NSCEB Statement on New FDA Rare Disease Framework Initiative
Emerging biotechnologies will not just treat, but cure, rare disease, and a revised regulatory framework can make accessing treatments easier for the patients and families who need them most.
19 October 2026
FOR IMMEDIATE RELEASE
Washington, DC – The National Security Commission on Emerging Biotechnology (NSCEB) is encouraged by the new Rare Disease Framework Initiative launched by the Food and Drug Administration (FDA).
In July, the NSCEB published analysis identifying the hurdles that hinder innovation in rare disease such as funding challenges, regulatory roadblocks, data quality and availability, and the difficulty of manufacturing. The paper highlighted NSCEB recommendations that address these issues.
Currently, thirty million Americans live with a rare disease but only 5% of recognized rare diseases have an FDA-approved treatment. Emerging biotechnology offers promising treatments for patients with rare disease, but a smoother pathway to market is necessary to ensure patients can access them.
“We’re entering an era where biotechnology can do more than manage rare diseases,” said NSCEB Executive Director Caitlin Frazer. “Emerging biotechnology offers paths to curing them. But groundbreaking science means little if patients can’t access treatments. The United States has the talent and innovation to lead in rare disease therapeutics and we are encouraged to see the FDA taking steps to create a regulatory environment that keeps pace with discovery.”
Read the NSCEB’s Bringing Rare Disease Treatments to Patients issue paper here.
Read the Future of Regulations discussion papers here.
Read the NSCEB’s April 2025 Action Plan here.
FDA’s Rare Disease Framework Initiative
- Provide greater regulatory clarity, consistency, and predictability for rare disease stakeholders, including patients, drug developers, and researchers.
- This reflects NSCEB’s recommendation to create simple pathways to market.
- Leverage external expertise to support customization of regulatory review.
- This reflects NSCEB’s recommendation to establish a Foundation for Enabling Biotechnology Innovation, introduced as the Foundation for Enabling Biotechnology Innovation Act (S.2696), that would bring together experts from industry, academia, and government to share information about regulatory challenges and suggest ways of solving them.
- Build shared resources, robust training, and institutional knowledge on rare disease drug development and review.
- This reflects NSCEB’s policy option in the paper Bringing Rare Disease Treatments to Patients ondeveloping and publishing resources for rare disease advocates (p. 4), as well as NSCEB’s policy options in the paper Modernizing Medical Biotechnology Regulation to hire, train and retain regulators (policy options 11, 12, and 13, on p. 6).
- Applies risk-based approaches, particularly to chemistry, manufacturing, and controls review and pre-approval inspections.
- This reflects NSCEB’s policy option in the paper Modernizing Medical Biotechnology Regulation to clarify manufacturing requirements (policy option 20, on p. 7).
- Champion the use of cutting-edge science, novel methods, and innovative statistical approaches in rare disease drug development.
- This reflects NSCEB’s policy option in the paper Modernizing Medical Biotechnology Regulation to validate modern testing methods and allow trial designs for small populations (Policy option 5 and 7, on p.3,5).
Learn more about the FDA’s announcement here.
About NSCEB: The National Security Commission on Emerging Biotechnology is a time-limited, high-impact legislative branch advisory entity whose purpose is to advance and secure biotechnology, biomanufacturing, and associated technologies for U.S. national security and to prepare the United States for the bioindustrial revolution. The Commission published a comprehensive report in April 2025, including recommendations for action by Congress and the federal government. The bipartisan Commission is composed of Congressionally-appointed Commissioners with members from both the Senate and the House of Representatives as well as experts from industry, academia, and government. For more information about the Commission and to view the report, visit: biotech.senate.gov.
Contacts:
NSCEB: press@biotech.senate.gov
