9th WORKSHOP ON PARTNERING FOR RARE
DISEASE THERAPY DEVELOPMENT
Sharing strategies and tools for access to diagnosis and treatment”
French Parliament - Assemblée Nationale
Paris, October 16-17, 2008
An official event of the
The French Presidency of the European Union
Under the High Patronage of
The Co-Chairs of the French Parliament’s Working
Group on Rare Diseases
|
Dr. Marc Laffineur |
Dr. Dominique Orliac |
|
|
|
|
PRELIMINARY PROGRAMME
Organising Committee co-chaired by:
| Yann Le Cam
EURORDIS representing Patients |
Kerstin
Westermark COMP Chairperson representing Science |
Wills
Hughes-Wilson Genzyme Europe representing Industry |
All sessions will be simultaneously translated in English and French
Une traduction simultanée Français/Anglais est prévue pout toutes les sessions
| Thursday, October 16, 2008 | |
| 8:30 | Registration |
| National Session - The First French National Plan for Rare Diseases: Conclusions and Perspectives | |
| 9:00 | Opening Dominique Orliac, Member of the National Assembly, Working Group on Rare Disorders, France Christian Lajoux, Les Entreprises du Médicament, France |
| National Session 1 - The French National Plan for Rare Diseases: main achievements / progress | |
| Moderator: Françoise Antonini, Alliance Maladies Rares (TBC), France | |
| 9.30 | Access to health care and its reimbursement (off-label
use/OTC use) Lionel Joubaud, Direction de la Sécurité Sociale (TBC), France |
| 9.40 | Evolution of the « Medical Service Rendered » evaluation François Meyer, HAS-Haute Autorité de Santé (TBC), France |
| 9.50 | Progress made: an Industry point of view Annick Schwebig, Actelion, Groupe Maladies Rares des Entreprises du Médicament, France |
| 10.00 | Discussion with the audience: Questions and Answers |
| 10.30 | Break
|
| National Session 2 - What can be improved in the Second National Plan for Rare Diseases? | |
| Moderator Marc Brodin, French National Consultative Committee for the Labelling of Centre of Reference for Rare Diseases, France | |
| 11.00 | Epidemiological Registries Ségolène Aymé, INSERM, Orphanet, ESHG, France |
| 11.10 | Information on Clinical Trials Chantal Belorgey, AFSSAPS (TBC), France |
| 11.20 | National Protocols for diagnosis and healthcare Marie-Claude Hittinger, HAS (TBC), France |
| 11.30 | Access to treatment for rare diseases patients OR The
Environment of Research Catherine Lassale, les Entreprises du Médicament, France |
| 11.40 | Discussion with the audience: Questions and Answers |
| 12.15 | Conclusions Paulette Morin, Alliance Maladies Rares, France |
| 12.30 | End of the National Session |
| 12.30 | Lunch (TBC) |
| European Sessions | |
| OPENING | |
| 13:30 | Opening: Setting the scene Marc Laffineur, Member of the French National Assembly, Working Group on Rare Disorders, France |
| 13:50 | Welcome note Alastair Kent, Chairman of EPPOSI, United Kingdom |
| 14:00 | Case study of a crisis: Hunter’s syndrome in Sweden –
what can we learn one year later? Veronica Hübinette, Swedish Mucopolysaccharidosis Society, Sweden |
| 14:10 | Case study of a public policy: what can we learn from
the French National Plan for Rare Diseases to improve access to therapies? François Meyer, HAS , France |
| SESSION 1.1 - Improving early diagnosis and access to care | |
| Chair:
Ségolène Aymé, INSERM, Orphanet, ESHG, France Rapporteur: Alastair Kent, Chairman of EPPOSI, United Kingdom In rare diseases, the success of treatment often depends on early diagnosis. The earlier the diagnosis, the more likely a successful treatment outcome. But how can we best achieve that early diagnosis? When and how should early diagnosis targeted programmes be developed? Should population screening programmes be used and, if so, how and when? This session will aim to explore the range of different approaches currently in place or in development in different European countries to facilitate early diagnosis with the objective of identifying measures to improve the outcomes for patients. |
