Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers (U01) Clinical Trial Optional

The purpose of this NOFO is to encourage new approaches to support therapeutic development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers.

Purpose and Research Objectives

The purpose of this FOA is to fund research to develop practical approaches to support drug development in ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers. The FOA focuses on supporting studies to improve therapeutic development in ultra-rare cancers with high unmet medical need where there is little economic incentive for commercial entities to conduct the research.

The primary focus of this FOA is to support research that incorporates new approaches that could be either be applied to facilitate the development of new drugs for the treatment of ultra-rare cancers in general, or one or more specific ultra-rare cancers, including but not necessarily limited to the examples below. 

Direct funding of interventional trials is outside the scope of this FOA. In addition, studies with a primary goal of developing a specific therapeutic product will not be supported.

1. • Platforms to screen and identify patients with ultra-rare cancers to facilitate enrollment in clinical trials
   • Studies to investigate the natural history of ultra-rare cancers. This work could involve analyses of registries and/or other real world data sources
   • Decentralized approaches for enhancing patient enrollment and facilitating patient assessments (e.g., collecting laboratory and/or imaging data from local facilities) in studies of ultra-rare cancers. OCE is particularly interested in studies focused on evaluating feasibility and implementation, including an analysis of the risks and benefits of different technologies, impact on clinical trial participation, and the quality of data collected.
   • Innovative approaches to identify new biologically-driven opportunities for clinical development of previously approved drugs or biologics (hereafter referred to as drugs), including drugs for which development has been discontinued, in ultra-rare cancers. This work could consider whether new or additional tumor assessment techniques may provide a more informative assessment of a drug’s effect on a tumor compared with traditional response criteria for a particular cancer.
   • 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