GAO to Investigate Orphan Drugs
Acting on a request from Sens. Orrin Hatch (R-UT), Charles Grassley (R-IA), and Tom Cotton (R-AR), the Government Accountability Office (GAO) will look for possible abuses in FDA’s orphan drug program. Kaiser Health News reports that GAO will take the next few months to determine the scope of its probe and a methodology before starting work.
In their letter requesting the GAO involvement, the senators raised the possibility that regulatory or legislative changes will be needed “to preserve the intent of this vital law.” They said they want to know whether the program is still “incentivizing product development for diseases with fewer than 200,000 affected individuals as intended.”
Kaiser says that in recent months, concerns have risen about five- and six-figure price tags for orphan drugs and the senators’ letter reflects those concerns. “While few will argue against the importance of the development of these drugs,” they wrote, “several recent press reports suggest that some pharmaceutical manufacturers might be taking advantage of the multiple designation allowance in the orphan drug approval process.”
Earlier this year, Kaiser published an investigation, also aired by NPR, showing that the orphan drug program is being manipulated by drug makers to maximize profits and to protect niche markets for medicines that are taken by millions of patients. The news service found that many drugs that now have orphan status are not entirely new. More than 70 were drugs first approved by FDA for mass market use, it says, including AstraZeneca’s Crestor and AbbVie’s Humira. Others were drugs that received multiple exclusivity periods for two or more rare conditions.
The senators asked GAO for a list of drugs approved for or denied orphan status by FDA and asked if agency resources have kept up with the number of requests from companies and whether there is consistency in reviews. They also said it will be important for GAO to include patient experiences.
Kaiser says that rare disease drugs have become increasingly popular with pharmaceutical and biotech companies due to the high prices that can be charged, and are expected to comprise 21.4% of worldwide prescription sales by 2022, not including generics. The story references EvaluatePharma’s 2017 orphan drug report showing that of the top 100 drugs in the U.S., the average cost per patient per year for an orphan drug was $140,443 in 2016, compared with $27,756 for a non-orphan drug.