|
| 14:30 | Is population screening an avenue to explore when a
treatment exists? Martina Cornel, VU University Medical Center, The Netherlands |
| 14.50 | Panel debate Adrian Quartel, BioMarin Europe, United Kingdom Yolande Adjibi, Drépa Action 13, France Olaf A. Bodamer, Austrian Neonatal Screening Programme, Division of Biochemical and Pediatric Genetics, University Children's Hospital Vienna, Austria |
| 15.30 | Discussion with the audience: Questions and concrete
recommendations è When and how should we assess screening strategies when a treatment exists? Which measures to improve the orientation of patients through the healthcare services? Which measures to improve early diagnosis in national plan? When and how to pool expertise and experience at the European level? |
| 16.30 | Coffee break |
| SESSION 1.2 - Improving early diagnosis and access to care | |
| 16:50 | Chair: Alexandra Fourcade, French Ministry for
Health, Youth and Sports, France Rapporteur: Alastair Kent, Chairman of EPPOSI, United Kingdom It is not just rare disease patients who are rare. So are expertise and experts. Such expertise is scattered across Europe and successful diagnosis and treatment too often depends on a lucky accident of geography. How can we build access to the best expertise in diagnosis and treatment via Centres of Expertise and European Reference Networks for rare diseases? And how can such Centres and Networks facilitate access to the best treatment? |
| 16.50 | How to optimise access to appropriate care? Edmund Jessop, National Health Service, United Kingdom |
| 17.10 | Panel discussion Alain Fischer, Hôpital Necker - Enfants Malades, Department of Immunology, Haematology and Rheumatology, France Laura Fregonese, Universiteit Leiden, The Netherlands Christel Nourissier, Prader-Willi France, EURORDIS, France Dennis Jackman, CSL Behring, U.S.A. |
| 17.50 | Discussion with the audience: Questions and concrete
recommendations è How to develop standard of diagnosis and care at national level? How to collaborate at European level to compare and upgrade standard of diagnosis and care? Which measures to optimize pre-marketing and post-marketing studies on orphan drugs through Centres of Expertise and European Reference Networks for rare diseases? |
| 18.50 | End |
| 20.00 | Networking Dinner Welcome note: Alastair Kent, Chairman of EPPOSI, United Kingdom |
| Friday, October 17, 2008 | |
| 08:30 | Doors open |
| SESSION 2.1 - Improving Access to Orphan Drugs | |
| Chair: Andrea Rappagliosi, Merck-Serono, joint task
force EBE-Europabio, Switzerland Rapporteur: Yann Le Cam, EURORDIS, COMP-EMEA, France Orphan drugs, by their very nature, are intended to treat only a small number of patients. This means that gathering clinical outcomes is only possible based on a small number of patients. When healthcare systems are used to dealing with dossiers based on thousands of patients, how can we tailor them to also meet the needs of dossiers based on just tens of patients? Bundling the fragmented know-how between different EU-level committees and the Member states would allow timely production of shared information reports. This session aims to identify methods for building information on clinical added value of orphan medicinal products. |
|
| 9.00 | How to assess clinical added value of orphan medicinal
products? Kerstin Westermark, COMP-EMEA, Sweden |
| 9.20 | Panel debate François Meyer, HAS-Haute Autorité de Santé, France (TBC) Birthe Holm, Rare Disorders Denmark, EURORDIS, COMP-EMEA, Denmark Hans-Georg Eichler, EMEA, United Kingdom Jens Grueger, Novartis, Switzerland |
| 10.00 | Discussion with the audience: Questions and concrete
recommendations è When, where and how to implement European collaboration for common scientific assessment report on the clinical added value of orphan medicines? Which measure in national plans for rare diseases? |
| 11.00 | Coffee break |
