title: 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.
authors.affiliation: Department of Psychiatry, Wayne State University and Lafayette Clinic.
authors.forename: C A
authors.initials: CA
authors.lastname: Bagne
authors.forename: R F
authors.initials: RF
authors.lastname: Lewis
mesh_terms: D000284:Administration, Oral; D002986:Clinical Trials as Topic / Q000379:methods*; D006220:Haloperidol / Q000008:administration & dosage / Q000009:adverse effects; D006801:Humans; D011569:Psychiatric Status Rating Scales / Q000706:statistics & numerical data; D011594:Psychometrics; D011619:Psychotropic Drugs / Q000009:adverse effects*; D011788:Quality of Life; D012559:Schizophrenia / Q000188:drug therapy; D012565:Schizophrenic Psychology; D012919:Social Behavior; D013881:Thioridazine / Q000008:administration & dosage / Q000009:adverse effects
chemical_list: D011619:Psychotropic Drugs; D006220:Haloperidol; D013881:Thioridazine