1
7th International Plasma Product Biotechnology Meeting, PPB11
Ruediger Gatermann
CSL Behring, Public Affairs Europe
Chair, PPTA Health Policy Steering Committee
May 12, 2011
European Policy Initiatives for Rare (Plasma) Diseases
2
Agenda for today
European challenges in health policy for rare diseases
Current healthcare challenges
European policy initiatives
National Plans for Rare Diseases
EUROPLAN project: National Conferences
Set-up of common indicators (spring 2011) for National Plans (until 2013)
European PID Recommendations
EU Expert Panel and political support
Overview on key recommendations
Other recent initiatives
European Principles of Hemophilia Care
European Voluntary Blood Donations Report
3
The CSL Group
4
PPTA* Mission: Plasma Industry‘s key goals
Protecting patients current access to therapy while expanding care worldwide
Promoting adequate reimbursement policies
Engaging with regulatory authorities to foster informed regulatory policies
Fostering awareness and diagnosis by working with patient advocacy organizations
Removing trade barriers
Expand quality of care worldwide
Assure Access to
Safe and Effective
Plasma Therapeutics
for Patients Worldwide
*Plasma Protein Therapeutics Association
5
Still many blank spots in detection and diagnosis of patients
Condition Organisation
European
National
Organisations
Known
Patients
Probable
European
Prevalence
Haemophilia EHC 44 45,000 50,000
WFH (Global) (131,000) (400,000)
Von Willebrands EHC 44 25,000 100,000
Primary Immunodeficiency IPOPI 27 7,000 60,000
Alpha-1 Antitrypsin Alpha Europe 13 125,000
Guillain-Barré GBS/CIDP
Foundation 14 5,700
Hereditary Angio-oedema HAEI 7,600
Idiopathic
Thrombocytopenic Purpura
ITP Support
Association 3,000 38,000
TOTAL 7 80,000 386,000
Source: PLUS – Plasma Users Group
6
Healthcare challenges increase in times of financial crisis
Overall healthcare costs are rising in response to key demographic and economic factors
Consequently, governments and payers across major markets are implementing numerous cost-containment policies with direct impact on drug margins
Health Technology Assessment is focusing on most expensive therapy areas, leading to new challenges for rare disorders including plasma therapies
Success of the plasma industry is determined, to a great extent, by the external environment in which we operate
Political interest for specific areas (innovation, biotech, rare diseases…) may lead to favourable sector policies despite general cost cutting efforts
Patient empowerment adds new options for alliances if mutually agreed and established in an open and transparent manner
7
Pricing controls are used extensively across major markets…
1. Profit controls
2. Reference pricing
3. Price cuts, freezes and ceilings
4. Discounts & rebates
… and reimbursement controls are becoming even more popular
Pharmacoeconomics, Health Technology Assessment (HTA)
Risk sharing
Volume limitations
Formulary positive / negative lists
8
EU outlook: On national level, cross fertilization in cost cutting goes on
• NICE* agree to risk-sharing after intense pressure from patient groups and
physicians
• NICE expected to expand its remit, undertaking pricing, de-investment and a
chargeable consultancy service to Pharma in designing clinical trials
• Referencing pricing has cut costs but it is partly responsible for a drop in
investor confidence in Spain as an R&D center
• Italy to pay increased attention to pharmaceutical innovation, after decreases in
pharmaceutical drug spending
• Reimbursement drug list reforms are considered insufficient in cutting costs
due to reported lack of physician compliance
• Germany continues to implement cost cutting policies that effect both branded
and generic pharma alike
*National Institute for Clinical Excellence
9
On European level, EU institutions become more influential in health policy
EU Commission and Parliament take more and more the lead in health policy agenda setting
Rare diseases are increasingly recognized as health priority
Plasma disorders are unique, treatable but still often neglected
Plasma industry’s public affairs efforts are focused on positioning Plasma disorders as a key area in the context of rare diseases
Recent EU policy initiatives: European Conferences on Rare Diseases
European Commission Task Force on Rare Diseases
Rare Diseases in EU Community Research Framework Programme (FP7)
New Communication and Council Recommendation on rare diseases
10
New Recommendation on Rare Diseases provides framework for further improvement
National plans for rare diseases in all
EU Member States
Establishment of EU reference networks
Common definition of rare diseases and revision of international classification (ICD)
Establishment of an inventory of rare diseases
Common databases and medical protocol for identification of rare diseases
Availability and accessibility of accurate diagnostic tests
Population screening strategies for rare diseases
11
The task for the next years:
