A Congressional advisory panel recommends the U.S. government put supportive policies in place for rare disease drug development, such as a finalized platform designation from the FDA and the allowance of novel trial designs for small patient populations.
While the U.S. continues to be the world leader in developing treatments for rare diseases, other countries—China especially—are catching up and could ultimately cause Americans to miss out on crucial medicines, a group of Congressional advisors argues.
“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,” according to a Tuesday report published by the National Security Commission on Emerging Biotechnology (NSCEB), a legislative advisory board convened by Congress to assess how advances in biotech could direct national defense initiatives.
While the U.S. still currently comes ahead of other countries in rare disease innovation, the NSCEB warned that the country’s “lead is eroding” due to its slow and expensive system of drug development. China, meanwhile, has rapidly grown as a key biotech innovator on the global stage, fueled largely by its “accelerated clinical trial pathways and active recruitment” of participants, the group wrote in the report.
Indeed, the Asian nation has hosted more clinical trials than the U.S. since 2020, up from less than 8% in 2010, according to an April report by Stanford University. If the U.S. fails to keep up with China’s progress, largely through policy changes, then “China’s competitive edge in biopharmaceuticals may soon expand into an insurmountable lead and point of geopolitical leverage,” the NSCEB cautioned.
The U.S. federal government has put in place some policies designed to tamp down on the industry’s Chinese dealmaking rather than bolster drug development. Last year, for instance, president Donald Trump greenlit the BIOSECURE Act, which targets certain Chinese “companies of concern,” flagged as those who could pose national security harms to the U.S., though the final text of the law didn’t name these companies.
But initiatives are also underway to expedite drug development in the U.S. Last month, the FDA announced a pilot program to improve clinical trial protocols, with an eye toward minimizing the time from drug identification to a first-in-human study, as well as improving the overall quality of new drug submissions.
The regulator has also recently unveiled rare disease–specific policies. In February, for instance, the agency released draft guidance for its plausible mechanism pathway for personalized genetic medicines for diseases affecting very few patients. This framework opens up the possibility of approval for drugs that can demonstrably target a condition’s underlying biological cause.
Although the report doesn’t mention these specific programs, the NSCEB does offer policy recommendations that are in line with the FDA’s approach, meant to remove barriers for the industry instead of restricting pipeline development.
The commission suggests, for example, that Congress urge the FDA to finalize its platform technology designation program, which allows for the carryover of validated data across programs based on a specific platform. The FDA launched this initiative in 2024 and named Sarepta as the first awardee in June 2025. The biotech’s award was revoked a month later, however, after three deaths were linked to Sarepta’s gene therapy platform. Krystal Biotech won the second platform designation last October.
Congress should also find ways to allow novel trial designs for small patient populations, the NSCEB suggests—otherwise a key stumbling block of many rare disease programs.
To this end, the FDA in September 2025 unveiled the Rare Disease Evidence Principles. Under this effort, the agency will work with drug sponsors, patients and experts to determine what evidence will be taken into consideration for approval. Only drugs that affect fewer than 1,000 patients in the U.S. are eligible for this program.
Likewise key to the rare disease sector is FDA stability, according to Tuesday’s report, which flagged the “persistent staffing shortages and knowledge gaps” at the regulator that render it unable to properly assess emerging technologies and therapies.
Notably, thousands of employees were laid off or voluntarily left the FDA last year amid Health Secretary Robert F. Kennedy Jr.’s efforts to overhaul the agency. Nearly 2,000 more staff were expected to get the boot this year, according to a fact-sheet of the regulator’s 2026 budget. The topmost ranks at the FDA have also undergone considerable churn in recent months, marked by the recent departure of Commissioner Marty Makary, as well as his controversial deputies Vinay Prasad and Tracy Beth Høeg. There is some light on this horizon, however, as the FDA is looking to hire some looking to hire some 2,200 people, Acting Chief of Staff and Deputy Commissioner Lowell Zeta said at BIO’s International Convention last month.
Under the previous FDA leadership, the rare disease space suffered from contentious decisions and inconsistencies. In February, for instance, Disc Medicine failed to secure an approval for bitopertin, being proposed for a rare blood disorder, amid reports of skepticism from Prasad.
In November 2025, the FDA said Phase 1/2 data were no longer enough to support a filing for its uniQure’s Huntington’s disease gene therapy—despite having previously signed off on the biotech’s protocols. The agency has since reversed that reversal, paving the way for uniQure to file for accelerated approval, which the company plans to do in the third quarter.
