14 Dec 2011

ICAB nv, Site Arsenaal, Witte Patersstraat 4, 1040 Brussel

There is a need for a transparent and evidence-based approach towards orphan drug pricing and reimbursement. Core issues re the access to orphan drugs are whether registration and reimbursement are evidence based and whether societal considerations can improve the decision making process re the availability of orphan drugs. A transparent approach should be targeted at demonstrating the relative effectiveness, cost-effectiveness and economic viability of orphan drugs with a view to informing pricing and reimbursement decisions.

During this workshop, we want to get an insight in the kind of evidence we have at the moment, the kind of evidence we would like to have and ways to improve the transparency on the pricing and reimbursement.



Organization: VUB CROSSTALKS in collaboration with the Fund Rare Diseases and Orphan Drugs, managed by the King Baudouin Foundation



Chairs: Jean-Jacques Cassiman (Prof. Em. KULeuven and President of the Fund for Rare Diseases and Orphan Drugs), Alain Dupont (Dean, Faculty Medicine & Pharmacology VUB) & Greet Musch (AFMPS-Agence Fédérale des Médicaments et des Produits de Santé/FAGG)

13:30 - 14:00 Registration, coffee and tea 

14:00 - 14:20 Intro: Setting the scene: Recommendations and proposed measures for the Belgian Plan for Rare Diseases & Orphan Drugs - Jean-Jacques Cassiman (President of the Fund - KULeuven)

14:20 - 14:50 Transparency I: Regular monitoring and the right incentive strategy - Sonja van Weely (Scientific officer Dutch Steering Committee Orphan Drugs)

14:50 - 15:20 Transparency II: High prices and low cost-effectiveness of orphan drugs: the reimbursement dilemma - Philippe Van Wilder (VUB & TiGenix)

15:20 - 16:00 Q&A followed by Coffee & tea

16:00 - 16:30 Transparency III: EU perspective on the market entry for orphan drugs - Steven Simoens (KULeuven)

16:30 - 17:00 Transparency IV: High R&D cost and market access - Rare Diseases, Orphan Drugs and their regulation: questions and misconceptions - Erik Tambuyzer (AbConsult, ex-Genzyme)

17:00 - 18:00 FINAL DISCUSSION

18:00 - 19:00 Drinks & Networking