Perceived benefit after participating in positive or negative/neutral heart failure trials: the patients' perspective

Background: Clinical trials, the gold standard for the evaluation of new th erapeutic strategies, may prove a drug to be beneficial, harmful or neutral according to its effect on the end-point(s) under study. Aims: To study th e reaction and perspective of the patients participating in a clinical hear t failure trial, particularly in relation to whether the trial subsequently proved to be positive, negative or neutral. Methods: Anonymous self-comple ted questionnaire uas sent to 78 and returned by 70 consecutive patients 1- 6 months after participating in six clinical heart failure trials. The tria l was neutral or negative regarding the primary end-point in four (47 patie nts) of the six studies (MACH-1 trial of mibefradil, REACH trial of bosenta n, CASCO trial of calcium sensitizer, ecadotril trial of neutral endopeptid ase inhibitor) and positive in two (23 patients) (ICARUS Israel carvedilol study, exercise study of candesartan cilexetil). Results: Most patients rep orted subjective global clinical benefit (78% for positive, 74% for negativ e or neutral trial, NS) after participating in a clinical trial. After adju stment for age, sex, level of education, previous research, perceived compr ehension, and treatment allocation (active drug/placebo) in a stepwise regr ession model, perceived global improvement was greater in older patients (P = 0.02), after participation in a positive trial (P = 0.05) and in females (P = 0.07). The major reason given by the patient for perceived clinical i mprovement was better follow-up, some believed it was due to change in medi cation, particularly those who had participated in a positive trial. Conclu sions: More than 70% of patients participating in clinical trials of new dr ugs for heart failure reported perceived global improvement. Clinical impro vement was greater in, but not limited to, patients who participated in pos itive trials. These salutary findings support the continued recruitment of patients to clinical heart failure trials. (C) 2001 European Society of Car diology. All rights reserved.