Defining a clinically meaningful effect for the design and interpretation of randomized controlled trials

Innovations in Clinical Neuroscience

Volumn 10, Issue 5-6, 2013, Pages

  Keefe, Richard S E ^a   Kraemer, Helena C ^b,c   Epstein, Robert S ^d   Frank, Ellen ^e   Haynes, Ginger ^f   Laughren, Thomas P ^g   McNulty, James ^h   Reed, Shelby D ⁱ   Sanchez, Juan ^j   Leon, Andrew C ^k  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b STANFORD UNIVERSITY   (United States)

^c UNIVERSITY OF PITTSBURGH   (United States)

^d University of Pittsburgh School of Medicine ^*

^e UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^f ELI LILLY AND COMPANY   (United States)

^g The Emmes Company   (United States)

^h MHCA OASIS RI   (United States)

ⁱ DUKE CLINICAL RESEARCH INSTITUTE   (United States)

^j Ladenburg Thalmann and Co   (United States)

^k WEILL CORNELL MEDICAL COLLEGE   (United States)

more..

Author keywords

Clinically meaningful effect; Psychopharmacology drug trials

Indexed keywords

4 AMINOPYRIDINE; CENTRAL NERVOUS SYSTEM AGENTS; DONEPEZIL; IMIPRAMINE; NEUROLEPTIC AGENT; PLACEBO; PSYCHOTROPIC AGENT; SEROTONIN UPTAKE INHIBITOR; THREO 3,4 DIHYDROXYPHENYLSERINE; ATYPICAL ANTIPSYCHOTIC AGENT; DULOXETINE; OLANZAPINE;

ALZHEIMER DISEASE; ARTICLE; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL DECISION MAKING; CLINICALLY MEANINGFUL EFFECT; COGNITIVE DEFECT; COMPARATIVE EFFECTIVENESS; COST CONTROL; COST EFFECTIVENESS ANALYSIS; COST UTILITY ANALYSIS; DEPRESSION; EFFECT SIZE; HEALTH CARE COST; HUMAN; MENTAL DISEASE; METABOLIC DISORDER; NUMBERS NEEDED TO TREAT; ORTHOSTATIC HYPOTENSION; OUTCOME ASSESSMENT; PERSONALIZED MEDICINE; PLACEBO EFFECT; POSITIVE AND NEGATIVE SYNDROME SCALE; PSYCHOPHARMACOLOGY; PSYCHOTHERAPY; QUALITY ADJUSTED LIFE YEAR; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REIMBURSEMENT; SAMPLE SIZE; SCHIZOPHRENIA; STATISTICAL SIGNIFICANCE; STUDY DESIGN; ATTENTION DEFICIT DISORDER; CLINICAL EFFECTIVENESS; CLINICAL RESEARCH; CLINICAL TRIAL (TOPIC); HAMILTON SCALE; HEALTH CARE SYSTEM; INVESTMENT; META ANALYSIS (TOPIC); RISK BENEFIT ANALYSIS; THERAPEUTIC MISCONCEPTION;

EID: 84886922537     PISSN: 21588333     EISSN: 21588341     Source Type: Journal    
DOI: None     Document Type: Article

References (28)

