Bilirakis Racks Up Three More Legislative Wins in Health Mark-Up

Washington, DC: During today's Health Subcommittee markup, three Bilirakis-authored measures were passed. This legislation now heads to the full Energy and Commerce Committee for further consideration. Each bill aims to provide much needed support for sick children or those patients suffering with a rare disease.

The first bill, the Gabriella Miller Kids First Research Act 2.0, which is coauthored by Bilirakis would reauthorize the National Institutes of Health's (NIH) Gabriella Miller Kids First Pediatric Research Program (Kids First) and Data Resource Center for the next five years. This Pediatric Research Initiative has funded dozens of research projects around the country to advance our knowledge and improve outcomes for children with cancer and a range of other rare diseases. "Pediatric cancer remains the number one disease that leads to the death of American children. While survival rates have improved for some types of pediatric cancers, thousands of children are lost to cancer each year and many more encounter life threatening complications related to harsh chemotherapies. Children have significantly fewer treatment options than adults and oftentimes must rely on treatment regimens developed for adults because pediatric-specific treatments simply do not exist for many pediatric cancers and rare diseases. The Gabriella Miller Kids First Pediatric Research Program at the National Institutes of Health (NIH) is working to develop a more clear understanding of pediatric cancers, and holds the promise for the development of better treatments and possible cures. As a longtime advocate for children and rare disease patients, I am proud to join my colleagues in pushing for the continuation of this crucial research by providing new funding sources," said Congressman Gus Bilirakis.

The second bill to pass was co-authored by Representatives Bilirakis and Fletcher. It would 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 third bipartisan bill, authored by Representatives Bilirakis and Butterfield, to 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 in this legislative package.