Sample size calculation in cost-effectiveness cluster randomized trials: Optimal and maximin approaches

Statistics in Medicine

Volumn 33, Issue 15, 2014, Pages 2538-2553

  Manju, Md Abu ^a   Candel, Math J J M ^a   Berger, Martijn P F ^a  

^a MAASTRICHT UNIVERSITY   (Netherlands)

Author keywords

Cluster randomized trials; Cost effectiveness analysis; Maximin design; Optimal design; Sample size calculation

Indexed keywords

ARTICLE; CALCULATION; CLUSTER ANALYSIS; CONTROLLED STUDY; COST; COST EFFECTIVENESS ANALYSIS; MATHEMATICAL COMPUTING; PROCESS OPTIMIZATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); SAMPLE SIZE; SAMPLING; STATISTICAL MODEL; VARIANCE; COST BENEFIT ANALYSIS; DEPRESSION, POSTPARTUM; ECONOMICS; FEMALE; HUMAN; PROCEDURES; QUALITY OF LIFE;

CLUSTER ANALYSIS; COST-BENEFIT ANALYSIS; DEPRESSION, POSTPARTUM; FEMALE; HUMANS; MODELS, STATISTICAL; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE;

EID: 84901929603     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.6112     Document Type: Article

References (33)

1. Drummond M, Sculpher M, Torrance GW, O'Brien BJ, Stoddart GL. Methods for the Economic Evaluation of Health Care Programmes. Oxford University Press: Oxford, 2005.
2. Morrell CJ, Slade P, Warner R, Paley G, Dixon S, Walters SJ, et al. Clinical effectiveness of health visitor training in psychologically informed approaches for depression in postnatal women: pragmatic cluster randomised trial in primary care. British Medical Journal 2009; 338:276-280.
3. Moerbeek M, Van Breukelen GJP, Berger MPF. Design issues for experiments in multilevel population. Journal of Educational and Behavioral Statistics 2000; 25:271-284.
4. Grieve R, Nixon R, Thompson SG. Bayesian hierarchical models for cost-effectiveness analyses that use data from cluster randomized. Medical Decision Making 2010; 30:163-175.
5. Raudenbush SW. Statistical analysis and optimal design for cluster randomized trials. Psychological Methods 1997; 2:173-185.
6. Raudenbush ST, Liu X. Statistical power and optimal design for multisite randomized trials. Psychological Methods 2000; 5:199-213.
7. Briggs A, Gray AM. Power and sample size calculations for stochastic cost-effectiveness analysis. Medical Decision Making 1998; 18:81-92.
8. Laska EM, Meisner M, Siegel C. Power and sample size in cost-effectiveness analysis. Medical Decision Making 1999; 19:339-343.
9. Zethraeus N, Johannesson M, Jönsson B, Löthgren M, Tambour M. Advantages of using the net-benefit approach for analysing uncertainty in economic evaluation studies. Pharmacoeconomics 2003; 21:39-48.
10. Manca A, Rice N, Sculpher MJ, Briggs AH. Assessing generalisability by location in trial-based cost-effectiveness analysis: the use of multilevel models. Health Economics 2005; 14:471-485.
11. Berger MPF, Wong WK. An Introduction to Optimal Designs for Social and Bio-Medical Research. Wiley: Chichester, 2009.
12. Atkinson AC, Donev AN, Tobias RD. Optimum Experimental Designs, with SAS. Oxford University Press: Oxford, 2007.
13. Wu CFJ. Efficient sequential designs with binary data. Journal of the American Statistical Association 1985; 80:974-984.
14. Spiegelhalter DJ, Abrams KR, Myles JP. Bayesian Approaches to Clinical Trials and Health-Care Evaluation. Wiley: Chichester, 2007.
15. Stinnett A, Mullahy J. Net health benefits: a new framework for the analysis of uncertainty in cost-effectiveness analysis. Medical Decision Making 1998; 18:S68-S80.
16. Verbeke G, Molenberghs G. Linear Mixed Models for Longitudinal Data. Springer: New York, 2000.
17. Tokola K, Larocque D, Nevalainen J, Oja H. Power, sample size and sampling costs for clustered data. Statistics and Probability Letters 2011; 81:852-862.
18. Gomes M, Edmond SWN, Grieve R, Nixon R, Carpenter R, Thompson SG. Developing appropriate methods for cost-effectiveness analysis of cluster randomized trials. Medical Decision Making 2012; 32:350-361.
19. Hampel FR. A general qualitative definition of robustness. The Annals of Mathematical Statistics 1971; 6:1887-1896.
20. Grieve R, Nixon R, Thompson SG, Cairns J. Multilevel models for estimating incremental net benefits in multinational studies. Health Economics 2007; 16:815-826.
21. Hoch JS, Briggs AH, Willan A. Something old, something new, something borrowed, something BLUE: a framework for the marriage of health econometrics and cost-effectiveness analysis. Health Economics 2002; 11:415-430.
22. Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. International Journal of Technology Assessment in Health Care 2001; 17:69-82.
23. Willan AR, Lin DY. Incremental net benefit in randomized clinical trials. Statistics in Medicine 2001; 20:1563-1574.
24. Murray DM, Varnell SP, Blitstein JL. Design and analysis of group-randomized trials: a review of recent methodological developments. American Journal of Public Health 2004; 94:423-432.
25. Snijders TAB, Bosker RJ. Multilevel Analysis. Sage Publications: London, 1999.
26. Cohen J. A power primer. Psychological Bulletin 1992; 112:155-159.
27. Liu X. Statistical power and optimum sample allocation ratio for treatment and control having unequal costs per unit of randomization. Journal of Educational and Behavioral Statistics 2003; 28:231-248.
28. Cheong YK, Fotiu RP, Raudenbush SW. Efficiency and robustness of alternative estimators for two- and three-level models: the case of NAEP. Journal of Educational and Behavioral Statistics 2001; 26:411-429.
29. Maas CJM, Hox JJ. The influence of violations of assumptions on multilevel parameter estimates and their standard errors. Computational Statistics and Data Analysis 2004; 46:427-440.
30. Candel MJJM, Van Breukelen GJP, Kotova L, Berger MPF. Optimality of unequal cluster sizes in multilevel studies with small sample sizes. Communications in Statistics: Simulation and Computation 2008; 37:222-239.
31. Van Breukelen GJP, Candel MJJM, Berger MPF. Relative efficiency of unequal versus equal cluster sizes in cluster randomized and multicentre trials. Statistics in Medicine 2007; 26:2589-2603.
32. Moerbeek M, Van Breukelen GJP, Berger MPF. Optimal experimental designs for multilevel models with covariates. Communications in Statistics-Theory and Methods 2001; 30:2683-2697.
33. Roberts C, Roberts SA. Design and analysis of clinical trials with clustering effects due to treatment. Clinical Trials 2005; 2:152-162.