Global regulatory workshop on COVID-19 therapeutics #2

Global regulatory workshop on COVID-19 therapeutics #2: agreement on acceptable endpoints for clinical trials

International regulators have published a report today on the acceptability of various primary endpoints in the clinical trials conducted for the development of treatments for COVID-19.

The report summarises the main outcomes of the second workshop on COVID-19 therapeutics and clinical trials organised under the umbrella of the International Coalition of Medicines Regulatory Authorities (ICMRA). The workshop was co-chaired by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and EMA on 20 July 2020.

Many developers of medicines for the treatment of COVID-19 have already or are in the process of conducting clinical trials and have approached their regulatory authorities with proposals for phase 3 clinical trials.

An agreement by regulators on acceptable endpoints will facilitate rapid and consistent implementation of future clinical trials for COVID-19 medicines across the world.

For hospitalised patients with moderate to severe COVID-19, a range of suitable primary endpoints is available to measure the clinical benefit of investigational therapeutics for COVID-19 and support regulatory decision-making. While the workshop participants agreed that mortality is not the sole acceptable primary endpoint for these patients, mortality data should still be collected as a key secondary endpoint in all trials that do not plan to primarily use this outcome.

For outpatients with mild COVID-19, regulators agreed that mortality as the primary endpoint may not be suitable. Instead, the rate of progression to severe disease and the proportion of patients not hospitalised at a pre-specified time point may be more appropriate, depending on the primary objective of the study.