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Prior effective sample size in phase II clinical trials with mixed binary and continuous responses

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  • Meghna Bose
  • Jean‐François Angers
  • Atanu Biswas

Abstract

The problem of finding Effective Sample Size (ESS) in Phase II clinical trials where toxicity and efficacy are the two components of the treatment response vector is considered. In particular, one of the components is assumed to be binary and the other is assumed to be continuous. The case of binary safety and continuous efficacy is studied for different prior distributions under different set up. Theoretical expressions are obtained in various situations. The methods are evaluated and compared by simulation studies. The proposed method is then illustrated by using some real life data on a phase II vaccine trial for Covid‐19.

Suggested Citation

  • Meghna Bose & Jean‐François Angers & Atanu Biswas, 2023. "Prior effective sample size in phase II clinical trials with mixed binary and continuous responses," Statistica Neerlandica, Netherlands Society for Statistics and Operations Research, vol. 77(2), pages 233-248, May.
    • Handle: RePEc:bla:stanee:v:77:y:2023:i:2:p:233-248
    DOI: 10.1111/stan.12283
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    References listed on IDEAS

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    1. Beat Neuenschwander & Sebastian Weber & Heinz Schmidli & Anthony O'Hagan, 2020. "Predictively consistent prior effective sample sizes," Biometrics, The International Biometric Society, vol. 76(2), pages 578-587, June.
    2. Cheng, Tsung-Chi & Biswas, Atanu, 2008. "Maximum trimmed likelihood estimator for multivariate mixed continuous and categorical data," Computational Statistics & Data Analysis, Elsevier, vol. 52(4), pages 2042-2065, January.
    3. Satoshi Morita & Peter F. Thall & Peter Müller, 2008. "Determining the Effective Sample Size of a Parametric Prior," Biometrics, The International Biometric Society, vol. 64(2), pages 595-602, June.
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