A federal report says it's too early to tell whether the Food and Drug Administration's pediatric voucher program has accomplished its goal of spurring the development of drugs that treat or prevent rare pediatric diseases, and the FDA wants to get rid of it.
Because it can take manufacturers over a decade to develop a drug, the U.S. Government Accountability Office says it can't gauge whether the 3-year-old program has been effective as it rewards drugmakers for developing treatments for rare diseases.
FDA officials told the GAO that the voucher program shouldn't continue after it expires in October. They said the program strains agency resources and undermines public health priorities by requiring regulators to give priority to new drug applications that wouldn't otherwise qualify for expediting given that they don't treat a serious condition or provide significant improvement in safety or effectiveness.
The FDA grants a reward voucher when a company develops and gets approval for a therapy to treat a rare pediatric condition that affects more than 50% of children under the age of 13 and has fewer than 200,000 cases. The voucher allows the company to have one of its drugs reviewed by the FDA in a six-month time frame as opposed to the standard 10-month period.
As of Dec. 31, there have been 11 pediatric voucher requests, six of which have been awarded, two that have been denied and three that remain under review. All six drugs for which vouchers were granted were in development before the program began, but four of the six vouchers have been sold to other companies, including a voucher sold by United Therapeutics Corp. to AbbVie for a record $350 million.
Vouchers were given to BioMarin Pharmaceutical, United Therapeutics, Asklepion Pharmaceuticals, Wellstat Therapuetics and Alexion Pharmaceuticals—which developed two drugs—for therapies designed to treat pediatric cancer, metabolic disorders and other diseases. These drugs fulfill otherwise unmet medical needs, the FDA and National Institutes of Health said in the report.
The FDA suggested that providing manufacturers with an additional period of "market exclusivity"—like the six-month exclusivity the agency grants for FDA-requested pediatric studies in some pediatric products—may provide a more sustainable incentive.
But drug manufacturers told the GAO that they plan to use some of the proceeds from voucher sales to fund research on rare pediatric diseases, even though the program doesn't require them to do so, and patient advocacy groups say the program could lead to development of needed drugs.
The GAO found that about 52 rare pediatric disease designations were requested for drugs in development prior to the submission of a new drug application, 29 of which have been granted. These drugs could have been pursued in response to the voucher program, but they also could have been in development before the program was established, the GAO said.
The report did not discuss the issuance of vouchers to companies that develop and gain approval for drugs that treat certain tropical diseases, another component of the priority review voucher program. The program was the subject of a critical Viewpoint in JAMA in September, in which Dr. Aaron Kesselheim of Harvard Medical School alleged that there is little reliable evidence that the program has encouraged development, particularly in tropical disease treatment.
Pharmaceutical Research and Manufacturers of America, the drug industry's lobby, said it is still reviewing the report and declined to comment Thursday afternoon. Several patient advocacy groups were not immediately available for comment.