OARSI Clinical Trials Recommendations: Key analytic considerations in design, analysis, and reporting of randomized controlled trials in osteoarthritis

Osteoarthritis and Cartilage

Volumn 23, Issue 5, 2015, Pages 677-685

  Losina, E ^a,b,c   Ranstam, J ^d   Collins, J E ^a,b   Schnitzer, T J ^e   Katz, J N ^a,b  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c BOSTON UNIVERSITY SCHOOL OF PUBLIC HEALTH   (United States)

^d LUND UNIVERSITY   (Sweden)

^e NORTHWESTERN UNIVERSITY   (United States)

Author keywords

Design; Economic evaluation; Missing data; Outcomes; Randomized controlled trials; Reporting

Indexed keywords

BEHAVIOR THERAPY; BODY WEIGHT MANAGEMENT; COMPARATIVE EFFECTIVENESS; DATA ANALYSIS; ECONOMIC EVALUATION; EXERCISE; FEASIBILITY STUDY; HEALTH CARE COST; HEALTH CARE UTILIZATION; HUMAN; INTERIM ANALYSIS; MATHEMATICAL COMPUTING; OSTEOARTHRITIS; OUTCOME ASSESSMENT; PATIENT SAFETY; PHYSIOTHERAPY; PRIORITY JOURNAL; PROCESS DEVELOPMENT; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SAMPLE SIZE; STRATIFIED SAMPLE; STUDY DESIGN; TOTAL KNEE REPLACEMENT; TREATMENT INDICATION; PRACTICE GUIDELINE; RESEARCH; STANDARDS; STATISTICS AND NUMERICAL DATA;

HUMANS; OSTEOARTHRITIS; PRACTICE GUIDELINES AS TOPIC; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH REPORT;

EID: 84933528472     PISSN: 10634584     EISSN: 15229653     Source Type: Journal    
DOI: 10.1016/j.joca.2015.03.011     Document Type: Review

References (61)

