Update on statistical power and sample size assessments for cost-effectiveness studies

References

• Commonwealth of Australia. Guidelines for the pharmaceutical industry on preparation of submissions to the Pharmaceutical Benefits Advisory Committee: including major submissions involving economic analyses. Australian Government Publishing Service, Canberra, Australia.
   Google Scholar
• National Institute for Clinical Excellence: technology appraisals work programme, new guidance documents (2001).
   Google Scholar
• Guidelines for Economic Evaluation of Pharmaceuticals: Canada. Second Edition. Canadian Co-ordinating Office for Health Technology Assessment. Ottawa, Canada (1997).
   Google Scholar
• Cost-Effectiveness in Health and Medicine. Gold MR, Siegel JE, Russell LB, Weinstein MC (Eds). Oxford University Press, NY, USA (1996).
   Google Scholar
• Guide to clinical preventive seMces Second Edition. US Preventive Services Task Force. Williams & Wilkins, MD, USA (1996).
   Google Scholar
• Blumenschein K, Johannesson M, Yokoyama KK, Freeman PR. Hypothetical versus real willingness to pay in the healthcare sector: results from a field experiment. J. Health Econ. 20 (3), 441–457 (2001).
   PubMed Web of Science ®Google Scholar
• Stinnett AA, Mullaby J. Net health benefits: a new framework for the analysis of uncertainty in cost-effectiveness analysis. Med. Decis. Making-18(2), S68-S80 (1998).
   Google Scholar
• ••Introduces the concept of net health costand benefit and utilizes them as a basis for inference in cost-effectiveness analyses.
   Google Scholar
• Glick HA, Briggs AH, Polsky D. Quantifying stochastic uncertainty and presenting results of cost-effectiveness analyses. Expert Rev. Pharmacoeconomics Outcomes Res. 1(1), 25–36 (2001).
   Google Scholar
• •Excellent review of statistical and practical issues in constructing confidence intervals for the cost-effectiveness ratio and net health benefit.
   Google Scholar
• Lothgren M, Zethraeus N. Definition, interpretation and calculation of cost-effectiveness acceptability curves. Health Econ. 9(7), 623–630 (2000).
   Google Scholar
• •Good reference for statistical definitions of summary measures used in cost-effectiveness analysis.
   Google Scholar
• Van Hout BA, Al MJ, Gordon GS, Ruten FFH. Costs, effects and C/E-ratios alongside a clinical-trial. Health Econ. 3(5), 309–319 (1994).
   Google Scholar
• Heitjan DF, Moskowitz AJ, Whang W Bayesian estimation of cost-effectiveness ratios from clinical trials. Health Econ. 8(3), 191–201 (1999).
   Google Scholar
• Heitjan DE Fieller's method and net health benefits. Health Econ. 9(4),327–335 (2000).
   Google Scholar
• Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis.Health Econ. 8(3), 257–261(1999).
   Google Scholar
• O'Hagan A, Stevens JW, Montmartin J. Inference for the cost-effectiveness acceptability curve and cost-effectiveness ratio. PharmacoEconomics 17 (4), 339–349 (2000).
   PubMed Web of Science ®Google Scholar
• Willan AR, Lin DY. Incremental net benefit in randomized clinical trials. Stat. Med. 20(11), 1563–1574 (2001).
   PubMed Web of Science ®Google Scholar
• Gardiner JC, Huebner M, Jetton J, Bradley CJ. On parametric confidence intervals for the cost-effectiveness ratio. Blamer. 43(3), 283–296 (2001).
   Google Scholar
• •Formal theoretical comparison of different parametric approaches to constructing confidence intervals for the cost-effectiveness ratio.
   Google Scholar
• Gardiner JC, Huebner M, Jetton J, Bradley CJ. Power and sample size assessments for tests of hypotheses on cost-effectiveness ratios. Health Econ. 9(3), 227–234 (2000).
   Google Scholar
• •Formal development of power and sample size expressions for hypotheses on the cost-effectiveness ratio.
   Google Scholar
• O'Brien BJ, Drummond ME, Labelle RJ, Willan A. In search of power and significance - issues in the design and analysis of stochastic cost-effectiveness studies in healthcare. Med. Care 32(2), 150–163 (1994).
   PubMed Web of Science ®Google Scholar
• ••Introduces a stochastic framework forassessing precision of an estimated cost-effectiveness ratio, including derivation of confidence intervals and formulation of tests of hypothesis. Very influential article in generating interest in the field.
   Google Scholar
• Sacristan JA, Day SJ, Navarro 0, Ramos J, Hernandez JM. Use of confidence-intervals and sample-size calculations in health economic-studies. Ann. Pharmacother. 29(7–8), 719–725 (1995).
   Google Scholar
• Al MJ, Van Hout BA, Michel BC, Rutten FFH. Sample size calculation in economic evaluations. Health Econ. 7(4), 327–335 (1998).
   Google Scholar
• Briggs AH, Gray AM. Power and sample size calculations for stochastic cost-effectiveness analysis. Med. Dec. Making- 18(2), S81-S92 (1998).
   Google Scholar
• Laska EM, Meisner M, Siegel C. Power and sample size in cost-effectiveness analysis. Med. Dec. Making 19 (3), 339–343 (1999).
   Google Scholar
• ••Introduces a nonpararnetric approach todetermination of power and sample size for cost-effectiveness studies based on net health cost.
   Google Scholar
• Backhouse ME. Use of randomised controlled trials for producing cost-effectiveness evidence - potential impact of design choices on sample size and study duration. PhannacoEconomics 20(15), 1061–1077 (2002).
