Improving the Decision to Pursue a Phase 3 Clinical Trial by Adjusting for Patient-Specific Factors in Evaluating Phase 2 Treatment Efficacy Data

Glenn Heller, Michael W. Kattan and Howard I. Scher
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Glenn Heller: Department of Epidemiology and Biostatistics, Memorial Sloan-Kettering Cancer Center, New York, NY, hellerg@mskcc.org
Michael W. Kattan: Department of Quantitative Health Sciences, Cleveland Clinic, Cleveland, Ohio
Howard I. Scher: Department of Medicine, Memorial Sloan-Kettering Cancer Center, New York, NY

Medical Decision Making, 2007, vol. 27, issue 4, 380-386

Abstract: Phase 2 clinical trials are undertaken to provide evidence of treatment efficacy and safety. A test statistic that accounts for individual patient risk in the patient population is proposed and applied to a phase 2 clinical trial for castrate metastatic prostate cancer. The test statistic is computed to compare, for each patient, the observed 2-year survival outcome to the predicted 2-year survival probability. A logistic regression model, developed using historical data in the same patient population, is used to adjust for patient risk in predicting the 2-year survival probability. Goodness-of-fit procedures are performed to ensure that a proper model is fit to the data. The test result is compared to the score test, the binomial exact test, and Fisher's exact test, all of which use the average 2-year survival probability in the population as the parameter of interest. The results demonstrate the benefit of risk adjustment in determining treatment efficacy in a single-arm phase 2 trial. By adjusting for patient risk, this method can provide a more precise assessment of phase 2 treatment efficacy, thereby improving the decision whether to proceed to a phase 3 clinical trial.

Keywords: Key words: analysis of phase 2 clinical trial data; heterogeneous patient population; model-based test statistic. (Med Decis Making 2007; 27:380—386) (search for similar items in EconPapers)
Date: 2007
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Persistent link: https://EconPapers.repec.org/RePEc:sae:medema:v:27:y:2007:i:4:p:380-386

DOI: 10.1177/0272989X07303826

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