Developing core outcome sets for clinical trials: issues to consider

Trials

Peter Tugwell¹  Elizabeth Gargon³  Declan Devane⁴  Mike Clarke⁶  Jane M Blazeby⁵  Douglas G Altman²  Paula R Williamson³ 

[1] University of Ottawa, Institute of Population Health, 1 Stewart Street, Ottawa, ON, K1N 6N5, Canada;University of Oxford, Centre for Statistics in Medicine, Wolfson College Annexe, Linton Road, Oxford, OX2 6UD, UK;Department of Biostatistics, University of Liverpool, Shelley’s Cottage, Brownlow Street, Liverpool,, L69 3GS, UK;National University of Ireland Galway, School of Nursing and Midwifery, University Road, Galway, Ireland;University of Bristol, School of Social and Community Medicine, 39 Whatley Road, Bristol, BS8 2PS, UK;Queens University Belfast, All-Ireland Hub for Trials Methodology Research, Institute of Clinical Sciences, Block B, Royal Victoria Hospital, Grosvenor Road, Belfast, BT12 6BJ, UK

关键词: Consensus;    Methodology;    Systematic review;    Clinical trials;    Outcome reporting bias;    Core outcome set;   

Others  :  1095420
DOI  :  10.1186/1745-6215-13-132

 received in 2012-03-13, accepted in 2012-07-13,  发布年份 2012

【 摘 要 】

The selection of appropriate outcomes or domains is crucial when designing clinical trials in order to compare directly the effects of different interventions in ways that minimize bias. If the findings are to influence policy and practice then the chosen outcomes need to be relevant and important to key stakeholders including patients and the public, health care professionals and others making decisions about health care. There is a growing recognition that insufficient attention has been paid to the outcomes measured in clinical trials. These issues could be addressed through the development and use of an agreed standardized collection of outcomes, known as a core outcome set, which should be measured and reported, as a minimum, in all trials for a specific clinical area. Accumulating work in this area has identified the need for general guidance on the development of core outcome sets. Key issues to consider in the development of a core outcome set include its scope, the stakeholder groups to involve, choice of consensus method and the achievement of a consensus.

