A Benefit–Risk Analysis Approach to Capture Regulatory Decision‐Making: Multiple Myeloma

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Regulatory decisions about drug approvability are benefit–risk assessments (as documented by representative quotations in Supplementary Table 1). Benefit–risk approaches1 have the potential to facilitate stakeholder decision‐making throughout the drug development lifecycle, and thereby support the accessibility of safe and effective medicines to patients.3 As part of the Prescription Drug User Fee Act (PDUFA V) and US Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA committed to implement a structured benefit–risk approach in assessments of new drug applications (NDAs) and biological license applications (BLAs). In prior work, a quantitative benefit–risk analysis approach was proposed6 and used for analysis of regulatory decision‐making in non‐small cell lung cancer (NSCLC).7
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.

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