procedure. A total of 131 EMA

dossiers for public information, including 111 Public Summaries of Opinion of Orphan Designations

(PSOs), 8 European Public Assessment Reports (EPARs) and 12 Package Leaflets (PLs), were reviewed by

EURORDIS staff members in order to ensure the quality of the information disseminated by the Agency

to the general public.


The EURORDIS Summer School, a four-day session on drug development, clinical trials and EU regulatory

process, is now a consolidated and consistently successful activity and has been extended last year to 43

selected volunteers. The 2011 edition focused on extending the competence of advanced patients’

advocates using formal presentations of the topics coupled with hands-on case studies from various

committees at the EMA, orphan drug designation dossiers from the Committee for Orphan Medicinal

Products and protocol assistance. An e-learning tool in English has been established based on the topics

discussed at the Summer School using different learning methods and covering methodologies, design,

statistics and ethics of clinical trials. Furthermore, three more patient’s advocates benefited from the

training opportunity on HTA last year in April, when they attended the four-day HTA Programme

organised by the UMIT University in Hall-im-Tyrol (Austria).

During 2011, EURORDIS continued its engagement to implement its “Charter for Clinical Trials in Rare

Diseases”, which has been signed by 7 pharmaceutical companies. In the framework of the Charter,

which aims to regulate the relationships between clinical trial promoters and the patient organisation

concerned by their studies, the first two Agreements of Understanding have been signed up.

Altogether, 2011 has been an important year for the consolidation of the role of EURORDIS staff and

volunteers in regulatory committees at the EMA and of the network of support provided by the TAG and

Task Forces. Capacity building for patients’ representatives was also strengthened thanks to consistently

successful activities such as the Summer School for patient advocates, workshops and trainings for

EURORDIS Networks (CNA, CEF), online learning tools and fact sheet developments.

Based on the initial assessment of potential risks, none has emerged over the year.

A comprehensive set of activity indicators was developed and data were collected throughout the year.

These indicators appear in the description of each activity and in table 2.4 of this Report.

The list of seminars and conferences, the list of travel and subsistence costs and the published

material and website addresses with publicity of Community funded are annexed to this Report.

Altogether the implementation of the work programme played an essential role in the accomplishment

of EURORDIS strategic objectives and paved the way to significant developments in the organisation and

to the reflection which led to the definition of the Work Plan 2012 along the lines of the new Strategy

2010-2015.

The final financial statements and comments, based on the Grant Agreement and amendment, are

reported separately. The financial statements are accompanied by the audit certificate of Deloitte &

Associates.



2.1. INTRODUCTION TO THE ACTIVITY TABLES

The following Activity Tables represent the core part of the technical implementation report. They are

meant to provide a list of the activities performed by EURORDIS in 2011 in compliance with the

Operating Grant, as well as an overview of the execution of the tasks in the Work Plan 2011 is detailed

activity by activity.

The Activity Tables are indeed based on the structure of EURORDIS Work Plan 2010, which can be found

in Annex I, page 24 of the Operating Grant Agreement. They are therefore organised along the two

Strategic Objectives and, in the second one, two main Objectives laid out therein:

A. Communication and Information to patients organisations, stakeholders and the public

B. Capacity building for patient representatives

1. Health policy, POs networking and empowerment

2. Research and Therapeutics Development

Each Table corresponds to one specific Activity and includes:

Title

Responsible and Supervising Staff

Deliverable(s), i.e. the outputs associated to each activity. Of course, a number of activities have

been performed that are not associated to a specific OPERA Deliverable. In any case, the full list

of Deliverables is provided in this report (section 2.3).

Indicators, which correspond to the metrics associated to each activity. Again, a table with the

overview of all Indicators is provided in section 2.4 of this report.

A Calendar of activities, summarising the main dates of the achievements relevant to the

concerned activity.

A qualitative Description of the Activity, which explains how the activity has been performed.

The Results achieved with each activity, described separately in order to highlight what has

been accomplished thanks to the Operating Grant by the end of the contract period.

Annexes, i.e. the published material related to the described activity, which is listed at the end

of the Activity Table in order to create a link between each activity and the material itself. The

latter is provided separately in the part C of this report, named “Contractual Annexes”.

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 2.1. Introduction to the Activity Tables 2011 - Page 7

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 2.1. Introduction to the Activity Tables 2011 - Page 8

Description of Work OPERA 2011

Description of Work 2011 Deliverables

A. Communication and information to POs, stakeholders and the public

A.a.1 EURORDIS Website DC D5 EURORDIS Website

A.a.2. Electronic newsletter PT D4 Electronic Newsletter

A.b. Preparation of ECRD 2012 SA

A.c. Update of reference documents on RD, reports, brochure; translations PT

A.d. RDD 2011 & media monitoring service. Preparation of RDD 2012 PT D9 Rare Disease Day 2011

A.e. Awareness raising through patient stories, video & photo contest JE‐PT

A.f. Maintenance of EURORDIS RD community databases PR D3 Update of EURORDIS RD community database

A.g. International dialogue AW

A.h. Promoting RD as an international priority FM

B. Capacity building for patient representatives:

B.1. Health Policy, POs networking and empowerment

B.1.a. +

B.1.b.

Outreach to POs and members, dissemination of information, consultation of members, focus on new MS incl. Fellowships

AH

B.1.c.Support to CNA; 2 Workshops; involvement in public awareness (RDD) and empowerment on RD national strategies

AH D2 Workshops of EU Networks

B.1.d.Support to CEF through dissemination and sharing good practices; 1 two‐day Workshop; awareness and empowerment on EU policies

AH D2 Workshops of EU Networks

B.1.e. Support to volunteers representing EURORDIS in EU committees AW

B.1.f. Fact Sheets for capacity‐building purposes on RD policy aspects PT‐FHz

B.2. Research and Therapeutics Development

B.2.a.1. Support to patient representatives in the EMA Committees and in Protocol Assistance MMD8 Participation in EMA Committees (COMP, PDCO, CAT, PCWP, PhVWP)

B.2.a.2.Support to patient representatives in the EMA Committees and WPs (PCWP) and implementation of the new legislation on Pharmacovigilance

FHzD8 Participation in EMA Committees (COMP, PDCO, CAT, PCWP, PhVWP)

B.2.a.3. Supporting the EURORDIS Therapeutic Action Group (TAG) MMD8: Participation in EMA Committees (COMP, PDCO, CAT, PCWP, PhVWP)

B.2.a.4. Monthly reports compiling feedback from each Committee MM

B.2.b.Review and validation of public information on RD therapies disseminated by EMA (PSOs, EPARs, PLs)

FHz

B.2.c.1.Support to EURORDIS Task Forces on Orphan Drugs, Paediatrics, Drug Information, Transparency & Access (DITA)

MM‐FHzD7 Meetings of the Task Forces Orphan Drugs, DITA, TAG, Paediatrics

B.2.c.2. Summer School 2011 MM D6 Summer School 2010 and online training tools

B.2.c.3. e‐Learning on drug development, clinical trials, regulatory affairs MM D6 Summer School 2010 and online training tools

B.2.d Support to capacity‐building activities of patient advocates in HTA MM

B.2.e.Supporting good practice relations between POs & sponsors on RD clinical trials basedon EURORDIS Charter on Clinical Trials

FB‐FHz

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 2.1.a. Table of Correspondance 2011 - Page 9

Activity A.a.1. EURORDIS website

Responsible and supervising staff

Responsible: Denis Costello Supervising: Yann Le Cam

Deliverable D4: Electronic Newsletter and EURORDIS website

Indicators Number of website visitors/month: approx. 18 891 Total number of website visits over the year: 226 692 Number of websites linking to EURORDIS website: 119 000 Number of Twitter.com/eurordis followers: 1152 Number of Facebook.com/eurordis likes: 2205 Number of Video views on YouTube.com/eurordis: 11 547

Calendar of activities Weekly Web Editorial Meeting. Monthly insertion of new articles in 6 languages. Regular updates of Homepage, Events calendar for both EURORDIS

Membership and the rare disease community. Regular Social Media updates via Twitter, Facebook & YouTube and

efforts to grow followers of each channel. September 2010 – Enhancements to Training Section of the website

with addition of e‐Learning and Videos from EURORDIS Summer School.

October 2010 ‐ Call for Tender to source developer to implement new features. Barcelona‐based development agency AteneaTech chosen to implement the following features: Update of software; Improved navigation; Addition of administrative features to grant greater autonomy

to Web Content Manager in creating new content; Addition of advanced search of EURORDIS membership by

disease area (linked to internal Contact Database see activity A.f).

November 2011 ‐ Addition of Russian Language homepage and monthly newsletter www.eurordis.org/ru

Description of the Activity The EURORDIS website provides information relating to the role of patient organisations in the development of rare disease and orphan drug policy in patient‐friendly language translated into 7 languages, while also outlining the activities provided by EURORDIS. During 2011 a new organisation of the workflow of publication was implemented which saw the EURORDIS Communications Director take the lead in managing the direction of content on the website, while the Web Communications Officer adopted a supporting role in providing the necessary technical enhancements.

Operating Grant EURORDIS n°20103205 Final Report 2.2. Activity Tables - Page 11

Additional language support for Russian was achieved in collaboration with the Russian Patients Union via a translated homepage and monthly newsletter. EURORDIS continues its commitment to rich media with the addition of regular video content to its YouTube channel, which is also included in relevant articles, as well as the addition of interactive e‐learning and on‐demand slidecast video content from the EURORDIS Summer Schools. The EURORDIS website is also an ideal platform to promote other relevant projects and activities such as: www.rareconnect.org, a social network of online patient

communities in 5 languages managed by EURORDIS; www.rarediseaseday.org (Activity A.d), an international awareness

day; and the European Conference on Rare Diseases and Orphan Products

(Activity A.b) www.rare‐diseases.eu. Each of these activities receives a prominent visibility on eurordis.org and as such receive traffic from eurordis.org.

Results achieved Weekly updates of the homepage and events calendar to reflect activities of EURORDIS and the rare disease community.

Regular updates and video content and growing social media community: twitter.com/eurordis & facebook.com/eurordis & youtube.com/eurordis

New website section providing information on e‐learning & videos from EURORDIS Summer School http://www.eurordis.org/training‐resources

Revamped section on National Policies and National Plans (final version online in early 2012).

Translation of the About Orphan Drugs section of the website into 6 languages (previously available only in English): http://www.eurordis.org/about‐orphan‐drugs

New website section to promote the Play Decide activity of the POLKA project: http://www.eurordis.org/playdecide

Promotion of RareConnect.org EURORDIS’ social network for rare disease patients via prominent presence throughout website

Addition of Russian homepage and monthly translation of newsletter www.eurordis.org/ru

Addition of new functionality allowing visitors to search for patient groups who are members of EURORDIS for a specific rare disease. This data is exported (daily) from the internal EURORDIS Contact Database (see Activity A.f)

Relevant Annexes EURORDIS website: www.eurordis.org EURORDIS Twitter feed: www.twitter.com/eurordis


EURORDIS Facebook page: www.facebook.com/eurordis EURORDIS YouTube channel: www.youtube.com/eurordis


Activity A.a.2. Electronic Newsletter


Responsible: Paloma Tejada Supervising: Yann Le Cam

Deliverable D4: Electronic Newsletter

Indicators Number of subscribers: 8189 Number of issues: 10

Calendar of activities Issues sent out in Jan, Feb, Mar, Apr, May, Jun, July, Oct, Nov, Dec

Description of the Activity In 2011, 10 monthly issues of the electronic newsletter were produced (no publication in August and September) in the 6 languages of the website (English, French, Spanish, Italian, German and Portuguese) and one additional languages (Russian) was added in November 2011. Three special e‐mailings in Russian were sent out to announce the new version of the Newsletter and to encourage Russian speaking rare disease patients and other stakeholders to subscribe. The e‐Newsletter was also promoted in key RD conferences in Russia. A landing page in Russian has been added to the EURORDIS website eurordis.org with basic information about EURORDIS in Russian; presentation brochure and Newsletter archive. Each issue of the e‐newsletter include the following sections: News, Insights, Report, Patient Profile, Learning From Each Other, designated Orphan Drug, authorised Orphan Drugs. Articles are published every month on: EU and international and RD and orphan drug policies; Progress on National Plans on RD in different EU Member States; Analysis of surveys and research activities; Case studies of RD patient advocates and patient organisations to

enhance exchanges of experience.

