Congressional Panel Says the United States Is Losing Its Lead on Rare Disease Cures

Rare disease information sits in scattered registries, individual academic centers and disconnected health systems. Both have already been introduced as legislation, including the National Biotechnology Initiative Act of 2025, and neither has been enacted. Many rare disease patient organizations maintain them, and a treating specialist can usually point to the right one. ClinicalTrials.gov lists trials by condition and can be checked periodically, since rare disease trials open and close with little publicity. It released a white paper on rare disease therapy development.