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Applications of Bayesian analysis to proof‐of‐concept trial planning and decision making
Author(s) -
Quan Hui,
Chen Xun,
Lan Yu,
Luo Xiaodong,
Kubiak Rene,
Bonnet Nicolas,
Paux Gautier
Publication year - 2020
Publication title -
pharmaceutical statistics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.421
H-Index - 38
eISSN - 1539-1612
pISSN - 1539-1604
DOI - 10.1002/pst.1985
Subject(s) - computer science , bayesian probability , influence diagram , proof of concept , decision analysis , decision theory , process (computing) , operations research , risk analysis (engineering) , artificial intelligence , decision tree , statistics , mathematics , medicine , operating system
Decision making is a critical component of a new drug development process. Based on results from an early clinical trial such as a proof of concept trial, the sponsor can decide whether to continue, stop, or defer the development of the drug. To simplify and harmonize the decision‐making process, decision criteria have been proposed in the literature. One of them is to exam the location of a confidence bar relative to the target value and lower reference value of the treatment effect. In this research, we modify an existing approach by moving some of the “stop” decision to “consider” decision so that the chance of directly terminating the development of a potentially valuable drug can be reduced. As Bayesian analysis has certain flexibilities and can borrow historical information through an inferential prior, we apply the Bayesian analysis to the trial planning and decision making. Via a design prior, we can also calculate the probabilities of various decision outcomes in relationship with the sample size and the other parameters to help the study design. An example and a series of computations are used to illustrate the applications, assess the operating characteristics, and compare the performances of different approaches.