Accelerating Rare Disease Innovation through an All-of-Government Strategy
The U.S. biotechnology ecosystem could lead the world in developing cures for rare disease, but without a unified national strategy to fund, develop, and regulate new therapies, that lead is eroding. Retaining America’s edge in rare disease therapeutics is a national security and health security imperative.
21 JULY 2026
Washington, D.C. – Today, the National Security Commission on Emerging Biotechnology (NSCEB) published new findings on treating and curing rare diseases in the United States’ biotechnology and regulatory ecosystem. The Commission identifies the hurdles that hinder rare disease innovation such as funding challenges, regulatory roadblocks, data quality and availability, and the difficulty of manufacturing, and highlights NSCEB recommendations that address these issues.
“Emerging biotechnology now enables us to cure rare diseases, not just manage their symptoms for life,” said NSCEB Vice Chair Michelle Rozo. “What we lack is a national strategy to channel the strength of our innovation. A comprehensive approach that aligns research, investment, regulation, and commercialization will ensure great American technology becomes great American capability.”
In its April 2025 Action Plan, the NSCEB found that an all-of-government approach to biotechnology will help address these challenges across applications of biotechnology, including rare disease treatments. The NSCEB recommends the creation of a National Biotechnology Coordination Office (NBCO) that would promote federal coordination and streamline the regulatory structures across the U.S. government currently inhibiting innovation. The NSCEB also recommends establishing a biopharmaceutical manufacturing center of excellence, which would develop advanced methods of manufacturing for innovative biopharmaceutical products. The center of excellence would also engage regulators to ensure alignment with regulatory requirements for new manufacturing methods.
Read the paper here: Bringing Rare Disease Treatments to Patients
Both the NBCO and biopharmaceutical manufacturing center of excellence recommendations are reflected in current bipartisan and bicameral legislation:
The National Biotechnology Initiative Act of 2025 (S.1387/H.R.2756)
This bill would direct the President to establish the National Biotechnology Initiative, a whole-of government approach to advancing U.S. national security, economic productivity, and competitiveness. This includes establishing a National Biotechnology Coordination Office in the Executive Office of the President to manage the coordinated strategy.
The Biomanufacturing Excellence Act of 2025 (S.3188/H.R.6089)
This bill would direct the National Institute of Standards and Technology (NIST) to establish a National Biopharmaceutical Manufacturing Center of Excellence (COE). This center would advance biopharmaceutical manufacturing methods to improve reliability and efficiency, engage regulators to ensure alignment with regulatory requirements, and provide workforce training opportunities.
Earlier this year, the Commission built upon its initial recommendations with 83 additional policy options to modernize and accelerate U.S. regulatory review of biotechnology products across the federal government. Streamlining regulation allows for more products to get to market faster while continuing to ensure safety, which will in turn unleash U.S. biotechnology innovation, enable economic growth, and support national security.
Read the Future of Biotechnology Regulation discussion papers 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
