Press Release

Representatives Ami Bera, M.D. (D-CA) and Mike Kelly (R-PA) today introduced the Data for Pediatric Brain Cancer Act of 2023, bipartisan legislation to strengthen the collection and use of real-world data to support pediatric cancer research, and to improve treatments for children facing this devastating disease.

“As a physician, I am proud to introduce the Data for Pediatric Brain Cancer Act of 2023, a significant step forward in our mission to understand the complexities in clinical trial design in rare conditions,” said Representative Ami Bera, who previously served as Chief Medical Officer for Sacramento County. “By leveraging real-world data, we are equipping researchers with the tools they need to make progress in understanding and treating pediatric brain cancer. A tremendous step in the right direction for rare diseases, this will help enhance patient access and holds the potential to usher in groundbreaking treatments.”

“Randomized clinical trial designs are hard to use in rare disease patient populations due to the inability to find large study populations. This legislation seeks to use Atypical Teratoid Rhabdoid Tumors as a stepping stone to work towards using real-world data registries as external control cohorts for pediatric brain tumor clinical trials,” said Representative Mike Kelly. “The Pediatric Brain Tumor Real World Data Registry Program would strengthen and expand activities related to the collection, sharing, and use of real world data for children with brain tumors. It is our hope that this legislation will improve both patient treatment and patient outcomes.”

The Data for Pediatric Brain Cancer Act of 2023 is endorsed by the Children’s Cancer Research Fund, Pediatric Brain Tumor Foundation, National Brain Tumor Society, Hope4ATRT, and the Pediatric Neuro-Oncology Consortium Foundation.

Click here to view the text of the legislation.

“Atypical teratoid/rhabdoid tumors (ATRT) are exceptionally rare, making them a challenging subject for research. A federal program aimed collecting data for use in clinical trials could significantly enhance research efforts, potentially leading to breakthroughs in understanding and treating this aggressive cancer. Families affected by ATRT have shown unwavering commitment, dedicating their time and resources to support research and find a cure. The Pediatric Brain Tumor Foundation wholeheartedly endorses this bill. Alongside Reps. Bera and Kelly, we will work to amplify the momentum needed to combat ATRT and provide hope to patients and their families,” said Ryne Debo, VP of Research for the Pediatric Brain Tumor Foundation.

"Patients and families confronting the diagnosis of a highly aggressive pediatric brain tumor experience an unimaginable urgency for more effective treatment options,” said David Arons, President and Chief Executive Officer of the National Brain Tumor Society. “Lacking more effective standard treatments for many patients with these malignant brain tumors, clinical trials are often the best hope for survival. The Data for Pediatric Brain Cancer Act stands to help researchers develop the capacity to leverage data and generate evidence in service of establishing external controls for pediatric brain tumor trials. Ultimately the bill benefits patient access, clinical trial design and the need to rigorously evaluate potential new treatments prior to approval. The National Brain Tumor Society commends Reps. Bera and Kelly for their leadership on this critical issue."