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Congress Boosts “Hope” for Infants with Rare Diseases

Congress needs to act to continue to spur new treatments for sick children with few options.   

The Creating Hope Reauthorization Act would extend an existing program that incentivizes the development of treatments for rare pediatric diseases by offering a voucher for expedited FDA review. Companies can then buy and sell these vouchers to capture a return on investment that rare drugs seldom deliver. 

There have been 49 vouchers issued under the program, generated by approval of treatments for 40 diseases impacting children, including a rare muscular dystrophy mutation, cystic fibrosis and hyperinflation. For most of these conditions, no approved drugs were available before the incentive program existed. 

Research and Rare Disease in Children 

A complex cost-benefit equation applies to treatments for rare diseases.  

Researchers and investors should prioritize technologies that can improve the well-being of children in addressing their unique diseases and challenges. The small number of total patients affected by rare conditions, though, makes it difficult to recover research and development costs, even if the treatment is wildly successful.  

The difficulty of conducting clinical trials for children, the effects of delayed diagnoses and a 15-year average for drug approval further complicate matters.  

Fully 95% of rare diseases have no FDA-approved treatment. These medications serve such a small patient population that they are difficult to develop, test and make available. The challenges are magnified for disease treatments for children, often discouraging traditional investment. 

Patients and Providers Urge Congress to Reauthorize Vouchers 

Just days after the Creating Hope Reauthorization Act was introduced, more than 600 patients, caregivers, providers and advocates converged on the U.S. Capitol to tell their stories and make a plea for more public investment in policy solutions to health care challenges.  

The bipartisan bill is sponsored by U.S. Representatives Gus Bilirakis (R-Fla.), Anna Eshoo (D-Calif.), Michael McCaul (R-TX), Lori Trahan (D-Mass.), Michael Burgess (R-TX) and Nanette Barragán (D-Calif.). In remarks announcing their sponsorship, several representatives referred to personal acquaintances or constituents whose children live with rare diseases.  

The voucher program, formally known as the Rare Pediatric Disease Priority Review Voucher Program, must be renewed before its September 2024 expiration, or programs currently developing treatments for America’s most vulnerable may be shuttered or delayed.