Posted by Michael Wonder on 29 Jun 2016
Letting post-marketing bridge the evidence gap: the case of orphan drugs
22 June 2016 - Post-approval studies seldom cover the deficit of knowledge about orphan drugs.
Developing medicines for rare diseases is difficult. Small target populations limit the potential to recover investments in research and development, and even when medicines get to clinical trials, there may be too few patients to support adequately sized trials. Trials for these drugs often also have other shortcomings—for example, the use of placebo as control, surrogate endpoints instead of hard clinical outcomes, or an inadequate length of follow-up. As a result, orphan drugs—those intended for rare diseases—are not only few but often have insufficient evidence of efficacy and safety at the time of approval.
