(R)evolution: toward a new paradigm of policy and patient advocacy for expanded access to experimental treatments

In life-threatening conditions such as cancer and rare diseases, where there is no cure and no U.S. Food and Drug Administration (FDA)-approved therapy, patients sometimes seek access to an unapproved, experimental therapy through expanded access programs as their last, best hope for treatment to save their lives. Since the 1980s, the policies and the practice of expanded access have evolved, but a common challenge remains that there is no obligation, and often little incentive, for manufacturers to offer expanded access programs, especially for individual patients. In recent years, online campaigns seeking access to an experimental therapy have become more common, paralleling growth in and representing an intersection of social media, digital health, and patient advocacy. Mackey and Schoenfeld have examined the evolution of expanded access policy, practice, and trends, as well as case studies of online campaigns to access experimental therapies, to arrive at several recommendations for the future of expanded access. This commentary puts their paper in context, examines their recommendations, and suggests further reforms. Please see related article: https://bmcmedicine.biomedcentral.com/articles/10.1186/s12916-016-0568-8