GAO to Investigate FDA’s Orphan Drug Program

Senators complain about drug makers’ abuses

Acting on a request from three influential senators, the Government Accountability Office (GAO) has confirmed that it will investigate potential abuses of the Orphan Drug Act (ODA), according to a report from Kaiser Health News (KHN). The GAO is expected to begin its work in about nine months. The delay is typical, as the agency has a queue of requests it is pursuing.

Earlier this month, Senators Orrin Hatch (R-Utah), Chuck Grassley (R-Iowa), and Tom Cotton (R-Arkansas) sent a letter to the GAO and raised the possibility that regulatory or legislative changes might be needed “to preserve the intent of this vital law” that gives drug makers lucrative incentives to develop medications for rare diseases.

The senators have asked the GAO to “investigate whether the ODA is still incentivizing product development for diseases with fewer than 200,000 affected individuals, as intended.”

In recent months, reports of five- and six-figure annual price tags for orphan drugs have amplified concerns about abuse of the law. The senators’ call for a GAO investigation reflects that sentiment, according to the KHN article.

“While few will argue against the importance of the development of these drugs, several recent press reports suggest that some pharmaceutical manufacturers might be taking advantage of the multiple designation allowance in the orphan drug approval process,” their letter said.

In January, KHN published an investigation that found the orphan drug program is being manipulated by drug makers to maximize profits and to protect niche markets for medications being used by millions of people.

That investigation found that many drugs with an orphan status aren’t entirely new. More than 70 were medications first approved by the FDA for mass-market use. They include the cholesterol blockbuster Crestor (rosuvastatin, AstraZeneca); Abilify (aripiprazole, Otsuka) for psychiatric disorders; and the rheumatoid arthritis drug Humira (adalimumab, AbbVie), the world’s best-selling medication. Others drugs have received multiple exclusivity periods for two or more rare conditions.

The senators asked the GAO for a list of drugs approved for or denied orphan status by the FDA. They also asked whether resources at the FDA, which oversees the ODA, have “kept up with the number of requests” from drug makers and whether there is consistency in the department’s reviews.

Drugs for rare diseases have become increasingly popular with pharmaceutical and biotech companies, according to KHN. They are expected to account for 21% of worldwide prescription sales by 2022, not including generics.

Source: Kaiser Health News; March 21, 2017.