The Data for Pediatric Brain Cancer Act of 2023 directs the federal government to establish a registry program to collect and share real-world medical data on children with atypical teratoid rhabdoid tumors, a rare and severe type of pediatric brain cancer. The program will work with academic institutions, industry partners, and the FDA to develop databases that can serve as comparison groups for clinical trials, particularly single-arm trials where finding enough patients for traditional study designs is difficult or where standard treatments are ineffective. The legislation authorizes two million dollars in funding for fiscal year 2025 to support data collection, integration, analysis, and the identification of how this real-world data can improve drug development and testing for pediatric brain tumors. This approach addresses a significant challenge in rare disease research by using actual patient treatment information to establish benchmarks for evaluating new treatments instead of relying solely on traditional clinical trial methods that are often impractical for ultra-rare cancers.
Take Action
Your position
Add a comment
to comment on this bill.
Annotate the text
Highlight any passage on the Summary or Full Text tab to attach a note. Annotations appear on the Annotations tab.