Statistical methods for cost-effectiveness analyses that use data from cluster randomized trials: A systematic review and checklist for critical appraisal

Medical Decision Making

Volumn 32, Issue 1, 2012, Pages 209-220

  Gomes, Manuel ^a   Grieve, Richard ^a   Nixon, Richard ^b   Edmunds, W J ^a  

^a LONDON SCHOOL OF HYGIENE AND TROPICAL MEDICINE   (United Kingdom)

^b NOVARTIS PHARMA AG   (Switzerland)

Author keywords

cluster analysis; cost effectiveness analysis; hierarchical models; statistical methods; systematic reviews

Indexed keywords

CHECKLIST; CLUSTER ANALYSIS; COST BENEFIT ANALYSIS; METHODOLOGY; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; STATISTICAL MODEL; STATISTICS;

CHECKLIST; CLUSTER ANALYSIS; COST-BENEFIT ANALYSIS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 84856599237     PISSN: 0272989X     EISSN: 1552681X     Source Type: Journal    
DOI: 10.1177/0272989X11407341     Document Type: Conference Paper

References (87)

1. Methods for Technology Appraisal. London, UK: NICE ; 2008 :
2. Guidelines for the Economic Evaluation of Health Technologies: Canada. 3 rd ed.Ottawa, Canada: CADTH; 2006. Ottawa, Canada: CADTH ; 2006 :
3. Guidelines for Preparing Submissions to the Pharmaceutical Benefits Advisory Committee. Canberra, Australia: Australian Government-Department of Health and Ageing ; 2008 :
4. Methods for Assessment of the Relation of Benefits to Costs in the German Statutory Health Care System. Cologne, Germany: IQWIG ; 2009 :
5. Methods for Development of NICE Public Health Guidance. London, UK: NICE ; 2009 :
6. Grieve R, Sekhon JS, Hu TW, Bloom JR. Evaluating health care programs by combining cost with quality of life measures: a case study comparing capitation and fee for service. Health Serv Res. 2008 ; 43 (4). 1204-22 (Pubitemid 352019148)
7. Hutchings A, Durand MA, Grieve R, et al. Evaluation of modernisation of adult critical care services in England: time series and cost effectiveness analysis. BMJ. 2009 ; 339: b4353
8. Drummond M, Sculpher M, Torrance GW, O'Brien BJ, Stoddart GL Methods for the Economic Evaluation of Health Care Programmes. 3 rd ed.Oxford, UK: Oxford University Press; 2005. Oxford, UK: Oxford University Press ; 2005 :
9. Glick HA, Doshi JA, Sonnad SS, Polsky D Economic Evaluation in Clinical Trials. Oxford, UK: Oxford University Press ; 2007 :
10. Gold MR Cost-Effectiveness in Health and Medicine. New York: Oxford University Press ; 1996 :
11. Willan A, Briggs A Statistical Analysis of Cost-Effectiveness Data. Chichester, UK: John Wiley ; 2006 :
12. Donner A, Klar N Design and Analysis of Cluster Randomization Trials in Health Research. London, UK: Hodder Arnold Publishers ; 2000 :
13. Donner A, Klar N. Pitfalls of and controversies in cluster randomization trials. Am J Public Health. 2004 ; 94 (3). 416-22 (Pubitemid 38269494)
14. Hayes R, Moulton L Cluster Randomised Trials. Keiding N Morgan B Wikle C van der Heijden P, ed. Boca Raton, FL: CRC Press, Taylor & Francis ; 2009 :
15. Klar N, Donner A. Current and future challenges in the design and analysis of cluster randomization trials. Stat Med. 2001 ; 20 (24). 3729-40
16. Murray DM, Varnell SP, Blitstein JL. Design and analysis of group-randomized trials: a review of recent methodological developments. Am J Public Health. 2004 ; 94 (3). 423-32 (Pubitemid 38269495)
17. Ukoumunne OC, Gulliford MC, Chinn S, Sterne JA, Burney PG. Methods for evaluating area-wide and organisation-based interventions in health and health care: a systematic review. Health Technol Assess. 1999 ; 3 (5). iii - 92
18. Campbell MJ, Donner A, Klar N. Developments in cluster randomized trials and Statistics in Medicine. Stat Med. 2007 ; 26 (1). 2-19 (Pubitemid 46099414)
19. Omar RZ, Thompson SG. Analysis of a cluster randomized trial with binary outcome data using a multi-level model. Stat Med. 2000 ; 19 (19). 2675-88
