Novel approaches to support therapeutic development in ultra-rare cancers

The Food and Drug Administration (FDA) is seeking novel approaches to support therapeutic development in ultra-rare cancers. The purpose of this program is to facilitate therapeutic development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. Specific areas of interest include the development of infrastructure for a coordination network and data repository, investigations to explore opportunities to develop and validate early clinical endpoints, and innovative approaches to identify new biologically-driven opportunities for clinical development of previously approved drugs or biologics. • Development of infrastructure for a coordination network and data repository for patient-level data across institutions and internationally to support drug development and regulatory decision-making for one or more ultra-rare cancers. • Investigations to explore opportunities to develop and validate early clinical endpoints and other novel efficacy endpoints for evaluation of treatments for ultra-rare cancers. • Development and implementation of a collaborative multi-stakeholder effort to support generation and use of real-world data leveraging a registry framework for use in development of new therapies for pediatric patients with diffuse midline glioma (DMG). • Innovative approaches to identify new biologically-driven opportunities for clinical development of previously approved drugs or biologics in ultra-rare cancers. • Research to develop novel approaches to preserve the availability of drugs for which commercial developers have discontinued adult development that have strong potential in ultra-rare cancers but lack financial incentives for commercial development.