1. Beavers K.M., Beavers D.P., Newman J.J., Anderson A.M., Loeser R.F., Nicklas B.J., et al. Effects of total and regional fat loss on plasma CRP and IL-6 in overweight and obese, older adults with knee osteoarthritis. Osteoarthritis and Cartilage 2015 Feb, 23(2):249-256.
2. FDA Document: Guidelines for Industry Statistical Principles for Clinical Trial E9. International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use 16 September 1998.
3. Miller F., Bjornsson M., Svensson O., Karlsten R. Experiences with an adaptive design for a dose-finding study in patients with osteoarthritis. Contemp Clin Trials 2014 Mar, 37(2):189-199.
4. Bland J.M., Altman D.G. Some examples of regression towards the mean. BMJ 1994 Sep 24, 309(6957):780.
5. Dobson F., Bennell K., Hinman R., Roos E., Abbott H., Stratford P., et al. OARSI recommended performance-based tests to assess physical function in osteoarthritis of the hip or knee: authors' reply. Osteoarthritis and Cartilage 2013 Oct, 21(10):1625-1626.
6. Bellamy N., Buchanan W.W., Goldsmith C.H., Campbell J., Stitt L.W. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. JRheumatol 1988 Dec, 15(12):1833-1840.
7. Hinchcliff M., Beaumont J.L., Thavarajah K., Varga J., Chung A., Podlusky S., et al. Validity of two new patient-reported outcome measures in systemic sclerosis: patient-reported outcomes measurement information system 29-item health profile and functional assessment of chronic illness therapy-dyspnea short form. Arthritis Care Res (Hoboken) 2011 Nov, 63(11):1620-1628.
8. Roos E.M., Lohmander L.S. The Knee injury and Osteoarthritis Outcome Score (KOOS): from joint injury to osteoarthritis. Health Qual Life Outcomes 2003, 1:64.
9. Jaeschke R., Singer J., Guyatt G.H. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989 Dec, 10(4):407-415.
10. Wells G., Beaton D., Shea B., Boers M., Simon L., Strand V., et al. Minimal clinically important differences: review of methods. JRheumatol 2001 Feb, 28(2):406-412.
11. Meinert C.L. Beyond CONSORT: need for improved reporting standards for clinical trials. Consolidated Standards of Reporting Trials. JAMA 1998 May 13, 279(18):1487-1489.
12. Moher D., Schulz K.F., Altman D.G. The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials. Ann Intern Med 2001 Apr 17, 134(8):657-662.
13. Altman D.G., Schulz K.F., Moher D., Egger M., Davidoff F., Elbourne D., et al. The revised CONSORT statement for reporting randomized trials: explanation and elaboration. Ann Intern Med 2001 Apr 17, 134(8):663-694.
14. Bland J.M., Altman D.G. Multiple significance tests: the Bonferroni method. BMJ 1995 Jan 21, 310(6973):170.
15. Holm S. Asimple sequential rejective multiple test procedure. Scand J Statist 1979, 6:65-70.
16. Brookes S.T., Whitely E., Egger M., Smith G.D., Mulheran P.A., Peters T.J. Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test. JClin Epidemiol 2004 Mar, 57(3):229-236.
17. Lagakos S.W. The challenge of subgroup analyses-reporting without distorting. NEngl J Med 2006 Apr 20, 354(16):1667-1669.
18. Scharfstein D.O., Hogan J., Herman A. On the prevention and analysis of missing data in randomized clinical trials: the state of the art. JBone Joint Surg Am 2012 Jul 18, 94(Suppl 1):80-84.
19. Little R.J., D'Agostino R., Cohen M.L., Dickersin K., Emerson S.S., Farrar J.T., et al. The prevention and treatment of missing data in clinical trials. NEngl J Med 2012 Oct 4, 367(14):1355-1360.
20. He P., Lai T., Liao O. Futility stopping in clinical trials. Stat Interface 2012, 5:415-423.
21. Committee for Medicinal Products for Human Use Reflection Paper on Methodological Issues in Confirmatory Clinical Trials Planned with an Adaptive Design October 2007, EMEA, London.
22. Broberg P. Sample size re-assessment leading to a raised sample size does not inflate type I error rate under mild conditions. BMC Med Res Methodol 2013, 13:94.
23. Chen Y.H., DeMets D.L., Lan K.K. Increasing the sample size when the unblinded interim result is promising. Stat Med 2004 Apr 15, 23(7):1023-1038.
24. Kieser M., Friede T. Simple procedures for blinded sample size adjustment that do not affect the type I error rate. Stat Med 2003 Dec 15, 22(23):3571-3581.
25. Lesaffre E. Superiority, equivalence, and non-inferiority trials. Bull NYU Hosp Joint Dis 2008, 66(2):150-154.
26. Evans S. When and how can endpoints be changed after initiation of a randomized clinical trial?. PLoS Clin Trials 2007, 2(4):e18.
27. Boutron I., Moher D., Tugwell P., Giraudeau B., Poiraudeau S., Nizard R., et al. Achecklist to evaluate a report of a nonpharmacological trial (CLEAR NPT) was developed using consensus. JClin Epidemiol 2005 Dec, 58(12):1233-1240.
28. Bromhead H.J., Goodman N.W. CONSORT statement on the reporting standards of clinical trials. Reporting of refusal of consent to take part in clinical trials is still poor. BMJ 1997 Apr 12, 314(7087):1126-1127.
29. Freemantle N., Mason J.M., Haines A., Eccles M.P. CONSORT: an important step toward evidence-based health care. Consolidated Standards of Reporting Trials. Ann Intern Med 1997 Jan 1, 126(1):81-83.
