FDA's rare disease pediatric voucher program could become permanent
- The Rare Pediatric Disease Priority Review Voucher Program, which was enacted in 2012 to encourage drug development, is due to expire in March 2016—but it may be made permanent if a new bill in Congress is approved, RAPS reports.
- The bill was intended to encourage development of drugs for pediatric diseases, which affect 200,000 children or fewer in the U.S. To date, the FDA has issued six priority review vouchers, three of which have been rare pediatric disease vouchers.
- Because of a sunset clause, the program is due to expire on March 17, 2016. However, the Advancing Hope Act of 2015 would make the program permanent if it is passed by Congress.
Between 2009 and 2015, a total of six priority review vouchers aimed at catalyzing drug development for rare diseases were given out. The companies receiving vouchers included Novartis, Janssen, BioMarin, Knight Therapeutics, United Therapeutics, and Asklepion Pharmaceuticals (BioMarin, United, and Asklepion are the three companies that received the pediatric vouchers, while the rest were for tropical diseases).
The benefit of receiving such a voucher is that the standard 10-month review process is expedited to six months. In fact, these vouchers are so valuable that two companies have sold them—one for $67.5 million and the other for $125 million.
Making this program permanent would definitely be a boon to all companies focused on addressing rare pediatric diseases.
- Regulatory Affairs Professional Society Bill would mae permanent FDA's rare pediatric voucher program