20. Spiegelhalter DJ. Bayesian methods for cluster randomized trials with continuous responses. Stat Med. 2001 ; 20 (3). 435-52 (Pubitemid 32125132)
21. Turner RM, Omar RZ, Thompson SG. Bayesian methods of analysis for cluster randomized trials with binary outcome data. Stat Med. 2001 ; 20 (3). 453-72 (Pubitemid 32125133)
22. Briggs AH, O'Brien BJ. The death of cost-minimization analysis?. Health Econ. 2001 ; 10 (2). 179-84
23. Hoch JS, Briggs AH, Willan AR. Something old, something new, something borrowed, something blue: a framework for the marriage of health econometrics and cost-effectiveness analysis. Health Econ. 2002 ; 11 (5). 415-30 (Pubitemid 34752613)
24. Nixon RM, Thompson SG. Methods for incorporating covariate adjustment, subgroup analysis and between-centre differences into cost-effectiveness evaluations. Health Econ. 2005 ; 14 (12). 1217-29 (Pubitemid 43013604)
25. Willan AR, Briggs AH, Hoch JS. Regression methods for covariate adjustment and subgroup analysis for non-censored cost-effectiveness data. Health Econ. 2004 ; 13 (5). 461-75 (Pubitemid 38660395)
26. Mihaylova B, Briggs A, O'Hagan A, Thompson SG. Review of statistical methods for analysing healthcare resources and costs. Health Econ. 2010 ; 27 (15). 101-9
27. Nixon RM, Thompson SG. Parametric modelling of cost data in medical studies. Stat Med. 2004 ; 23 (8). 1311-31 (Pubitemid 38514826)
28. Thompson SG, Nixon RM. How sensitive are cost-effectiveness analyses to choice of parametric distributions?. Med Decis Making. 2005 ; 25 (4). 416-23 (Pubitemid 41023168)
29. Bachmann MO, Fairall L, Clark A, Mugford M. Methods for analyzing cost effectiveness data from cluster randomized trials. Cost Eff Resour Alloc. 2007 ; 5: 12
30. Flynn TN, Peters TJ. Cluster randomized trials: another problem for cost-effectiveness ratios. Int J Technol Assess Health Care. 2005 ; 21 (3). 403-9 (Pubitemid 41099223)
31. Grieve R, Nixon R, Thompson SG. Bayesian hierarchical models for cost-effectiveness analyses that use data fromcluster randomized trials. Med Decis Making. 2010 ; 30 (2). 163-75
32. Philips Z, Bojke L, Sculpher M, Claxton K, Golder S. Good practice guidelines for decision-analytic modelling in health technology assessment: a review and consolidation of quality assessment. Pharmacoeconomics. 2006 ; 24 (4). 355-71
33. Evers S, Goossens M, de Vet H, van Tulder M, Ament A. Criteria list for assessment of methodological quality of economic evaluations: Consensus on Health Economic Criteria. Int J Technol Assess Health Care. 2005 ; 21 (2). 240-5 (Pubitemid 40656554)
34. Graves N, Walker D, Raine R, Hutchings A, Roberts JA. Cost data for individual patients included in clinical studies: no amount of statistical analysis can compensate for inadequate costing methods. Health Econ. 2002 ; 11 (8). 735-9 (Pubitemid 36018689)
35. Hjelmgren J, Berggren F, Andersson F. Health economic guidelines similarities, differences and some implications. Value Health. 2001 ; 4 (3). 225-50 (Pubitemid 32661267)
36. Ofman JJ, Sullivan SD, Neumann PJ, et al. Examining the value and quality of health economic analyses: implications of utilizing the QHES. J Manag Care Pharm. 2003 ; 9 (1). 53-61
37. Ramsey S, Willke R, Briggs A, et al. Good research practicesfor cost-effectiveness analysis alongside clinical trials: the ISPOR RCT-CEA Task Force report. Value Health. 2005 ; 8 (5). 521-33 (Pubitemid 41727304)
38. Willan A. Statistical analysis of cost-effectiveness data from randomised clinical trials. Expert Rev Pharmacoecon Outcomes Res. 2006 ; 6: 337-46 (Pubitemid 43984125)
39. Moerbeek M, van Breukelen GJ, Berger MP. Design issues for experiments in multilevel populations. J Educ Behav Stat. 2000 ; 25 (3). 271-84
40. Donner A. Some aspects of the design and analysis of cluster randomization trials. Appl Stat. 1998 ; 47: 95-113 (Pubitemid 128366703)
41. Hayes RJ, Bennett S. Simple sample size calculation for cluster-randomized trials. Int J Epidemiol. 1999 ; 28 (2). 319-26 (Pubitemid 29176663)