【 授权许可】

   
2012 Williamson et al.; licensee BioMed Central Ltd.

【 参考文献 】

• [1]Tovey D: Impact of Cochrane Reviews [editorial]. 2010. http://www.thecochranelibrary.com/details/editorial/756937/The-Impact-of-Cochrane-Reviews-by-Dr-David-Tovey.html webcite
• [2]Dwan K, Altman DG, Arnaiz JA, Bloom J, Chan AW, Cronin E, Decullier E, Easterbrook PJ, von Elm E, Gamble C, Ghersi D, Ioannidis JP, Simes J, Williamson PR: Systematic review of the empirical evidence of study publication bias and outcome reporting bias. PLoS One 2008, 3:e3081.
• [3]Kirkham JJ, Dwan KM, Altman DG, Gamble C, Dodd S, Smyth R, Williamson PR: The impact of outcome reporting bias in randomised controlled trials on a cohort of systematic reviews. BMJ 2010, 340:c365.
• [4]Clarke M: Standardising outcomes for clinical trials and systematic reviews. Trials 2007, 8:39. BioMed Central Full Text
• [5]Rosenbaum SE, Glenton C, Nylund HK, Oxman AD: User testing and stakeholder feedback contributed to the development of understandable and useful Summary of Findings tables for Cochrane reviews. J Clin Epidemiol 2010, 63:607-619.
• [6]Rosenbaum SE, Glenton C, Oxman AD: Summary-of-findings tables in Cochrane reviews improved understanding and rapid retrieval of key information. J Clin Epidemiol 2010, 63:620-626.
• [7]WHO: WHO Handbook for Guideline Development. http://wwwwhoint/hiv/topics/mtct/grc_handbook_mar2010_1pdf webcite
• [8]Serrano-Aguilar P, Trujillo-Martin MM, Ramos-Goni JM, Mahtani-Chugani V, Perestelo-Perez L, Posada-de la Paz M: Patient involvement in health research: a contribution to a systematic review on the effectiveness of treatments for degenerative ataxias. Soc Sci Med 2009, 69:920-925.
• [9]Mease PJ, Arnold LM, Crofford LJ, Williams DA, Russell IJ, Humphrey L, Abetz L, Martin SA: Identifying the clinical domains of fibromyalgia: contributions from clinician and patient Delphi exercises. Arthritis Rheum 2008, 59:952-960.
• [10]Sinha I, Gallagher R, Williamson PR, Smyth RL: Development of a core outcome set for clinical trials in childhood asthma-a survey of clinicians, parents and young people. Trials 2012, 13:103. BioMed Central Full Text
• [11]Kirwan JR, Hewlett SE, Heiberg T, Hughes RA, Carr M, Hehir M, Kvien TK, Minnock P, Newman SP, Quest EM, Taal E, Wale J: Incorporating the patient perspective into outcome assessment in rheumatoid arthritis-progress at OMERACT 7. J Rheumatol 2005, 32:2250-2256.
• [12]Oliver S, Gray J: A bibliography of research reports about patients’, clinicians’ and researchers’ priorities for new research. James Lind Alliance, London; 2006.
• [13]Tugwell P, Boers M, Brooks P, Simon L, Strand V, Idzerda L: OMERACT: an international initiative to improve outcome measurement in rheumatology. Trials 2007, 8:38. BioMed Central Full Text
• [14]Turk DC, Dworkin RH, Allen RR, Bellamy N, Brandenburg N, Carr DB, Cleeland C, Dionne R, Farrar JT, Galer BS, Hewitt DJ, Jadad AR, Katz NP, Kramer LD, Manning DC, McCormick CG, McDermott MP, McGrath P, Quessy S, Rappaport BA, Robinson JP, Royal MA, Simon L, Stauffer JW, Stein W, Tollett J, Witter J: Core outcome domains for chronic pain clinical trials: IMMPACT recommendations. Pain 2003, 106:337-345.
• [15]Devane D, Begley CM, Clarke M, Horey D, Oboyle C: Evaluating maternity care: a core set of outcome measures. Birth 2007, 34:164-172.
• [16]Sinha I, Jones L, Smyth RL, Williamson PR: A systematic review of studies that aim to determine which outcomes to measure in clinical trials in children. PLoS Med 2008, 5:e96.
• [17]The COMET Initiative. http://www.comet-initiative.org webcite
• [18]Potter S, Brigic A, Whiting PF, Cawthorn SJ, Avery KN, Donovan JL, Blazeby JM: Reporting clinical outcomes of breast reconstruction: a systematic review. J Natl Canc Inst 2011, 103:31-46.
• [19]Potter S, Harcourt D, Cawthorn S, Warr R, Mills N, Havercroft D, Blazeby J: Assessment of cosmesis after breast reconstruction surgery: a systematic review. Ann Surg Oncol 2011, 18:813-823.
• [20]Sinha IP, Williamson PR, Smyth RL: Outcomes in clinical trials of inhaled corticosteroids for children with asthma are narrowly focussed on short term disease activity. PLoS One 2009, 4:e6276.
• [21]Cowan K, Oliver S: The James Lind Alliance Guidebook. 4th edition. James Lind Alliance, Oxford; 2012.
• [22]Schmitt J, Langan S, Stamm T, Williams HC: Harmonizing Outcome Measurements in Eczema Delphi panel. Core outcome domains for controlled trials and clinical recordkeeping in eczema: international multiperspective Delphi consensus process. J Invest Dermatol 2011, 131:623-630.
• [23]Arnold LM, Crofford LJ, Mease PJ, Burgess SM, Palmer SC, Abetz L, Martin SA: Patient perspectives on the impact of fibromyalgia. Patient Educ Counsel 2008, 73:114-120.
• [24]Sanderson T, Morris M, Calnan M, Richards P, Hewlett S: What outcomes from pharmacologic treatments are important to people with rheumatoid arthritis? Creating the basis of a patient core set. Arthritis Care Res Hoboken 2010, 62:640-646.
• [25]Kirwan J, Heiberg T, Hewlett S, Hughes R, Kvien T, Ahlmèn M, Boers M, Minnock P, Saag K, Shea B, Suarez Almazor M, Taal E: Outcomes from the Patient Perspective Workshop at OMERACT 6. J Rheumatol 2003, 30:868-872.
• [26]Staley K: Exploring Impact: Public involvement in NHS, public health and social care research. INVOLVE, Eastleigh; 2009.
• [27]Mokkink LB, Terwee CB, Patrick DL, Alonso J, Stratford PW, Knol D, Bouter LM, de Vet HC: The COSMIN checklist for assessing the methodological quality of studies on measurement properties of health status measurement instruments: an international Delphi study. Qual Life Res 2010, 19:539-549.
• [28]Reddel HK, Taylor DR, Bateman ED, Boulet L-P, Boushey HA, Busse WW, Casale TB, Chanez P, Enright PL, Gibson PG, de Jongste JC, Kerstjens HAM, Lazarus SC, Levy ML, O’Byrne PM, Partridge MR, Pavord ID, Sears MR, Sterk PJ, Stoloff SW, Sullivan SD, Szefler SJ, Thomas MD, Wenzel SE: American Thoracic Society/European Respiratory Society Task Force on Asthma Control and Exacerbations: An official American Thoracic Society/European Respiratory Society statement: asthma control and exacerbations: standardizing endpoints for clinical asthma trials and clinical practice. Am J Respir Crit Care Med 2009, 180:59-99.
• [29]Sinha IP, Smyth RL, Williamson PR: Using the delphi technique to determine which outcomes to measure in clinical trials: recommendations for the future based on a systematic review of existing studies. PLoS Med 2011, 8:e1000393.
• [30]The CONSORT Statement. http://www.consort-statement.org/consort-statement/ webcite
• [31]The AGREE Collaboration: Appraisal of Guidelines for Research & Evaluation (AGREE) Instrument. www.agreecollaborationorg webcite
• [32]Kirwan JR, Hewlett S: Patient perspective: reasons and methods for measuring fatigue in rheumatoid arthritis. J Rheumatol 2007, 34:1171-1173.