Securing aligned national plans that tackle key issues
Best practices on rare diseases care
Equal access to therapies, including orphan drugs
Development of national/regional centres of reference
Health Technology Assessment of orphan drugs
Coordinated compassionate use programmes
Networks of research for rare diseases
Common approach to the empowerment of patient organisations
12
Rare Diseases National Plans Development
13
EUROPLAN Timetable: Here we are…
EUROPLAN Workshop in Krakow (May 2010)
with Health Authorities:
EUROPLAN recommendations approved
Discuss the recommendations with
stakeholders throughout National Conferences
organized by EURORDIS (National Alliances) in 15 countries
Present the final version of documents
(recommendations and indicators) at the
international conference (Roma, Spring 2011)
14
Calendar of EUROPLAN Conferences
Date in 2010 Country City Organiser
1 28-29-30 May BULGARIA Sofia NAPRD National Alliance of People with RD
2 18-19 June ROMANIA Bucarest RONARD Romanian National Alliance for RD
3 18-19-20 September CROATIA Dubrovnik Hrvatska udruga bolesnikas rijetkim bolestima
4 30 September FRANCE Paris Alliance Maladies Rares
5 13-14 October GERMANY Berlin ACHSE Allianz Chronischer Seltener Erkrankungen
6 18-19 October HUNGARY Budapest HUFERDIS Rare Disease Hungary
7 5-6 November SPAIN Burgos FEDER Federación Española de Enfermedades Raras
8 11 November SWEDEN Stockholm Sällsynta diagnoser
9 11-12-13 November ITALY Rome UNIAMO Federazione Italiana Malattie Rare
10 16 November UNITED KINGDOM London Genetic Interest Group - Rare Diseases UK
11 19 November NETHERLANDS The Hague VSOP Dutch Genetic Alliance
12 19 November DENMARK Copenhagen Rare Disorders Denmark
13 26-27 November GREECE Athens PESPA Greek Alliance for RD
14 3 December IRELAND Dublin GRDO Genetic and Rare Disorders Organisation
15 To be confirmed LUXEMBOURG Luxembourg Groupe de Travail Maladies Rares
16 To be confirmed POLAND To be
confirmed To be confirmed
15
EUROPLAN: Thematic Areas
Five main themes…
1. Methodology and Governance of a National Plan / Strategy (NP)
2. Definition, codification and inventorying of Rare Diseases (RD)
3. Research on RD
4. Standards of care for RDs – Centres of Expertise / European Reference
Networks
5. Patient Empowerment and Specialised Services
… and two horizontal themes 1. Sustainability
2. Gathering expertise at the EU level
16
Industry Trade Associations: EuropaBio/EBE* – Proposed Input for National Plans on Rare Diseases (I) For Use in Discussions at Member State Level
A focus on a small number of core actions will serve rare disease patients
best. Once these are up and running, the programme could be expanded.
Multi-stakeholder advisory groups should be established at national level.
National plans should foresee a system that grants timely and equitable
access to patients where treatment exists.
The National Plans should also foresee the possibility of a reimbursed,
temporary access prior to Marketing Authorisation in specific situations of
a high medical need (similar to the current “ATU”** system in France)
*European Biopharmaceutical Enterprises
** Temporary Authorization for Use
17
Industry Trade Associations: EuropaBio/EBE – Proposed Input for National Plans on Rare Diseases (II) For Use in Discussions at Member State Level
Better and timely diagnosis as well as
screening programmes should be appropriately implemented
in all Member States based on guidelines and protocols
drawn up with EU experts in the field.
Centres of excellence for diagnosis, research and treatment should be
established in Member States and networked between one Member State and
another.
Registries are a vital tool but should be linked at a European level to derive
the maximum benefit. A public-private partnership model is the best way
forward, to avoid losing data and investment in existing registries.
Treatments for rare diseases should be funded at a separate, centralised,
preferably national level.
18
EU Actions on Primary Immunodeficiency
European Awareness Day for Primary Immunodeficiency (23 October 2002)
STOA Workshop on Primary Immunodeficiency (17 March 2004) hosted in the
European Parliament by Antonios Trakatellis MEP* and Giuseppe Nistico MEP.
EU PID Consensus Conference (19-20 June 2006), supported by the European
Commission at the Paul Ehrlich Institute in Germany. A consensus document
representing the views of the European Commission, physicians, patients groups and
industry was adopted.
European Parliament lunch debate on Rare Plasma Disorders (January 2008)
hosted by Miroslav Mikolasik MEP where 9 members of the European Parliament
debated policy options for tackling rare plasma disorders such as PID.
European Parliament debate on European Commission Rare Diseases proposals
and the implications for patients with Rare Plasma Disorders (December 2008)
hosted by Jorgo Chatzimarkakis MEP and Miroslav Mikolasik MEP. Launch of Call for
Action document, headed by both host MEPs and developed by the stakeholders.