Results achieved The e‐Newsletter subscription rate went up 4 % in 2011 compared to 2010 (3671 English, 1249 French, 920 Spanish, 1097 German, 776 Italian, 395 Portuguese, 81 Russian), leading to a total of 8189 subscribers.

Articles from EURORDIS e‐Newsletter are often reproduced and reused by patient organisations in their own printed or electronic newsletters.

Relevant Annexes 1 example of e‐Newsletter (email)


Activity A.b. Preparation of ECRD 2012


Responsible: Sharon Ashton Supervising: Yann Le Cam

Deliverable No specific deliverable is associated with this activity

Indicators

Calendar of activities Q1 2011 ‐ Site inspections and selection of conference venue Q2 2011 – Programme Committee members appointed September 2011 ‐ 1st Programme Committee meeting held in Paris December 2011 ‐ 2nd Programme Committee meeting held in Paris November 2011 ‐ Official ECRD 2012 website launched in English December 2011 ‐ Official ECRD 2012 website translated into 6

languages (English, Spanish, French, German, Dutch and Russian) November 2011 ‐ Call for Poster abstracts launched November 2011 ‐ ECRD‐EURORDIS Patient Advocate Fellowship

programme launched

Description of the Activity The European Conference on Rare Diseases & Orphan Products is the unique platform/forum across all rare diseases, across all European countries, bringing together all stakeholders ‐ academics, health care professionals, industry, policy makers, patients’ representatives. It is a bi‐ annual event, providing the state‐of‐the‐art of the rare disease environment, monitoring and benchmarking initiatives. It covers research, development of new treatments, health care, social care, information, public health and support at European, national and regional levels. It is synergistic with national and regional conferences, enhancing efforts of all stakeholders. There is no competition with them, but efforts are complementary, fully respecting initiatives of all, rather aiming at integrating EU and national policies and actions. The ECRD 2012 Programme Committee started to work in 2011. It is composed of 14 members, of which 3 Co‐Chairs (Kate Bushby, EUCERD Vice Chair, Kerstin Westermark, COMP Chair, Avril Daly, EURORDIS Board Member). The Programme Committee members represent 12 different European countries, including 3 Central European countries. Advisors to the Programme Committee include 7 members representing 7 different European countries. Overall, the Committee and its Advisors represent 11 members of EUCERD, 9 members of COMP and OMPs, 1 ORPHANET member, 9 patient representatives. The outcomes of the first Programme Committee meeting in September 2011 included deciding upon the conference themes and appointing the Theme Leaders to develop the programme sessions. This is the first time ECRD has appointed Theme Leaders. Theme Leaders are selected from among the Programme Committee members and advisors to the Programme Committee. 80% of the session topics were defined during the first Programme Committee meeting. Tutorial topics


were also identified. A series of conference calls with the Theme Leaders took place in November 2011 to define each session and name speakers/session chairs. The outcomes of the second Programme Committee meeting in December 2011 included finalising each session topic, highlighting overlaps/gaps in the programme and naming the missing speakers/session chairs. Plenary sessions were defined and speakers named. Informal invitations to speakers were sent in December 2011 by Theme Leaders and Programme Committee members.

Results achieved Consolidated partnership for ECRD: EURORDIS (organiser), DIA (co‐organiser) and partners: EUCERD, EMA (including COMP), ORPHANET, ESHG, EuropaBio‐EBE/EFPIA, NORD.

On‐site inspections conducted in Brussels, call for tender processed and venue selected.

Call for tender processed, negotiation with PCOs (Professional Conference Organisers) and PCO selected to handle the registrations for the conference.

Official ECRD 2012 website launched in November 2011 in English. Official ECRD 2012 website translated in December into 6 languages

(English, Spanish, French, German, Dutch and Russian). ECRD‐EURORDIS Patient Advocate Fellowship launched via the

ECRD 2012 official website. Regular targeted electronic emails to different categories of

participants scheduled. Negotiated tax exemption on revenues for the conference with the

Belgian Fiscal Administration.

Relevant Annexes Composition of Programme Committee and Advisors ECRD 2012 website: www.rare‐diseases.eu ECRD 2012 Overall programme matrix Call for Poster Abstracts Flyers to different categories of participants Belgian Fiscal Administration official response confirming tax

exemption for ECRD 2012


Activity A.c. Update of reference documents on RD, reports, brochure; translations


Responsible: Paloma Tejada Supervising: Jill Bonjean

Deliverable No specific deliverable is associated to this activity

Indicators External communication materials: 3

Calendar of activities EURORDIS presentation brochure produced in 5 additional languages

Become a Member Guide

Description of the Activity In 2011, the EURORDIS presentation brochure, which had been published in English and French in 2010, was produced in 5 additional languages (German, Spanish, Italian, Portuguese and Russian). A new Become a Member 8‐page booklet was produced in 7 languages: English, French, German, Spanish, Italian, Portuguese and Russian. It describes membership benefits and the administrative procedure to become a member of EURORDIS. It includes an application form in Adobe Pro. The booklet is available in printed form and on‐line at eurordis.org

Results achieved Improved communication with members and other key stakeholders of the rare disease community.

Relevant Annexes Presentation Brochure Become a Member Guide


Activity A.d. Rare Disease Day 2011 and media monitoring service. Preparation of RDD 2012


Responsible: Paloma Tejada Supervising: Jill Bonjean

Deliverables D9: Rare Disease Day 2011

Indicators Number of participating countries: 55 (27 European) Number of events: around 1000 Number of visits to the website: 49 387 from 150 countries from 1st

January to 3rd March, 2011 (38 000 in the same period last year)

Number of visits to the website (all 2011): 74 963

Number of individuals, patient organisations, industry, health professionals, research and public authorities signed up as ‘Friends of the Rare Disease Day’ in the dedicated section of the RDD 2011 website: 292

Fans who joined RDD 2011 Facebook: more than 14 000 People who followed the campaign on Tweeter: almost 1500 Posters printed and sent out (to member patient organisations,

National Alliances, European Federations, researchers, policy‐makers, industry and other relevant stakeholders): 986

Calendar of activities On and around 28 February 2011 – Hundreds of awareness‐raising events all across Europe and in many countries outside Europe

28 February 2011 – “European Symposium Rare but Equal‐ Addressing Health Inequalities for Rare Disease Patients in Europe”, Brussels

Description of the Activity Rare Disease Day 2011 took place on 28 February 2011. The campaign has been implemented 55 countries, of which 27 European countries (Austria, Belgium, Bulgaria, Croatia, Cyprus, Denmark, France, Georgia, Germany, Greece, Hungary, Ireland, Italy, Finland, Latvia, Luxembourg, Netherlands, Norway, Poland, Portugal, Romania, Russia, Serbia, Slovenia, Spain, Sweden, UK). EURORDIS and 25 Rare Disease National Alliances, together with 26 patient groups acting as country organisers, mobilised hundreds of patient organisations throughout 5 continents. Outside of Europe, RDD 2011 was organised in USA, Canada, China, India, Japan, Brazil, Argentina, Australia, South Africa, amongst other. Newcomers in 2011 were Mexico, Thailand, Panama, Armenia, Uruguay, Nepal, Dubai, Morocco, Peru, Iran. EURORDIS has established a RDD 2011 Steering Committee composed of a subgroup of the Council of National Alliances from France, UK, Ireland, Spain, Italy, Germany, Canada, USA to develop methodology and tools. The main tools were: ‐ The common theme “Equality of Access” and the slogan “Rare but


Equal” ‐ The Information Pack ‐ The Poster ‐ The RDD 2011 website The RDD 2011 website contained 55 country pages (including one for Europe), with a calendar of events organised in each country. The website included a downloadable tool kit with an Information Pack, logo, poster and other materials accessible to organisers; a section for patients to upload photos (432) and videos (100); press releases for journalists and links to 4 social media networks (Facebook, YouTube, Flickr, and Tweeter) especially created for the Day. A dedicated section ‘Friends of Rare Disease Day’ was created, where sympathisers could register and describe their contribution to the campaign. Other interesting features in 2011 included: a special section on the website on “Rare Diseases and Health Inequalities” featuring more than 50 testimonies from patients about their experience of inequality of access to treatments, care and social services. Rare Disease Day 2011 European Symposium. In line with this year’s slogan: “Rare but Equal”, EURORDIS organised a one‐day symposium in Brussels in partnership with the European Commission, to address the issue of health inequalities and discuss mitigating measures. The event, entitled ‘European Symposium‐ Rare but Equal‐ Addressing Health Inequalities for Rare Disease Patients in Europe’, attracted over 90 attendants to the International Press Centre in Brussels. Ms. Antonyia Parvanova, MEP and shadow rapporteur on the new EU Directive on Cross‐Border Health Care and Patient Mobility, gave a key note speech. The programme highlighted examples of inequalities, such as the absence of widespread neonatal screening in Romania for phenylketonuria, a rare disease that is systematically screened for in most of Europe; the disparities in life expectancy for cystic fibrosis patients between new and old Member States; the unequal access to new therapeutic approaches for rare cancer patients leading to variations in survival rates across Europe. Participants included patient representatives, health professionals, industry representatives, policy makers and high‐level officials of the European Commission. The morning panel was devoted to the presentation of the three EU initiatives to address health inequalities: The EU Solidarity in Health initiative, the EU Disability Strategy and the new Directive on Cross Border Health Care and Patient Mobility. The European perspective was illustrated with key findings from the EurordisCare surveys on patients' experiences and expectations regarding access to diagnosis and to medical and social services, as well as EURORDIS’ latest survey on orphan drugs availability in Europe. A


presentation, featuring a study on the Public Health Situation and Needs of People with Rare Diseases in Spain conducted by the Spanish Alliance (FEDER) illustrated inequalities within Member States. Patient testimonies included a patient representative from PKU Life Romania and the wife of a Multiple Myeloma patient from the UK. EURORDIS Chief Executive Officer, Yann Le Cam, closed the event with the announcement of key results of the Eurobarometer survey on public opinion about rare diseases. The event was streamlined live and broadcasted on the EURORDIS website and on the RDD Facebook Group throughout the day (380 people from 39 countries tuned in). Preparation of RDD 2012. EURORDIS has established a RDD 2011 Steering Committee composed of a subgroup of the Council of National Alliances from France, Spain, Italy, Germany and USA to develop methodology and tools. The main tools are: ‐ The common theme “Solidarity” and the slogan “Rare but Strong

Together” ‐ The Information Pack, including a statement on RD and solidarity ‐ The Poster ‐ Website printable documents, logos, banners, badges ‐ A completely revamped RDD website, including new features to

ease access to country information ‐ An international video clip in 10 languages

Results achieved Improved knowledge via public awareness on rare diseases, improved access to information for patient representatives, patients and their families, as well as enhanced patient to patient exchange of information on their disease through social networking and new online tools.

The ability of EURORDIS to involve National Alliances from around the world for RDD has been consolidated.

A growth of RDD awareness campaign in number of countries (55), number of events (around 1000).

Broad media coverage, with hundreds of news articles collected through a media monitoring service. A newsfeed displayed those media clippings on a loop on the homepage of rarediseaseday.org.

