Establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorisation

This reflection paper is intended to reflect the current thinking of EMA's Committee for Medicinal Products for Human Use (CHMP) on single-arm trials (SATs) that are submitted as pivotal evidence for establishing efficacy in a marketing authorisation application. The reflection paper discusses considerations in relation to the design, planning, conduct, analysis and interpretation of results derived from single-arm trials. It is applicable across different therapeutic areas, including for rare diseases.

Keywords: Single-arm trials, non-randomised trials, regulatory decision making

Current version

Reflection paper on establishing efficacy based on single-arm trials submitted as pivotal evidence in a marketing authorisation

Consultation dates: 21/04/2023 to 30/09/2023Draft: consultation closedReference Number: EMA/CHMP/564424/2021