Comparison of Treatments with Multiple Outcomes

  • Pascale Tubert-Bitter
  • Daniel A. Bloch
  • Tze L. Lai


Treatment comparisons in clinical studies often involve several endpoints, particularly those related to quality of life of patients suffering from diseases like cancer and arthritis. We review traditional statistical methods for this problem and describe some new approaches. One approach is based on a new formulation of the null hypothesis that incorporates the essential univariate and multivariate features of the treatment effects. Another approach is based on assigning benefit scores to different regions of the toxicity-efficacy outcome space. A third approach involves patient thresholds for tolerating different treatments. Bootstrap methods are used to circumvent the analytic and computational complexities of the new approaches. We illustrate these approaches using data from patients with rheumatoid arthritis. In this setting, quality of life involves trade-offs between efficacy and toxicity of treatments.


Null Hypothesis Rheumatoid Arthritis Patient Error Probability Multivariate Normal Distribution Multiple Outcome 
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Copyright information

© Springer Science+Business Media Dordrecht 2002

Authors and Affiliations

  • Pascale Tubert-Bitter
    • 1
  • Daniel A. Bloch
    • 2
  • Tze L. Lai
    • 2
  1. 1.INSERM U. 472USA
  2. 2.Stanford UniversityUSA

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