OKC Standard

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 today. The legislation aims to ensure that pharmaceutical companies continue developing drugs to treat rare diseases affecting children, including various cancers.

The bipartisan bill seeks to extend the Food and Drug Administration’s (FDA) Rare Pediatric Disease Priority Review Voucher (PRV) program. This program incentivizes drugmakers by expediting the market introduction of new treatments for rare pediatric conditions.

“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.”

Since its inception in 2012, the PRV program has been pivotal for hundreds of thousands of children living with rare medical conditions, awarding 53 vouchers for 39 different pediatric diseases leading to innovations benefitting over 200,000 patients. Of these diseases, 36 had no previously approved therapies at their time of approval.

The Creating Hope Reauthorization Act proposes extending the PRV program through September 30, 2030—longer than previous reauthorizations—to provide greater stability for innovators and encourage investment in neglected diseases that disproportionately affect children.

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

The legislation has garnered endorsements from several organizations including 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.

More information about the Creating Hope Reauthorization Act can be found here.

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