The European Medicines Agency gave its official endorsement to the campaign and featured Rare Disease Day 2011 on its website.

Relevant Annexes Poster 2011 Information Pack 2011 RDD 2011 website Agenda RDD 2011 Symposium Brussels Video of RDD 2011 Symposium Brussels



Activity A.e. Awareness raising through patient stories, photo and video contest


Responsible: Justine Evans/Paloma Tejada Supervising: Yann Le Cam

Deliverable No specific deliverable associated to this activity

Indicators Number of photo entries: 94 Number of countries represented (photos): 20 Number of diseases (photos): 25 Number of photo viewings: more than 2000 Number of video entries: 43 Number of countries represented (videos): 11 Number of diseases (videos): 21 Number of viewings of videos: 3 016 Number of visits to winning video: 845

Calendar of activities 1 March 2011 ‐ Contest launched 6 May 2011 ‐ Contest closed

Description of the Activity The EURORDIS Photo & Video Contest was held from 1 March 2011 to 6 May 2011. The contest was promoted through the EURORDIS website (www.eurordis.org), the EURORDIS and Rare Disease Day Social medias (Facebook and Twitter) as well as the EURORDIS e‐news. People from all over the world, representing a variety of rare diseases, ‘told their story’ with the help of a photo or video camera.

The contest attracted 94 photo entries and 43 video entries. The entries were submitted via the EURORDIS website then uploaded to Flickr and YouTube. Photo contest. Photo submissions were received from 20 different countries, and showcased over 25 different rare diseases. The photos have been viewed more than 2000 times on Flickr. All of the photos submitted to the contest can be found in the EURORDIS Photo Contest gallery on Flickr. The photo of Emilia (Achondroplasia) taken by Anna Spindelndreier of Germany was chosen as the winner by a jury of 10 persons. The winning photo can be found in the Flickr photo gallery. Video contest. The winning video, entitled “Y tu ¿Sabes Moebius?” (“And you? Do you know about Moebius?”), features the daily life of Susana Romero who suffers from Moebius Syndrome. Susana’s story received more than 800 visits during the contest. The winning video can be found on the EURORDIS YouTube channel. Video submissions were received from more than 25 different


countries, and showcased more than 21 different rare diseases. The videos have been viewed more than 3000 times. Viewers connect from around the globe, from India and China, the Americas and especially from every country in Europe. The final selection of videos submitted to the contest can found on the EURORDIS YouTube channel.

Results achieved For both contests, there were fewer entries than in previous years due to the fact that the contest was not held in conjunction with Rare Disease Day photo wall; the time period lasted two months instead of four; there was a greater focus on achieving a higher quality of image (photos) and quality subject matter (videos). Nevertheless, important results were achieved: Improved public awareness on rare diseases via photo and videos. Enhanced patient‐to‐patient exchange of information on their

disease through social networking and new online tools. Greater higher quality of photos.

Relevant Annexes EURORDIS website page of Photo and Video Contest 2011 Winning photo and information about the winner All photos Winning video and information about the winner Final video entries


Activity A.f. Maintenance of the EURORDIS RD community database


Responsible: Patrice Régnier

Deliverable D3: Update of EURORDIS RD community database

Indicators Number of entries in the patient organisation section of the contact database: 1656 (112 new entries in 2011)

Number of entries in the member organisation section: 492 Contact database entries: 5405 (535 additional entries in 2011)

contacts, 4146 (226 additional entries in 2011) organisations

Calendar of activities All year ‐ ongoing

Description of the Activity The community database gathers data of 5405 contacts in 4146 organisations, 8000 diseases, 1656 patient organisations, including 492 EURORDIS member organisations, 23 EURORDIS projects and 60 events.The database allows: ‐ E‐mailing or paper mailing to all contacts or sub‐groups of contacts; ‐ Search of patient groups or experts by disease; ‐ Direct link to ORPHANET database by disease; ‐ Follow‐up of member fees payments & statistics; ‐ Recording of participation of contacts in events & projects,

including role (i.e. speaker, chair, participant, etc.); ‐ Recording of activities performed (i.e. mailing, meeting, trainings,

etc.). The database is used and updated daily by all EURORDIS staff members.

Results achieved 2nd update of the emailing system (send html emailings without entering the html code directly, send emailings from organisations)

Enhancement of the templates sections Feasibility study of update of diseases directly from orphadata.org Feasibility study of import export of contacts and enhancement of

donors section

Relevant Annexes 1. Html emailing 2. Disease search: http://eurordisprod.ateneatech.com/disease_search? 3. OrphaData.org 4. Feasibility study


Activity A.g. International dialogue


Responsible: Ariane Weinman Supervising: Yann Le Cam


Indicators International conferences attended by EURORDIS representatives in 2011: 111

Calendar of activities Please refer to Annex 1: List of European and international conferences attended by EURORDIS’ representatives in 2011

Description of the Activity EURORDIS has developed partnerships with several European and international not‐for‐profit organisations to work on transversal issues relevant for patients affected with rare diseases. Staff and EURORDIS volunteers engage in a range of different activities depending on the level and type of involvement with international NGO partners. They are supported and coordinated by Ariane Weinman. The partners are: ‐ NORD – The US Organization for Rare Disorders ‐ CORD – The Canadian Organization for Rare Disorders ‐ EPF – The European Patients’ Forum ‐ EPPOSI – The European Platform for Patients’ Organisation, Science

and Industry ‐ DIA – Drug Information Association ‐ ICORD – The International Conference on Rare Diseases and

Orphan Drugs NORD: For more than a decade, EURORDIS and NORD have been joining efforts to improve the life of rare disease patients on both sides of the Atlantic. NORD is a member of the EURORDIS Council of National Alliances of rare disease patient organisations. A partnership, aimed at converging strategies between the two organisations for the period 2009 – 2015, was sealed on 4th July 2009. Each organisation brings mutual support to develop the following activities: ‐ Exchange of information and advice on EU and US national policies

on orphan drugs and rare diseases, notably orphan drugs development in the EU and the US, work on the harmonisation of marketing authorisation process for ODs;

‐ Raising international awareness of rare diseases as a public health priority on the occasion of the Rare Disease Day; NORD leads Rare Disease Day in the USA;

‐ Developing online rare disease patients’ communities with a common strategy as well as co‐branded blogs; in EURORDIS, throughout 2011 this activity has been coordinated by Robert Pleticha.

‐ Bringing together more closely the NORD Corporate Council and the EURORDIS Round Table of Companies (ERTC); coordinate the


topics addressed, cooperate to promote companies membership in each other's forum.

In 2011: EURORDIS advised NORD to create a US Conference on Rare

Diseases and Orphan Products based on the model of the European Conference on Rare Disorders and Orphan Products (ECRD). After establishing a partnership between NORD, EURORDIS and the DIA, NORD organised a conference on rare diseases, which took place on 11‐13 October 2011 in Washington D.C.

NORD shared its experience in organising its annual Gala to support EURORDIS in organising its EURORDIS Black Pearl Dinner Gala, on 29 February 2012.

EURORDIS and NORD jointly developed online patient communities (service “RareConnect”, rareconnect.org) with the launch of 13 additional communities, totalling 14, and new features.

Finally, EURORDIS and NORD have collaborated on a weekly basis in many policy areas, including orphan drug development, regulatory affairs and access to medicines; research policy and participation in the International Consortium on RD Research (IRDiRC); registries.

CORD: the Canadian Organization on Rare Diseases collaborates with EURORDIS on a number of areas: CORD leads the Rare Disease Day in Canada. CORD participated in the IRDiRC meeting in Montreal, Québec,

Canada, in October 2011, thanks to EURORDIS. Active participation of CORD’s President, Durhan Wong, as

Moderator at the EURORDIS General Assembly 2011 in Amsterdam. Regular exchange and support on policy and national plans. Sharing

all tools and results from EURORPLAN‐EURORDIS Conferences. EPF: Anders Olauson, former EURORDIS Board member, has been officially appointed by our Board to represent EURORDIS in EPF. Anders Olauson is the current President of EPF. He was the co‐founder of Agrenska in Sweden, a centre for disabled people, and was EURORDIS’ president from 1999 to 2001. EURORDIS works on transversal issues with EPF: EU Directive on cross‐border healthcare; information to patients; animal welfare; EMA Reflection Paper on Ethical and GCP Aspects of Clinical Trials Conducted in Third Countries. EPPOSI: Yann Le Cam is a Board member of EPPOSI and the Treasurer until October 2011. EURORDIS participates in the governance of EPPOSI and provides inputs to its programmes. DIA: “The DIA patient fellowship programme” was established in 2006 with the support of EURORDIS. This programme provides each year 47 fellowships to European patient advocates across diseases to speak in sessions or attend the DIA Annual EuroMeeting. The successful fellows are selected based on their involvement in clinical trials and advocacy track records. Since 2006, 119 patients’ representatives received a fellowship, 68 are members of EURORDIS. At the EuroMeeting 2011 in Geneva (28‐30 March), the special DIA booth for patients was manned by Ariane Weinman.


EURORDIS has initiated the partnership with DIA and NORD for the organisation of the Conferences on Rare Diseases and Orphan Products in both the US and the EU. ICORD: The ICORD mission is to improve the welfare of patients with rare diseases and their families world‐wide through better knowledge, research, care, information, education and awareness. It brings together international rare diseases’ organisations and stakeholders. EURORDIS brings its longstanding experience as a patient organisation involved in the field of orphan drugs and rare diseases policy making at the European level. EURORDIS and NORD have both agreed to play pivotal and coordinated role in ICORD to help expand the movement of rare diseases / disorders at the international level. In particular, they have collaborated to develop the ICORD Position Paper of "Rare Diseases: an International Public Health Priority", and the international network of patient organisations. This paper is under consultation by rare disease umbrella groups outside Europe. Beyond EU: In 2011, EURORDIS supported new patients groups in Russia, Armenia, Georgia and Ukraine to organise international rare diseases awareness‐raising conferences in their respective countries. EURORDIS’ representatives attended conferences showing our strong support on a long term basis.

Results achieved Successful workshops and great participation of patients’ representatives.

Greater visibility of patients’ representatives in international conferences and workshops.

Common work with NGO Partners to produce recommendations on healthcare policies relevant for people affected with rare diseases.

Partnership EURORDIS‐NORD‐DIA.

Relevant Annexes List of European and international conferences attended by EURORDIS’ representatives in 2011

Memorandum of Understanding: Strategic Partnership between NORD and EURORDIS – July 4th 2009

DIA ANNUAL EUROMEETINGS 2006 to 2011: List of patient fellows including members of EURORDIS


Activity A.h. Promoting RD as an international priority


Responsible: Flaminia Macchia Supervising: Yann Le Cam


Indicators Number of consulted parties: 23 (including the European Public Affairs Committee of EURORDIS)

Number of countries replying: EU countries + 8 non EU

Calendar of activities Broad consultation launched in March 2010 September 2011 – Consultation within the EURORDIS’ EPAC and

external experts November 2011 – Circulation and consultation to an international

group of experts Q2 2012 ‐ Adoption

Description of the Activity EURORDIS has been starting elaborating a first draft of the so‐called “International Paper” in 2009. In March 2010, in view of the ICORD meeting in Buenos Aires, NORD (National Organization for Rare Diseases, US) and EURORDIS have been working together on the draft International Paper with the aim at circulating it widely to Patients’ Organisations worldwide and making it a Joint Declaration from the rare diseases patients’ movement, at global level. The idea at that time was to be able to present the “Joint Declaration: Rare Diseases as an International Public Health Priority” at the ICORD meeting in 2011. Gathering input from different Regions in the world has proven to be very difficult and the paper seemed to be still too Europe/US focused. In 2011, the process was resumed. In September 2011, the draft was circulated to the EPAC (European Public Affairs Committee) within EURORDIS and to a group of experts in the field from Denmark, Russia, Italy, Ireland, the UK, Germany, the US, Canada, New‐Zealand and Taiwan. In November 2011, it has been circulated further to some additional experts identified in Singapore, Japan, Malaysia, New Zealand, Australia, Canada, Thailand, South Africa, China, Hong King, Taiwan and the U.S. The objectives of the Joint Declaration are 1. to directly promote rare diseases as an international public health priority towards relevant institutions at international level; 2. to provide an advocacy tool for patient groups to advocate towards their national authorities thereby serving as a basis for patient empowerment locally; and 3. to enhance international cooperation in the field of rare diseases. By the end of 2011, the last draft before finalisation and adoption, is


structured as follows: ‐ The first part of the Joint Declaration aims at setting up the

background and objectives of the Joint Declaration, as well as identifying underline common features of rare diseases and common challenges facing the rare diseases community worldwide.

