The PRV program offers vouchers that companies can win for gaining approval of new rare pediatric treatments affecting fewer than 200,000 people, more than half of whom have to be under the age of 18. The vouchers can be used to speed FDA’s review of a new drug application by four months or sold to other companies. So far, the vouchers have sold for as much as $350 million.
FDA Pushback
FDA officials, however, are not pleased with the program so far. Unidentified officials at the agency told GAO 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.”
FDA even told GAO it does not support the PRV program’s continuation after its current authorization expires 1 October 2016. “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,” GAO said. “Additionally, FDA officials said that the additional workload from the program strains the agency’s resources.”
Review staff shortages is another concern raised by FDA as the agency says it cannot move reviewers from one division to another on an ad hoc basis to complete priority reviews for the application based on the vouchers. “According to FDA, it cannot predict which review divisions will need additional staff to complete the additional priority reviews, making anticipatory hiring infeasible,” GAO explained. “Although FDA receives a special user fee from a drug sponsor when the sponsor redeems a voucher, in addition to the regular user fee that accompanies a new drug application, the Agency noted that FDASIA did not authorize resources beyond the user fees—funding or staff—to administer the program, including determining rare pediatric disease designations.” Others were more positive about the program. Drug sponsors that sold the vouchers said they plan to reinvest portions of the proceeds they received into additional research on rare pediatric diseases while patient advocacy groups told GAO that the program could lead to the development of needed drugs. The GAO report did not include any information on FDA’s other PRV program for rare tropical diseases.
Statistics
Currently, there have been 11 requests for a pediatric PRV, and of these, six have been awarded, two denied and three remain under review, GAO says. The six drugs for which vouchers were awarded were in development prior to the program’s implementation and are indicated to treat a rare pediatric cancer and other rare metabolic diseases affecting children for which no other treatments exist.
Four (4) of the six (6) awarded pediatric vouchers have been sold to other drug sponsors for prices ranging from $67.5 million to $350 million. One (1) of the six (6) vouchers awarded has been redeemed and was used by Sanofi to obtain a priority review of a new drug application for a drug to treat adults with high cholesterol, which was approved in July 2015.
Sanofi has since used another PRV to speed up the review of a drug to treat Type 2 diabetes.
REFERENCE: RAPS; 02 MAR 2016; Zachary Brennan