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

J Consult Clin Psychol. 1992 Apr;60(2):225-39. doi: 10.1037//0022-006x.60.2.225.

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.

Publication types

  • Clinical Trial
  • Comparative Study
  • Randomized Controlled Trial

MeSH terms

  • Administration, Oral
  • Clinical Trials as Topic / methods*
  • Haloperidol / administration & dosage
  • Haloperidol / adverse effects
  • Humans
  • Psychiatric Status Rating Scales / statistics & numerical data
  • Psychometrics
  • Psychotropic Drugs / adverse effects*
  • Quality of Life*
  • Schizophrenia / drug therapy
  • Schizophrenic Psychology
  • Social Behavior*
  • Thioridazine / administration & dosage
  • Thioridazine / adverse effects

Substances

  • Psychotropic Drugs
  • Haloperidol
  • Thioridazine