GAO Issues Report on Pediatric Voucher Program, Findings Inconclusive

March 3, 2016

By Alexander J. Varond

On March 2, 2016, GAO released its report, “RARE DISEASES – Too Early to Gauge Effectiveness of FDA’s Pediatric Voucher Program.” While the report nicely summarizes the history of the program, it contains little new information. Highlights from the report include:

  • FDA officials interviewed for the GAO report expressed “concern” about the program and do not support its continuation after the program expires on October 1, 2016;
  • 3 PRV requests are currently pending before FDA;
  • 52 rare pediatric disease designation requests have been submitted;
  • 29 rare pediatric disease designation requests have been granted; and
  • To date, no voucher has been awarded for a new drug application to prevent a rare pediatric disease.

The report provides an overview of the drugs that received pediatric vouchers and describes the extent to which they filled unmet need. It also identifies how the pediatric PRVs are being used. Beyond compiling these historical facts, GAO interviewed FDA officials, sponsors, patient advocacy groups, trade groups, and other interested organizations and individuals.

The report’s conclusion is simple: it’s too early to gauge if the pediatric voucher program stimulates drug development (although there are hints that it is).


GAO provides the following table summarizing the issued pediatric vouchers:


GAO also notes that, as of December 31, 2015, there have been 11 requests for pediatric vouchers. Of these, 6 were granted, 2 were denied, and 3 remain under review. The 2 denials were because the diseases “did not satisfy the definition of a rare pediatric disease.”

Too Early to Gauge if Program Stimulates Drug Development

GAO concludes in its report that “[g]iven that the typical drug development process often exceeds a decade, insufficient time has elapsed to gauge whether the 3-year-old pediatric voucher program has been effective at encouraging the development of drugs for rare pediatric diseases. [GAO] found that each of the drugs awarded pediatric vouchers were in development prior to the voucher program’s implementation. Any sponsors motivated by this relatively new program to attempt to develop drugs for such diseases would likely be years away from submitting their new drug applications to FDA.”

The report also mentions that “[a]lthough it is too early to gauge whether the program stimulates drug development, a potential indication of sponsor interest in the program may be reflected by the number of requests that have been submitted for a pediatric voucher or a rare pediatric disease designation.”

Of the 6 drugs that received pediatric vouchers, “[o]fficials from both NIH and FDA agree that these drugs are meaningful for patients with the rare pediatric diseases as the drugs may, for example, increase life expectancy, alleviate certain symptoms, or otherwise improve quality of life.”

FDA Officials Resistant to the Pediatric Voucher Program

What may come as a bit of surprise, the FDA officials interviewed by GAO were resistant to the pediatric voucher program. The report states:

FDA officials expressed concern about the pediatric voucher program, and do not support its continuation after its current authorization expires October 1, 2016. In written responses to our questions, FDA officials reported that they have seen no evidence that the program has encouraged increased development of drugs for rare pediatric diseases. The agency also indicated that while it strongly supports the goal of the program—incentivizing the development of drugs for rare pediatric diseases—it has not seen evidence that the program has yet been effective in achieving this goal. Instead, the agency suggested that companies may consider that other incentives, such as provision of an additional period of “market exclusivity,” may be more effective at incentivizing drug development than the priority review vouchers. FDA specifically cited its authority to provide an additional 6 months of market exclusivity for FDA requested pediatric studies in products that may produce health benefits in the pediatric population—known as pediatric exclusivity—as providing an effective incentive to drug sponsors.

FDA’s concerns include:

  • The pediatric voucher program interfering with the agency’s ability to determine its public health priorities (i.e., “the program allows sponsors to ‘purchase’ a priority review at the expense of other important public health work in FDA’s portfolio.”)
  • The pediatric voucher program is resource intensive and makes it difficult for FDA to manage its workload.

Other Groups Generally Support the Pediatric Voucher Program

In general, feedback from stakeholders was mostly positive. Sponsors indicated that voucher program incentivized drug development. One way it does this is by channeling money from pharmaceutical companies with more cash to companies focusing on developing drugs for rare pediatric diseases. A significant commentary from stakeholders was that the program should be reauthorized for even longer or permanently. This would provide the certainty needed for companies to invest significant long term resources to developing drugs for rare pediatric diseases.

In Other News, Gilead Successfully Redeems a Priority Review Voucher

On March 1, FDA approved Odefsey® (emtricitabine 200 mg/rilpivirine 25 mg/tenofovir alafenamide 25 mg or R/F/TAF) for the treatment of HIV-1 infection in certain patients. Gilead used the tropical disease voucher it acquired from Knight for $125 million to reduce the review time by 4 months. Thomson Reuters has estimated that peak sales of Odefsey may exceed $1.6 billion per year.