Skip to main content
Erschienen in: PharmacoEconomics 5/2016

01.05.2016 | Review Article

Conducting Economic Evaluations Alongside Randomised Trials: Current Methodological Issues and Novel Approaches

verfasst von: Dyfrig Hughes, Joanna Charles, Dalia Dawoud, Rhiannon Tudor Edwards, Emily Holmes, Carys Jones, Paul Parham, Catrin Plumpton, Colin Ridyard, Huw Lloyd-Williams, Eifiona Wood, Seow Tien Yeo

Erschienen in: PharmacoEconomics | Ausgabe 5/2016

Einloggen, um Zugang zu erhalten

Abstract

Trial-based economic evaluations are an important aspect of health technology assessment. The availability of patient-level data coupled with unbiased estimates of clinical outcomes means that randomised controlled trials are effective vehicles for the generation of economic data. However there are methodological challenges to trial-based evaluations, including the collection of reliable data on resource use and cost, choice of health outcome measure, calculating minimally important differences, dealing with missing data, extrapolating outcomes and costs over time and the analysis of multinational trials. This review focuses on the state of the art of selective elements regarding the design, conduct, analysis and reporting of trial-based economic evaluations. The limitations of existing approaches are detailed and novel methods introduced. The review is internationally relevant but with a focus towards practice in the UK.
Literatur
1.
Zurück zum Zitat Sculpher MJ, Claxton K, Drummond M, McCabe C. Whither trial-based economic evaluation for health care decision making? Health Econ. 2006;15(7):677–87.CrossRefPubMed Sculpher MJ, Claxton K, Drummond M, McCabe C. Whither trial-based economic evaluation for health care decision making? Health Econ. 2006;15(7):677–87.CrossRefPubMed
2.
Zurück zum Zitat Glick H, Doshi J, Sonad S, Polsky D. Economic evaluation in clinical trials. 1st ed. Oxford: Oxford University Press; 2007. Glick H, Doshi J, Sonad S, Polsky D. Economic evaluation in clinical trials. 1st ed. Oxford: Oxford University Press; 2007.
3.
4.
Zurück zum Zitat Ramsey SD, Willke RJ, Glick H, Reed SD, Augustovski F, Jonsson B, et al. Cost-effectiveness analysis alongside clinical trials II. An ISPOR Good Research Practices Task Force report. Value Health. 2015;18(2):161–72.CrossRefPubMed Ramsey SD, Willke RJ, Glick H, Reed SD, Augustovski F, Jonsson B, et al. Cost-effectiveness analysis alongside clinical trials II. An ISPOR Good Research Practices Task Force report. Value Health. 2015;18(2):161–72.CrossRefPubMed
5.
Zurück zum Zitat Ridyard CH, Hughes DA. Methods for the collection of resource use data within clinical trials: a systematic review of studies funded by the UK Health Technology Assessment program. Value Health. 2010;13(8):867–72.CrossRefPubMed Ridyard CH, Hughes DA. Methods for the collection of resource use data within clinical trials: a systematic review of studies funded by the UK Health Technology Assessment program. Value Health. 2010;13(8):867–72.CrossRefPubMed
6.
Zurück zum Zitat Ahonkallio S, Santala M, Valtonen H, Martikainen H. Cost-minimisation analysis of endometrial thermal ablation in a day case or outpatient setting under different anaesthesia regimens. Eur J Obstet Gynecol Reprod Biol. 2012;162(1):102–4.CrossRefPubMed Ahonkallio S, Santala M, Valtonen H, Martikainen H. Cost-minimisation analysis of endometrial thermal ablation in a day case or outpatient setting under different anaesthesia regimens. Eur J Obstet Gynecol Reprod Biol. 2012;162(1):102–4.CrossRefPubMed
8.
Zurück zum Zitat Evans WK, Coyle D, Gafni A, Walker H, National Cancer Institute of Canada Clinical Trials Group-Working Group on Economic Analysis. Which cancer clinical trials should be considered for economic evaluation? Selection criteria from the National Cancer Institute of Canada’s Working Group on Economic Analysis. Chronic Dis Can. 2003;24(4):102–7.PubMed Evans WK, Coyle D, Gafni A, Walker H, National Cancer Institute of Canada Clinical Trials Group-Working Group on Economic Analysis. Which cancer clinical trials should be considered for economic evaluation? Selection criteria from the National Cancer Institute of Canada’s Working Group on Economic Analysis. Chronic Dis Can. 2003;24(4):102–7.PubMed
9.
