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FDA guidance and MRD
Using MRD as primary endpoint in MM drug trials to support accelerated approval
- On January 20, 2026, the FDA issued a draft guidance for industry that provides recommendations to sponsors about using MRD as a primary endpoint in trials evaluating drugs and biologics intended to treat patients with MM to support approval under the accelerated approval regulations
- These recommendations include general drug development considerations, trial design and statistical considerations, and assay considerations for MRD evaluation
- MRD endpoint refers to MRD negativity rate as assessed in the bone marrow by either flow cytometry- or sequencing-based methods in patients who achieved a CR*
- A robust trial design, including clear assessment schedules, minimal missing data, pre-specified analysis timepoints (e.g., 9-12 months) and rigorous Type 1 error control is necessary
*CR is defined to include patients who achieved CR or stringent CR
FDA, United States Food and Drug Administration; MM, multiple myeloma; MRD, minimal residual disease; ODAC, Oncologic Drugs Advisory Committee.
U.S. Food and Drug Administration. (2026, February 20). Minimal residual disease and complete response in multiple myeloma: Use as endpoints to support accelerated approval [Draft guidance]. https://www.fda.gov/media/190647/download.
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