GOA to investigate FDA’s orphan drug program

The Government Accountability Office (GAO) has confirmed it will investigate the U.S. Food and Drug Administrations (FDA) Orphan Drug program following a letter from senators citing possible exploitation.

The letter written by Sens. Orrin Hatch (R-Utah), Chuck Grassley (R-Iowa) and Tom Cotton (R-Arkansas) urges the GAO to investigate the ODA and its incentivization of drugs for diseases affecting fewer than 200,000 people. The ODA, introduced in 1983, aimed at increasing the production of drugs through incentives for patients with less common diseases. As a result, more than 600 orphan drugs and 4,000 orphan drug destinations have been approved by the FDA and saved the lives of patients who would have otherwise died without the drugs being developed.

According to Kaiser Health News (KHN), “in recent months, reports of five- and six-figure annual price tags for orphan drugs have amplified long-simmering concerns about abuse of the law.” KHN subsequently published its findings in an investigation accusing drug makers of abusing the ODA for profit.