‐ The second part is made of ten recommendations: 1. Enhance visibility of RD at international level 2. Improve timely diagnosis 3. Coordinate research efforts on RD 4. Improve universal access to high quality healthcare for RD

patients 5. Improve access to orphan drugs 6. Patients and families support and empowerment 7. Promote international cooperation in the field of services and

support for patients and families 8. Develop, gather and disseminate information on RD 9. Support specific policy on RD at national/regional level through

action plans on RD 10. Elaborate RD related policies based on common values of social

justice, solidarity and equity. Besides, Yann Le Cam, EURORDIS’ CEO, as ICORD President Elect has initiated it in 2009 as a ICORD Policy Statement on Rare Diseases. This Draft Paper has been further elaborated, finalised and adopted by ICORD Board in 2011 and presented at ICORD 2012 meeting in Tokyo, Japan (February).

Results achieved Extensive consultation with rare disease stakeholders and experts worldwide.

Advanced draft before finalisation.

Relevant Annexes Joint Declaration on Rare Diseases as an International Public Health Priority


Activity B.1.a. & B.1.b. Outreach to POs and members, dissemination of information, consultation of members, focus on new EU Member States including Fellowships

Responsible staff, supervising staff

Responsible: Anja Helm Supervising: François Houyez


Indicators Total number of patient organisations members of EURORDIS (December 2011): 492

New members in 2011: 50, including 43 new members in EU Member States and 4 in candidate EU Member States

Number of countries where there are EURORDIS members: 46 Number of EU Member States where there are members: 24 Internal consultations with members: 21 Lost members: 5

Calendar of activities All year – ongoing

Description of the Activity EURORDIS is a grassroots movement of patient organisations active in the field of rare diseases. The membership of EURORDIS (492 patient organisations) is involved in the organisation’s activities and in its decision‐making process through regular mailings sent out to all members in 6 languages, surveys (on‐line surveys or simple e‐mail consultations) and face‐to‐face meetings. EURORDIS staff and volunteers participate in a great number of conferences and meetings across Europe and notably in new Member States (on volunteers, see also Activity Table B.1.d.). Patient representatives attended EURORDIS events such as: the General Assembly (172), Membership Meeting (244 participants), Summer School Training (43) and different workshops, such as the Council of National Alliances, the Council of European Rare Disease Federations, the Eurordis Round Table of Companies or workshops for Online Communities. Altogether, this makes nearly 1000 person/days from EURORDIS membership mobilised to participate to such events. For the majority of workshops and trainings, patient representatives’ travel and hotel expenses are covered. At the EURORDIS Membership Meeting 2011 (Amsterdam, 12‐14 May), 33 participants benefited from 3 types of fellowships (Fellowships for patient representatives from Eastern & Central Europe, Fellowships for Play Decide session organisers and fellowships offered by the ISS –the Italian Institute for Health‐ for participants of the New‐Born Screening Workshop). The 5 members that withdrew in 2011 are: ‐ Danish Cystic Fibrosis Association(financial difficulties)


‐ La Chainette, France (association closed) ‐ HAE Belgium (association closed) ‐ Interessengemeinschaft Sichelzellkrankheit und Thalassaemie eV,

Germany (President left) ‐ AIDES, France (change of strategy)

Results achieved EURORDIS membership grows by an average of 12% per year. The trend was confirmed by the 50 new members that joined in 2011.

At least 1000 person/days from EURORDIS membership were mobilised to participate to events, conferences, trainings and workshops organised throughout the year.

A growing number of patient representatives attend conferences, workshops and trainings, building their capacities and acquiring skills.

Relevant Annexes List of EURORDIS members Programme of EURORDIS Membership Meeting 2011 (Amsterdam) List of 33 Fellows who benefited from Fellowships toattend the

Membership Meeting and its Workshops


Activity B.1.c. Support to CNA; 2 Workshops; involvement in public awareness (RDD) and empowerment on RD national strategies


Responsible: Anja Helm Supervising: François Houÿez, Yann le Cam

Deliverable D2: Workshops of the EU Networks

Indicators

National Rare Disease Alliances that are members of the Council of National Alliances (CNA): 25

Number of National Alliances that are members of EURORDIS: 28 Participants to the 1st one‐day Workshop of the CNA (12 May 2011):

29 Participants to the 2nd one‐day Workshop of the CNA (8 November

2011): 22 [Reminder from the EUROPLAN project: representatives of National

Alliances worked as Advisors for EUROPLAN: 6

Calendar of activities 12 May 2011 ‐ One‐day Workshop, Amsterdam 8 November 2011 ‐ One‐day Workshop, Paris

Description of the Activity National RD Alliances serve to bring together all the many RD organisations in a particular country. The CNA (Council of National RD Alliances), established by EURORDIS, allows national representatives of rare disease patients to work together on common European actions. Currently, 17 EU countries participate in the CNA, plus 6 non EU countries (Croatia, Georgia, Russia, Switzerland, Canada and the USA). In both Portugal and Russia, two National Alliances per country are members of the CNA. One of the main achievements of the CNA is the creation and implementation of the Rare Disease Day.

The CNA’s main activities in 2011 were the analysis of the outcomes of the national conferences on national plans for rare diseases, that were organised under the EUROPLAN project; the preparation of future conferences “EUROPLAN II”, in the framework of the Joint Action on Rare Diseases 2012‐2014; the debriefing on RDD 2011 as well as the preparation and coordination of the Rare Disease Day 2012. In 2011, two CNA Workshops have been held. The workshop held on 12 May 2011, held in Amsterdam at the occasion of the EURORDIS Membership Meeting, gathered 29 representatives of National Alliances. The following topics were discussed: ‐ Rare Disease Day 2011 and preparation of 2012; ‐ National Plans: EUROPLAN update and national conferences ‐ EUCERD (the EU Committee of Experts on Rare Diseases).

The workshop of 8th November 2011 gathered 22 representative and


focused on the following topics: ‐ Information on EUROPLAN II (Joint Action on Rare Diseases 2012‐

2014); ‐ Rare Disease Day 2012 – preparation; ‐ Discussion of the role of National Alliances in the transposition of

the Cross Border Health Care Directive.

Results achieved Involvement of patient organisations in the development of national plans and strategies for rare diseases at the national level, the establishment and consolidation of relations with national stakeholders and the participation in national decision‐making bodies.

Networking and information sharing at European level of National Alliances respective experiences.

Increasing awareness at the national level on rare diseases via the Rare Disease Day.

Relevant Annexes Report of the CNA Workshops of 12 May 2011 Report of the CNA Workshops of 8 November 2011 List of members of the CNA (December 2011) Brochure on National Alliances and CNA (July 2007)


Activity B.1.d. Support to CEF through dissemination and sharing good practices; 1 two‐day Workshop; awareness and empowerment on EU policies


Responsible: Anja Helm Supervising: Yann le Cam

Deliverables D2: Workshops of the EU Networks

Indicators European Federations and Networks members of the CEF: 35 Participants to a one‐day Workshop and a one‐day training: 18

Calendar of activities 30 June 2011 – Workshop of the Council of European RD Federations 1 July 2011 ‐ Training session “Clinical Trials”

Description of the Activity

Representatives of European Rare Disease Federations gathered in Paris on 30 June 2011 to discuss issues that are important across Europe and across diseases: ‐ Rare Disease Day‐ How can European Federations get involved ‐ Case study: PHA Europe campaign ‐ Impact of Cross Border Health Care Directive on RD patients ‐ Presentation of www.clinicaltrialsregister.eu ‐ Presentation of Online Patient Communities The next day, July 1st, 2011, the representatives participated in a one‐day capacity building session entitled “Clinical Trials”. EURORDIS continued as well the pilot project “EURORDIS Grant Programme to support of European Rare Disease Federations”. The smallest and/or youngest organisations often have great difficulties in financing their network meetings (Board meetings, Network meetings, conferences etc). EURORDIS has given 7 European RD Federations financial support to help them organise their different meetings. A total of 11,727 € has been granted for the following meetings: ‐ Aniridia Europe Board meeting; ‐ 7th International Conference of the European Chromosome 11

Network; ‐ ECHDO (Congenital Heart Diseases) Board Meeting; ‐ European Network for Ichthyosis Board Meeting; ‐ Euro‐HSP (Hereditary Spastic Paraplegia) Conference; ‐ European Fragile X Network meeting; ‐ Meeting of OIFE and national OI (Osteogenesis Imperfecta)

associations & Scientific Conference.

Results achieved European Federations have benefited from sharing experiences at the workshop and increased their knowledge of clinical trials.

The 7 beneficiaries of the pilot project could not have organised their meetings without our support and are very satisfied with the results


(see some testimonies attached).

Relevant Annexes List of members of the CEF Report of the meeting of the Council of European Federations Testimonies of European Federations on the “EURORDIS Grant

Programme”


Activity B.1.e. Support to volunteers representing EURORDIS in EU committees


Responsible: Ariane Weinman Supervising: Yann Le Cam


Indicators Volunteers total number in 2011: 59 Volunteer patient advocates: 52 Office volunteer who assist the EURORDIS Therapeutic team: 1 Volunteer translators for 5 languages: French, German, Italian,

Portuguese and Spanish: 6 Volunteers in projects: 13 Volunteers involved in Task Forces: 22 Total number of participations of EURORDIS volunteers in

European, international conferences and workshops: 111 Total number of days/volunteers: 941 Number of EPAC consultations: 8

Calendar of activities Please refer to Activity Table B.2.a.1 and B.2.a.2 for the participation of EURORDIS staff and volunteers in EMA Committees.

‐ COMP: 11 volunteers for 2‐3 days each ‐ CAT: 11 volunteers for 2‐3 days each ‐ PDCO: 12 volunteers for 3 days each ‐ PCWP: 4‐5 volunteers for 1 day each 31 May ‐ 1 June 2011 ‐Joint Informal Meeting of EMA Committees

CHMP, COMP, PDCO, CAT, Budapest, Hungary 22‐23 March; 24‐25 October 2011 ‐ EUCERD Meeting dates in

Luxembourg 21‐22 March; 8 September 2011 ‐ EUCERD Workshops on Centres

of Expertise and European Reference Networks in Luxemburg 4 October 2011 ‐ EUCERD & EMA joint Workshop on Registries in

London 4 November 2011 ‐ EUCERD Workshop on Healthcare data

management in Montpellier

Description of the Activity As stated in its mission, EURORDIS voices rare disease patients’ views at the EU level, in EU Institutions and in European and international conferences. EURORDIS has altogether 17 representatives + 3 observers who seat on EMA scientific committees and on the EUCERD: 14 of them are volunteers from 9 different countries and 14 different diseases. EURORDIS volunteers are considered as experts. Since the creation of the organisation, they have greatly contributed to shaping EU rare disease policies. Most of them are either patients or parents of patient. Thanks the Operating Grant, EURORDIS is enabled to cover travel and accommodation’s expenses of alternates and observers to allow them to participate in EMA Committees and EUCERD meetings. Therefore,


both full members and alternates can team up to support EURORDIS’ views and opinions. Selection of the volunteers to represent EURORDIS in EU institutions: EURORDIS selects potential representatives based on a call for expression of interests and rigorous selection criteria. The volunteers must: a) be committed to the cause of rare diseases, b) master English, c) have a long‐standing advocacy track record in the field of rare diseases. Our strict rules have enabled us to propose good candidates with a good expertise to EMA and EC Committees. As a result, these candidates have been nominated by the European Commission. Representation of EURORDIS in EMA and EC Committees Over the past ten years, EURORDIS has always been able to identify high‐level volunteers able to represent the rare disease patients’ community in EU committees: ‐ EMA Committee for Orphan Medicinal Products: Two EURORDIS’

representatives out of three patients’ representatives: Birthe Byskov Holm (Denmark), Vice‐Chair of the COMP, and Lesley Greene (UK). Both of them are full members and volunteers. Dr Maria Mavris, staff, is observer.

