Zhaohui Su, VP of Biostatistics at Ontada, shared a post on LinkedIn about a recent article by Lockwood Taylor et al published in Blood Cancer Journal:
“This editorial from Blood Cancer Journal (2025) discusses the growing role of real-world evidence (RWE) in regulatory approvals for multiple myeloma (MM) therapies.
MM is a rare, incurable blood cancer with frequent relapses, making traditional randomized clinical trials (RCTs) increasingly challenging. Regulatory agencies like the FDA and EMA have issued guidelines supporting the use of RWE—data derived from routine clinical practice, such as electronic health records—in regulatory submissions, especially for rare diseases and advanced treatment lines where RCTs may be impractical or unethical.
Between January 2021 and April 2025, 44% of MM drug marketing applications approved by the FDA and EMA included RWE, mainly in advanced lines of therapy. RWE was used to demonstrate unmet medical need, support single-arm trials, and provide external comparator arms. However, the quality and reliability of RWE are critical; regulators have sometimes rejected RWE due to methodological limitations or data heterogeneity.
The article emphasizes that sponsors should engage early with regulators and ensure data sources are fit-for-purpose, reliable, and generalizable. High-quality RWE is expected to remain essential for advancing MM therapies, especially where patient needs are most urgent.”
Title: Utilization of real-world evidence in regulatory approvals for multiple myeloma therapies
Authors: Lockwood Taylor, Angela Chen, Amy Pierre
Read the Full Article on Blood Cancer JournalÂ
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