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Monitoring of a pilot toxicity study with two adverse outcomes
Author(s) -
Etzioni Ruth,
Pepe Margaret Sullivan
Publication year - 1994
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/sim.4780132203
Subject(s) - frequentist inference , univariate , bivariate analysis , bayesian probability , adverse effect , confidence interval , clinical trial , adverse outcome pathway , outcome (game theory) , statistics , posterior probability , multivariate statistics , medicine , computer science , bayesian inference , mathematics , mathematical economics , computational biology , biology
We consider monitoring a pilot toxicity study in which the adverse outcome is bivariate and the goal is to terminate the trial if evidence of excessive toxicity is encountered. We develop a Bayesian monitoring rule, based on the posterior probability that the frequency of either adverse outcome exceeds that observed under standard therapy. This rule is intuitive and ethical, and extends in a straightforward fashion from the univariate to the multivariate case. Since p ‐values and confidence intervals are standard methods for reporting the results of clinical trials, we also suggest how frequentist inferences may be drawn at the conclusion of a study monitored in this fashion. This work thus represents an integration of Bayesian and frequentist methodology for sequential clinical trials.