Optimizing multiple Myeloma clinical trials: research direction, addressing limitations, and strategies for improvement

Despite significant advancements in multiple myeloma (MM) treatment, including novel therapies and combination strategies, the translation of findings from randomized controlled trials (RCTs) into real-world clinical practice has been associated with several challenges. Specifically, the principles and criterion that shape the current design of MM RCTs have left out a sizable portion of patients that would particularly benefit from trial inclusion. In addition, RCTs may use primary outcomes which only partially cover patient-relevant endpoints important for evaluating treatment efficacy and quality of life. In this review, we explore the current MM RCT landscape and suggest possible solutions to improve generalizability of trial results, mitigate logistical pitfalls, and integrate real-world evidence into trials. Together, these strategies are designed to refine MM treatment guidelines and improve outcomes for all patient populations.