- A new report from the Government Accountability Office revealed the FDA does not support the reauthorization of the priority review voucher program for pediatric rare diseases after the current authority lapses October 1, 2016.
- Since the program's inception, pharmaceutical companies have made 11 requests for a pediatric voucher, six of which were awarded while two were denied. Three requests remain under review.
- Although the GAO report said it was too early to assess the program's effectiveness, FDA officials are concerned the program requires the agency to award priority reviews to new drugs which wouldn't otherwise qualify.
Established by the FDA Safety and Innovation Act of 2012, the pediatric priority review program was designed to incentive the development of rare pediatric diseases. When a pharmaceutical company's new drug meets the criteria for priority review, the company can request a pediatric voucher in its new drug application. If the drug is approved, the FDA awards a priority review voucher, which can either be redeemed by the company on another one of its drugs, or sold.
These vouchers are highly coveted as they slice four months off of the standard 10-month regulatory review. While four months may not seem significant, it can help a drug company beat a competitor to market or boost a drug's initial revenue.
Since the program has only existed for three years, the GAO determined it was too soon to appropriately measure the program's effectiveness as the drug development process usually stretches longer than ten years. All of the six pediatric priority review vouchers awarded were granted for the approval of drugs already in development before 2012.
In responding to the GAO, the FDA indicated it has seen "no evidence that the program has encouraged increased development of drugs for rare pediatric diseases." Furthermore, officials felt the program may negatively impact public health priorities. They were concerned the priority reviews awarded would used on drugs treating prevalent conditions with existing treatment options rather than on unmet medical needs.
Indirectly, the report also shed light on how the six pediatric priority review vouchers have been used. Four of the six were sold for prices ranging from $67.5 million to $350 million. One voucher, awarded to BioMarin for Vimizim, was sold to Regeneron Pharmaceuticals and then redeemed for Praluent by Regeneron and Sanofi.
Sanofi also purchased one of the vouchers from Retrophin for $245 million, while AbbVie and AstraZeneca bought the other two. These three vouchers have not been redeemed as of yet.