SEVENTH WORKSHOP ON PARTNERING FOR RARE
DISEASE THERAPY DEVELOPMENT
Madrid, 26-27 October 2006
“Positioning Rare Diseases on the Healthcare Agenda”
Venue:
IMSERSO
Spanish Ministry of Labour and Social Services
Calle Ginzo de Limia 58
28029 Madrid
DRAFT PROGRAMME
Chairs of the Workshop:
| Fernando Royo Genzyme |
Segolène Aymé Orphanet |
Rosa Sanchez de Vega FEDER |
| DAY 1 - Thursday, October 26, 2006 | |
| 8.30 | Registration opens |
| 9.00 | Opening session: Welcome and Introductory Note Michael Griffith, EPPOSI, Chairman Introductory remarks Rosa Sanchez de Vega, FEDER Welcome remarks Alfonso Jiménez Palacios, Director General of Quality - Ministry of Health & Consumer Affairs |
| 9.30 | SESSION 1 – How to enable the appropriate assessment of new therapies? |
| Chaired by: | Michael Griffith, Chair EPPOSI Alfonso Jimenez Palacios, Director General of Quality - Ministry of Health & Consumer Affairs |
| 9.30 | Impact of the trial directive and of the advanced
therapy regulation on clinical trials for rare diseases: the point of view
of Academia Bruce Morland, Birmingham Children's Hospital, United Kingdom |
| 9.50 | Impact of the clinical trial directive and of the draft
Advanced Therapy Regulation on clinical trials for rare diseases: the point of view
of Industry Maria Pascual-Martinez, Cellerix |
| 10.10 | Interactive discussion |
| 11.00 | Coffee Break |
| 11.30 | SESSION 2 – How to assess the benefit of therapies for patients and their families? |
| Chaired by: | Pauline Evers, EGAN Josep Torrent-Farnell, COMP |
| 11.30 | Assessment of the therapeutic added value: methods and
their limits Panos Kanaos or R. Ömer Saka, London School of Economics |
| 11.50 | Assessment of the therapeutic added value: the point of
view of Industry Andrea Rappagliosi, EU Commission Pharmaceutical Forum (representing EuropaBio) |
| 12.10 | Assessment of the therapeutic added value: the point of
view of Regulators François Meyer, HAS - Haute Autorité de Santé, France |
| 12.30 | Assessment of the therapeutic added value: the point of
view of Patients Andrea Buzzi, Italian Haemophilia Society |
| 12.50 | Assessment of the therapeutic added value: the point of
view of Payers Ad Schuurman, chairman of the Medicine Evaluation Committee (MEDEV) of the European Social Insurance Fund |
| 13.10 | Interactive discussion |
| 13.45 | Lunch |
| 15.00 | SESSION 3 – How to enable the development of orphan indications of marketed drugs? |
| Chaired by: | Jan Inge Henter, Karolinska Institute Miguel Angel Izquierdo, Pharmamar |
| 15.00 | Orphan indications which could be developed Valérie Thibaudeau, Orphanet |
| 15.20 | The development of orphan indications : the point of
view of EMEA Channa Debruyne, EMEA |
| 15.40 | The development of orphan indications : the point of
view of FDA Marlene Haffner, FDA |
| 16.00 | Coffee Break |
| 16.30 | The development of orphan indications: the point of view
of Industry Pierre Vankan, VP Drug Development, Santhera |
| 16.50 | Support to the development of orphan indications by
academic groups Segolene Aymé, Orphanet |
| 17.10 | The development of orphan indications: the point of view
of the Mutual Benefit Societies Heidi Goethals, AIM - International Association of Mutual Benefit Society |
| 17.30 | Interactive discussion |
| 18.30 | End of session |
| 21.00 | Dinner with all participants |
| DAY 2 - Friday, October 27, 2006 | |
| 9.30 | SESSION 4 – How to ensure access to optimal treatment for patients |
| Chaired by: | Dianne Dorman, NORD Kerstin Westermark, COMP |
| 9.30 | Would Centres of Reference contribute to better treat
patients? Ségolène Aymé, Rare Diseases Task Force |
| 9.50 | How to speed up access to orphan drugs in Europe Yann Le Cam, Eurordis |
| 10.10 | Reimbursement policies: what can be shared between
health technology assessment agencies? Finn Børlum Kristensen, Danish Centre for Evaluation and Health Technology Assessment National Board of Healh |
| 10.30 | Responsibility of Industry in facilitating access to
drugs Erik Tambuyzer, chair joint task force OMP EBE / EuropaBio |
| 10.50 | Interactive discussion |
| 11.20 | Coffee Break |
| 11.50 | SESSION 5 – Positioning Rare Diseases on the healthcare agenda |
| Chaired by: | Lisa Wise, Genetic Alliance of America |
| 11.50 | Where do we go from here? |
| Round table with representatives from: - EMEA: Channa Debruyne - COMP: Kerstin Westermark - Office of Rare Diseases: Steve Groft, NIH - FDA: Marlene Haffner - Genetic Alliance of America: Lisa Wise - NORD: Dianne Dorman - Rare Diseases Task Force: Ségolène Aymé - Eurordis: Yann Le Cam, - FEDER: Rosa Sanchez de Vega - French Ministry of Health for the French Action Plan: Alexandra Fourcade (TBC) - Co-chair of the organising committee: Fernando Royo |
|
| 12.30 | Interactive discussion |
| 13.00 | Summary of Recommendations & Outcomes of the Workshop Ségolène Aymé |
| 13.15 | Closing remarks by Maria Teresa Pagès, General Director of
Pharmacy and Health Products - Ministry of
Health & Consumer Affairs Lunch |