Zurück zum Zitat Revicki DA, Paramore C, Rentz AM. Incorporating pharmacoeconomic and health outcomes into randomized clinical trials. Expert Rev Pharmacoecon Outcomes Res. 2005;5(6):695–703.CrossRefPubMed Revicki DA, Paramore C, Rentz AM. Incorporating pharmacoeconomic and health outcomes into randomized clinical trials. Expert Rev Pharmacoecon Outcomes Res. 2005;5(6):695–703.CrossRefPubMed
10.
Zurück zum Zitat Marshall DA, Hux M. Design and analysis issues for economic analysis alongside clinical trials. Med Care. 2009;47(7 Suppl 1):S14–20.CrossRefPubMed Marshall DA, Hux M. Design and analysis issues for economic analysis alongside clinical trials. Med Care. 2009;47(7 Suppl 1):S14–20.CrossRefPubMed
11.
Zurück zum Zitat Ramsey SD, McIntosh M, Sullivan SD. Design issues for conducting cost-effectiveness analyses alongside clinical trials. Annu Rev Public Health. 2001;22:129–41.CrossRefPubMed Ramsey SD, McIntosh M, Sullivan SD. Design issues for conducting cost-effectiveness analyses alongside clinical trials. Annu Rev Public Health. 2001;22:129–41.CrossRefPubMed
13.
Zurück zum Zitat Gomes M, Grieve R, Nixon R, Edmunds WJ. Statistical methods for cost-effectiveness analyses that use data from cluster randomized trials: a systematic review and checklist for critical appraisal. Med Decis Mak. 2012;32(1):209–20.CrossRef Gomes M, Grieve R, Nixon R, Edmunds WJ. Statistical methods for cost-effectiveness analyses that use data from cluster randomized trials: a systematic review and checklist for critical appraisal. Med Decis Mak. 2012;32(1):209–20.CrossRef
14.
Zurück zum Zitat Edwards RT, Charles JM, Lloyd-Williams H. Public health economics: a systematic review of guidance for the economic evaluation of public health interventions and discussion of key methodological issues. BMC Public Health. 2013;13:1001.CrossRefPubMedPubMedCentral Edwards RT, Charles JM, Lloyd-Williams H. Public health economics: a systematic review of guidance for the economic evaluation of public health interventions and discussion of key methodological issues. BMC Public Health. 2013;13:1001.CrossRefPubMedPubMedCentral
16.
Zurück zum Zitat Payne K, McAllister M, Davies LM. Valuing the economic benefits of complex interventions: when maximising health is not sufficient. Health Econ. 2013;22(3):258–71.CrossRefPubMed Payne K, McAllister M, Davies LM. Valuing the economic benefits of complex interventions: when maximising health is not sufficient. Health Econ. 2013;22(3):258–71.CrossRefPubMed
17.
Zurück zum Zitat Weatherly H, Drummond M, Claxton K, Cookson R, Ferguson B, Godfrey C, et al. Methods for assessing the cost-effectiveness of public health interventions: key challenges and recommendations. Health Policy. 2009;93(2–3):85–92.CrossRefPubMed Weatherly H, Drummond M, Claxton K, Cookson R, Ferguson B, Godfrey C, et al. Methods for assessing the cost-effectiveness of public health interventions: key challenges and recommendations. Health Policy. 2009;93(2–3):85–92.CrossRefPubMed
18.
Zurück zum Zitat Fischer AJ, Threlfall A, Meah S, Cookson R, Rutter H, Kelly MP. The appraisal of public health interventions: an overview. J Public Health (Oxf). 2013;35(4):488–94.CrossRef Fischer AJ, Threlfall A, Meah S, Cookson R, Rutter H, Kelly MP. The appraisal of public health interventions: an overview. J Public Health (Oxf). 2013;35(4):488–94.CrossRef
19.
Zurück zum Zitat Martin A, Jones A, Mugford M, Shemilt I, Hancock R, Wittenberg R. Methods used to identify and measure resource use in economic evaluations: a systematic review of questionnaires for older people. Health Econ. 2012;21(8):1017–22.CrossRefPubMed Martin A, Jones A, Mugford M, Shemilt I, Hancock R, Wittenberg R. Methods used to identify and measure resource use in economic evaluations: a systematic review of questionnaires for older people. Health Econ. 2012;21(8):1017–22.CrossRefPubMed
21.
