SIXTH WORKSHOP ON PARTNERING FOR RARE
DISEASE THERAPY DEVELOPMENT
London, 25-27 October 2005
“People with Rare Diseases – No Longer Alone in the
World”
Venue:
Department of Trade and Industry Conference Centre
1, Victoria Street, London SW1
DRAFT PROGRAMME
Chair of the Workshop: Alastair Kent, Genetic Interest Group & European Alliance of Patient & Parent Organisations for Genetic Services & Innovation in Medicine
| DAY 1 - Tuesday, October 25, 2005 | |
| 11:30 | Registration Opens |
| 12:00 | Light Lunch |
| 13:00 | Introductory remarks Alastair Kent, Conference Chair, United Kingdom |
| 13:30* | SESSION 1 – Encouraging Research and Setting Priorities |
| Chaired by: | Alastair Kent, Genetic Interest Group, United Kingdom - Board Member of EPPOSI |
| 13:35* | What is a Priority from the Patients’ Point of View?
Terkel Andersen, Danish Haemophilia Society & Eurordis, France |
| 13:45* |
What are
the Priorities from the Academic Point of View? Ketty Schwartz, INSERM, Institut de Myologie, France |
| 13.55* | Encouraging Research into Rare Diseases – a Global View Steve Groft, Office of Rare Diseases, National Institute of Health, USA |
| 14:15* | Streamlining Research & Methodologies for Setting
Priorities Sir Michael Rawlins, National Institute for Clinical Excellence (NICE), UK |
| 14.35* | What Influences Industry’s Priority-Setting in rare disease
R&D? Paul Herrling, Novartis AG, Switzerland |
| 14:55* | Interactive discussion |
| 15:20* | Coffee break |
| 15:40* | SESSION 2 – Facilitating Development |
| Chaired by: | Erik Tambuyzer, Genzyme Europe, Belgium - Board member EPPOSI |
| 15:45* | What prevents designations turning into products? 20 Years
Experience Marlene Haffner, FDA, USA |
| 16:05* |
What do we
need to secure investment in rare disease therapy developments? Jane Fisken, Scottish Equity Partners, United Kingdom |
| 16:25* | The OrphanXChange experience Ségolène Aymé, INSERM & Orphanet, France |
| 16:45* | The impact of Regulation 141/2000 on the EU Economic
Activity in the Field of Rare Diseases Alejandro Manchado, EuropaBio/EBE Orphan Drugs Task Force, Belgium |
| 17:05* | What does EMEA do to facilitate development of Rare
Disease Therapies Spiros Vamvakas, European Medicines Agency (EMEA), United Kingdom |
| 17:25* | Interactive discussion |
| 17:45* | Closing remarks of Day 1 Workshop Chair |
| 19:00* | Cocktail Reception at the Crowne Plaza |
| 20:00 | Dinner for all at the Crowne Plaza |
| * please note the changes in alloted time slots | |
| DAY 2 - Wednesday, October 26, 2005 | |
| 08:30 | Coffee |
| 9:00 | SESSION 3 – Improving Diagnosis and Earlier Treatment |
| Chaired by: | Ségolène Aymé, Orphanet, France – Board Member EPPOSI
Bruce Trapnell, Cincinnati Children's Hospital Medical Center |
| 9:05 | Where are we and where do we want to go? The Eurogentest project Jean-Jacques Cassiman, University of Leuven, Belgium – Board Member EPPOSI |
| 9:30 | The US Rare Disease Laboratory Network– How did we make it happen? Joe Boone, CDC, Atlanta GA, USA |
| 9:50 | Clinical Service Delivery in Rare Diseases Dian Donnai, University of Manchester, United Kingdom |
| 10:10 | Clinical Utility and Validation of Genetic Tests for Rare Diseases –
academic view Ron Zimmern, Public Health Genetics Unit, Cambridge, United Kingdom |
| 10:20 | Clinical Utility and Validation of Genetic Tests for Rare Diseases –
industry view Andy Bufton, European Diagnostics Manufacturers Association (EDMA), Belgium |
| 10:30 | Interactive discussion |
| 11.00 | Coffee Break |
| 11:15 | SESSION 4 – Facilitating Clinical Trials |
| Chaired by: | Bruce Morland, Birmingham Children’s Hospital, United Kingdom |
| 11:20 | The Impact of the Clinical Trials Directive outside the Pharmaceutical
Industry Richard Sullivan, Cancer Research, United Kingdom |
| 11:40 | Biomarkers and clinical endpoints Elisabeth Svanberg, Serono International & University of Göteborg, Sweden |
| 12:00 | Statistical Issues in Designing & Analysing Trials in Small Populations Wolfgang Köpcke, University of Münster, Germany |
