A Benchmark for Dose Finding Studies with Continuous Outcomes
share
There is a growing interest in dose finding studies involving continuous outcomes. An important tool to evaluate the performance of any design is a benchmark that provides an upper bound on the performance. While the benchmark for dose finding studies with discrete endpoint is known, no such benchmark for studies with continuous endpoint has been developed. We show that the original benchmark by O'Quigley et al. (2002) and Cheung (2014), when looked at a different perspective, can be generalised to various settings with both discrete and continuous outcomes. We illustrate and compare the benchmark performance in the setting of a Phase I clinical trial with continuous endpoint and in the setting of Phase I/II clinical trial with continuous efficacy. It is found that the proposed benchmark provides an accurate upper bound for model-based dose finding methods and serves as a powerful tool for evaluating designs.
READ FULL TEXT
