Abstract
In the summer of 2012, a large public debate took place in The Netherlands on the reimbursement of two expensive orphan drugs for Pompe and Fabry disease. On September 21, 2012 the appraisal committee (ACP) of the National Health Care Institute Package (ZIN) had to take a decision on this issue. Since its installation in 2008, I have been a member of the ACP as the patient representative. In this article, I will first outline the main discussions in the ACP of the scientific assessment and the societal appraisal process. Secondly, I will add some learning points for the international patient community. The overall message is that, right from the start, patient groups should be involved in all stages of decision making concerning both registration and reimbursement. This would make decisions more appropriate for patients, for the general public and for the healthcare system. Concerning rare diseases, the importance of a single European reimbursement procedure is discussed.
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