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Abstract:
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It is essential that both efficacy and safety be considered when evaluating the merit of drugs to ensure that patients are only exposed to drugs when the benefit outweighs the risk. Historically, efficacy and safety analyses are performed separately when assessing the value of a new drug or dose. However, it is also of interest to assess the comparative benefit-risk (BR). Several methods have been proposed that use ordered categorical outcomes combining both efficacy and safety components to attain this goal. Evans and Follmann (2016) recently advocated for a unified composite of benefit and risk as a pragmatic endpoint in a clinical trial setting, using the desirability of outcome ranking (DOOR, Evans et al., 2015). In this paper we adapted the win ratio concept, developed to address composite endpoints, (Pocock et al. 2012) for a potential application in BR analysis. We applied a simple set of rules to identify winners and losers comparing matched or unmatched pairs of patients. We will discuss statistical considerations for active control comparisons and dose response. We applied this approach retrospectively to evaluate the BR of a Phase III clinical trial.
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