Study: FDA Flexibility Helps With Orphan Drug Approvals

Monday, May 11, 2015

Source: FDA News

The FDA is not requiring ordinary proof of effectiveness as it strives to approve orphan drugs for some 7,000 diseases, only 300 of which currently have treatment options, a new study concludes.

The authors, Frank J. Sasinowski, Erika B. Panico and James E. Valentine, looked at 27 noncancer orphan drugs approved between July 1, 2010, and June 30, 2014. They found that two-thirds of sponsors bringing these drugs through the FDA won approvals, despite not providing the gold standard of proof of safety and effectiveness, according to the study in Therapeutic Innovation & Regulatory Science.

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