Ethical, legal and social implications of rare diseases and orphan drugs in Europe

Day 1:

Opening: Aim and scope of the symposium (09:00-09:10)
I. Setting the scene (09:10-11:00)
a. Steven Simoens
Arguments against current orphan drug regulations and practice
b. David Cassiman
Arguments pro current orphan drug regulations and practice
c. Eline Picavet
Clinical trials of orphan drugs: quality of evidence
d. Carla Hollak
Cost-effectiveness issues in enzyme replacement therapy for lysosomal storage diseases Coffee break (11:00-11:30)
II. Industry and Patient perspectives (11:30-13:00)
a. Fabrizia Bignami
Stakeholder involvement as a key for success in orphan drug development
b. Tsveta Schyns
Patient advocacy for rare diseases in the EU
c. Cees Smit
HTA for rare diseases and the cost-effectiveness of treatment: a patient perspective

Lunch (13:00 – 14:00)

III. Policy and regulation (14:00-16:00)
a. Ségolène Aimé:
The impact of initiatives and incentives for rare diseases and orphan drugsw
b. Marc Dooms
Orphan Drugs: Experiences from the Hospital Pharmacy
c. Heidi Howard:
Databases and biobanks for rare diseases
d. Thomas Morel:
Market access and reimbursement policies

Tea break (16:00-16:30)

IV. Ethics (16:30-18:00)
a. Wim Pinxten
Distributive Justice and Resource allocation
b. Hans-Joerg Ehni:
Vulnerability and protection
c. Nils-Eric Sahlin:
Medical Ethics, Rare diseases and priority Setting

Day 2:

Practical arrangements for the Symposium Proceedings (09:00-09:15)

Introduction to the workshops (09:15-09:30)

Parallel Workshops (09:30-11:00)

• Workshop I: Symposium recommendations regarding clinical trials and patient databases

• Workshop II: Symposium recommendations regarding cost-effectiveness and distributive justice

• Workshop III: Symposium recommendations regarding patient and industry involvement

Coffee break (11:00-11:30)

Voting on the Symposium recommendations from the workshops (11:30-12:00)

Policy Forum (12:00-13:00)