‐ EMA Paediatric Committee: One EURORDIS’ representative, Dr Tsveta Schyns (Bulgaria/Belgium), volunteer, and her alternate, Dr. Gérard Nguyen (France), volunteer, out of three patients’ representatives.

‐ EMA Committee for Advanced Therapies: One EURORDIS’ representative, Dr Fabrizia Bignami, staff, and her alternate Dr. Michele Lipucci di Paola (Italy), volunteer, out of two patients’ representatives.

‐ EMA Patients’ and Consumers Working Party: The Co‐Chair, Lise Murphy (Sweden), is a volunteer EURORDIS’ representative. Richard West (UK), volunteer as well, joined the PCWP in September 2011. François Houÿez, staff, also seats on the EMA PCWP.

‐ EU Committee of Experts on Rare Diseases: The 8 seats (four full members and four alternates) for patients are hold by EURORDIS’ representatives (selected based on their expertise, advocacy track records, representativeness of large disease groups and geographic outreach). Yann Le Cam is the only staff member, other representatives are volunteers: Dorica Dan (Romania), Jan Geissler (Germany), Torben Gronnebaek (Denmark), Alastair Kent (UK), Christel Nourissier (France), Bianca Pizzera (Italy) and Gabor Pogany (Hungary). Two observers, Flaminia Macchia and Ariane Weinman, attend the EUCERD meetings to assist and support the volunteers.

EURORDIS organisation to support volunteers involved in EU committees EURORDIS has established different working groups, mainly composed by volunteers and coordinated by EURORDIS staff. These working groups are aimed at supporting the work performed by


EURORDIS’ volunteers in EU committees as well as developing initiatives in favour of rare disease patients in different fields (therapeutics, public health, social services…) ‐ The Therapeutic Action Group (TAG): Please refer to Activity Table

B.2.a.3. ‐ The Policy Action Group (PAG): Composed by all EURORDIS

representatives on the EUCERD (seven volunteers and EURORDIS’ CEO, Yann Le Cam), Flaminia Macchia, European Public Affairs Director and Ariane Weinman, Volunteers and NGO Partners Coordinator. The group organises regular conference calls to better follow up the work of the EUCERD and exchange emails on a regular basis.

‐ The European Public Affairs Committee (EPAC): This internal EURORDIS committee plays an active and key role in EURORDIS’ advocacy activities. It is composed of 30 members: 21 volunteers (Board members, TAG members and full members representing patients on the EUCERD), as well as 10 staff members (CEO, Directors and managers involved in advocacy). The EPAC members communicate regularly by emails. In 2011, they discussed important issues related to improving care for rare disease patients such as cross‐border healthcare, revision of the EU Directive on Clinical Trials, information to patients, rare disease research, etc…The exchanges of opinions are coordinated by Flaminia Macchia. Each EPAC member is entitled to vote for the adoption of a position of EURORDIS on specific issues.

‐ The EURORDIS Task Forces: Please refer to Activity Table B.2.c.1: Existing Task Force (TFs) include the DITA‐TF (Drug Information, Transparency and Access), the OD‐TF (Orphan Drugs) and the P‐TF (Paediatrics). The Task Forces support and/or advise the EURORDIS representatives who participate in EMA scientific committees and working parties. They are also consulted on papers prepared by EURORDIS.

European Council Recommendation and National Plans and Strategies on Rare Diseases EURORDIS’ volunteers on rare diseases work to translate the EC Commission Communication and the Council Recommendation on Rare disease into national policies and plans for people living with a rare disease. Volunteer patient advocates have been able to catalyse the participation of all stakeholders in strategies for rare diseases at the national level. This is one of the key success factors to develop a sustainable national policy. [Link with activities of the EUROPLAN project, covered by the EUROPLAN project and not by EURORDIS FY2011 In 2010 and 2011, EURORDIS coordinated the organisation of 15 national conferences on national plans for rare diseases within the framework of EUROPLAN, a DG Sanco co‐funded project. Yann Le Cam, Valentina Bottarelli, European Public Affairs Advisors, and Ariane Weinman, teamed up with six Advisors, patient advocates, who were in charge of coordinating two to three national conferences each.


Relevant activities in support of National Plans from other sections of EURORDIS FY2011 include: the new section of the EURORDIS website on National Plans; the Fact Sheets for patients advocates; the capacity‐building workshops organised at EURORDIS Membership Meeting in Amsterdam, e‐Newseltter articles. In 2011, EURORDIS team prepared th e second phase of EUROPLAN, due to start in March 2012. It will be co‐financed within the Joint Action on Rare Diseases and will involve the supervision of the organisation of 20 national conferences on national plans for rare diseases with 9 Advisors. Preparatory activities from March to December 2011 were not covered by EUROPLAN or Joint Action funds.

Results achieved EMA: Please refer to Activity Table B.2.a.1, B.2.a.2, B.2.c.1. EUCERD: Contribution to the first EUCERD Recommendation on

“Quality criteria for centres of expertise on rare diseases in Member States”

EPAC communication. Over 195 exchanges of emails (apart from consultations). 8 EPAC Consultations took place to provide an expert opinion on: 1. Concepts of availability, affordability, accessibility of Orphan

Drugs (19 April) 2. Inputs needed on the EURORDIS Electronic Health Record Draft

Check List (28 April) 3. EURORDIS response to the Commission's consultation on the

revision of the Clinical Trials Directive (11 May) 4. Position Paper on Priorities for Rare Disease Research 2014‐

2020, 1st round of comments (29 June) 5. Inputs for the draft Joint Declaration "Rare Diseases: an

International Public Health Priority" (5 September) 6. Position Paper on Priorities for Rare Disease Research 2014‐

2020, 2nd round of comments (21 September) 7. Inputs to deepen/complete/modify the definition used by the

EMA and the FDA (two different definitions) of the concept "Unmet Medical Need". (2 October)

8. Comment ‐ IRDiRC Policy Paper and Report from the Montreal meeting (21 October)

Relevant Annexes List of EPAC members in 2011 Example of email launching an EPAC consultation


Activity B.1.f. Fact Sheets for capacity‐building purposes on RD policy aspects


Responsible: Anna Kole / Paloma Tejada Supervising: Yann Le Cam


Indicators Number of new Fact Sheets produced in 2011: 1 Total Number of Fact Sheets: 10

Calendar of activities Distribution to new members and participants of: February 2011 – EUROPLAN final conference May 2011 ‐ EURORDIS Membership Meeting Amsterdam July 2011 ‐ Council of European RD Federations, CEF November 2011 – Council of National RD Alliances, CNA

Description of the Activity To provide the Council of National Alliances, the Council of European Federations and individual patient groups and representatives with advocacy tools encouraging the implementation of key recommendations in the European Commission’s Communication on Rare Diseases & Council Recommendation on Rare Diseases, and facilitating their transposition into national plans and strategies on rare diseases. Specific objectives include: Create Fact Sheets with input from patient advocates and experts

for patient advocates; Create Policy Fact Sheets corresponding to the main pillars of the

Commission Communication & Council Recommendation on RD to support their implementation at the national level and their transposition into national plans;

Provide these Policy Fact Sheets as downloadable documents on the EURORDIS website;

Provide these Policy Fact Sheets in a format that may be reproduced in hard copy in an easy and cost‐effective manner.

Results achieved 1 Fact Sheet produced in 2011 on New Born Screening Dissemination of hard copies to new members, CNA and CEF and

others. Dissemination of hard copies at EUROPLAN conference and at other

important conferences. Publication of all Fact Sheets on EURORDIS website. Development of Advocacy Pack for patient advocates including Fact

Sheets, the Commission Communication and the Council Recommendation on Rare Diseases.

Continued positive feedback from patient advocates on the utility of these supportive documents.

Relevant Annexes (The Fact Sheet on New Born Screening was not yet published at the moment of drafting the Operating Grant report).


Activity B.2.a.1. Support to the participation of patient representatives in the EMA committees (COMP, PDCO, CAT, PCWP) and in Protocol Assistance


Responsible: Maria Mavris Supervising: Fabrizia Bignami, Yann Le Cam

Deliverable D8: Participation in EMA Committees (COMP, PDCO, CAT, PCWP, PhVWP)

Indicators EURORDIS nominated patients’ representatives in EMA (European Medicines Agency) Committees and Working Parties: (COMP – Committee for Orphan Medicinal Products PDCO – Paediatric Committee CAT – Committee for Advanced Therapies PCWP – Patients’ and Consumers’ Working Party) Number of patients representatives in COMP: 2

(Birthe Byskov‐Holm, Vice Chair of COMP, Lesley Greene, member + Maria Mavris, observer)

Number of patients representatives in PDCO: 1 (Tsveta Schyns, member + Gérand Nguyen, alternate)

Number of patients’ representatives in CAT: 1 (Fabrizia Bignami, member + Michele Lipucci di Paola, alternate)

Number of patients’ repres. in PCWP: 3 (Lise Murphy, Co‐Chair, Richard West &François Houyez, members)

Number of Protocol Assistance (PA) dossiers received: 75 Number of patients’ representatives included in PA: 16 Number of days of EURORDIS volunteers at COMP: 45 Number of days of EURORDIS volunteers at PDCO: 43 Number of days of EURORDIS volunteers at CAT: 30 Number of days of EURORDIS volunteers at PCWP: 24 Total number of meeting days over the year: 142 Number of dossiers examined: 377, distributed as follows: COMP: 166 dossiers CAT : 24 dossiers PDCO: 187 dossiers

Calendar of activities Monthly meetings for COMP (2 days/month), PDCO (3 days/month) and CAT

Four meetings annually for PCWP (2 days/month) Participation in Protocol Assistance dependent upon dossiers 30 September 2011 ‐ Commission Call for Expression of Interest for

the position of Member of the Management Board of the EMA and for the position of member of the CAT, the COMP and the Pharmacovigilance Risk Assessment Committee ('PRAC'), with deadline on 1st December 2011.


Description of the Activity Patients’ representatives involved in the scientific committees and working parties at the EMA dedicate their time, experience and expertise to the task of: Evaluating dossiers of medicinal products applying for orphan drug

designation; Reviewing Public Summaries of Opinion on orphan designation and

Significant Benefit Assessment reports; Evaluation for use in paediatric patients; Evaluation of advanced therapies; Ensuring accurate, transparent and available information to

patients on authorised medicinal products (EPARs, PLs). The permanent members of the scientific committees and PCWP make up the Therapeutic Action Group (see Activity Table B.2.a.3.). Participants in Protocol Assistance are selected from our membership database and as often as possible have received training via the EMA in Quality Review of Documents or via the EURORDIS Summer School (B.2.c.2). Applications responding to the Commissions Call for Expression of Interest were submitted for extension of mandate for the COMP for both Birthe Holm and Lesley Greene. An application proposing Michele Lipucci di Paola as member and Monica Ensini as alternate was submitted in response to the Call for the CAT. No candidates were put forward for the Management Board or the PRAC. However, strong candidates from other patients’ associations were contacted and support was offered in the form of invitation to join EURORDIS’ TAG (Therapeutic Advisory Group) upon their eventual nomination by the Commission.

