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Design and analysis of clinical trials 

Design and analysis of clinical trials
Design and analysis of clinical trials

Daniel Sargent

and Qian Shi


March 29, 2019: This chapter has been re-evaluated and remains up-to-date. No changes have been necessary.

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date: 31 July 2021

This chapter addresses the statistical design and analysis of oncology clinical trials. Traditional trial designs are described by each of three phases—phase I, II, and III studies. It elaborates on the concepts of single-arm versus randomized (screening and selection) phase II trials. Critical aspects of randomized phase III studies are discussed, including randomization, stratification, blinding, and the intent-to-treat principle. Furthermore, the chapter introduces innovative designs, emphasizing the incorporation of biomarkers into the study design. Careful considerations of endpoints, essential to power and sample size when planning a study, are considered. In addition to discussing standard statistical analysis methods, we particularly discuss the critical element of controlling for multiple comparisons.

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