A Benefit–Risk Analysis Approach to Capture Regulatory Decision‐Making: Non‐Small Cell Lung Cancer

Drug regulators around the world make decisions about drug approvability based on qualitative benefit–risk analyses. There is much interest in quantifying regulatory approaches to benefit and risk. In this work the use of a quantitative benefit–risk analysis was applied to regulatory decision‐making about new drugs to treat advanced non‐small cell lung cancer (NSCLC). Benefits and risks associated with 20 US Food and Drug Administration (FDA) decisions associated with a set of candidate treatments submitted between 2003 and 2015 were analyzed. For benefit analysis, the median overall survival (OS) was used where available. When not available, OS was estimated based on overall response rate (ORR) or progression‐free survival (PFS). Risks were analyzed based on magnitude (or severity) of harm and likelihood of occurrence. Additionally, a sensitivity analysis was explored to demonstrate analysis of systematic uncertainty. FDA approval decision outcomes considered were found to be consistent with the benefit–risk logic.