Dive Brief:
- The clock ticks: Congress has only a few weeks to consider legislation to extend the controversial pediatric priority review voucher (PRV) program, or it would expire before Oct. 1 after four years. The vouchers are given to companies to speed the review of pediatric rare disease drugs, but can be sold to other companies and used to decrease the review time for any drug.
- It comes as Sarepta contemplates selling the voucher it was awarded to get more money for commercialization of its recently approved Duchenne muscular dystrophy drug Exondys 51 (eteplirsen). Ending the program could possibly increase the demand for Sarepta's PRV.
- The Food and Drug Administration, which runs the program, opposes its continuation as being ineffective since many of the vouchers are sold and not used for pediatric rare disease drugs as intended. Meanwhile, advocates say it delivers on promises by hastening reviews for needed medicines in rare pediatric treatments.
Dive Insight:
Whether the program continues remains to be seen. Against the backdrop of possible political stagnation, millions of dollars are at stake for drugmakers as well as the speed of some medications going to market. While only less than a dozen drugmakers have held the vouchers, the pot of funds could grow for those who already have been awarded the vouchers if there are fewer companies to share it with, like a lottery winner, especially if the legislation dies. The price, the FDA has said in an overview of the program, "will depend on supply and demand."
Sarepta Therapeutics is one company that may be sitting on a potentially lucrative sale of the PRV after it won the award from the FDA last week, at the same time it received conditional approval for its long-sought DMD drug. It was the first company given the go-ahead by the FDA to treat the disease.
Sarepta Chief Financial Officer Sandy Mahatme said the company is considering selling the PRV to assist with financing of Exondys 51 and clinical development. Unloading the voucher would be no surprise because drug companies are seeing great returns by doing so. As of Dec. 31, 2015, there were 11 requests for the PRV, with six awarded, and four were sold to others at prices ranging from $67.5 million to $350 million, according to a General Accountability Office (GAO) report on the program.
In the meantime, there are the political issues that may eventually determine the fate of the program itself.
Previously, Congress extended the PRV until this fall, and if it does not review the program, then the FDA will stop awarding the vouchers. In a report, the FDA observed, "Members of Congress seem to like the voucher program (although also seeing scope for improvement), as evidenced by votes to extend the program to additional diseases, which suggests that Congress will probably renew the program."
Whether Congress supports the position of Casey and other PRV advocates is unknown thus far. It is possible the program could be included in other legislation, although no steps have been made to do that.