US HHS, FDA Support Regulatory Flexibility for Rare Disease Gene Therapies

Under President Donald Trump's administration, Health and Human Services Secretary Robert F. Kennedy Jr. and FDA Commissioner Marty Makary have committed to accelerating the development and approval of gene and cell therapies, particularly for rare diseases. They plan to maintain and enhance regulatory flexibility to facilitate faster access to innovative treatments, focusing on therapies that address the root causes of diseases. Kennedy emphasized reallocating funds from administrative costs to support innovation despite recent budget cuts to health agencies. Additionally, Kennedy has expressed support for expanding public access to experimental and alternative medical treatments, including stem cells, vitamins, peptides, and chelation therapy, while acknowledging potential risks and the presence of charlatans in the field. This approach marks a significant shift from previous FDA policies, raising concerns among experts about balancing patient safety with broader availability of unproven therapies. The administration's stance aims to position the U.S. as a hub of biotechnology innovation but has sparked debate over regulatory oversight and public health implications.