Bilirakis Pediatric and Rare Disease Bills Pass the House

Washington, DC: Three more Bilirakis-authored measures passed the House last night as part of the Food and Drug Amendments of 2022. This comprehensive legislative package now heads to the Senate for final approval. Each provision aims to provide much needed support for children and patients suffering with a rare disease.

The pediatric provisions of the bill, co-authored by Representatives Bilirakis and Fletcher, will advance the development of pediatric medical devices by reauthorizing the Pediatric Device Consortia (PDC) grant program and extending the Humanitarian Device Exemption (HDE) incentive pathway. These programs provide additional opportunities to improve the number of medical devices in pediatric populations. "Manufacturing devices specifically made for pediatric patients have presented unique challenges and innovation has not kept pace with those developed for adults, leaving many children with rare diseases without devices that appropriately meet their individual needs," Bilirakis added. "Our bill will help address this shortage by continuing to incentivize the development of child-specific medical devices that account for variables like size and body chemistry. By reauthorizing these successful pediatric and humanitarian device programs, we are taking an important step forward in ensuring that our youngest Americans have access to high quality health care.

The rare disease provisions of the bill, authored by Representatives Bilirakis and Butterfield, will reauthorize the Food and Drug Administration's (FDA) Orphan Drug Grant Program for another five years also passed out of Subcommittee. This program funds studies that address knowledge gaps, support clinical trials and advance rare disease medical product development. The larger healthcare package also contained a provision from Bilirakis's Speeding Therapy Access Today (STAT) Act, which further expands this grant program to include new methods in developing regulatory science of individualized therapies for those with rare diseases and conditions. "With these important measures to reauthorize and expand the Orphan Drug Grant Program, we will facilitate the continued research and development of treatments and acceleration of cures for the more than 30 million people in the U.S. with rare diseases or conditions. This health package, with the addition of our bipartisan language, will help patients across the nation have better access to care,"Bilirakis noted. While Bilirakis expressed disappointment that the entirety of the STAT Act was not included, he lauds his colleagues for advancing key measures to accelerate development of therapies across the spectrum of rare diseases and disorders, and better facilitate patient access to such therapies, and he remains hopeful to continue to advance policies championed in the STAT Act through an alternative legislative package.