Post-authorisation assessment of orphan drugs

The EU regulation of orphan drugs has promoted the development of new treatments for rare disorders.1 However, the high cost of most orphan drugs threatens the sustainability of public health care. Unfortunately, the effectiveness of treatment is often unclear for part, if not all, of the patient population, especially for patients with very rare diseases, such as inherited metabolic disorders. We believe that the system of post-authorisation assessment for orphan drugs needs to be reformed to address these problems.