POLICIES FOR RARE DISEASES

Our client was the Belgian Health Care Knowledge Centre (KCE)

In this study, we analysed policies on orphan drugs: drugs for patients with a rare disease. More specifically, the study aimed at providing an overview of the commonly used definitions for orphan drugs at an international level, describing the current Belgian policy with regard to the reimbursement of orphan drugs and comparing the procedures that exist in other countries for decision-making about the reimbursement of orphan drugs.

The study found that European stimuli for the development of orphan drugs, such as 10 years of market exclusivity, are successful. The KCE concluded that when these drugs appear to be clearly profitable, this exclusivity should be reconsidered. Furthermore, setting up European patient registers would allow a better documentation of the effects of these (often) expensive treatments. Lastly, the procedures for obtaining reimbursements for individual patients can be simplified.

Based on the study findings in 2009, recommendations were formulated for the improvement of the Belgian policy.

https://kce.fgov.be/en/policies-for-rare-diseases-and-orphan-drugs