Evaluating the effects of drugs on behavior and quality of life: an alternative strategy for clinical trials

Abstract

Conventional clinical trials involve tests of hypotheses with statistics computed from values of dependent variables alone. An alternative is to test hypotheses with statistics computed from benefit/harm scores that measure longitudinal associations between dose and values of the dependent variables. The proposed standardized measure of benefit/harm quantifies the strength of evidence that a patient did either better or worse while on treatment. A benefit/harm score, particularly when obtained from a randomized, N-of-1 trial, indicates a beneficial or harmful treatment effect for the individual. Benefit/harm scores from samples of patients are evaluated with standard statistical tests, with or without group comparisons, to make inferences about populations. The proposed alternative strategy can yield within-patient indicators of treatment effect that are more reliable, valid, comprehensive, and detailed. This, in turn, could help make many population-based clinical trials more informative, cost-effective, and clinically useful for participants.