Authors: Roberta Joppi Vittorio Bertele’ Silvio Garattini
Publish Date: 2012/10/23
Volume: 69, Issue: 4, Pages: 1009-1024
Abstract
From 2000 up to 2010 809 of the 845 candidate orphan drug designations received a positive opinion from the European Medicines Agency EMA’s Committee on Orphan Medicinal Products Of the 108 OMP marketing authorizations applied for 63 were granted Randomised clinical trials were done for 38 OMPs and placebo was used as comparator for nearly half the licensed drugs One third of the OMPs were tested in trials involving fewer than 100 patients and more than half in trials with 100–200 cases The clinical trials lasted less than one year for 429 of the approved OMPsAlthough there may have been some small improvements over time in the methods for developing OMPs in our opinion the number of patients studied the use of placebo as control the type of outcome measure and the followup have often been inadequate The present system should be changed to find better ways of fostering the development of effective and sustainable treatments for patients with orphan diseases Public funds supporting independent clinical research on OMPs could bridge the gap between designation and approval More stringent criteria to assess OMPs’ efficacy and cost/effectiveness would improve the clinical value and the affordability of products allowed onto the market
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