How to reimburse and pay orphan drugs for rare diseases?

Orphan drugs are very expensive drugs that are used to treat rare diseases. Although there are between 5,000 and 7,000 rare diseases affecting a total of 30-40 million patients in Europe, only 151 orphan drugs are available to treat such patients. As a result, market access is an issue of high priority. Of those 151 orphan drugs, only 86 drugs are fully reimbursed by RIZIV/INAMI in Belgium, implying that the patient does not have to pay for these drugs. This observation raises two issues, which this project wishes to address.

First, we need to develop approaches that assess the value of orphan drugs, calculate the budget impact of orphan drugs and follow up the market entry of orphan drugs, in order to make sure that only innovative and valuable orphan drugs are reimbursed.

Second, the sizeable budget impact associated with orphan drugs raises the question of how much money we can spend on orphan drugs and on other drugs, and calls into question the sustainability of orphan drug expenditure in the long run. Therefore, new approaches are needed to pay for R&D and reimbursement of innovative and valuable orphan drugs.

Although reimbursement and payment of orphan drugs is high on the political agenda, few studies have actually provided advice on how these challenges can be addressed. Thus, this project wishes to contribute to developing a sustainable, balanced, transparent and ethical approach to reimburse and to pay for orphan drugs for rare diseases.