Federal auditors are looking into whether the Orphan Drug Act still serves its intended purpose, and whether drugmakers may be abusing its incentives to maximize profits.
The Government Accountability Office said Wednesday that it's initiating the investigation at the request of Sens. Orrin Hatch (R-Utah), Chuck Grassley (R-Iowa) and Tom Cotton (R-Ark.), who sent a letter to the head of the agency earlier this month. The senators are inquiring whether changes may be needed to preserve the law's initial intent to encourage the development of drugs for rare diseases that affect less than 200,000 people.
The senators said they're responding to media reports suggesting some pharmaceutical manufacturers may be “taking advantage” of the act's lucrative incentives. An investigation by Kaiser Health News in January found that many orphan drugs have exorbitant price tags, while some manufacturers may be obtaining orphan status for drugs that have already been on the market, giving them millions of dollars in incentives and seven-year exclusivity on drugs that aren't entirely new.
“It is essential that we maintain the integrity of this important law and continue to stimulate the pipeline for rare disease drugs,” the senators wrote.
The GAO accepted the senators' request, but staff on the healthcare team are currently working on other reports and won't be able to begin the investigation until December, the GAO told the senators. A. Nicole Clowers, managing director of healthcare at the GAO, has been assigned to oversee the request, according to a letter from the agency.
Before the GAO can give a timeline when the investigation will be complete, auditors must determine the scope of the report and what methodology it will use to examine the issue, a GAO spokesman said.