Muaiad Kittaneh, Senior Director at ICON, shared a post on LinkedIn:
“The FDA has released a draft guidance proposing Minimal Residual Disease (MRD) negativity and Complete Response (CR) as primary endpoints to support accelerated approval in multiple myeloma…. This is a very meaningful regulatory evolution for hematologic oncology drug development.
From a drug development standpoint, this guidance reflects the reality of contemporary multiple myeloma trials, where modern therapies routinely achieve ORRs >70%. As a result, traditional response endpoints are becoming less discriminating and increasingly inefficient for trial design. MRD and CR provide greater sensitivity to depth of response, better alignment with long-term outcomes (PFS and OS), and a more realistic path to differentiation in today’s myeloma landscape.
Importantly, FDA grounds this shift in robust evidence including pooled analyses and unanimous support from the ODAC 2024, and provides concrete recommendations on trial design, statistical considerations, and MRD assay methodology to enable regulatory-grade use of these endpoints.
For drug developers, this guidance opens the door for:
- Smaller, more efficient trials
- Earlier regulatory decision-making
- Deeper integration of biomarker-driven endpoints into registrational strategies
Public comments are open through March 23 2026, and thoughtful industry engagement will be critical to shaping how MRD and CR are operationalized across future myeloma trials.
Regulatory science continues to evolve and this is a clear signal that the depth of response now matters as much as response itself.”
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