As a result of  these challenges and others, drug sponsors may be hesitant to attempt to  develop drugs to treat rare pediatric diseases. FDA and the Pediatric Voucher Program

FDA, an agency within the Department of Health and Human Services  (HHS), is responsible for overseeing the safety and efficacy of drugs sold  in the United States. In making this decision, FDA determines  whether the drug sponsor has met all of the eligibility criteria for a  pediatric voucher, which includes determining that the drug is for a rare  pediatric disease as well as reviewing the clinical data examining the  drug’s use in a pediatric population included in the drug application. Once a drug sponsor is awarded a voucher, it can later be redeemed by  that sponsor with the submission of another new drug application for a  drug to treat any disease or condition in adults or children, making the  sponsor automatically eligible for a 6-month priority review. If a rare pediatric disease designation is not  requested prior to a drug sponsor submitting its new drug application,  FDA officials may determine through their reviews of a new drug  application and discussions with a drug sponsor that a certain drug may  be eligible for a voucher. Too Early to Gauge if Pediatric Voucher Program Stimulates Drug Development

		Drugs for Which Vouchers Were Awarded Were in Development Prior to the Program’s Implementation, though They Helped Fulfill Unmet Medical Need

Given 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. As of December 31, 2015,  there have been 11 requests for a pediatric voucher. Of these, 6 have  been awarded, 2 denied, and 3 are still under review. Specifically, these six drugs were the first drugs  approved by FDA to treat the seven rare pediatric diseases for which they  are indicated. No other drugs had been previously approved for these  diseases. As of December 31, 2015, four of the six pediatric vouchers—for Vimizim,  Unituxin, Cholbam, and Xuriden—have been sold or transferred to other  drug sponsors. Sale prices of the pediatric vouchers have ranged from  $67.5 million to $350 million. It was used to  expedite the review of Praluent, a new drug to treat adults with high  cholesterol. 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. According to FDA,  the program interferes with its ability to set priorities on the basis of public  health needs by requiring FDA to provide priority reviews of new drug  applications that would not otherwise qualify, based on the merits of those  applications. The agency noted that an application for a drug will receive  priority review designation if it is for a drug that treats a serious condition  and, if approved, would provide a significant improvement in safety or  effectiveness. Four  of five sponsors that were awarded or transferred and later sold vouchers  told us that they plan to reinvest a portion of the proceeds they received  into additional research and development of drugs to treat other rare  pediatric diseases. Although sponsors and patient advocacy groups were generally positive  about the voucher program, some also expressed concerns related to the  uncertain future of the program and FDA’s interpretation of what diseases  are considered rare pediatric diseases, concerns also expressed by  organizations representing physicians and the health insurance industry. For example, some of the sponsors, patient advocacy groups, and other  organizations that we contacted said that the FDASIA provision providing  for termination of FDA’s authority to award pediatric vouchers one year  after the award of the third voucher under the program (March 2016)  created ambiguity for industry that therefore diminishes the program’s  appeal. He indicated, similar to FDA’s concern, that  the program could instead lead to unintended consequences. Agency Comments

We provided a draft of this report for comment to HHS. HHS provided  technical comments, which we incorporated as appropriate. Children's Hospital Association 3.
Why GAO Did This Study

Almost 7,000 rare diseases, most of which are serious or life-threatening, affect more than 25 million Americans. About half of all rare diseases affect children, and few of these diseases have viable treatments. To encourage the development of drugs to treat or prevent rare pediatric diseases, the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012 authorized FDA to award a priority review voucher to a drug sponsor upon approval of that sponsor's drug to treat a rare pediatric disease. A drug sponsor can later redeem the voucher when submitting another new drug application to treat any disease or condition in adults or children, or sell or transfer the voucher to another sponsor. A voucher entitles a sponsor to a 6-month priority review by FDA rather than the 10-month standard review.
FDASIA included a provision for GAO to study the pediatric voucher program. GAO examined what is known about the effectiveness of the program in encouraging the development of drugs to prevent or treat certain rare pediatric diseases. GAO reviewed relevant laws and documentation related to the program and its management, and identified drug sponsors who were awarded vouchers, the diseases their drugs were approved to treat, and whether the vouchers were redeemed, sold, or transferred. GAO also interviewed FDA officials, drug sponsors, patient advocacy groups, and organizations representing physicians and children's hospitals, among others.

What GAO Found

It is too early to gauge whether the Food and Drug Administration's (FDA) pediatric voucher program has stimulated the development of drugs to treat or prevent rare pediatric diseases. Given that the typical drug development process often exceeds a decade, insufficient time has elapsed to determine whether the 3 year-old program has been effective. Any drug sponsors motivated by the program to attempt to develop a drug for a rare pediatric disease may be many years from submitting new drug applications—which contain scientific and clinical data about safety and effectiveness—to FDA for review.
As of December 31, 2015, there have been 11 requests for a pediatric voucher. Of these, six have been awarded, two denied, and three remain under review. The six drugs for which vouchers were awarded were in development prior to the program's implementation and these drugs helped fulfill unmet medical needs. One drug is indicated to treat a rare pediatric cancer, and the other five drugs treat rare metabolic diseases affecting children. No other drugs had been previously approved by FDA for these conditions. Four of the six awarded pediatric vouchers have been sold to other drug sponsors for prices ranging from $67.5 million to $350 million. One of the six vouchers awarded has been redeemed and was used to obtain a priority review of a new drug application for a drug to treat adults with high cholesterol. FDA approved this new drug application in July 2015.
FDA officials stated that, while they strongly support the goal of incentivizing drug development for rare pediatric diseases, they have seen no evidence that the program is effective. The program's authorization, as amended, is set to terminate October 1, 2016, and FDA officials said they do not support the program's continuation. They expressed concern that the program adversely affects the agency's ability to set its public health priorities by requiring FDA to provide priority reviews of new drug applications that would not otherwise qualify if they do not treat a serious condition or provide a significant improvement in safety or effectiveness. Additionally, FDA officials said that the additional workload from the program strains the agency's resources. However, other stakeholders provided generally positive feedback on the program. For example, drug sponsors that sold these vouchers said they plan to reinvest portions of the proceeds they received into additional research on rare pediatric diseases, although there is no requirement to do so. Patient advocacy groups told GAO that the program could lead to the development of needed drugs.
We provided a draft of this report for comment to the Department of Health and Human Services (HHS). HHS provided technical comments, which we incorporated as appropriate.