Collins, Bipartisan Group Introduce Bill to Spur Development of New Drugs to Treat Rare Childhood Diseases

Washington, D.C. - U.S. Senator Susan Collins joined Senators Bob Casey (D-PA), Markwayne Mullin (R-OK), and Sherrod Brown (D-OH) in introducing the Creating Hope Reauthorization Act to encourage the development of drugs to treat rare diseases affecting children. The bipartisan legislation would extend the Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) program, which incentivizes drug makers to create these novel treatments by bringing them on the market on an expedited timeline.  Senator Collins is a member of the Senate Health, Education, Labor, and Pensions (HELP) Committee.

“The FDA’s Priority Review Voucher program supports the critical yet challenging work of developing new treatments for rare pediatric diseases,” said Senator Collins. “By extending this program, we can continue to provide hope to children and families affected by these devastating conditions and encourage the pharmaceutical industry to invest in innovative solutions.”

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 more than 200,000 patients. Thirty-six 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 would provide greater stability to innovators, encourage investment, and spur innovation in rare and neglected diseases that disproportionately impact children.

This legislation is endorsed by EveryLife Foundation for Rare Diseases, Haystack Project, Kids v Cancer, National Organization for Rare Disorders, Nationwide Children’s Hospital, and the Rare Disease Company Coalition.

Read more about the Creating Hope Reauthorization Act here.

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