Two groups of senators have launched investigations into orphan drugs to examine whether drugmakers are misusing the designation and overcharging for treatments.
Sens. Chuck Grassley (R-Iowa), Tom Cotton (R-Ark.) and Orrin Hatch (R-Utah) have asked the Government Accountability Office to evaluate whether the FDA’s resources have kept up with the demand for orphan drug designations, and whether the agency’s evaluation process for orphan drugs is consistent across reviews.
The senators have requested a comprehensive list of orphan drugs approved since 1983 — when the Orphan Drug Act was enacted — and a catalog of drugs seeking the designation over the past year. The GAO should make recommendations for regulatory or legislative changes to preserve the intent of the law, the letter said.