Reflection paper on the use of measurable residual disease as a clinical endpoint in multiple myeloma studies - Scientific guideline

Current version

Reflection paper on the use of measurable residual disease as a clinical endpoint in multiple myeloma studies

AdoptedReference Number: EMA/49401/2022

The reflection paper aims to address the use of undetectable minimal residual disease (MRD) as an intermediate efficacy endpoint in controlled randomised clinical studies in patients with multiple myeloma (MM), adequately designed to demonstrate efficacy by relevant hard endpoints. MRD as an endpoint in this context would allow earlier approval of new drugs pending final confirmatory data.

Keywords: Minimal residual disease (MRD), Myeloma

Superseded documents

Draft guideline on the use of minimal residual disease as a clinical endpoint in multiple myeloma studies – Superseded

Consultation dates: 06/08/2018 to 31/10/2018Draft: consultation closedReference Number: EMA/CHMP/459559/2018Summary:

The guideline aims to address the use of undetectable minimal residual disease (MRD) as an intermediate efficacy endpoint in controlled randomised clinical studies in patients with multiple myeloma (MM), adequately designed to demonstrate efficacy by relevant hard endpoints. MRD as an endpoint in this context would allow earlier approval of new drugs pending final confirmatory data.