Using a Benefit-Risk Analysis Approach to Capture Regulatory Decision Making: Melanoma.

Drug regulators seek to make decisions regarding drug approvals based on analysis of the relevant benefits and risks. In this work, 25 US Food and Drug Administration (FDA) decisions on melanoma drugs were identified and analyzed based on clinical trial results published between 1999 and 2017. In each case, the benefits and risks of the new drug in each clinical trial relative to a comparator (typically the control arm of the same clinical trial) were quantified. The benefits and risks were analyzed using a common scale to allow for direct comparison. A sensitivity analysis was conducted using vemurafenib to explore the magnitude of uncertainty in the quantitative assessments. The associated FDA decision outcomes of the new drugs were consistent with the benefits and risks quantified in this work.