Advancing Pediatric Cancer Research and Drug Development Through Multi-stakeholder Collaboration

This report explores the persistent challenges in developing treatments for childhood cancers and highlights how collaboration among nonprofits, academia, industry, and regulators can drive progress. Limited financial incentives and misaligned goals have long slowed research, but determined families and advocates have become catalysts for change.

A standout example is the development of tovorafenib, a therapy born from parent-led advocacy that achieved FDA approval in 2024. To build on this success, Day One Biopharmaceuticals convened a workshop that produced the Framework for Multistakeholder Collaboration in Pediatric Cancer Research and Drug Development, outlining key principles for effective partnerships. The lessons extend beyond pediatric oncology to other rare diseases facing similar barriers.