A Benefit–Risk Analysis Approach to Capture Regulatory Decision‐Making: Multiple Myeloma
In this work, that approach is applied to multiple myeloma (MM). Analyzing benefit–risk for MM requires extension beyond the analysis applied to NSCLC. In particular, the NSCLC analysis used overall survival (OS) as a common metric of benefit. In MM, FDA decisions pertaining to drug approval8 are mostly based on non‐OS metrics. Instead, they are based on metrics of time to progression (TTP), progression‐free survival (PFS), and/or overall response rate (ORR). Although these metrics are not the only possible measures of benefit, they are commonly used by regulatory reviewers.
MM represents about 13% of hematological cancers and is the second most common hematologic malignancy.9 Estimated median OS for newly diagnosed patients was less than 3 years in the year 2000 and exceeds 6 years in 2016.