| 12:20 | The Challenge for Paediatric Research – Clinical Trials in a Key
Population Gerard Pons, René Descartes University, Paris, France |
| 12.40 | Involving the Patient in Clinical Trials Design Nancye Buelow of the Genetic Alliance Board of Directors, USA |
| 13:00 | Interactive discussion |
| 13:45 | Lunch |
| 14:15 | SESSION 5 – Harmonising Reference Frames |
| Chaired by: | Rodney Elgie, GAMIAN - Europe, United Kingdom – Board member EPPOSI |
| 14:20 | Harmonising EU and US Regulatory Requirements – What is Needed? Alison Lawton, Genzyme, USA |
| 14:40 | Cooperation between the Agencies supporting orphan therapy development
– A European View Agnès Saint-Raymond, European Medicines Agency (EMEA), United Kingdom |
| 15:00 | Cooperation between the Agencies supporting orphan therapy development
- A View from the US Marlene Haffner, FDA, USA |
| 15.20 | The European Commission’s Vision on international collaboration for
orphan drugs and orphan devices Martin Terberger, European Commission, Pharmaceuticals Unit of DG Enterprise and Industry, Belgium |
| 15:40 | Interactive discussion |
| 16:00 | Coffee break |
| 16:20 | SESSION 6 – Facilitating Access to Treatment |
| Chaired by: | Andrea Rappagliosi, Serono International, Switzerland – Board Member EPPOSI |
| 16:25 | Securing Treatment for Patients – the Dutch Experience Bert Leufkens, Chair Dutch Steering Committee Orphan Drugs, The Netherlands |
| 16:45 | How to improve patient's access to orphan drugs in
Europe? Yann le Cam, Eurordis, France |
| 17:05 | How to optimize value for orphan drugs? The social value of orphan
medicinal products Rod Taylor – University of Birmingham, United Kingdom |
| 17:25 | Interactive discussion |
| 17:50* | Closing remarks of Day 2 & Invitation to Submit Recommendations Workshop Chair |
| Free evening | |
| 19:00* | Cocktail & Dinner for Speakers & Guests |
| * please note the changes in alloted time slots | |
| DAY 3 - Thursday, October 27, 2005 | |
| 08:00* | Coffee |
| 8:15* | SESSION 7 – The Global Perspective |
| Chaired by: | Ijsbrand Poortman, Chair of EPPOSI |
| 8:20* | Where do patients with rare diseases stand on a global scale? Bart Wynberg, Dutch Ministry of Health, Netherlands |
| 8:40* | The Indian perspective on rare and congenital disorders Jai Rup Singh, Guru Nanak Dev University, Amritsar, India |
| 9:10* | Rare diseases and orphan drugs in Northern Africa Sonia Abdelhak, Institut Pasteur Tunis, Tunesia |
| 9:30* | Patients’ access to orphan drugs in Russia Nicolai P. Bochkov, Vice-President of the Medical Academy, Russia |
| 9:50* | Interactive discussion |
| 10.15* | Coffee Break |
| 10:30* | SESSION 8 – Generating Action and Securing Commitment |
| Moderated by: | Geoff Watts, BBC Radio, UK |
| 10:35* | “White Paper” – Steps towards an Optimal Orphan System in Europe Catarina Edfjäll, Actelion, EBE/EuropaBio Orphan Drugs Task Force, Belgium |
| 10:40* | The Joint Technology Initiative for Innovative Medicines in Europe –
Where Do Rare Diseases Fit? Paul Herrling, EFPIA Research Director’s Group, Belgium |
| 10:45* | The views of the rare disease research and treating
community Tim Cox, University of Cambridge, United Kingdom |
| 10.55* | What do the patients define as priorities? Diane Dorman, NORD, USA |
| 11:00* | Where do we go from here? Recommendations by the audience discussed Panellists: - Catarina Edfjäll, Actelion - Paul Herrling, EFPIA - Tim Cox, University of Cambridge - Diane Dorman, NORD - Steve Groft, NIH - Thomas Lönngren, EMEA - Marlene Haffner, FDA - Alastair Kent, Co-chair of the organising committee - Segolène Aymé, Co-chair of the organising committee - Erik Tambuyzer, Co-chair of the organising committee |
| 11.45* | Summary of Recommendations & Outcomes of the Workshop Workshop Chair |
| 12:00* | Close of Workshop |
| 12:15* | Press Conference (meeting point registration desk) |
| * please note the changes in alloted time slots | |