Senate Leaders Push for Reauthorization of Program to Combat Rare Pediatric Diseases

A physician examining her patientPhoto by Los Muertos Crew on

WASHINGTON, D.C. — On Thursday, U.S. Senators Bob Casey (D-PA) and Markwayne Mullin (R-OK) introduced the Creating Hope Reauthorization Act, a bipartisan effort aimed at encouraging pharmaceutical companies to develop drugs for rare diseases affecting children, including various forms of cancer. The proposed legislation seeks to extend the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, which incentivizes drugmakers to bring novel treatments to market more quickly.

Addressing a Critical Need

Senator Casey, Chairman of the Senate Health, Education, Labor, and Pensions (HELP) Subcommittee on Children and Families, emphasized the importance of continuing this program. “Too many children suffer from rare diseases with few treatment options,” he said. “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.”

Senator Mullin, a member of the same subcommittee, added that the PRV program has been crucial over the past decade. “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.”

The Impact of the PRV Program

The PRV program has served as a lifeline for children with rare medical conditions since its inception in 2012. Senator Casey was instrumental in creating the program, which has since awarded 53 vouchers for 39 rare pediatric diseases, benefiting over 200,000 patients. Notably, 36 of these diseases had no previously approved therapies available at the time of approval.

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The Creating Hope Reauthorization Act aims to extend the PRV program through September 30, 2030. This longer extension period is intended to provide greater stability for innovators, encourage investment, and spur development in treating rare and neglected diseases that disproportionately affect children.

Broader Support and Implications

The bill has garnered support from both sides of the aisle, with U.S. Senators Sherrod Brown (D-OH) and Susan Collins (R-ME) cosponsoring the legislation. This bipartisan backing highlights the widespread recognition of the PRV program’s value.

Several prominent organizations endorse the bill, including the 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.

Creating Hope Reauthorization Act: Accelerating Treatments for Rare Pediatric Diseases

The Creating Hope Reauthorization Act has far-reaching implications for public health. Extending the PRV program will likely lead to faster development of treatments for rare pediatric diseases, offering hope to countless families. By incentivizing pharmaceutical companies to focus on these conditions, the legislation aims to fill a critical gap in the healthcare system where few options currently exist.

In conclusion, the bipartisan effort to reauthorize the PRV program underscores the urgent need for continued innovation in the treatment of rare pediatric diseases. By extending this vital program, lawmakers hope to ensure that children suffering from these conditions have a better chance at receiving life-saving treatments.

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