Description
The purpose of this Notice of Funding Opportunity (NOFO) is to support new approaches that can be applied to facilitate therapeutic development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. The FDA Oncology Center of Excellence (OCE) aims to advance the development and regulation of oncology products for patients with cancer. The key areas of interest include development of infrastructure for a coordination network and data repository, investigations to explore opportunities to develop and validate early clinical endpoints, and development of methods to incorporate use of telemedicine and/or pragmatic trial design elements.
Some of the key points to consider are:
• 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.