Results achieved Contribution to the scientific evaluation of 377 dossiers at the EMA. Richard West elected as member of PCWP by EURORDIS Board of

Directors. The participation of patients’ representatives in 16 Protocol

Assistance procedures altered the outcome of the procedure in 6 cases.

Relevant Annexes Protocol Assistance participation is referenced in the summary section of the monthly Therapeutic Reports (see Annexes of B.2.a. 4.)

Protocol Assistance information sent to patients’ representatives: Protocol Assistance letter of invitation and explanation EURORDIS Protocol Assistance flyer Regulatory diagram to explain Protocol Assistance


Activity B.2.a.2. Support to the participation of patient representatives in the EMA committees (PCWP) and implementation of the new legislation on Pharmacovigilance


Responsible: François Houÿez Supervising: Yann Le Cam


Indicators Working Party meetings in 2011: 4

Joint meetings with health care professionals in 2011: 3

Stakeholders meeting on pharmacovigilance: 2

Training on product information review: 1

Meeting on EUDRACT, database on clinical trials: 1

Calendar of activities 22 February, 16 June, 13 September, 30 November ‐ Three meetings for PCWP.

April 2011 ‐ Presentation by Lise Murphy, PCWP co‐chair, on the interaction between patients’ organisations and the EMA at the Heads of Medicines Agency meeting.

April 2011 and September 2011 ‐ Presentation on the patients’ expectations in pharmacovigilance, stakeholders’ meeting on pharmacovigilance by respectively Lise Murphy and François Houÿez.

2 December 2011 – Presentation by François Houÿez on pharmacovigilance and the role of patients, at the Irish Medicine Board conference, Dublin.

Description of the Activity EURORDIS nominated patients’ representatives in EMA Committees and working parties (see Activity Table B.2.a.1.). In particular, three patient representatives seat at the PCWP (Patients’ and Consumers’ Working Party) on behalf of EURORDIS: Lise Murphy (PCWP co‐chair), volunteer, Richard West, volunteer, and François Houÿez, EURORDIS staff. The PCWP provides recommendations to the EMA and its human scientific committees on all matters of interest to patients in relation to medicinal products.

Main activities in 2011 included:

Participation in the EMA Eudravigilance users groups to advise on the development of a web interface for the public.

Consultation and guidance on the pilot phase for participation of patient representatives in Scientific Advisory Group (SAG) meetings.

Advice on the European standard form to collect spontaneous adverse drug reactions reports from the patients.

Consultation by the CHMP (Committee for Medicinal Products for Human Use) on the evaluation of benefit/risks for some products, and involvement of relevant patients’ organisations (i.e. Fabry


International Network). Consultation by PhVWP (Pharmacovigilance Working Party) and

CHMP on press releases for risk communication (product shortages).

Training of 4 new volunteers for the review of EMA documents for the public (package leaflets, European Public Assessment Report summaries, press releases…).

Brainstorming on training strategy for patients and consumers. Participation in the working group on clinical trials in third

countries. Follow‐up meeting with Thalidomide Patients and Victims

Organisations. Discussion on the Proposal for legislation on information to patients

with Patricia Brunko (European Commission). Revision of the criteria for eligibility of patients’ and consumers’

organisations working with the EMA During 2011, François Houÿez continued to be topic leader on risk communication and started to be topic leader on CHMP consultation on benefit/risk.

Results achieved The input of our participation in the PCWP and related activities is monitored annually by the Agency, and the results are presented to the EMA Management Board, for adoption (see annexe, 4th progress report).

Pilot consultations on the benefit/risk by the CHMP will help defining possible interaction between CHMP and patients’ organisations on relevant topics identified during the pilot phase.

Richard West appointed as member of PCWP by EURORDIS Board of Directors after an internal selection process with 12 candidates.

Relevant Annexes EMA Outcome report on pilot phase for participation of patient representatives in Scientific Advisory Group (SAG) meetings.

4th EMA report on the progress of the interaction with patients' and consumers' organisations (2010) and Results/analysis of the degree of satisfaction of patients and consumers involved in EMA activities during 2010.

EMA paper on the role of paper as members of the EMA Human Scientific Committees.


Activity B.2.a.3. Supporting the EURORDIS Therapeutic Advisory Group (TAG)




Indicators Number of members of TAG: 12

Number of consultations/meetings of TAG: 12

Calendar of activities Monthly conference call with all members 19 November 2011 – TAG Meeting in Paris 19 November 2011 – TAG Meeting in Paris with EURORDIS Board of

Directors

Description of the Activity The Therapeutic Action Group (TAG) is composed of 12 EURORDIS representatives in the scientific committees and working party at the EMA (PDCO, PCWP, CAT, COMP): Fabrizia Bignami (CAT), Lesley Greene (COMP), Birthe Byskov Holm (COMP), François Houÿez (PCWP), Lise Murphy (PCWP), Richard West (PCWP), Michele Lipucci di Paola (CAT), Tsveta Schyns (PDCO), Gerard Nguyen (PDCO), Maria Mavris (COMP, observer), François Houÿez (PCWP) and Yann Le Cam (CEO of EURORDIS). These patients’ representatives dedicate their time, experience and expertise to the task of evaluating dossiers of medicinal products requesting orphan drug designation, evaluation for use in paediatric patients, evaluation of advanced therapies as well as ensuring accurate, transparent and available information to patients on authorised medicinal products. A monthly report of activities of EMA Committees and EURORDIS’ patients’ representatives in these Committees is produced and disseminated to all members of TAG, Task Forces, Eurordis Summer School participants and EURORDIS’ Board (see Activity Table B.2.a.4).

Results achieved The TAG was created in January, 2009 and has successfully established a monthly conference call and a yearly face to face meeting to discuss matters related to regulatory affairs and therapeutic development.

A face‐to‐face meeting on 19 November 2011 took place in Paris after the EURORDIS Board of Directors meeting. The members of the TAG present included Fabrizia Bignami, Lesley Greene, Michele Lipucci di Paola, Lise Murphy, Richard West and Tsveta Schyns.

Relevant Annexes Agenda from TAG face‐to‐face meeting


Activity B.2.a.4. Monthly reports compiling feedback from each Committee




Indicators Number of monthly reports: 11

Calendar of activities Monthly conference call with all members to exchange information and receive updates to include in monthly report

Publishing and dissemination of monthly Therapeutic Report on activities of patients’ representatives in regulatory activities in the EU.

Description of the Activity A monthly report of activities of EMA Committees and EURORDIS’ patients’ representatives in these Committees is produced and disseminated to all members of the TAG (Therapeutic Action Group, see Activity Table B.2.a.3), EURORDIS Task Forces, EURORDIS Summer School participants and EURORDIS’ Board. The report is compiled based on feedback received from members of the TAG, monthly reports published by the European Medicines Agency, searching of the EMA website for relevant news and information, meetings attended relating to regulatory affairs by TAG members and presence of Maria Mavris at COMP monthly meetings.

Results achieved Eleven monthly “Therapeutic Reports” were published in 2011 Targeted dissemination of the Reports to:

- EURORDIS Summer School alumni - Targeted EMA staff - EURORDIS Board of Directors - Targeted staff of French Muscular Dystrophy Association (AFM) - EURORDIS staff

Relevant Annexes Three examples of Therapeutic Reports are provided: - May 2011 - September 2011 - December 2011


Activity B.2.b. Review and validation of public information on RD therapies disseminated by EMA (PSOs, EPARs, PLs)


Responsible: Maria Mavris, François Houÿez Supervising: François Houÿez, Fabrizia Bignami


Indicators Public Summaries of Opinion of orphan designation (PSO), European Public Assessment Report (EPAR) summaries and Package Leaflets (PL) reviewed by EURORDIS staff members: Number of PSOs: 111 Number of EPARs: 8 Number of PLs: 12 Total number of EMA dossiers for public information: 131

Calendar of activities PSOs validated monthly upon positive opinion given by COMP and designation awarded by EC

EPARs and PLs – at time of Marketing Authorisation

Description of the Activity The European Medicines Agency (EMA) is responsible for providing information about medicines authorised via the centralised procedure that includes information directed to the patient and the public. During the preparation of this information, the Agency interacts with patients and consumers’ organisations to ensure that it is adequately formulated and comprehensible to the target audience. EURORDIS is extensively involved in these activities. In 2011, EURORDIS staff has reviewed a total of 131 EMA dossiers for public information this year. Documents that are destined for the general public include: Public Summary of Opinion (PSO) of orphan drug designations explain

in lay terms the disease, the number of individuals potentially affected in the EU, the medicinal product and the stage of development. A link to the Sponsor and to EURORDIS and ORPHANET are provided in the PSOs to help patients obtain more information.

EPAR summaries (EPAR) are shorter documents based on the European Public Assessment Reports that are published at the time of Marketing Authorisation. The EPARs contain information about the development of the product and how the committee reached it recommendations.

The Package Leaflet (PL) contains information on what the drug is, what it is used for, how to take the drug, possible side effects and how to store the drug. It is important that this information is easily understandable for the general public.

The EMA evaluates yearly the added value of the review of these documents by patients and consumers, and this analysis, described in a report, is approved by the EMA management board.

Results achieved A total of 131 EMA dossiers for public information were reviewed by EURORDIS staff members in order to ensure the quality of the information


disseminated by the EMA to the general public. In particular, 8 European Public Assessment Reports (EPAR) for the public and 12 package leaflets (PL) were reviewed, for new marketing authorisations or for renewals. As not all of the decisions have been officially published by the European Commission, the names of the products and their indication remain confidential at this stage. Cumulatively, this represents 54 documents (28 PL, 26 EPARs) since 2006, with an average of 5 comments for each EPAR and 6 comments for each PL.

Relevant Annexes Link to PSOs on EMA website: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/ orphan_search.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d12b

Link to EPAR summaries and PL on EMA website: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/epar_search.jsp&murl=menus/ medicines/medicines.jsp&mid=WC0b01ac058001d125

The EMA report on the interaction with patients and consumers in 2010 can be found here: http://www.ema.europa.eu/docs/en_GB/document_library/Report/2011/10/WC500116866.pdf

At the time of writing this Activity Table, the 2011 report is not available yet.


Activity B.2.c.1. Support to EURORDIS Task Forces on Orphan Drugs, Paediatrics, Drug Information, Transparency & Access (DITA)


Responsible: Maria Mavris Supervising: Fabrizia Bignami, François Houÿez

Deliverable D7: Meetings of the Task forces: Orphan Drugs, DITA and Paediatrics

Indicators Number of consultations with Task Forces: DITA‐TF: 9 Number of volunteers involved in Task Forces: 34

Calendar of activities 18 June – DITA Task Force meeting, Paris 27 November – DITA Task Force meeting, London

Description of the Activity The Task Forces represent a group of volunteers who are trained (via the EURORDIS Summer School) and active in issues concerning therapeutic development of orphan drugs. The Task Forces support and/or advise the EURORDIS representatives who participate in EMA scientific committees and working parties. They are also consulted on papers prepared by EURORDIS. Existing Task Force (TFs) include: - DITA‐TF, Drug Information, Transparency and Access - OD‐TF, Orphan Drugs - P‐TF, Paediatrics The General Terms of Reference, which apply to all TFs, were approved by the Board of Officers on April 20, 2009. The Task Forces participate in conference calls when necessary (if certain issues need to be discussed) and at least one face‐to‐face meeting per year for DITA. As of 2012, the Orphan Drugs TF and the Paediatric TF will be just considered as groups of volunteers for consultation and no more meetings will be scheduled for them.