42. Feng ZD, McLerran D, Grizzle J. A comparison of statistical methods for clustered data analysis with Gaussian error. Stat Med. 1996 ; 15 (16). 1793-806 (Pubitemid 26280309)
43. Ukoumunne OC, Thompson SG. Analysis of cluster randomized trials with repeated cross-sectional binary measurements. Stat Med. 2001 ; 20 (3). 417-33 (Pubitemid 32125131)
44. Panageas KS, Schrag D, Russell Localio A, Venkatraman ES, Begg CB. Properties of analysis methods that account for clustering in volume-outcome studies when the primary predictor is cluster size. Stat Med. 2007 ; 26 (9). 2017-35 (Pubitemid 46534090)
45. Al MJ, van Hout BA, Michel BC, Rutten FF. Sample size calculation in economic evaluations. Health Econ. 1998 ; 7 (4). 327-35 (Pubitemid 28339961)
46. Briggs A. Economic evaluation and clinical trials: size matters. BMJ. 2000 ; 321 (7273). 1362-3
47. Drummond M, O'Brien B. Clinical importance, statistical significance and the assessment of economic and quality-of-life outcomes. Health Econ. 1993 ; 2 (3). 205-12
48. O'Sullivan AK, Thompson D, Drummond MF. Collection of health-economic data alongside clinical trials: is there a future for piggyback evaluations?. Value Health. 2005 ; 8 (1). 67-79 (Pubitemid 40293643)
49. Bland JM. The tyranny of power: is there a better way to calculate sample size?. BMJ. 2009 ; 339: b3985
50. Campbell MK, Fayers PM, Grimshaw JM. Determinants of the intracluster correlation coefficient in cluster randomized trials: the case of implementation research. Clin Trials. 2005 ; 2 (2). 99-107 (Pubitemid 40637892)
51. Grieve R, Nixon R, Thompson SG, Normand C. Using multilevel models for assessing the variability of multinational resource use and cost data. Health Econ. 2005 ; 14 (2). 185-96 (Pubitemid 40253633)
52. Thompson SG, Nixon RM, Grieve R. Addressing the issues that arise in analysing multicentre cost data, with application to a multinational study. J Health Econ. 2006 ; 25 (6). 1015-28 (Pubitemid 44572505)
53. O'Hagan A, Stevens JW, Montmartin J. Bayesian cost-effectiveness analysis from clinical trial data. Stat Med. 2001 ; 20 (5). 733-53 (Pubitemid 32177846)
54. Briggs A, Nixon R, Dixon S, Thompson S. Parametric modelling of cost data: some simulation evidence. Health Econ. 2005 ; 14 (4). 421-8 (Pubitemid 40514754)
55. Manning WG, Basu A, Mullahy J. Generalized modeling approaches to risk adjustment of skewed outcomes data. J Health Econ. 2005 ; 24 (3). 465-88 (Pubitemid 40461909)
56. Manning WG, Mullahy J. Estimating log models: to transform or not to transform?. J Health Econ. 2001 ; 20 (4). 461-94 (Pubitemid 32515166)
57. Grieve R, Nixon R, Thompson SG, Cairns J. Multilevel models for estimating incremental net benefits in multinational studies. Health Econ. 2007 ; 16 (8). 815-26 (Pubitemid 47215132)
58. Morrell CJ, Warner R, Slade P, et al. Psychological interventions for postnatal depression: cluster randomised trial and economic evaluation. The PoNDER trial. Health Technol Assess. 2009 ; 13 (30). 1-153
59. Goldstein H Multilevel Statistical Models. 3 rd ed.Oxford, UK: Oxford University Press; 2003. Oxford, UK: Oxford University Press ; 2003 :
60. Leyland A, Goldstein H Multilevel Modelling of Health Statistics. Chichester, UK: John Wiley ; 2001 :
61. Hardin JW, Hilbe JM Generalized Estimating Equations. Boca Raton, FL: Chapman & Hall/CRC ; 2003 :
62. Davison AC, Hinkley DV Bootstrap Methods and Their Application. Gill R Ripley BD Ross S Stein M Williams D, ed. Cambridge, UK: Cambridge University Press; ; 1997 :
63. Lipsitz S, Fitzmaurice G, Ibrahim J, Sinha D, Parzen M, Lipshultz S. Joint generalized estimating equations for multivariate longitudinal binary outcomes with missing data: an application to acquired immune deficiency syndrome data. J Roy Stat Soc A. 2009 ; 172 (pt 1). 3-20
64. Liang KY, Zeger S. Longitudinal data analysis using generalized linear models. Biometrika. 1986 ; 73 (1). 13-22
