The Continuing Impact of the 'Right to Try' Legislation on Patient Access to Experimental Treatments

The 'Right to Try' legislation, a notable achievement promoted during President Trump's administration, allows terminally ill patients to access experimental treatments not yet approved by the FDA. Despite ongoing debates, this law continues to yield promising outcomes. For instance, Naval aviator Matt Bellina, diagnosed with Lou Gehrig's disease, experienced significant health improvements after receiving an investigational therapy under this law, extending his life beyond expectations. While the right to try offers hope, it's important to recognize that outcomes vary, and not all patients benefit. The law has become especially vital in the context of limited treatment options for rare diseases, such as certain genetic disorders. However, challenges remain, particularly in the realm of gene therapy. For example, the FDA's recent suspension of shipments for Elevidys, a gene therapy for Duchenne muscular dystrophy, has temporarily restricted access for patients, illustrating the ongoing regulatory hurdles. Additionally, there are concerns about how FDA leadership makes decisions on approving or denying experimental treatments. Reports suggest that some promising therapies might be...