Review and comparison of clinical evidence submitted to support European Medicines Agency market authorization of orphan-designated oncological treatments

Conclusions:
Clinical trials of orphan treatments are often limited by low patient numbers and inadequate follow-up. Pooling of expertise in single centres and the establishment of rare disease reference networks and patient registries may facilitate appropriate trial design for orphan-designated treatments. This analysis found that the pivotal clinical trial for mifamurtide in osteosarcoma had the largest number of patient-years of follow-up, despite a small eligible patient population, showing that it is possible to conduct studies with an adequate patient population size and duration of follow-up in patient-years, and a comparative design with clinical, survival-based, endpoints.

Source: Orphanet Journal of Rare Diseases - October 28, 2015 Category: Internal Medicine Authors: Julie WinstoneShkun ChaddaStephen RalstonPeter Sajosi Source Type: research