Sample sizes for phase II clinical trials derived from Bayesian decision theory

Stat Med. 1994 Dec;13(23-24):2493-502. doi: 10.1002/sim.4780132312.

Abstract

In early phase clinical trials of a new medical treatment, patients are treated to decide whether there is sufficient promise to justify additional studies. A decision theoretic approach is proposed to help determine the number of patients that should be treated. The optimal sample size is obtained by maximizing a utility function which incorporates both the number of 'gained successes' and the costs of treatment. The method extends work of Sylvester and Staquet, and adopts a Bayesian formulation. Numbers of patients in later studies and in eventual routine use of the treatment are taken into account. We allow for the possibility that a later study might lead to an erroneous conclusion. The effects of these various influences on the recommended sampling plan for the early phase clinical trial are explored.

MeSH terms

  • Bayes Theorem*
  • Chi-Square Distribution
  • Clinical Trials, Phase II as Topic / economics
  • Clinical Trials, Phase II as Topic / statistics & numerical data*
  • Costs and Cost Analysis
  • Decision Theory*
  • Humans
  • Sample Size*
  • Treatment Outcome