Zurück zum Zitat Chisholm D, Knapp MR, Knudsen HC, Amaddeo F, Gaite L, van Wijngaarden B. Client Socio-Demographic and Service Receipt Inventory-European Version: development of an instrument for international research. EPSILON Study 5. European Psychiatric Services: inputs linked to outcome domains and needs. Br J Psychiatry Suppl. 2000;177(39):s28–33.CrossRef Chisholm D, Knapp MR, Knudsen HC, Amaddeo F, Gaite L, van Wijngaarden B. Client Socio-Demographic and Service Receipt Inventory-European Version: development of an instrument for international research. EPSILON Study 5. European Psychiatric Services: inputs linked to outcome domains and needs. Br J Psychiatry Suppl. 2000;177(39):s28–33.CrossRef
22.
Zurück zum Zitat Clarke PM, Fiebig DG, Gerdtham UG. Optimal recall length in survey design. J Health Econ. 2008;27(5):1275–84.CrossRefPubMed Clarke PM, Fiebig DG, Gerdtham UG. Optimal recall length in survey design. J Health Econ. 2008;27(5):1275–84.CrossRefPubMed
23.
Zurück zum Zitat Brown JB, Adams ME. Patients as reliable reporters of medical care process. Recall of ambulatory encounter events. Med Care. 1992;30(5):400–11.CrossRefPubMed Brown JB, Adams ME. Patients as reliable reporters of medical care process. Recall of ambulatory encounter events. Med Care. 1992;30(5):400–11.CrossRefPubMed
24.
Zurück zum Zitat Evans CJ, Crawford B. Data collection methods in prospective economic evaluations: how accurate are the results? Value Health. 2000;3(4):277–86.CrossRefPubMed Evans CJ, Crawford B. Data collection methods in prospective economic evaluations: how accurate are the results? Value Health. 2000;3(4):277–86.CrossRefPubMed
25.
Zurück zum Zitat Bhandari A, Wagner T. Self-reported utilization of health care services: improving measurement and accuracy. Med Care Res Rev. 2006;63(2):217–35.CrossRefPubMed Bhandari A, Wagner T. Self-reported utilization of health care services: improving measurement and accuracy. Med Care Res Rev. 2006;63(2):217–35.CrossRefPubMed
26.
Zurück zum Zitat Richards SH, Coast J, Peters TJ. Patient-reported use of health service resources compared with information from health providers. Health Soc Care Community. 2003;11(6):510–8.CrossRefPubMed Richards SH, Coast J, Peters TJ. Patient-reported use of health service resources compared with information from health providers. Health Soc Care Community. 2003;11(6):510–8.CrossRefPubMed
27.
Zurück zum Zitat Ridyard CH, Hughes DA, DIRUM Team. Development of a database of instruments for resource-use measurement: purpose, feasibility, and design. Value Health. 2012;15(5):650–5.CrossRefPubMed Ridyard CH, Hughes DA, DIRUM Team. Development of a database of instruments for resource-use measurement: purpose, feasibility, and design. Value Health. 2012;15(5):650–5.CrossRefPubMed
28.
Zurück zum Zitat McPherson CJ, Addington-Hall JM. Judging the quality of care at the end of life: can proxies provide reliable information? Soc Sci Med. 2003;56(1):95–109.CrossRefPubMed McPherson CJ, Addington-Hall JM. Judging the quality of care at the end of life: can proxies provide reliable information? Soc Sci Med. 2003;56(1):95–109.CrossRefPubMed
30.
Zurück zum Zitat Lyons RA, Jones KH, John G, Brooks CJ, Verplancke JP, Ford DV, et al. The SAIL databank: linking multiple health and social care datasets. BMC Med Inform Decis Mak. 2009;9:3.CrossRefPubMedPubMedCentral Lyons RA, Jones KH, John G, Brooks CJ, Verplancke JP, Ford DV, et al. The SAIL databank: linking multiple health and social care datasets. BMC Med Inform Decis Mak. 2009;9:3.CrossRefPubMedPubMedCentral
31.
Zurück zum Zitat Hounsome N, Orrell M, Edwards RT. EQ-5D as a quality of life measure in people with dementia and their carers: evidence and key issues. Value Health. 2011;14(2):390–9.CrossRefPubMed Hounsome N, Orrell M, Edwards RT. EQ-5D as a quality of life measure in people with dementia and their carers: evidence and key issues. Value Health. 2011;14(2):390–9.CrossRefPubMed
32.
Zurück zum Zitat Noyes JP, Williams A, Allen D, Brocklehurst P, Carter C, Gregory JW, et al. Evidence into practice: evaluating a child-centred intervention for diabetes medicine management. The EPIC Project. BMC Pediatr. 2010;10:70.CrossRefPubMedPubMedCentral Noyes JP, Williams A, Allen D, Brocklehurst P, Carter C, Gregory JW, et al. Evidence into practice: evaluating a child-centred intervention for diabetes medicine management. The EPIC Project. BMC Pediatr. 2010;10:70.CrossRefPubMedPubMedCentral
33.
