Designing historical control studies with survival endpoints using exact statistical inference.

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Tytuł:: Designing historical control studies with survival endpoints using exact statistical inference.
Autorzy:: Han G; Department of Epidemiology and Biostatistics, School of Public Health, Texas A&M University, College Station, Texas, USA.
Źródło:: Pharmaceutical statistics [Pharm Stat] 2021 Jan; Vol. 20 (1), pp. 4-14. Date of Electronic Publication: 2020 Aug 03.
Typ publikacji:: Journal Article; Research Support, Non-U.S. Gov't
Język:: English
Imprint Name(s):: Original Publication: Chichester, UK : Wiley, c2002-
MeSH Terms:: Models, Statistical*
Research Design*
Bias ; Historically Controlled Study ; Humans ; Sample Size
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Contributed Indexing:: Keywords: historical control trials; randomized clinical trials; statistical power; survival analysis; uniformly most powerful test
Entry Date(s):: Date Created: 20200804 Date Completed: 20211125 Latest Revision: 20211125
Update Code:: 20240105
DOI:: 10.1002/pst.2050
PMID:: 32743949
: Czasopismo naukowe

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Historical control trials compare an experimental treatment with a previously conducted control treatment. By assigning all recruited samples to the experimental arm, historical control trials can better identify promising treatments in early phase trials compared with randomized control trials. Existing designs of historical control trials with survival endpoints are based on asymptotic normal distribution. However, it remains unclear whether the asymptotic distribution of the test statistic is close enough to the true distribution given relatively small sample sizes in early phase trials. In this article, we address this question by introducing an exact design approach for exponentially distributed survival endpoints, and compare it with an asymptotic design in both real examples and simulation examples. Simulation results show that the asymptotic test could lead to bias in the sample size estimation. We conclude the proposed exact design should be used in the design of historical control trials.
(© 2020 John Wiley & Sons Ltd.)

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