An important tool to evaluate the performance of any design is an optimal benchmark proposed by O'Quigley and others (2002. Non-parametric optimal design in dose finding studies. Biostatistics3, 51-56) that provides an upper bound on the performance of a design under a given scenario. The original benchmark can only be applied to dose finding studies with a binary endpoint. However, there is a growing interest in dose finding studies involving continuous outcomes, but no benchmark for such studies has been developed. We show that the original benchmark and its extension by Cheung (2014. Simple benchmark for complex dose finding studies. Biometrics70, 389-397), when looked at from a different perspective, can be generalized to various settings with several discrete and continuous outcomes. We illustrate and compare the benchmark's performance in the setting of a dose finding Phase I clinical trial with a continuous toxicity endpoint and a Phase I/II trial with binary toxicity and continuous efficacy endpoints. We show that the proposed benchmark provides an accurate upper bound in these contexts and serves as a powerful tool for evaluating designs.

中文翻译：

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具有连续结果的剂量查找研究的基准。

评估任何设计性能的重要工具是O'Quigley等人（2002年。剂量寻找研究中的非参数最优设计。Biostatistics3，51-56）提出的最佳基准，它为设计的性能提供了上限。在给定场景下进行设计。原始基准只能用于具有二进制终点的剂量查找研究。然而，对涉及连续结果的剂量寻找研究的兴趣日益浓厚，但尚未建立此类研究的基准。我们显示，Cheung提出的原始基准及其扩展（2014年，复杂剂量查找研究的简单基准。Biometrics70，389-397），从不同的角度来看，可以推广到具有多种离散和连续结果的各种环境。我们说明并比较基准 在具有连续毒性终点的I期临床试验和具有二元毒性和连续功效终点的I / II期剂量设定中的表现。我们表明，提出的基准在这些情况下提供了准确的上限，并且可以用作评估设计的强大工具。