Introduction: Minimal residual disease (MRD) negativity has emerged as a surrogate marker in multiple myeloma (MM), correlating with deeper responses and improved progression-free survival. In April 2024, the FDA’s Oncologic Drugs Advisory Committee (ODAC) endorsed MRD negativity as an early endpoint for accelerated treatment approval in MM. While this regulatory shift may shorten drug approval timelines, patient perspectives on MRD as a treatment goal and trial endpoint remain underexplored. Understanding these views is critical to aligning clinical trial design, regulatory strategies, and patient values.
Methods: A cross-sectional online survey was conducted among adult MM patients (≥18 years) enrolled in the HealthTree Cure Hub registry. The IRB-approved survey included 13 MRD-related questions and six demographic questions. Patients were asked about MRD testing experience, treatment perceptions, and regulatory awareness. The survey included a comparative evaluation of the patient’s insights regarding MRD-based accelerated approval, both prior to and following their awareness of the ODAC vote concerning MRD. Responses were de-identified and analyzed descriptively.
Results: Among 192 respondents, 163 (85%) reported an MM diagnosis. Of 149 patients who answered the testing question, 106 (71%) had undergone MRD testing. Among those tested, 42 (41%) achieved 10⁻⁶ sensitivity, 19 (19%) reached 10⁻⁵, and 24 (24%) did not achieve MRD negativity. Regarding efficacy perceptions, 105 of 136 (77%) rated sustained MRD negativity (≥12 months) as “excellent,” while only 1 (1%) rated it “below average” or worse. Despite achieving complete response, 58 of 134 (43%) indicated they would be “above average likely” or more to discuss changing therapy to pursue MRD negativity. After being informed of MRD-based regulatory approval by an expert, 87 of 128 (68%) rated it as “very important,” compared to 64 of 130 (49%) before reading the explanation. Sixty-three of 127 (50%) said the decision gave them “a great deal” of hope; 55 (44%) believed it would greatly accelerate treatment access. When considering future therapies, 106 of 124 (85%) said MRD negativity held “a lot” or “a great deal” of importance, despite recognizing the limited long-term safety data.
Conclusions: Patients with MM strongly view MRD negativity, particularly when sustained, as a meaningful indicator of efficacy. A substantial proportion reported willingness to modify treatment strategies to achieve MRD negativity, and educational content significantly increased support for MRD-based regulatory pathways. These findings highlight the importance of incorporating patient-reported preferences into clinical trial endpoints and regulatory decision-making, reinforcing MRD’s relevance not only as a biomarker but as a patient-valued measure of treatment success.
