The current approval indications for pembrolizumab are complex, reflecting the inclusion criteria of numerous clinical trials that led to approvals. Here we argue that allowing the use of pembrolizumab to any advanced solid tumor in any tumor type in any line of therapy for a fixed duration may be preferable to the current assortment of indications. The aggregate response rate in landmark clinical trials for approved indications of pembrolizumab is low and even lower in real-world populations. Due to heterogeneity of response to checkpoint inhibitors and limited predictive biomarkers, there are subsets of patients without approved indications for pembrolizumab that may have response to checkpoint inhibitors. The current regulatory framework of numerous overlapping clinical trials leading to complex approval indications is redundant and inefficient. We conclude that giving pembrolizumab in any metastatic solid tumor in any setting may lead to better outcomes with minimal increase in cost. Randomized clinical trials should focus more on optimal duration of treatment based on tumor type and initial response to checkpoint inhibitors.

pembrolizumab 目前的批准适应症很复杂，反映了导致批准的众多临床试验的纳入标准。在这里，我们认为允许在固定持续时间内对任何肿瘤类型的任何晚期实体瘤使用 pembrolizumab 可能比目前的各种适应症更可取。在具有里程碑意义的临床试验中，针对派姆单抗批准适应症的总反应率很低，在现实世界人群中甚至更低。由于对检查点抑制剂反应的异质性和有限的预测性生物标志物，有一些没有批准派姆单抗适应症的患者亚群可能对检查点抑制剂有反应。导致复杂批准适应症的众多重叠临床试验的当前监管框架是多余且低效的。我们的结论是，在任何情况下对任何转移性实体瘤给予派姆单抗可能会带来更好的结果，而成本增加最少。随机临床试验应更多地关注基于肿瘤类型和对检查点抑制剂的初始反应的最佳治疗持续时间。