Pediatric populations historically suffer from a dearth of medical therapies; few new medications are tested on and approved specifically for infants and young children. Now a new bill introduced in the U.S. Senate hopes to change all that. S. 2041, “The Promoting Life-saving New Therapies for Neonates Act of 2015,” offers pharmaceutical developers an intriguing trade-off: a one-year, transferrable extension on patent exclusivity in exchange for a new medical therapy that treats a critical need condition for pre-term or full-term infants
The bill, sponsored by Sen. Bill Cassidy, MD, (R-LA), and Sen. Robert Casey (D-PA), prioritizes medical therapies for conditions that qualify as “critical need.” As Dr. Cassidy explained in a recent press release, ““Many of us know a child who was born prematurely, or a family whose child was born sick and unfortunately there were not enough treatment options available to save that child. Hundreds of thousands of children born in the U.S every year require intensive care after birth, but in the past 16 years, only one drug has been approved for these children—that doesn’t add up.” Lawmakers plan to collaborate with the National Institutes of Health and patient advocates to identify which conditions are most critical for neonates.
The patent extension granted for producing a therapy to treat one of these conditions is described by the bill as a “Neonatal Drug Exclusivity Voucher.” The extension is transferrable, even subject to sale by the recipient, with certain limitations.
S. 2041 builds upon prior efforts to increase medical therapies for infants. In 2012, the FDA introduced Rare Pediatric Disease Priority Review Vouchers, which developers of medical therapies for infants and children can use to shorten the approval timeframe for a subsequent therapy. Regulators and lawmakers intend such efforts to incentivize more options for the often overlooked patient population.
