NEW YORK – The US House Energy & Commerce Committee on Wednesday voted unanimously to pass the Give Kids a Chance Act (H.R. 3433), which aims to encourage more drug development for children with rare diseases and contains language that will reauthorize the rare pediatric disease priority review voucher program until 2029.
The priority review voucher program offers drugmakers incentives that encourage them to develop treatments for rare pediatric disease, which are small commercial markets. If legislators don't vote to continue funding the program, it will expire on Sept. 30.
Recently, several developers of rare disease cell and gene therapies announced that they'd applied for rare pediatric disease designation for certain precision medicine products from the US Food and Drug Administration. However, these developers are uncertain if they will be eligible for priority review vouchers if the drugs receive regulatory approval pending reauthorization of the program.
For example, the Muscular Dystrophy Association recently launched the MDA Kickstart for Ultra-Rare Neuromuscular Disease program and announced plans to develop a gene therapy for congenital myasthenic syndrome caused by mutations in the CHAT gene, which affects approximately 200 children and adults in the US. The association said it had applied for orphan drug and rare pediatric disease designation for this investigational gene therapy, but it also urged the public to petition legislators to reauthorize the priority review voucher program.
"MDA's Kickstart program, and our efforts to develop a gene therapy for congenital myasthenic syndrome caused by mutations in the CHAT gene, are the exact kinds of programs that rely on the rare pediatric disease priority review voucher incentivizing for-profit biopharmaceutical companies to bring these therapies to market," Paul Melmeyer, executive VP of public policy and advocacy at the Muscular Dystrophy Association, said in a statement earlier this month. "This is why the rare pediatric disease PRV program must be extended beyond Sept. 30, and a long-term reauthorization must pass by the end of the year."
The bill's passage through the House E&C Committee improves its chances of being included in federal government funding legislation. The US Congress has yet to approve a new funding bill and is under pressure to come to a compromise by the end of the government's annual funding cycle at the end of the third quarter.
In addition to language reauthorizing the rare pediatric disease priority review voucher program, the Give Kids a Chance Act features other provisions focused on ensuring that rare diseases are studied in children, including the Innovation for Pediatric Drugs Act and the Rare Act. The former would strengthen the FDA's ability to enforce post-market requirements related to pediatric uses for approved therapies, and the latter would cement the agency's interpretation of how to award orphan drug exclusivity.
"In 2024, half of the estimated 30 million Americans living with rare diseases are children and many see their lives cut short by these devastating and progressive conditions," Heidi Ross, VP of policy and regulatory affairs at the National Organization for Rare Disorders, said in a statement. "We are pleased to see the House Energy and Commerce Committee take this important step toward addressing the urgent and often life-threatening needs in rare disease. … However, if Congress does not pass legislation to reauthorize the RPD PRV program, it will lapse on Sept. 30, 2024, which would be devastating to pediatric rare disease patient communities desperate for new and innovative therapies."