* Member of the European Parliament
19
Policy endorsement through EU meetings and events: Rare plasma disorders enter the EU agenda
Results from
multi stakeholder
policy projects
20
European PID Expert Recommendations (2010/2011)
21
European PID Expert Group
Steffen Ball
Deutsche Selbsthilfe Angeborene
Immundefekte (German Support Group for
Primary Immune Deficiencies, DSAI)
Professor Helen Chapel
Professor of Clinical Immunology, University
of Oxford
Gabriele Gründl
Deutsche Selbsthilfe Angeborene
Immundefekte (German Support Group for
Primary Immune Deficiencies, DSAI)
Sven Fandrup
IPOPI (International Patient Organisation for
Primary Immunodeficiencies)
Professor Lennart Hammarström
Karolinska Institutet, Stockholm
Fred Modell
Jeffrey Modell Foundation
Vicki Modell
Jeffrey Modell Foundation
Professor Tim Niehues
Professor of Pediatrics, HELIOS Klinikum
Krefeld
Brian O’Mahony
PLUS (Plasma Users’ Group)
Martine Pergent
IPOPI (International Patient Organisation for
Primary Immunodeficiencies)
Professor Reinhold Schmidt
Director, Dept. of Clinical Immunology,
Hannover Medical School
Larry Warren
PLUS (Plasma Users’ Group)
David Watters
IPOPI (International Patient Organisation for
Primary Immunodeficiencies)
Chair:Jorgo Chatzimarkakis, Member of the European Parliament
22
Expert Recommendations – Primary Immunodeficiency
1. Funding research into better understanding of Primary Immunodeficiency is
vital in terms of both diagnosis and management of the conditions and general
understanding of the immune system. This must be done at both EU and
national level.
2. Patients must be provided with the therapies that they need to live a more
comfortable and productive life with a near-normal life expectancy.
3. An appropriate supply of safe plasma for immunoglobulin extraction to be
collected each year, from both blood donations and plasmapheresis, in order
to ensure patients can receive safe and effective blood therapies that they
need.
4. General Practitioners, Pulmonary Specialists, Hematologists, Ear Nose and
Throat specialists and other physicians need to be made more aware of PID
through training in medical school and awareness campaigns to ensure
better diagnosis of patients.
23
Options for transfer of Recommendations to national level
Translation (Adaptation?) of Recommendations
Foreword from high level national policy maker
Distribution of PID Recommendations (by PPTA WG, Experts…)
Channeling of PID Recommendations to Health Minister (by MEP or Expert)
Preparation of national Parliament event - MP hosts, speakers, meeting
content, PR etc
Follow-Up to national Parliament event with attendants
Involvement of PID in national parliament e.g debates/committees/reports
Awareness campaign to parents on PID diagnosis (via children and family
associations)
Create incentive for PID diagnosis for physicians and ensure allocation of
appropriate funding for treatment of PID patients
24
Policy endorsement through new consensus documents:
The European principles of haemophilia care
Cooperation between
medical KOL and
patient associations
25
EU: Voluntary Blood Donations Report (April 2011)
1st report (2006): Basically casuistic compilation (potentially misleading)
from individual member states’ collection and compensation practices
2nd report (2011): Main progress is that this 2nd report can be seen as an
objective general inventory
Thus, constant policy efforts have borne fruits:
(1) The differentiation between blood and plasma donations is clearly
recognized throughout the text;
(2) The compilation of incentives clearly indicates the various options to
enhance donations while not favoring one approach against the other.
26
Summary (I): Global challenges for rare plasma disorders
1. Lack of awareness and (early) diagnosis
2. Many patients are untreated, insufficient access
3. Limited source material
4. Lack of harmonization
5. Tenders: Price versus quality
6. Barriers to trade
7. Need for paradigm shift: costs = investment in health
8. Need for level playing field
27
Summary (II): What do we need? – Requirements
Good Data – Registries
Reference Centres for treatment
Consensus Treatment Protocols
Clinical Freedom and Patient involvement
Funding for Diagnosis and Treatment
Patient organisation involvement / consultation before decisions made
28
Summary (III): What do we need? – Stakeholder alignment
Successful healthcare programs on national level need active involvement of 5 parties:
1. Patients (Consumer Groups)
2. Physicians (Treaters)
3. Experts (Research)
4. Industry (Manufacturers, Distributors, Providers)
5. Government/Officials (Regulators, Politicians, Payors)
29
THANK YOU FOR YOUR ATTENTION !
Contact:
Ruediger Gatermann
Director, Public Affairs Europe
CSL Behring Biotherapies for Life ™
P.O. Box 1230 | 35002 Marburg | Germany
+49 (0) 6421 39 4264 phone | +49 (0) 6421 39 5550 fax
+49 (0) 171 3035465 mobile
