When entering the market, orphan drugs are associated with substantial prices and a high degree of uncertainty regarding safety and effectiveness. This makes decision making about the reimbursement of these drugs a complex exercise. To advance on this, the Dutch government introduced a...

Considerations beyond cost-effectiveness are important in reimbursement decision making. We assessed the importance of disease severity in drug reimbursement decision making in Belgium, France, The Netherlands and Sweden. We investigated scientific literature and policy documents and conducted...

To investigate the desirability and feasibility of a cyclic reimbursement process to address uncertainty accompanying initial decision making.