Zurück zum Zitat Noyes JP, Lowes L, Whitaker R, Allen D, Carter C, Edwards RT, et al. Developing and evaluating a child-centred intervention for diabetes medicine management using mixed methods and a multicentre randomised controlled trial. Health Serv Deliv Res. 2014;2(8). doi:10.3310/hsdr02080. Noyes JP, Lowes L, Whitaker R, Allen D, Carter C, Edwards RT, et al. Developing and evaluating a child-centred intervention for diabetes medicine management using mixed methods and a multicentre randomised controlled trial. Health Serv Deliv Res. 2014;2(8). doi:10.​3310/​hsdr02080.
34.
Zurück zum Zitat Thorrington D, Eames K. Measuring health utilities in children and adolescents: a systematic review of the literature. PLoS One. 2015;10(8):e0135672.CrossRefPubMedPubMedCentral Thorrington D, Eames K. Measuring health utilities in children and adolescents: a systematic review of the literature. PLoS One. 2015;10(8):e0135672.CrossRefPubMedPubMedCentral
35.
Zurück zum Zitat Drummond MF, Sculpher M, Torrance GW, O’Brien BJ, Stoddart GL. Methods for the economic evaluation of health care programmes. 3rd ed. Oxford: Oxford University Press; 2005. Drummond MF, Sculpher M, Torrance GW, O’Brien BJ, Stoddart GL. Methods for the economic evaluation of health care programmes. 3rd ed. Oxford: Oxford University Press; 2005.
36.
37.
Zurück zum Zitat Mulhern B, Rowen D, Jacoby A, Marson T, Snape D, Hughes D, et al. The development of a QALY measure for epilepsy: NEWQOL-6D. Epilepsy Behav. 2012;24(1):36–43.CrossRefPubMed Mulhern B, Rowen D, Jacoby A, Marson T, Snape D, Hughes D, et al. The development of a QALY measure for epilepsy: NEWQOL-6D. Epilepsy Behav. 2012;24(1):36–43.CrossRefPubMed
38.
Zurück zum Zitat Marson AG, Appleton R, Baker GA, Chadwick DW, Doughty J, Eaton B, et al. A randomised controlled trial examining the longer-term outcomes of standard versus new antiepileptic drugs. The SANAD Trial. Health Technol Assess. 2007;11(37):iii–iv, ix–x, 1–134. Marson AG, Appleton R, Baker GA, Chadwick DW, Doughty J, Eaton B, et al. A randomised controlled trial examining the longer-term outcomes of standard versus new antiepileptic drugs. The SANAD Trial. Health Technol Assess. 2007;11(37):iii–iv, ix–x, 1–134.
39.
Zurück zum Zitat Versteegh MM, Leunis A, Uyl-de Groot CA, Stolk EA. Condition-specific preference-based measures: benefit or burden? Value Health. 2012;15(3):504–13.CrossRefPubMed Versteegh MM, Leunis A, Uyl-de Groot CA, Stolk EA. Condition-specific preference-based measures: benefit or burden? Value Health. 2012;15(3):504–13.CrossRefPubMed
40.
Zurück zum Zitat Clarke P, Gray A, Holman R. Estimating utility values for health states of type 2 diabetic patients using the EQ-5D (UKPDS 62). Med Decis Mak. 2002;22(4):340–9.CrossRef Clarke P, Gray A, Holman R. Estimating utility values for health states of type 2 diabetic patients using the EQ-5D (UKPDS 62). Med Decis Mak. 2002;22(4):340–9.CrossRef
41.
Zurück zum Zitat Briggs AH, Bousquet J, Wallace MV, Busse WW, Clark TJ, Pedersen SE, et al. Cost-effectiveness of asthma control: an economic appraisal of the GOAL study. Allergy. 2006;61(5):531–6.CrossRefPubMed Briggs AH, Bousquet J, Wallace MV, Busse WW, Clark TJ, Pedersen SE, et al. Cost-effectiveness of asthma control: an economic appraisal of the GOAL study. Allergy. 2006;61(5):531–6.CrossRefPubMed
42.
Zurück zum Zitat Ryan M, Gerard K, Amaya-Amaya M. Using discrete choice experiments to value health and health care, vol. 11. Dordrecht: Springer; 2008. Ryan M, Gerard K, Amaya-Amaya M. Using discrete choice experiments to value health and health care, vol. 11. Dordrecht: Springer; 2008.
43.
