Expanded Access Programs, compassionate drug use, and Emergency Use Authorizations during the COVID-19 pandemic

Highlights • Expanded Access (EA) is the use of an investigational drug outside of a clinical investigation. • Emergency Use Authorizations (EUAs) are issued during public health emergencies. • The pandemic has forced us to recognize weaknesses associated with our current EA and EUA system.
The US Food and Drug Administration (FDA) Expanded Access (EA) Program, which allows for compassionate uses of unapproved therapeutics and diagnostics outside of clinical trials, has gained significant traction during the Coronavirus 2019 (COVID-19) pandemic. While development of vaccines has been the major focus, uncertainties around new vaccine safety and effectiveness have spawned interest in other pharmacological options. Experimental drugs can also be approved under the FDA Emergency Use Authorization (EUA) program, designed to combat infectious disease and other threats. Here, we review the US experience in 2020 with pharmacological EA and EUA approvals during the pandemic. We also provide a description of, and clinical rationale for, the EA- or EUA-approved drugs during the pandemic and concluding reflections on the EA program and its potential future uses.