ABSTRACT: Patients with life-threatening disorders such as amyotrophic lateral sclerosis, for which only minimally effective medical therapies currently exist, often seek treatments not proven to be effective and not approved by regulatory agencies for use outside of experimental treatment trials. The expanded access (compassionate use) provisions of the US Food and Drug Administration (FDA) for access to such therapies are often perceived as being inadequate. In response, states have passed right-to-try laws designed to improve access to experimental therapies for patients willing to assume the risks associated with such treatments. This situation has resulted in conflicts between those who perceive access to such treatments as their right as autonomous individuals and those who believe that the principles of beneficence and nonmaleficence justify actions of physicians and regulators in controlling access to such treatments. A variety of factors also contribute to the inequitable distribution of such treatments. Better systems are needed to improve access to promising new treatments while protecting these vulnerable patients from the abuses associated with human research in the preregulatory era.

Address correspondence to Dr Zachary Simmons, Department of Neurology, Penn State Hershey Medical Center, Department of Neurology, EC 037, 30 Hope Dr, Hershey, PA 17033, zsimmons@psu.edu.

Relationship Disclosure: Dr Simmons serves as editor-in-chief of Muscle & Nerve and as a consultant for Cytokinetics, Inc, and Neuralstem, Inc. Dr Simmons receives research/grant support from the ALS Association; Biogen; Cytokinetics, Inc; and GlaxoSmithKline plc.

Unlabeled Use of Products/Investigational Use Disclosure: Dr Simmons discusses the controversies surrounding legislation to permit access to therapies not yet approved by the US Food and Drug Administration.