Results achieved DITA Task Force meetings held in Paris on 18 June, and in London on 27 November, 2011. In addition, DITA exchanged information on:

- Fabrazyme and Replagal shortages; - Involvement of patients’ organisations in the activities of their

national agencies; - Self‐reporting of adverse drug reactions; - Off‐label use of medicines for rare diseases; - Compassionate use programmes for orphan drugs; - The situation regarding some treatments for Friedreich Ataxia in

France; - The EMA workshop on clinical development and scientific advice in


ophthalmology; - The SAG meeting about a new medicine for Addison’s diseases; - EURORDIS contribution to the DG Enterprise consultation on a

possible revision of the Transparency Directive 89/105 EEC; - The new EC pharmacovigilance legislation.

An important and ongoing discussion was started with the Agency

regarding the new policy on Conflicts of Interest. The DITA Task Force also advised the EMA on its new functions for

the public part of its European register on clinical trials and its European pharmacovigilance database Eudravigilance.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) requested an opinion for a benefit/risk evaluation on 4 occasions.

Kerry Leeson‐Beevers (Alstrom Syndrome UK) and Camelia Lazar Livieratou (Williams Syndrome, Romania) were nominated by the EURORDIS Board of Directors as observers at PDCO to ensure continuation of patient representative presence and support for PDCO member.

The DITA Task Force is in itself a capacity‐building activity preparing volunteers to handle responsibilities in scientific committees.

Relevant Annexes Agenda of DITA meeting 18 June 2011 Report of DITA meeting 27 November 2011


Activity B.2.c.2. Summer School 2011


Responsible: Maria Mavris Supervising: Fabrizia Bignami

Deliverable D6: Summer School 2011 and online training tools

Indicators Number of training sessions: 1 session of 4 days Total number of trainees: 43 Total number of person‐days in training: 172 Total number of trainers: 11

Calendar of activities 12‐16 June 2011 ‐ Summer School Programme held, Barcelona 15 September 2011 – Report finished


The Summer School is a 4‐day capacity building programme for rare disease patient representatives in the areas of drug development, clinical trials and aspects of the EU regulatory process. Its main objectives are: To provide patient advocates with an initial introduction to the basic

processes of clinical trials including the essential aspects of statistics and ethics. In addition, a special mention of the problems with working with small populations such as rare and paediatric is made.

To familiarise patients’ advocates with the regulatory processes in the EU with respect to the different scientific committees and working parties at the European Medicines Agency and the role that patients can play. This includes formal presentations of each committee and real case studies from the Committee for Orphan Medicinal Products (COMP) and the Paediatric Committee (PDCO).

Results achieved The 2011 Summer School provided participants with a fundamental understanding of the processes involved in drug development, the time required and the different stages of clinical trials. Coupled with formal presentations of the topics, the participants were divided into small groups and encouraged to share their experiences and knowledge in the context of documents provided to complement the lectures.

In addition, practical ‘hands‐on’ examples of the work performed by the representatives at the EMA scientific committees and working parties is presented using: Orphan drug designation dossiers from the Committee for Orphan

Medicinal Products; An example of a waiver request from the Paediatric Committee.

The Summer School session in 2011 innovated by introducing a one‐day initiation on Health Technology Assessment (HTA).

Forty‐three participants attended representing 19 countries and 24 different rare diseases. The Summer Scholl 2011 participants have been selected based on a call for candidates. EURORDIS received 76 applications from which 43 candidates were selected.

Speakers were once again recorded and edited footage has been linked to


the slide presentations and made available to all participants on the EURORDIS website.

Relevant Annexes 2011 Summer School Programme Flyer Summer School 2011 List of attendees Application form Web link on Summer School: http://www.eurordis.org/training‐resources


Activity B.2.c.3. e‐Learning on specific and advanced aspects of drug development, clinical trials and regulatory affairs

Responsible and upervising staff

Responsible: Maria Mavris Supervising: François Faurisson, Fabrizia Bignami

Deliverable D6: Summer School and online training tool

Indicators Total number of slides: approximately 400

Calendar of activities An online tool has been launched based on the content of the EURORDIS Summer Schools. The various modules have been validated and uploaded and are available for general use via the EURORDIS website.

Description of the Activity As part of the EURORDIS Summer School, a 4‐day capacity building programme for rare disease patient representatives in the areas of drug development, clinical trials and aspects of the EU regulatory process, an on‐line e‐learning tool for continued education has been established. The on‐line e‐learning tool is made up of three modules and was developed using PowerPoint presentations. The layout of the slides was designed by a graphic designer and then uploaded. All slides were elaborated based on presentations from the Summer School and reviewed for language and coherence by Lesley Greene (CLIMB, Metabolic Diseases) on a volunteer basis. The e‐learning tool will cover the topics of described during the Summer School using different learning methods. The topics that are included are presented as modules: - Methodology and Designing of Clinical Trials - Statistics for Clinical Trials - Ethics of Clinical Trials The user can access the different modules and learn by using the lessons, case studies, vocabulary tests, quizzes or specific documents. Once inside a particular module, the user can move between case studies to lessons to vocabulary or relevant documents.

Results achieved The slides are completed and are currently being uploaded and validated in a web tool. The tool can be found in the newly revamped Training Resources section on the EURORDIS website: http://www.eurordis.org/training‐resources.

Annex Online e‐learning tool: http://www.eurordis.org/training‐resources


Activity B.2.d. Support to capacity‐building activities of patient advocates in HTA


Responsible: Maria Mavris Supervising: Fabrizia Bignami


Indicators Patient advocates who received the HTA training: 3

Calendar of activities

April 2011 ‐ Four‐day training course in Hall‐im‐Tyrol, Austria by UMIT (The Health & Life Sciences University)


In 2011, three more patients’ representatives had the opportunity to be trained in Health Technology Assessment (HTA): Christos Sotirelis (Thalassaemia UK), Fabrizia Bignami and Maria Mavris. They participated in a four‐day course focusing on Health Technology Assessment, Health‐Economic Evaluation and Outcomes Research. This introductory course, delivered by the Health & Life University (UMIT) in Hall‐im‐Tyrol, Austria, covers the key elements and methods of HTA and combines lectures, discussions, case study group work, and hands‐on computer lab session. This course covers key elements and methods of HTA and combines, lectures, discussions, case study group work and hands‐on computer lab sessions. Case examples of the course include technologies from different areas such as pharmaceuticals, devices, public health and prevention strategies, management programmes and health information systems.

Results achieved Three patients’ advocates were trained for 4 days in the HTA training. This adds to the five patients trained in 2010.

Involvement of RD patient representatives is becoming part of core recurrent activities of EURORDIS, as much as their involvement in EU regulatory affairs. A critical mass of patient representatives needs to be trained.

Relevant Annexes Web link on HTADS: http://www.umit.at/page.cfm?vpath=departments/public_health/ htads_continuing_education_program_d/short_courses_d/modeling_for_hta_d


Activity B.2.e. Supporting good practice relations between POs & sponsors on RD clinical trials based on EURORDIS Charter on Clinical Trials


Responsible: Rob Camp Supervised by : Fabrizia Bignami, François Houÿez


Indicators Number of companies having signed up to the Charter: 7 Number of Memorandum of Understanding signed: 2

Calendar of activities

Description of the Activity The general aim of the “EURORDIS Charter for Clinical Trials in Rare Diseases” (CCTRD) is to encourage good practices in the relationship between a clinical trial promoter and the patient organisations representing the disease concerned by the study. Since 2009, EURORDIS engaged in the implementation phase of the Charter. During 2010 and 2011 this activities implied the following steps: Formal adhesion of pharmaceutical companies developing

medicinal products for rare diseases to the EURORDIS Charter for Clinical Trials in Rare Diseases.

Implementation of the Charter at the occasion of any clinical trial performed by the signatories in a rare indication. EURORDIS offers its support to help implementing the Charter, and in particular:

- Upon request of a sponsor having signed the EURORDIS Clinical Trials Charter or of a patient group, EURORDIS contacts and gathers together all EU patient groups concerned by a specific clinical trial.

- Then, EURORDIS provides methodological support to help all involved parties reach an agreement on the level and modalities of the collaboration they all wish to establish for a specific clinical trial.

- Such agreement is officially described in a dedicated MoU (Memorandum of Understanding) signed by the parties and published on the EURORDIS website.

N.B.: Discussions on content and results of a clinical trial are not in the remit of EURORDIS but of the concerned patient groups involved. A specific section on the EURORDIS website provides information on the EURORDIS Charter for Clinical Trials in Rare Diseases, on the companies who adhered to the Charter and lists the MoU signed (http://www.eurordis.org/content/eurordis‐charter‐clinical‐trials‐rare‐diseases) A work plan for 2012‐2013 has been developed.

Results achieved The Charter Project has been “good, in that we have met a couple of times and got to know each other better and better. Some meetings were carefully organised and went smoothly, some less so. It is helpful for us to see how TSC groups in other countries are organised and how


they raise funding.” On the negative side: There has been “nothing dramatically bad but it would be helpful to have an agreed schedule of upcoming meetings of our group over a longer period of time, two years, maybe, at least. It would also be helpful for us to know more about Eurordis and to meet further people working there.”

The acting president of E‐TSC, Bo Karlsen, Sweden

“The opportunity Advocates have been given to learn about Clinical Trials and orphan drugs, etc, by EURORDIS … is a brilliant initiative. I feel these should be implemented to fine tune the little knowledge we have in this field. From a patient’s perspective I noticed that a lot of people (myself included) found the amount of information … to take in a little overwhelming. (This is) a great launching pad for getting us involved in proceedings.”

Carla Fladrowski, Italy

“The result of all our meetings that we have participated during these two years is the experience and the knowledge we got for clinical trials and orphan drugs. Until now, although we have a very warm relationship with all our members, even with those that don’t live in Athens, the subject of Clinical Trials and the new orphan drug (everolimus) gave us the excellent opportunity to bring us all together in a very well organised meeting which took place in Athens and patients participated from all the parts of Greece (Nov 12, 2011).

Because of this success we organised a second meeting on December 17 2011 generally for Clinical Trials where, we had the opportunity to repeat the lecture about the new orphan drug (with speaker Prof. Yiouroukos, Child Neurologist). We are very satisfied from those two meeting because parents and patients learned about clinical trials and feel more optimistic. As about the new orphan drugs there is a real hope and light for them for the future. Also we have done everything we could in order to inform as many doctors as we could about TSC and Clinical Trials.

The “bad” could be the fact that in the meetings that you invited us were only for patient representatives and our opinion is that it would be very helpful if doctors were also invited in order to learn by the patients the real situation and the everyday problems that they confront.

So, it would be a chance, at least in our country for more doctors to be informed about TSC and able to participate in the clinical trials for new drug(s), as in our country we have a lack of Doctors that know TSC, especially for adult patients”.

