Overview
Real-world evidence (RWE) is increasingly being incorporated into regulatory submissions for multiple myeloma (MM) treatments in the US and EU. This study analyzed MM drug approvals by the FDA and EMA between January 2021 and November 2024 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 42% of MM marketing applications, primarily for relapsed/refractory MM. The most common uses of RWE were as an external comparator arm and to demonstrate the natural history of MM. While regulators noted some clinical and treatment-related differences between RWE studies and registrational trials, they generally accepted RWE as supportive evidence in these marketing applications to contextualize unmet medical need.
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.