Novel Approaches to Support Therapeutic Development in Ultra-Rare Cancers supports the advancement of new tools, methods, or therapeutic approaches for ultra-rare pediatric and adult cancers, including molecularly-defined subsets of more common cancers.

Overview

The FDA Oncology Center of Excellence (OCE) aims to advance the development and regulation of oncology products for patients with cancer. The Pediatric Oncology Program and Rare Cancers Program were established to facilitate and expedite drug development for pediatric and other rare cancers. OCE’s Project Catalyst connects scientific knowledge, creative insight, and medical professionals to foster early-stage product innovation, which is particularly important to address the challenges related to product development for ultra-rare cancers. In collaboration with the National Institutes of Health (NIH) and the OCE, the Foundation for the National Institutes of Health (FNIH) recently launched the design phase of the Ultra-Rare Cancer Treatment Advancement Program (ULTRA), a new public-private partnership dedicated to accelerating the development of innovative ultra-rare cancer treatments.

For the purposes of this Notice of Funding Opportunity (NOFO), the FDA OCE refers to cancers with an approximate annual incidence in the U.S. of 300 to 400 people or less as ultra-rare.

Research Areas or Activities

Specific areas of interest include, but are not limited to, the following:

• 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) (including diffuse intrinsic pontine glioma, DIPG).
• 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.
• 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.
• Development of methods to incorporate use of telemedicine and/or pragmatic trial design elements (e.g., collecting laboratory and/or imaging data from local facilities) for patient assessments to facilitate enrollment of patients with ultra-rare cancers.
• Development of nanoparticle-based delivery approaches for therapeutic nucleic acids targeting onco-fusion transcription factors in meta-molecular level, providing new opportunities for targeted drug development.

Funding and Benefits

Estimated funding: 1,500,000

Award ceiling: 500,000

Award floor: none

Number of awards: 3

Eligibility

The programme supports development efforts related to ultra-rare pediatric and adult cancers.

Deadline and Timeline

Posted Date: 2026-05-04-00-00-00

Open Date (Earliest Submission Date): May 01, 2026

Letter of Intent Due Date(s): May 18, 2026

Application Due Date(s): June 15, 2026

All applications are due by 11:59 PM local time of applicant organization. No late applications will be accepted.

Expiration Date: July 31, 2026

Application Process

Applicants must adhere to all requirements detailed in the Research (R) Instructions How to Apply - Application Guide and in the NOFO. When program-specific instructions deviate from those in the How to Apply - Application Guide, the program-specific instructions must be followed. Applications that do not comply with these instructions may be delayed or not accepted for review.

The official full-announcement attachment is available as RFA-FD-26-004.pdf.