Summary Points

• Time-sensitive access to unapproved experimental interventions should be permitted on humanitarian grounds when patients or communities are facing death or irreversible disease progression and no other efficacious diagnostic, preventive, or therapeutic alternative exists.
• Regulatory deficits could stymie time-sensitive efforts to contain public health threats when no efficacious curative, therapeutic, or preventive interventions exist to counter the threat in question.
• United States regulatory mechanisms may provide useful guidance from a regulatory perspective to policy makers grappling with how to adequately prepare for, or respond to, potential or emerging public health emergencies.
• Access to unapproved experimental interventions should be underpinned by a robust monitoring and evaluation component that will inform product development and licensure.
• A global-level rapid-response governance framework for the employment of unapproved interventions in humanitarian contexts should be established as a matter of urgency.