The United States remains a global leader in developing treatments for rare diseases, but other countries—particularly China—are rapidly closing the gap and could eventually surpass the U.S., according to a July 22 report by the National Security Commission on Emerging Biotechnology, a legislative advisory board convened by Congress.
“Developing novel rare disease treatments in the United States is critical to improving U.S. health outcomes, as well as ensuring U.S. national security,” the commission said in its report. The group warned that while the U.S. still leads in rare disease innovation, this advantage is diminishing due to what it described as a slow and expensive drug development system.
China has expanded its role as a biotech innovator through accelerated clinical trial pathways and active recruitment of participants, according to the report. The commission cited data showing that since 2020, China has hosted more clinical trials than the U.S., compared with less than 8% in 2010, based on an April report by Stanford University. The commission cautioned that without policy changes to keep pace with China’s progress, “China’s competitive edge in biopharmaceuticals may soon expand into an insurmountable lead and point of geopolitical leverage.”
Recent federal actions have included both restrictions and efforts at expediting drug development domestically. Last year, President Donald Trump approved the BIOSECURE Act targeting certain Chinese companies identified as potential national security risks; however, these policies focused more on limiting Chinese involvement rather than bolstering American innovation.
Meanwhile, initiatives at the Food and Drug Administration aim to speed up domestic drug approvals. Last month, the FDA announced a pilot program designed to improve clinical trial protocols and reduce time from drug identification to first-in-human studies. In February, draft guidance was released for personalized genetic medicines for very small patient populations—a framework allowing approval for drugs targeting specific biological causes of diseases.
The National Security Commission recommended further policy steps aligned with these FDA approaches: urging Congress to finalize platform technology designation programs—which allow validated data carryover across products—and supporting novel trial designs for small patient groups common among rare diseases. Stability at FDA leadership was also highlighted as essential; recent staff shortages and departures were noted as challenges affecting regulatory oversight.
