Study Finds Priority Review Voucher Program Ineffective

Despite its lucrative promise, the FDA’s priority review voucher program has done little to spur development of novel treatments for tropical and rare pediatric diseases, a new study in JAMA concludes.

That’s due in part to the fact that companies can sell the vouchers for huge sums, rather than put them toward an accelerated review of another pediatric or tropical disease treatment — and many of them do.

Through August 2015, the FDA has issued six vouchers — three for neglected tropical diseases and three for rare pediatric diseases. Of those, four were sold, and one was used by the firm that received it. The sixth, a tropical disease voucher awarded to Johnson & Johnson in 2012, is being held by the company.

Novartis received the first tropical disease voucher in 2009 for its antimalarial drug Coartem (artemether-lumefantrine), and used it for a review of Ilaris (canakunimab) for gouty arthritis in 2011, but the indication was rejected. The third tropical disease voucher, given to Knight Therapeutics in 2014 for Miltex (miltefosine) to treat leishmaniasis, was sold to Gilead for $125 million.

All three of the rare pediatric disease vouchers were sold. BioMarin sold its voucher to Regeneron and Sanofi last year for $67.5 million, which then used it to spur a review and subsequent approval of Praluent (alirocumab) for high cholesterol. Asklepion’s, received for its cholic acid drug Cholbam, went to Sanofi for $245 million, while United Therapeutics’ voucher for Unituxin (dinutuximab) went to Abbvie last month for $350 million. Both of those vouchers have yet to be used.

The researchers also argue that the RVS program doesn’t require drugmakers to be heavily invested in a product to earn the reward. They note, for example, that Unituxin was developed, tested and manufactured by the National Cancer Institute through the Biopharmaceutical Development Program, but a voucher was awarded to United Therapeutics.

To ensure the PRV program achieves its true goal, firms should have to show some degree of involvement in the drug development process before receiving the windfall that comes with a voucher, the researchers say.

Congress authorized the tropical disease RVP in 2007, adding the incentive for rare childhood diseases in 2012. The fee for cashing in a voucher goes up $165,000 to $2.727 million, starting Oct. 1. — Kellen Owings