30. Junker C., Egger M., Schneider M., Zellweger T., Antes G. The CONSORT statement. JAMA 1996 Dec 18, 276(23):1876-1877. author reply 7.
31. Meade T.W., Wald N., Collins R. CONSORT statement on the reporting standards in clinical trials. Recommendations are inappropriate for trial reports. BMJ 1997 Apr 12, 314(7087):1126. author reply 7.
32. Moher D., Schulz K.F., Altman D. The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials. JAMA 2001 Apr 18, 285(15):1987-1991.
33. O'Toole L.B. Consort statement on the reporting standards of clinical trials. MRC uses checklist similar to CONSORT's. BMJ 1997 Apr 12, 314(7087):1127.
34. Ross S.D. The CONSORT statement. JAMA 1996 Dec 18, 276(23):1877. author reply.
35. Schulz K.F. The quest for unbiased research: randomized clinical trials and the CONSORT reporting guidelines. Ann Neurol 1997 May, 41(5):569-573.
36. World Medical Association Declaration of Helsinki - Ethical Principles for Medical Research Involving Human Subjects - adopted October, 2013. In: last accessed on 04Jun2014. Ferney-Voltaire, France: 'World Medical Association'. http://www.wma.net/en/30publications/10policies/b3/.
37. Food and Drug Administration. Amendments Act of 2007. Public Law No 110-85, 2007.
38. U.S. Food and Drug Administration. Food and Drug Administration Modernization Act (FDAMA) of 1997 [cited 2015 March 20]. Available from: . http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDAMA/.
39. International Committee of Medical Journal Editors. Recommendations for the Conduct, Reporting, Editing, and Publication of Scholarly Work in Medical Journals. 2014. Available from: . http://www.icmje.org/icmje-recommendations.pdf.
40. Ghersi D., Clarke M., Berlin J., Gulmezoglu A., Kush R., Lumbiganon P., et al. Reporting the findings of clinical trials: a discussion paper. Bull World Health Organ 2008 Jun, 86(6):492-493.
41. Tse T., Williams R.J., Zarin D.A. Reporting "basic results" in. Chest 2009 Jul, 136(1):295-303. http://ClinicalTrials.gov.
42. Schulz K.F., Altman D.G., Moher D. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. Int J Surg 2011, 9(8):672-677.
43. Moher D., Hopewell S., Schulz K.F., Montori V., Gotzsche P.C., Devereaux P.J., et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. Int J Surg 2012, 10(1):28-55.
44. CONSORT Consolidated Standards of Reporting Trials. [cited 2015 March 20]. Available from: . http://www.consortstatement.org/.
45. The International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH). ICH Guidelines [cited 2015 March 20]. Available from: . http://www.ich.org/products/guidelines.html.
46. Gillespie W.J., Pekarsky B., O'Connell D.L. Evaluation of new technologies for total hip replacement. Economic modelling and clinical trials. JBone Joint Surg Br 1995 Jul, 77(4):528-533.
47. Jonsson B., Weinstein M.C. Economic evaluation alongside multinational clinical trials. Study considerations for GUSTO IIb. Int J Technol Assess Health Care 1997, 13(1):49-58. Winter.
48. Morris S. Acomparison of economic modelling and clinical trials in the economic evaluation of cholesterol-modifying pharmacotherapy. Health Econ 1997 Nov-Dec, 6(6):589-601.
49. Siegel JE, Weinstein MC, Russell LB, Gold MR. Recommendations for reporting cost-effectiveness analyses. Panel on costeffectiveness in health and Medicine. JAMA 1996 Oct 23e30;276(16):1339e41.
50. Weinstein MC, Siegel JE, Gold MR, Kamlet MS, Russell LB. Recommendations of the panel on cost-effectiveness in health and medicine. JAMA 1996 Oct 16;276(15):1253e8.
51. Edwards RT, Hounsome B, Linck P, Russell IT. Economic evaluation alongside pragmatic randomised trials: developing a standard operating procedure for clinical trials units. Trials 2008;9:64.
52. Haycox A, Drummond M, Walley T. Pharmacoeconomics: integrating economic evaluation into clinical trials. Br J Clin Pharmacol 1997 Jun;43(6):559e62.
53. Tsevat J, Weeks JC, Guadagnoli E, Tosteson AN, Mangione CM, Pliskin JS, et al. Using health-related quality-of-life information: clinical encounters, clinical trials, and health policy. J Gen Intern Med 1994 Oct;9(10):576e82.
54. Weinstein MC. A QALY is a QALYeor is it? J Health Econ 1988 Sep;7(3):289e90.
55. Torrance GW. Measurement of health state utilities for economic appraisal. J Health Econ 1986 Mar;5(1):1e30.
56. Brazier J, Jones N, Kind P. Testing the validity of the Euroqol and comparing it with the SF-36 health survey questionnaire. Qual Life Res 1993 Jun;2(3):169e80.
57. Brazier JE, Walters SJ, Nicholl JP, Kohler B. Using the SF-36 and Euroqol on an elderly population. Qual Life Res 1996 Apr;5(2): 195e204.
58. Selai C, Rosser R. Eliciting EuroQol descriptive data and utility scale values from inpatients. A feasibility study. Pharmacoeconomics 1995 Aug;8(2):147e58.
59. Wolfe F, Hawley DJ. Measurement of the quality of life in rheumatic disorders using the EuroQol. Br J Rheumatol 1997 Jul;36(7):786e93.
60. Dolan P. Modeling valuations for EuroQol health states. Med Care 1997 Nov;35(11):1095e108.
61. Claxton KP, Sculpher MJ. Using value of information analysis to prioritise health research: some lessons from recent UK experience. Pharmacoeconomics 2006;24(11):1055e68.