Pediatric Voucher Program Expires October 1
Attorney Alexander Varond (Hyman, Phelps & McNamara) says that efforts are underway to reauthorize the FDA rare pediatric disease priority review voucher program that is set to expire 10/1. But it is not clear that those efforts will succeed in time, he writes in his firm’s FDA Law Blog. “Expiration of the pediatric voucher program would mean the end (at least temporarily) of Congress’ programs to encourage investment and development of new drugs for rare pediatric diseases,” Varond says. “A number of pieces of legislation have been proffered to stave off the program’s imminent sunset. Notably, each piece of legislation has proposed changes to the definition of ‘rare pediatric disease,’ which is currently defined, in part, as a ‘disease primarily affect[ing] individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.’”
The primary vehicles that could lead to reauthorization, the article says, are the 21st Century Cures Act (H.R. 6) and Senate and House versions of the Advancing Hope Act (S. 1878 and H.R. 1537).
The 21st Century Cures Act passed the House 7/10/15, Varond says, and has been stalled in the Senate HELP Committee. If passed, it would extend the pediatric voucher program such that marketing applications submitted by 12/31/18, provided that they meet the law’s requirements, would be eligible for a voucher. The bill amends the definition to “a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents.”
The Senate version of the Advancing Hope Act would extend the voucher program to 9/30/22, with the caveat that if a drug is designated as a drug for a rare pediatric disease by that date, it would remain eligible for a pediatric voucher as long as it is approved by 9/30/27. The definition would be changed by using the wording from the 21st Century Cures Act.
The House version of the Advancing Hope Act would make the voucher program permanent and would specifically designate pediatric cancers and sickle cell anemia as rare pediatric diseases.
Varond says that as 10/1 draws close, it appears increasingly unlikely that the 21st Century Cures Act or the Senate’s version of it will be passed in time to reauthorize the program. “The best hope for reauthorization may be a last push for passage of the standalone Senate version of the Advancing Hope Act via the ‘hotline’ process or by attaching reauthorization to the continuing resolution to fund the government,” he says. “If efforts to reauthorize the pediatric voucher program before 10/1 fail, the program could be reinstated as part of the 2017 user fee reauthorization package or other legislation. Whether this would retroactively extend the program to eliminate a gap between the sunset date and subsequent reauthorization remains to be seen.”