   Google Scholar
• O'Hagan A, Stevens JW. Bayesian assessment of sample size for clinical trials of cost-effectiveness. Med. Dec. Making 21(3), 219–230 (2001).
   Google Scholar
• •Addresses the Bayesian approach to sample size determination using prior distributions on means of cost and effectiveness.
   Google Scholar
• O'Hagan A, Stevens JW, Montmartin J. Bayesian cost-effectiveness analysis from clinical trial data. Stat. Med. 20(5), 733–753 (2001).
   PubMed Web of Science ®Google Scholar
• •Provides a Bayesian interpretation of the cost-effectiveness acceptability curve.
   Google Scholar
• Willan AR, O'Brien BJ. Sample size and power issues in estimating incremental cost-effectiveness ratios from clinical trials data. Health Econ. 8 (3), 203–211 (1999).
   Google Scholar
• Willan AR, Obrien BJ. Confidence intervals for cost-effectiveness ratios: an application of Fieller's theorem. Health Econ. 5(4), 297–305 (1996).
   Google Scholar
• Cook JR, Heyse JE Use of an angular transformation for ratio estimation in cost- effectiveness analysis. Stat. Med. 19(21), 2989–3003 (2000).
   PubMed Web of Science ®Google Scholar
• Lawless JE Statistical Models and Methods for Lifetime Data. John Wiley & Sons, NY, USA (1982).
   Google Scholar
• Bang H, Tsiatis AA. Estimating medical costs with censored data. Biometrika 87(2), 329–343 (2000).
   Web of Science ®Google Scholar
• Carides GW, Heyse JW, Iglewicz B. A regression-based method for estimating mean treatment cost in the presence of right-censoring. Biastatistics1(3), 299–313 (2000).
   Google Scholar
• Diehr P, Yanez D, Ash A, Hornbrook M, Lin DY. Methods for analyzing healthcare utilization and costs. Ann. Rev Pub. Health 20,125–144 (1999).
   PubMed Web of Science ®Google Scholar
• Etzioni RD, Feuer EJ, Sullivan SD, Lin DY, Hu CC, Ramsey SD. On the use of survival analysis techniques to estimate medical care costs. .1. Health Econ. 18 (3), 365–380 (1999).
   Google Scholar
• Gardiner JC, Polverejan E. Longitudinal models for the analysis of healthcare costs and outcomes. Proceedings of the American Statistical Association, Section on Health Policy Statistics [CD-ROM] (2002).
   Google Scholar
• Lin DY. Linear regression of censored medical costs. Biostatistics 1,35–47 (2000).
   PubMedGoogle Scholar
• Willan AR, Lin DY, Cook RJ, Chen EB. Using inverse-weighting in cost-effectiveness analysis with censored data. Stat. Methods Med. Res. 11(6), 539–551 (2002).
   Google Scholar
• •Summary of methods for estimating costs and effectiveness from censored survival and costs data.
   Google Scholar
• Willan AR, Chen EB, Cook RJ, Lin DY. Incremental net benefit in randomized clinical trials with quality-adjusted survival. Stat. Med. 22(3), 353–362 (2003).
   Google Scholar
• Gardiner J, Hogan A, Holmes-Rovner M, Rovner D, Griffith L, Kupersmith J. Confidence-intervals for cost-effectiveness ratios. Med. Dec. Making 15 (3), 254–263 (1995).
   Google Scholar
• Gardiner J, Holmes-Rovner M, Goddeeris J, Rovner D, Kupersmith J. Covariate-adjusted cost-effectiveness ratios. J. Stat. Ran. Inference 75 (2), 291–304 (1999) .
   Google Scholar
• Polsky D, Glick HA, Willke R, Schulman K. Confidence intervals for cost-effectiveness ratios: a comparison of four methods. Health Econ. 6(3), 243–252 (1997).
   Google Scholar
• Gardiner JC, Indurkhya A, Luo Z. The performance of estimation procedures for cost-effectiveness ratios. In: Advances on Methodological, and Applied Aspects of Probability and Statistics. Balakrishnan N (Ed.). Taylor & Francis, NY, USA, 547–559 (2002).
   Google Scholar
• Chaudhary MA, Stearns SC. Estimating confidence intervals for cost-effectiveness ratios: An example from a randomized trial. Stat. Med. 15(13), 1447–1458 (1996).
   Google Scholar
• Tambour M, Zethraeus N. Bootstrap confidence intervals for cost-effectiveness ratios: some simulation results. Health Econ. 7(2), 143–147 (1998).
   Google Scholar
• 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. 18(23), 3245–3262 (1999).
   Google Scholar
• Severens JL, De Boo TM, Konst EM. Uncertainty of incremental cost-effectiveness ratios - a comparison of Fieller and bootstrap confidence intervals. Irk. j Tech. Assess: Health. 15(3), 608–614 (1999).
   Google Scholar
• Jiang G, Wu J, Williams GR. Fieller's interval and the bootstrap-Fieller interval for the incremental cost-effectiveness ratio. Health Serv. Outcomes Res. Method.1 (3–4), 291–303 (2000).
   Google Scholar
• 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. 11(5), 415–430 (2002).
   PubMed Web of Science ®Google Scholar
• Gardiner JC, Luo Z, Bradley CJ, Polverejan E, Holmes-Rovner M, Rovner D. Longitudinal assessment of cost in healthcare interventions. Health Serv. Outcomes Res. Method. 3,149–168 (2002).
   Google Scholar
• Lin DY. Regression analysis of incomplete medical costs data. Stat. Med. 22, 1181–1200 (2003).
   Google Scholar