FDA Moves To Rein In Drugmakers’ Abuse Of Orphan Drug Law
The FDA is changing the way it approves orphan drugs after realizing drugmakers may be abusing the Orphan Drug Act. FDA Commissioner Scott Gottlieb said he wants to ensure that financial incentives are granted the way Congress intended when the act was passed in 1983. This law offered incentives, including tax credits, user fee waivers and seven years of market exclusivity if drugmakers developed medicines for rare diseases.
An investigation by Kaiser Health News found that many drugs with orphan status aren’t exactly new. Of about 450 drugs that have won orphan approval, over 70 were drugs first approved for mass-market use.
Gottlieb plans to close a loophole that allows manufacturers to skip pediatric testing requirements when developing a mass-market drug for treating rare diseases in children, and he signaled that bigger actions are being considered.
