Single-patient expanded access: A primer for pharmacists.

Abstract

The process of providing treatment with investigational drugs through expanded access is explained. Roles and informational resources for pharmacists are discussed. Expanded access is a regulatory pathway for the treatment of serious or life-threatening diseases or conditions with investigational agents outside of clinical trials. In the setting of no available therapies or ineligibility for clinical trials, a patient and their treating physician may pursue therapies that are not approved by the Food and Drug Administration (FDA). The drug manufacturer, FDA, and institutional review boards are required stakeholders in the expanded access process. Other pathways for obtaining investigational agents outside of clinical trials, including federal Right to Try and emergency use authorization, exist but differ in their level of involvement of these key stakeholders. Pharmacists are equipped to be involved in therapy identification, risk vs benefit evaluations, therapy preparation and administration, supportive care, transitions of care, and regulatory compliance. Specific websites, publications, and organizations can aid in navigating expanded access. Combining elements of traditional clinical care and research, expanded access involves direct treatment with non-FDA-approved agents outside of a clinical trial. Healthcare providers should be aware of the possibility of providing investigational treatments after all approved options have been exhausted.