The Pediatric Voucher Program and Its Impending Renewal DeadlineSeptember 22, 2016
By Alexander J. Varond –
FDA’s rare pediatric disease priority review voucher program is set to expire on October 1. If this happens, FDA will no longer award pediatric vouchers to otherwise eligible sponsors. Expiration of the pediatric voucher program would mean the end (at least temporarily) of Congress’s program to encourage investment and development of new drugs for rare pediatric diseases.
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.”
Recap of the Pediatric Voucher’s Evolution
Before diving into a discussion of the current legislation, a recap of the pediatric voucher program is in order.
Key Priority Review Voucher Milestones
September 27, 2007
FDAAA creates the tropical disease priority review voucher program
July 9, 2012
FDASIA creates the rare pediatric disease voucher program
February 14, 2014
FDA awards the first pediatric voucher to BioMarin
FDA awards two pediatric vouchers to United Therapeutics and Asklepion; the third voucher award triggers a 1-year sunset provision on the pediatric voucher program; GAO is tasked with issuing a report on the program within a year
August 19, 2015
A pediatric voucher is sold for $350M
December 18, 2015
Congress reauthorizes the pediatric voucher program until September 30, 2016
March 2, 2016
GAO issues its report on the pediatric voucher program
September 19, 2016
FDA awards a pediatric voucher to Sarepta
October 1, 2016
Pediatric voucher program set to expire
The sales price of pediatric vouchers has ranged between $67.5 million and $350 million.
The primary drivers for reauthorization have been the 21st Century Cures Act (H.R. 6) and Senate and House versions of the Advancing Hope Act (S. 1878 and H.R. 1537). We previously discussed versions of these proposals here and here.
21st Century Cures Act
The 21st Century Cures Act was passed in the House on July 10, 2015, but it has subsequently stalled and the Senate HELP Committee has proposed instead to issue less sweeping legislation. If passed, the 21st Century Cures Act would extend the pediatric voucher program such that marketing applications submitted by December 31, 2018, provided they meet the requirements in the statute, would be eligible for a pediatric voucher. The definition of a qualifying “rare pediatric disease” would also be amended 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.”
Advancing Hope Act (S. 1878)
Introduced on July 28, 2015, the Senate version of the Advancing Hope Act has had considerably more interest from stakeholders than the House version (discussed below). On April 5, 2016, the bill was amended and reported by Senator Alexander of the Committee on Health, Education, Labor, and Pensions. If passed, the pediatric voucher program would be extended until September 30, 2022, with the caveat that if a drug is designated as a drug for a rare pediatric disease by September 30, 2022, it remains eligible for a pediatric voucher so long as it is approved by September 30, 2027. The definition of “rare pediatric disease” would be amended 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 omits the controversial ex-US bar on the tropical disease voucher program contained in the House version of the bill.
Advancing Hope Act (H.R. 1537)
The House version of the Advancing Hope Act was introduced on March 23, 2015. It proposes to make the pediatric voucher program permanent and to specifically designate pediatric cancers and sickle cell anemia as rare pediatric diseases. It also modifies the tropical disease voucher program, making drugs approved outside of the U.S. for more than 24 months prior to submission of a marketing application to FDA ineligible for a tropical disease voucher.
Where Do We Go From Here?
As the week draws to an end, it appears increasingly unlikely that the 21st Century Cures Act or the Senate’s accompanying effort will be passed in time to reauthorize the pediatric voucher program. The best hope for authorization may be a last push for the 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. If efforts to reauthorize the pediatric voucher program before October 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.