65. Austin PC. A comparison of the statistical power of different methods for the analysis of cluster randomization trials with binary outcomes. Stat Med. 2007 ; 26 (19). 3550-65 (Pubitemid 47180697)
66. Turner RM, White IR, Croudace T. Analysis of cluster randomized cross-over trial data: a comparison of methods. Stat Med. 2007 ; 26 (2). 274-89 (Pubitemid 46099400)
67. Lee AY, Nelder J. Conditional and marginal models: another view. Stat Sci. 2004 ; 19 (2). 219-38 (Pubitemid 40300430)
68. Briggs AH, Mooney CZ, Wonderling DE. Constructing confidence intervals for cost-effectiveness ratios: an evaluation of parametric and non-parametric techniques using Monte Carlo simulation. Stat Med. 1999 ; 18 (23). 3245-62
69. Nixon RM, Wonderling D, Grieve RD. Non-parametric methods for cost-effectiveness analysis: the central limit theorem and the bootstrap compared. Health Econ. 2010 ; 19 (3). 316-33
70. O'Hagan A, Stevens JW. Assessing and comparing costs: how robust are the bootstrap and methods based on asymptotic normality?. Health Econ. 2003 ; 12 (1). 33-49 (Pubitemid 36149858)
71. Flynn TN, Peters TJ. Use of the bootstrap in analysing cost data from cluster randomised trials: some simulation results. BMC Health Serv Res. 2004 ; 4: 33-43
72. Moher D, Liberati A, Tetzlaff J, Altman DG. Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement. BMJ. 2009 ; 339: b2535
73. Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. Health Econ. 1999 ; 8 (3). 257-61 (Pubitemid 29219090)
74. Philips Z, Ginnelly L, Sculpher M, et al. Review of guidelines for good practice in decision-analytic modelling in health technology assessment. Health Technol Assess. 2004 ; 8 (36). 1-158
75. Sculpher MJ, Pang FS, Manca A, et al. Generalisability in economic evaluation studies in healthcare: a review and case studies. Health Technol Assess. 2004 ; 8 (49). 1-192
76. Rivero-Arias O, Gray A. The multinational nature of cost-effectiveness analyses alongside multinational clinical trials. Value Health. 2010 ; 13 (1). 34-41
77. Turner RM, Omar RZ, Thompson SG. Modelling multivariate outcomes in hierarchical data, with application to cluster randomised trials. Biom J. 2006 ; 48 (3). 333-45 (Pubitemid 43973039)
78. Briggs AH, Gray AM. Handling uncertainty when performing economic evaluation of healthcare interventions. Health Technol Assess. 1999 ; 3 (2). 1-134
79. Eldridge SM, Ashby D, Feder GS, Rudnicka AR, Ukoumunne OC. Lessons for cluster randomized trials in the twenty-first century: a systematic review of trials in primary care. Clin Trials. 2004 ; 1 (1). 80-90
80. Manca A, Palmer S. Handling missing data in patient-level cost-effectiveness analysis alongside randomised clinical trials. Appl Health Econ Health Policy. 2005 ; 4 (2). 65-75 (Pubitemid 41324092)
81. Willan AR, Lin DY, Manca A. Regression methods for cost-effectiveness analysis with censored data. Stat Med. 2005 ; 24 (1). 131-45 (Pubitemid 40074070)
82. Donner A, Klar N. Methods for comparing event rates in intervention studies when the unit of allocation is a cluster. Am J Epidemiol. 1994 ; 140 (3). 279-89 (Pubitemid 24231914)
83. Feng Z, Diehr P, Peterson A, McLerran D. Selected statistical issues in group randomized trials. Annu Rev Public Health. 2001 ; 22: 167-87 (Pubitemid 32452504)
84. Efron B, Tibshirani R An Introduction to Bootstrap. New York: Chapman & Hall ; 1993 :
85. Manca A, Lambert PC, Sculpher M, Rice N. Cost-effectiveness analysis using data from multinational trials: the use of bivariate hierarchical modeling. Med Decis Making. 2007 ; 27 (4). 471-90 (Pubitemid 47106941)
86. Briggs A, Gray A. The distribution of health care costs and their statistical analysis for economic evaluation. J Health Serv Res Policy. 1998 ; 3 (4). 233-45 (Pubitemid 28479263)
87. Manning WG. The logged dependent variable, heteroscedasticity, and the retransformation problem. J Health Econ. 1998 ; 17 (3). 283-95 (Pubitemid 28300217)