Commission Calls for National Strategy on Rare Disease Drug Development

Rare Daily Staff

A bipartisan legislative advisory commission is calling for a unified federal strategy to speed rare disease drug development, warning that the United States is losing ground in an area it describes as both a health and national security priority.

The National Security Commission on Emerging Biotechnology (NSCEB), in a new paper, says funding gaps, regulatory bottlenecks, data limitations, and manufacturing challenges are slowing progress on therapies that could shift rare disease care from symptom management toward cures.

The commission argues that the U.S. biotechnology ecosystem can still lead the world in rare disease innovation, but only if research, regulation, and commercialization are better coordinated. It frames the issue as urgent for more than 30 million Americans living with a rare disease and says current systems are too slow and costly to support therapies for small patient populations.

“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.”

Among the commission’s main recommendations are creation of a National Biotechnology Coordination Office in the Executive Office of the President and a National Biopharmaceutical Manufacturing Center of Excellence at NIST. The commission says those bodies would improve interagency coordination, streamline regulatory structures, and help regulators adapt to new manufacturing methods for advanced therapies.

The paper links those recommendations to two bipartisan bills already in Congress: the National Biotechnology Initiative Act of 2025 and the Biomanufacturing Excellence Act of 2025. According to the commission, the first would help establish a whole-of-government biotechnology strategy, while the second would create a center focused on manufacturing reliability, efficiency, and workforce training.

The rare disease paper builds on NSCEB’s April 2025 report, which laid out dozens of recommendations for strengthening U.S. biotechnology, with additional policy options aimed at modernizing federal biotech review. The commission says faster—but still safe—regulation would help bring more products to market and support U.S. innovation and competitiveness in the face of growing global competition.

The paper notes that rare disease drug development is uniquely difficult because patient populations are small, natural history data are limited, and traditional trial designs often do not fit these constraints. It also says developers face expensive manufacturing and scale-up requirements, especially for personalized therapies, while regulators may lack specialized expertise in emerging modalities such as gene and cell therapies.

The commission argues that other countries are moving faster in some areas and warns that without a more unified U.S. approach, American leadership in rare disease therapeutics could erode. That warning sits at the center of NSCEB’s broader effort to frame biotechnology policy as a national security issue as well as a health and economic one.