Congressional panel warns US rare-disease lead is eroding and calls for supportive policies

A Congressional advisory panel has urged the U.S. government to adopt policies that would help rare disease drug development, including a finalized FDA platform designation and the use of novel trial designs for small patient groups.

The panel notes that while the United States remains the global leader in creating treatments for rare diseases, its progress is slowing due to a costly and lengthy development system.

China is rapidly advancing in biotech, leveraging accelerated clinical trial pathways and active patient recruitment, which the report says could allow it to overtake the U.S. in delivering critical medicines.

The National Security Commission on Emerging Biotechnology frames the erosion of the U.S. lead as a national security concern, emphasizing the need for policy changes to maintain health outcomes and strategic advantage.