Navigating Compassionate Use: From Patients to Policies and Beyond
Two publications from the New York Academy of Sciences examine pre-approval access to investigational drugs from a range of stakeholders and perspectives.
The Food and Drug Administration (FDA) has granted the use of investigational drugs outside of clinical trials for decades, but in the past several years this practice has attracted significant attention in the news and on social media. Under expanded access (also called compassionate use), patients who suffer from serious or immediately life-threatening diseases for whom no comparable or satisfactory alternative therapy is available can access drugs and medical devices that are not approved by the FDA and are currently being tested in clinical trials. A total of 29 states have also passed "right-to-try" laws allowing terminally ill patients to access experimental therapies, but there are many questions about the safety and efficacy of such treatments that are not FDA-approved.
Recently the FDA announced significant changes to shorten and simplify the application process used by physicians to request expanded access to investigational drugs for individual patients. Some are predicting that the FDA may not approve a drug to treat Duchenne muscular dystrophy that is currently under review, but will allow compassionate use while additional studies are conducted. Regardless of the FDA decision, the debate over compassionate use will continue and evolve as additional treatments are brought to the forefront of experimental medicine and research.