Marianna Lambrou, Greece

Relevant Annexes EURORDIS Clinical Trials Charter


2.3. DELIVERABLES Operating Grant EURORDIS_FY2011 n° 2010 32 05

Number Title Nature Dissemination outline Actual delivery date

D1 Activity Report OPERA Report Public February 2012

D2 Workshops of EU Networks Meetings Representatives of Networks

November 2011

D3 Update of EURORDIS RD community database

Database Public December 2011

D4 Electronic Newsletter Web communication

8189 subscribers December 2011

D5 EURORDIS Website Web communication

Public December 2011

D6 Summer School 2011 and online training tools

Training and Web communication

Selected participants (Summer School)

Public (online training)

12-16 June 2011

D7 Meetings of the Task Forces Orphan Drugs, DITA, TAG, Paediatrics

Meetings Members of Task Forces + Patient Organisations

November 2011

D8 Participation in EMA Committees (COMP, PDCO, CAT, PCWP, PhVWP)

Meetings Members of the Task Forces

December 2011

D9 Rare Disease Day 2011 Event, web and media communication

Public February 2011

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 2.3. List of Deliverables 2011 - Page 56

Operating Grant EURORDIS FY_2011 n°20103205 Final Report 2.3. List of Deliverables 2011 - Page 57

INDICATORS Who Periodicity Reliability 2 011A.a.1. EURORDIS Website

www.eurordis.org visitors DC Q 226 692Numbers of websites linking to EURORDIS website DC Q 119 000

A.a.2. Electronic NewsletterNewsletter subscribers/ total PT Q 8 189

A.d. European RDD 2011 and media monitoring serviceRare Disease Day 2011 nb of participating countries PT Y 55Rare Disease Day 2011 number of events (=nb of associations participating as of 2011) PT Y approx. 1 000Rare Disease Day 2011 website visitors DC Q 49 387

A.f. Maintenance of EURORDIS RD community databasesPO database entries AH Q 1 656Contact database entries AH Y 5 405

A.g. Strengthening international dialoguePresentations at meetings / workshops / conferences AW Q 111

B.1.a&b Outreach to POs and members, dissemination of information, consultation of members, focus on new MS including fellowshipMembers total number AH Q 492Number of countries where there are members (max 196) AH Q 46Number of EU Member States where there are members (max 27) AH Q 24Internal consultations with members AH Q approx. 21

B.1.c. Support to the EU network of National AlliancesNumber of National Alliances AH Q 28

B.1.d. Support and strengthening of the Network of EU RD specific FederationsNumber of European Federation for Rare Diseases members of EURORDIS AH Q 35CEF Workshop participants AH Y 18

B.1.e. Support to volunteers representing EURORDIS in EU CommitteesVolunteers total number AW Y 59Volunteers in projects AW Y 14Volunteers in task forces AW,MM Y 22Total nbr days volunteers AW Y 941Number of consultations with EPAC AW,FM Q 8

B.2.a. Patient involvement in EMA activitiesNumber of Protocol Assistance dossiers MM Q 75Number of participation of patients in Protocol Assistance MM Q 16Number of days at EMA committees x number of EURORDIS representatives MM Q 142Number of days at COMP x number of EURORDIS representatives MM Q 45Number of days at CAT x number of EURORDIS representatives FB Q 30Number of days at PDCO x number of EURORDIS representatives MM Q 43Number of days at PCWP x number of EURORDIS representatives FHZ Y 24Scientific dossiers studied MM Q 377Orphan drug applications studied (COMP) MM Q 166Advanced therapy applications studied (CAT) FB Y 24Paediatric applications validated (PDCO) MM Y 187Number of consultations or meetings of TAG MM Q 12

B.2.b. Review and validation of public information on RD therapies disseminated by EMA (PSOs, EPARs, PLs)Public Summary of Opinions (PSOs) reviewed by EURORDIS staff MM Q 111European Public Assessment Reports (EPARs) reviewed by EURORDIS staff FHZ Y 8Package Leaflets (PLs) reviewed by EURORDIS staff FHZ Y 12

B.2.c.1. Support to EURORDIS Task Forces on Drug information, Transparency & Access (DITA)Number of volunteers in Task Forces MM Q 34Number of consultations with DITA TF MM Q 9

B.2.c.2. Summer School 2011Number of trainees MM Y 43Number of person-day in training MM Y 172Number of trainers MM Y 11

B.2.e. Supporting good practice relations between POs & sponsors on RD clinical trials based on EURORDIS Nb of companies signed up for Clinical Trial Charter (CTC) FB Y 7Number of assistance for CTC between POs and Companies FB Y 2

Operating Grant EURORDIS n°20103205 Final Report 2.4. Indicators 2011 FINAL - Page 58

Operating Grant EURORDIS n°20103205 Final Report 2.4. Indicators 2011 FINAL - Page 59

Staff Invitees

Travels Subsistence Allowances Travels Subsistence

Allowances0. Overall Management (Audit) 132,00 27,50 159,50

Preparation Operating grant 2012 Brussels 2 05/05/11 EURORDIS 132,00 27,50 159,50 B.1.c. Two European Workshops of National Alliances of Rare Diseases 311,00 1 193,43 402,00 314,43 2 220,86

Eurordis Membership Meeting /CNA meeting Amsterdam 35 12/05/11 EURORDIS 311,00 1 193,43 138,00 301,73 1 944,16 CNA meeting Paris 22 08/11/11 EURORDIS 264,00 12,70 276,70

B.1.b. Fellowships & country visits 940,30 600,47 2 533,57 1 716,50 5 790,84 Eurordis Membership Meeting (fellowships) Amsterdam 33 12/05/11 EURORDIS 2 119,71 1 716,50 3 836,21 Opening for the pilot center for Rare Diseases (NoRo Project) Zalau ~ 150 27/06/11 EURORDIS 413,86 413,86 All Russian Congress Samara ~ 200 03/11/11 Russian Patients Union 940,30 600,47 1 540,77

B.1.d. Support to volunteers in EUCERD 454,20 115,20 3 109,10 1 644,94 5 323,44 EUCERD on ERN's Luxembourg ~ 60 21/03/11 DG SANCO 35,60 458,31 409,60 903,51 EUCERD work on Criteria CoE Luxembourg ~ 60 08/09/11 DG SANCO 115,20 828,85 84,00 1 028,05 EUCERD Plenary assembly Luxembourg ~ 60 24/10/11 DG SANCO 225,80 - 1 388,14 1 086,34 2 700,28 EUCERD / JARD Nps & strat. Luxembourg ~ 60 08/03/11 DG SANCO 192,80 76,80 269,60 Attendance of EUCERD member to ERTC Paris ~ 50 21/11/11 EURORDIS 357,00 65,00 422,00

B.3.a. TAG reports and attendance to EMA workshops & committees 2 966,31 5 605,32 3 362,90 4 639,53 16 574,06 COMP Meeting # 1 London ~ 40 11/01/11 EMA 240,00 445,63 685,63 COMP Meeting # 2 London ~ 40 08/02/11 EMA 350,00 647,99 997,99 CAT Meeting # 1 London ~ 55 11/02/11 EMA 157,33 228,96 386,29 PCWP Meeting # 1 London ~ 35 22/02/11 EMA 260,25 225,78 486,03 COMP Meeting # 3 London ~ 40 08/03/11 EMA 241,50 383,82 625,32 EUDRA CT meeting at EMA London ~ 40 17/03/11 EMA 11,48 11,48 COMP Meeting # 4 London ~ 40 04/05/11 EMA 350,00 450,56 800,56 CAT Meeting # 2 London ~ 55 11/05/11 EMA 87,38 252,58 339,96 PDCO meeting # 1 London ~ 55 18/05/11 EMA 822,74 1 139,10 1 961,84 COMP Meeting # 5 London ~ 40 08/06/11 EMA 311,81 855,07 1 166,88 COMP Meeting # 6 London ~ 40 06/07/11 EMA 197,00 579,92 776,92 COMP Meeting # 7 London ~ 40 06/09/11 EMA 147,00 239,10 386,10 PDCO meeting # 2 London ~ 55 07/09/11 EMA 305,00 447,53 752,53 CAT Meeting # 3 London ~ 55 14/09/11 EMA 139,22 170,58 309,80 COMP Meeting # 8 London ~ 40 14/10/11 EMA 305,00 875,21 1 180,21 CAT Meeting # 4 London ~ 55 14/10/11 EMA 89,18 268,01 357,19 CAT Satellite meeting ESGCT Brighton ~ 55 26/10/11 EMA 263,08 395,06 658,14 COMP Meeting # 9 London ~ 40 08/11/11 EMA 344,00 430,80 774,80 PDCO meeting # 3 London ~ 55 09/11/11 EMA 113,00 113,00 TAG meeting Paris 7 19/11/11 EURORDIS 1 125,72 1 511,93 2 637,65 EMA training London ~ 40 28/11/11 EMA 69,73 69,73 COMP Meeting # 10 London ~ 40 06/12/11 EMA 240,00 420,66 660,66 CAT Meeting # 4 London ~ 55 08/12/11 EMA 240,00 195,35 435,35

B.3.c.1. Task forces 253,00 419,62 2 282,72 1 578,88 4 534,22 DITA Meeting # 1 London 6 18/06/11 EURORDIS 253,00 367,30 1 061,96 847,70 2 529,96 DITA Meeting # 2 London 8 28/11/11 EURORDIS 52,32 1 220,76 731,18 2 004,26

B.3.c.2. Summer School 2 340,38 5 211,60 10 053,04 28 561,77 46 166,79 Preparation Summer School Barcelona 2 11/04/11 EURORDIS 194,40 437,63 632,03 Eurordis Summer School 2011 Barcelona ~ 50 12/06/11 EURORDIS 2 032,00 4 707,07 10 053,04 28 561,77 45 353,88 Preparation Summer School 2012 Barcelona 1 23/11/11 EURORDIS 113,98 66,90 180,88

B.3.e. Clinical Trial Charter 139,30 139,30 Clinical Trial Directive Brussels ~ 100 01/12/11 EUROPABIO 139,30 139,30

B.3.d. HTA training 1 108,10 1 412,52 717,56 1 078,30 4 316,48 DIA Euromeeting Geneva ~ 2500 27/03/11 DIA 595,53 463,31 98,48 186,05 1 343,37 HTA Training Tirol ~ 25 05/04/11 UMIT 243,19 490,30 619,08 892,25 2 244,82 Post Approval Summit Zurich ~ 100 21/09/11 Post Approval Summit 269,38 458,91 728,29

A.d. RDD 2011 & media monitoring and Video contest 1 512,26 1 193,93 3 466,62 1 764,02 7 936,83 Preparation European Workshop RDD 2011 Brussels 1 23/02/11 EURORDIS 180,00 180,00 European Workshop RDD 2011 Budapest ~ 40 26/02/11 Huferdis 372,26 83,48 455,74 European Workshop RDD 2011 Brussels 86 28/02/11 EURORDIS 960,00 1 110,45 3 466,62 1 764,02 7 301,09

A.b. Preparation of ECRD 2012 4 079,66 1 865,44 4 695,57 1 177,57 11 818,24 Preparation ECRD 2012 Basel 2 06/05/11 EURORDIS 417,11 85,23 502,34 Preparation PC meeting ECRD 2012 Brussels 1 31/05/11 EURORDIS 156,00 38,90 194,90 Preparation PC meeting ECRD 2012 Brussels 1 05/07/11 EURORDIS 90,00 85,00 175,00 Preparation PC meeting ECRD 2012 Brussels 1 23/09/11 EURORDIS 132,00 132,00 1st ECRD 2012 Programm Committee Paris 12 26/09/11 EURORDIS 112,00 75,50 2 714,50 726,51 3 628,51 NORD-DIA Global RD Conf. Washington D.C. Washington ~ 350 11/10/11 NORD-DIA 3 060,55 1 400,22 4 460,77 Preparation ECRD 2013 and 2014 Barcelona 1 29/11/11 EURORDIS 107,79 107,79 2nd ECRD 2012 Programm Committee Paris 12 09/12/11 EURORDIS 112,00 72,80 1 981,07 451,06 2 616,93

Grand Total 14097,21 17645,03 30762,38 42475,94 104980,56

TotalPurpose sorted by activity and by date Place # of participants Date Organiser

Operating Grant EURORDIS n°20103205 Final Report 2.5. List of conferences, seminars,etc. - Page 60

Operating Grant EURORDIS n°20103205 Final Report 2.5. List of conferences, seminars,etc. - Page 61

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