2009 issue 2


Volume 18, issue 2

World Psychiatry Forum

Effectiveness as an outcome measure for treatment trials in psychiatry

W. Wolfgang Fleischhacker1, Guy M. Goodwin2
1. Department of Psychiatry and Psychotherapy, Biological Psychiatry Division, University of Innsbruck, Innsbruck, Austria
2. University Department of Psychiatry, Warneford Hospital, Oxford, OX3 7JX, UK
Postępy Psychiatrii i Neurologii 2009; 18 (2): 111-116
Keywords: clinical trials, methodology, schizophrenia, mood disorders, bipolar disorder, depression, antipsychotics, antidepressants, mood stabilizers, efficacy, effectiveness, pragmatic trials


There is at present some confusion about the relative value of clinical trials performed to investigate efficacy vs. those designed to investigate effectiveness. This is particularly challenging when studies performed as experiments for regulators by companies are used to shape and inform clinical practice, especially if studies conducted under more real life conditions fail to support predicted benefits. We review the field in relation to the new antipsychotics, inparticular. Other indications, including mood disorders, which are also briefly touched upon, have so far received less definitive attention, but are likely to encounter the same difficulties. We conclude that, where the results of efficacy trials arepositive and an effectiveness trial is negative, one should not necessarily prefer the effectiveness trial - it may simply have failed. Where efficacy trials and effectiveness trials point to similar conclusions, then the findings are mutually supportive.