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Abstract:
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Understanding the value of a new therapeutic agent in treatment of cancer is important to both caregivers and patients. The recently developed ASCO and ESMO value framework attempts to address this complex problem using a multi-dimensional approach that evaluates benefit in terms of efficacy, safety and QoL. Here we compare their recommendations on measuring clinical benefit in terms of efficacy, for different classes of anti-cancer agents (immunotherapy, chemotherapy, small molecules). In particular, we focus on the emerging immuno-oncology agents where there is a possible underlying delayed treatment effect. We compare the performance of commonly used statistics (hazard ratio, median, restricted mean survival and survival at landmark time points) with the composite scores proposed by ASCO and ESMO-MCBS for clinical benefit assessment under different treatment scenarios (PH, delayed treatment effect, belly shaped KM curves). A novel method to measure clinical benefit is proposed using a weighted combination of these standard measures. The existing statistics/scores and the proposed method are evaluated using both simulated and real clinical trial data.
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