The Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) has unanimously voted to recommend the use of minimal residual disease (MRD) as an accelerated approval endpoint in clinical trials for multiple myeloma. The 12-0 vote by the committee, which serves as a recommendation to the FDA rather than a binding decision, followed a review of data indicating a correlation between 12-month MRD negativity and traditional endpoints such as overall survival (OS) and progression-free survival (PFS).<\/p>\n\n\n\n
The University of Miami\u2019s Sylvester Comprehensive Cancer Center conducted research into MRD negativity as a potential efficacy proxy, performing a meta-analysis across over 5,000 patients in nine different studies to assess the correlation between MRD and OS\/PFS. Additionally, i2TEAMM, affiliated with the International Myeloma Foundation, presented data on the associations between 9- and 12-month MRD negativity and PFS.<\/p>\n\n\n\n
While i2TEAMM’s analysis supported MRD negativity as a prognostic for PFS in multiple myeloma patients, the group concluded that PFS should remain a key endpoint in Phase III trials to confirm clinical benefit.<\/p>\n\n\n\n
Ravi A. Madan, MD, a senior clinician at the National Cancer Institute and a committee member, emphasized the FDA’s need for caution, noting that the new guidance “will change the incentive structure for preclinical modeling, clinical development, and early clinical trials, requiring the FDA to be vigilant.”<\/p>\n\n\n\n