Overview
Real-world evidence (RWE) is increasingly being incorporated into regulatory submissions for multiple myeloma (MM) treatments in the US and EU. The authors analyzed MM drug approvals by the FDA and EMA between January 2021 and April 2025 to understand how RWE has been used in marketing applications. Researchers reviewed regulatory documents to identify key themes in how RWE was leveraged and the feedback provided by regulators.
Findings showed that RWE was included in 44% of MM marketing applications, primarily for relapsed/refractory MM. The most common uses of RWE were as an external comparator arm and to demonstrate unmet need using a natural history study.. MM approvals including RWE in their application were predominantly in advanced lines of therapy. Regulators specifically noted RWD must be both relevant and reliable, completely capturing critical treatment variables such as line of therapy, eligibility criteria that indicate evidence of progression or disease stage, and endpoints such as real-world overall response rate and progression-free survival.
Why this matters
As the role of RWE in regulatory decision-making continues to grow, understanding how it has been used in past MM drug approvals provides valuable insights for future submissions. This study highlights the acceptance of RWE by the FDA and EMA and underscores its potential to complement traditional clinical trial data. By identifying key regulatory considerations, this research can help optimize the use of RWE in future drug marketing applications for the treatment of MM as well as other types of rare cancers, ultimately supporting more informed regulatory decisions and improving patient access to new treatments.