S. 204 amends the Federal Food, Drug, and Cosmetic Act to exempt, from specified requirements and restrictions under that Act and other laws, the provision of certain unapproved, investigational drugs to a terminally ill patient who has exhausted approved treatment options and is unable to participate in a clinical trial involving the drugs. The manufacturer or sponsor of an eligible investigational drug must report annually to the Food and Drug Administration (FDA) on any use of the drug in accordance with these provisions. The FDA shall post an annual summary report of such use on its website.
The bill limits the liability of a sponsor, manufacturer, prescriber, or dispenser that provides, or declines to provide, an eligible investigational drug to an eligible patient in accordance with the bill.
The Food and Drug Administration (FDA) regulates the U.S. sale of drugs and biological products, basing approval or licensure on evidence of the safety and effectiveness for a product's intended uses. Without that approval or licensure, a manufacturer may not distribute the product except for use in the clinical trials that will provide evidence to determine that product's safety and effectiveness.
Under certain circumstances, however, FDA may permit the sponsor to provide an unapproved or unlicensed product to patients outside that standard regulatory framework. Two such mechanisms are expanded access to investigational drugs, commonly referred to as compassionate use, and emergency use authorization. Although FDA granted over 99% of the expanded access requests it has received since 2010, patients and others point to what they see as FDA-created obstacles to access. In February 2015, FDA released draft guidance and a new form that, when finalized, would reduce the amount of information required from the physician. Since 2014, 38 states have passed so-called right to try laws to bypass FDA permission for access to an investigational drug.