| SESSION 2.2 - Improving Access to Orphan Drugs | |
| 11:20 | Chair: Andrea Rappagliosi, Merck-Serono, joint task
force EBE-Europabio, Switzerland Rapporteur: Yann Le Cam, EURORDIS, COMP-EMEA, France Rare disease patients deserve the same access to care as those suffering from more common conditions. But Member States are under pressure to manage healthcare budgets effectively. This means paying only for “things that work”. Established methodologies are not tailored to suit the orphan medicinal products model. There is often a lack of understanding of rare diseases, orphan products and the process by which they are authorised. Do Member States’ decision-makers have the tools they need to make decisions for rare disease treatments? If not, what needs to be changed or developed to help them evaluate this special sub-set of treatments? |
| 11.20 | How should reimbursement policies support access to
treatment? Erik Tambuyzer, Genzyme Corporation, joint task force EBE-EuropaBio, Belgium |
| 11.40 | Panel debate Stefaan Van der Spiegel, European Commission, DG Enterprise, Belgium (TBC) Christine Lavery, Mucopolysaccharidosis Society, United Kingdom Stanislav Primožič, JAZMP - Agency for Medicinal Products and Medical Devices of the Republic of Slovenia, Slovenia (TBC) Andrea Laslop, AGES Austrian Agency for Health and Food Safety, Austria (TBC) |
| 12.20 | Discussion with the audience: Questions and concrete
recommendations è How to share common information to base pricing and reimbursement decision making by Member states? How to implement parallel requests for pricing and reimbursement to all Member states? How to fast track decision making procedures for orphan drugs? How to improve the reimbursement system for orphan medicinal products in the current regulatory framework? |
| 13.20 | Lunch |
| SESSION 3.2 - Awareness and solidarity | |
| Chair: Bert Leufkens, WGM - Dutch Steering Committee
on Rare Diseases and Orphan Drugs, The Netherlands Rapporteur: Wills Hughes-Wilson, Genzyme, Co-Chair of the Workshop, Belgium Care for rare disease patients is delivered at a national level. But rare diseases often need special attention and a specific approach. France implemented a tailored approach for rare diseases over the past 4 years. And multi-stakeholder, expertise-based working groups in several Member States are developing their own strategies and action plans. The Commission Communication on Rare Diseases is providing a policy framework covering the wide spectrum of needed actions, including national plans for rare diseases as a cornerstone. So which measures should Member States deliver in their National Plans for Rare Diseases to deliver equitable access and social justice? |
|
| 14.30 | How to deliver equitable access and social justice for
rare diseases patients and their families? Yann Le Cam, EURORDIS, COMP-EMEA, France |
| 14.50 | Panel debate Domenica Taruscio, ISS, EuroPlan, Italy Mirosław Zielinski, Polish National Forum for Rare Disease Therapy - Krajowe Forum na rzecz terapii chorob rzadkich, Poland Yves Juillet, LEEM, France Maria Puiu, University of Medicine and Pharmacy Victor Babes, Romanian National Organization of Rare Diseases, Romania |
| 15.30 | Discussion with the audience: Questions and concrete
recommendations è How can stakeholders partner best to raise public awareness on rare diseases? Which measures in National Plans for Rare Diseases would we expect to deliver equitable access and social justice? |
| Closing Session | |
| 16.30 | Key messages & recommendations Wills Hughes-Wilson, Genzyme, Co-Chair of the Workshop, Belgium |
| 16.50 | Closing remarks Roselyne Bachelot-Narquin, Minister for Health, Youth and Sports, France (TBC) |
For additional information about our activities:
European Platform for Patients' Organisations, Science and Industry
Rue de l’Industrie, 4 - B1000 Brussels
Tel: +32 2 503 13 07 - Fax: + 32 2 503 31 08
info@epposi.org -
www.epposi.org
