Mullin, Casey Introduce Bipartisan Bill to Spur Development of New Drugs to Treat Rare Childhood Diseases

Washington, D.C. – Today, U.S. Senators Markwayne Mullin (R-OK) and Bob Casey (D-PA), members of the Senate Health, Education, Labor, and Pensions (HELP) Subcommittee on Children and Families, introduced the Creating Hope Reauthorization Act to ensure that pharmaceutical companies continue to develop drugs to treat rare diseases affecting children, including types of cancer. The bipartisan legislation would extend the Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) program, which incentivizes drugmakers to create these novel treatments by bringing them on the market on an expedited timeline.

“Over the past decade, the Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program has successfully helped incentivize and expedite the development of new treatments and cures for young children with rare conditions including pediatric cancer and rare genetic disorders,” said Senator Mullin. “Our commonsense bill will reauthorize and extend the PRV to provide greater stability and fuel innovation to benefit a far greater number of pediatric patients living with rare diseases. One in ten Americans are living with a rare disease, but less than 10 percent of all rare diseases have an approved treatment option. It is important we do all we can to improve access to innovative health care options — especially for children. Thank you to Sen. Casey for joining me on this important legislation.”

“Too many children suffer from rare diseases with few treatment options,” said Senator Casey. “Our bipartisan bill will keep this critical voucher program going so drug companies don’t stop innovating new treatments to help sick kids. This is an investment in finding treatments and cures for rare diseases so that children get the care they need.”

The PRV program has been a lifeline for hundreds of thousands of children living with rare medical conditions. Since its creation in 2012, this program has awarded 53 vouchers for 39 rare pediatric diseases that have led to innovations benefitting over 200,000 patients. 36 of those rare diseases had no previously approved therapies on the market at the time of approval.

The Creating Hope Reauthorization Act would extend the PRV program through September 30, 2030, a longer period than previous reauthorizations. This will provide greater stability to innovators, encourage investment, and spur innovation in rare and neglected diseases that disproportionately impact children.

In addition to Casey and Mullin, the bill is cosponsored by U.S. Senators Sherrod Brown (D-OH) and Susan Collins (R-ME).

This legislation is endorsed by Children’s Hospital of Philadelphia, EveryLife Foundation for Rare Diseases, Haystack Project, Kids v Cancer, Life Sciences Pennsylvania, National Organization for Rare Disorders, Nationwide Children’s Hospital, Penn State Hershey Children’s Hospital, and the Rare Disease Company Coalition.

Read more about the Creating Hope Reauthorization Act here.