Expanded Access to Drug and Device Products for Clinical Treatment

Historically, patient access to investigational products (drugs or devices) has been limited to enrollment in clinical trials. However, enrollment of a patient into a clinical trial is not always possible for various reasons. Demand from patients and physicians for access to investigational products is due to the (sometimes remote) potential benefit to patients who have no other therapeutic alternatives. There are various options for the use of investigational drugs and devices in patient treatment through the Food and Drug Administration’s (FDA) expanded access program. These are outlined, along with criteria for use, submission requirements, approval processes, and associated practical guidance. There are four expanded access pathways for the use of investigational drugs: single-patient emergency use, single-patient treatment use, intermediate-size population treatment use, and treatment use. There are three expanded access pathways for the use of investigational devices: single-patient emergency use, single-patient or small group compassionate use, and treatment use. Additionally, treatment of a rare disease or condition with a Humanitarian Use Device (HUD) under a Humanitarian Device Exemption (HDE) is also possible.