Life Sciences Horizons Brochure 2025 - Flipbook - Page 113
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2025 Horizons Life Sciences and Health Care
Scaling back: Changes to FDA’s pediatric drug development incentives
The Best Pharmaceuticals for Children Act (BPCA)
and the Pediatric Research Equity Act (PREA)
Rare Pediatric Disease Priority Review
Voucher Program
BPCA and PREA have played pivotal roles in pediatric drug
development. The BPCA, enacted in 2002, extends the period
during which competitor products cannot be approved due to
certain exclusivities and patents by six months for conducting
pediatric studies based on a written request (WR). PREA, enacted
in 2003, mandates pediatric studies for certain pharmaceuticals
and biologics. The two statutes are often referred to as the “carrot”
(BPCA) and “stick” (PREA). While the two programs have
historically overlapped in many ways, in 2023, FDA issued a draft
guidance proposing to limit the issuance of WRs only to sponsors
who conduct additional pediatric studies beyond what is required
under PREA, a change to FDA’s longtime practice. In other words,
sponsors would no longer qualify for pediatric exclusivity based
solely on PREA-required studies. Many commenters criticized the
proposal, and it remains to be seen how or if it will be finalized.
Nonetheless, we have already observed in recent years a trend
toward increasing difficulty for obtaining a WR.
Under the rare pediatric disease PRV program, companies that
receive approval for a rare pediatric disease drug may qualify for
a voucher granting priority review for a future drug application.
These vouchers may be used by the company, transferred, or sold,
and vouchers have sold on the market for over a hundred million
dollars. Currently, a drug must have received rare pediatric
disease designation by 20 December 2024 and must be approved
by 30 September 2026 to be eligible for a voucher. While the bill
extending the December 2024 sunset date ultimately did not pass,
FDA continues to review designation requests in the event the
program is reauthorized in future legislation.
The agency will be holding a public meeting on 15 May 2025 to
gather input from stakeholders on pediatric drug development
and labeling. The meeting will involve discussions on the public
health impact of BPCA and PREA, challenges in conducting
pediatric studies, and the impact of scientific advancements
on pediatric drug development. The agency has invited public
comments, and we encourage interested parties to share with
FDA their perspectives to help shape the pediatric drug
regulatory framework.
Komal Karnik Nigam
Partner
Washington, D.C.
Deborah Cho
Counsel
Washington, D.C.
Bryan Walsh
Senior Associate
Washington, D.C.