1. Kraemer HC, Kupfer DJ. Size of treatment effects and their importance to clinical research and practice. Biol Psychiatry. 2006;59(11):990-966.
2. Cohen J. Statistical Power Analysis for the Behavioral Sciences. Hillsdale, New Jersey: Lawrence Erlbaum Associates, Inc.; 1977.
3. Appelbaum PS, Roth LH, Lidz CW, et al. False hopes and best data: consent to research and the therapeutic misconception. Hastings Cent Rep. 1987;17(2):20-24.
4. Fisher R. The arrangement of field experiments. J Min Agric Gr Br. 1926;33:505-513.
5. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10(4):407-415.
6. Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res. 1993;2(3):221-226.
7. Jha AK, Aubert RE, Yao J, et al. Greater adherence to diabetes drugs is linked to less hospital use and could save nearly $5 billion annually. Health Aff (Millwood). 2012;31(8):1836-1846.
8. Sokol MC, McGuigan KA, Verbrugge RR, Epstein RS. Impact of medication adherence on hospitalization risk and healthcare cost. Med Care. 2005;43(6):521-530.
9. Zhu CW, Leibman C, Townsend R, et al. Bridging from clinical endpoints to estimates of treatment value for external decision makers. J Nutr Health Aging. 2009;13(3):256-259.
10. Hurley MV, Walsh NE, Mitchell H, et al. Long-term outcomes and costs of an integrated rehabilitation program for chronic knee pain: a pragmatic, cluster randomized, controlled trial. Arthritis Care Res (Hoboken). 2012;64(2):238-247.
11. Jonsson L, Gerth W, Fastbom J. The potential economic consequences of cognitive improvement with losartan. Blood Press. 2002;11(1):46-52.
12. Wolstenholme JL, Smith SJ, Whynes DK. The costs of treating breast cancer in the United Kingdom: implications for screening. Int J Technol Assess Health Care. 1998;14(2):277-289.
13. Lopez-Pousa S, Vilalta-Franch J, Garre-Olmo J, et al. Effectiveness of donepezil on several cognitive functions in patients with Alzheimer's disease over 12 months]. Neurologia. 2001 Oct;16(8):342-347.
14. Weinstein MC, Stason WB. Foundations of cost-effectiveness analysis for health and medical practices. N Engl J Med. 1977;296(13):716-721.
15. Baltussen R, Niessen L. Priority setting of health interventions: the need for multi-criteria decision analysis. Cost Eff Resour Alloc. 2006;4:14.
16. National Institute for Health and Clinical Excellence. Depression in adults. Manchester, England: National Institute for Health and Clinical Excellence; 2009 [cited 2013 2 March]; Guidance]. Available from: http://www.nice.org.uk/nicemedia/live/12329/45888/45888.pdf.
17. Leon AC. Comparative effectiveness clinical trials in psychiatry: superiority, noninferiority, and the role of active comparators. J Clin Psychiatry. 2011;72(10):1344-1349.
18. Leucht S, Arbter D, Engel RR, et al. How effective are second-generation antipsychotic drugs? A meta-analysis of placebo-controlled trials. Mol Psychiatry. 2009;14(4):429-447.
19. Meehl P. Theoretical risks and tabular asterisks: Sir Karl, Sir Ronald, and the slow progress of soft psychology. J Consult Clin Py. 1978;46:806-834.
20. Jones L, Tukey J. A sensible formulation of the significance test. Psychol Methods. 2000;5(4):411-414.
21. Baron RM, Kenny DA. The moderator-mediator variable distinction in social psychological research: conceptual, strategic, and statistical considerations. J Pers Soc Psychol. 1986;51(6):1173-1182.
22. National Institute of Mental Health. The Multimodal Treatment of Attention Deficit Hyperactivity Disorder Study (MTA): Questions and Answers. Rockville, MD: National Institute of Mental Health; 2009 [updated November 2009; cited 2013 3 March]; Available from: http://www.nimh.nih.gov/trials/practical/mta/the-multimodal-treatment-of-attention-deficit-hyperactivity-disorder-study-mta-questions-and-answers.shtml.
23. Frank E, Kupfer DJ, Perel JM, et al. Three-year outcomes for maintenance therapies in recurrent depression. Arch Gen Psychiatry. 1990;47(12):1093-1099.
24. Kraemer HC, Frank E, Kupfer DJ. How to assess the clinical impact of treatments on patients, rather than the statistical impact of treatments on measures. Int J Methods Psychiatr Res. 2011;20(2):63-72.
25. Frank E, Cassano GB, Rucci P, et al. Predictors and moderators of time to remission of major depression with interpersonal psychotherapy and SSRI pharmacotherapy. Psychol Med. 2011;41(1):151-162.
26. Kraemer HC, Frank E, Kupfer DJ. How to assess the clinical impact of treatments on patients, rather than the statistical impact of treatments on measures. Int J Methods Psychiatr Res.2011;20:63-72.
27. Frank E, Kupfer DJ, Rucci P, et al. Simultaneous evaluation of the harms and benefits of treatments in randomized clinical trials: demonstration of a new approach. Psychologic Med. 2012;42:865-873.
28. Wallace ML, Frank, E, Kraemer, HC. A novel approach for developing and interpreting treatment moderator profiles in randomized clinical trials. JAMA Psychiatry (in press).