By signing a government funding bill that ended a partial shutdown, President Donald Trump has also reauthorized a beloved program meant to speed the development of new drugs for rare childhood diseases.
The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon Give Kids a Chance Act and reviving the rare pediatric disease (RPD) priority review voucher program. The program will now be funded through September 2029.
These vouchers slash the FDA’s review time of new drug applications and can also be sold by recipients for steep prices.
Established in 2012, 63 RPD vouchers have been awarded across 47 rare diseases, ranging from Duchenne muscular dystrophy to hemophilia A, according to the National Organization for Rare Disorders (PDF).
The program’s initial authorization expired on Dec. 20, 2024, and Congress has struggled to reauthorize it since then.
“The passage of the Mikaela Naylon Give Kids a Chance Act is a bipartisan victory for children and families living with rare diseases,” John Crowley, president and CEO of the Biotechnology Innovation Organization, said in a Feb. 3 statement.
The Rare Disease Company Coalition (RDCC), a group of biopharmas including Kyowa Kirin, BioMarin, BridgeBio, Alkermes, argenx, Sarepta Therapeutics and more, also expressed excitement at the act’s passage.
“This milestone brings renewed hope to families awaiting lifesaving treatments for children with rare diseases and reaffirms a bipartisan commitment to advancing innovation for pediatric patients who urgently need therapeutic breakthroughs,” Stacey Frisk, executive director of the RDCC, said in a Feb. 3 statement.
U.S. Representative Michael McCaul, R-Texas, introduced legislation to bring back the vouchers in February 2025, with the House finally okaying the measure by unanimous assent in December. But that victory was short-lived, with the bill ultimately stalled in the Senate.