Zurück zum Zitat Latimer NR. Survival analysis for economic evaluations alongside clinical trials–extrapolation with patient-level data: inconsistencies, limitations, and a practical guide. Med Decis Mak. 2013;33(6):743–54.CrossRef Latimer NR. Survival analysis for economic evaluations alongside clinical trials–extrapolation with patient-level data: inconsistencies, limitations, and a practical guide. Med Decis Mak. 2013;33(6):743–54.CrossRef
44.
Zurück zum Zitat Grieve R, Hawkins N, Pennington M. Extrapolation of survival data in cost-effectiveness analyses: improving the current state of play. Med Decis Mak. 2013;33(6):740–2.CrossRef Grieve R, Hawkins N, Pennington M. Extrapolation of survival data in cost-effectiveness analyses: improving the current state of play. Med Decis Mak. 2013;33(6):740–2.CrossRef
45.
Zurück zum Zitat Bagust A, Beale S. Survival analysis and extrapolation modeling of time-to-event clinical trial data for economic evaluation: an alternative approach. Med Decis Mak. 2014;34(3):343–51.CrossRef Bagust A, Beale S. Survival analysis and extrapolation modeling of time-to-event clinical trial data for economic evaluation: an alternative approach. Med Decis Mak. 2014;34(3):343–51.CrossRef
46.
Zurück zum Zitat Bilcke J, Beutels P, Brisson M, Jit M. Accounting for methodological, structural, and parameter uncertainty in decision-analytic models: a practical guide. Med Decis Mak. 2011;31(4):675–92.CrossRef Bilcke J, Beutels P, Brisson M, Jit M. Accounting for methodological, structural, and parameter uncertainty in decision-analytic models: a practical guide. Med Decis Mak. 2011;31(4):675–92.CrossRef
47.
Zurück zum Zitat Jackson CH, Bojke L, Thompson SG, Claxton K, Sharples LD. A framework for addressing structural uncertainty in decision models. Med Decis Mak. 2011;31(4):662–74.CrossRef Jackson CH, Bojke L, Thompson SG, Claxton K, Sharples LD. A framework for addressing structural uncertainty in decision models. Med Decis Mak. 2011;31(4):662–74.CrossRef
48.
Zurück zum Zitat Eddy DM, Hollingworth W, Caro JJ, Tsevat J, McDonald KM, Wong JB, et al. Model transparency and validation: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force–7. Value Health. 2012;15(6):843–50.CrossRefPubMed Eddy DM, Hollingworth W, Caro JJ, Tsevat J, McDonald KM, Wong JB, et al. Model transparency and validation: a report of the ISPOR-SMDM Modeling Good Research Practices Task Force–7. Value Health. 2012;15(6):843–50.CrossRefPubMed
49.
Zurück zum Zitat Noble SM, Hollingworth W, Tilling K. Missing data in trial-based cost-effectiveness analysis: the current state of play. Health Econ. 2012;21(2):187–200.CrossRefPubMed Noble SM, Hollingworth W, Tilling K. Missing data in trial-based cost-effectiveness analysis: the current state of play. Health Econ. 2012;21(2):187–200.CrossRefPubMed
50.
Zurück zum Zitat White IR, Royston P, Wood AM. Multiple imputation using chained equations: issues and guidance for practice. Stat Med. 2011;30(4):377–99.CrossRefPubMed White IR, Royston P, Wood AM. Multiple imputation using chained equations: issues and guidance for practice. Stat Med. 2011;30(4):377–99.CrossRefPubMed
51.
Zurück zum Zitat White IR, Thompson SG. Adjusting for partially missing baseline measurements in randomized trials. Stat Med. 2005;24(7):993–1007.CrossRefPubMed White IR, Thompson SG. Adjusting for partially missing baseline measurements in randomized trials. Stat Med. 2005;24(7):993–1007.CrossRefPubMed
52.
Zurück zum Zitat Sterne JA, White IR, Carlin JB, Spratt M, Royston P, Kenward MG, et al. Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls. BMJ. 2009;338:b2393.CrossRefPubMedPubMedCentral Sterne JA, White IR, Carlin JB, Spratt M, Royston P, Kenward MG, et al. Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls. BMJ. 2009;338:b2393.CrossRefPubMedPubMedCentral
53.
Zurück zum Zitat Ramsey S, Willke R, Briggs A, Brown R, Buxton M, Chawla A, et al. Good research practices for cost-effectiveness analysis alongside clinical trials: the ISPOR RCT-CEA Task Force report. Value Health. 2005;8(5):521–33.CrossRefPubMed Ramsey S, Willke R, Briggs A, Brown R, Buxton M, Chawla A, et al. Good research practices for cost-effectiveness analysis alongside clinical trials: the ISPOR RCT-CEA Task Force report. Value Health. 2005;8(5):521–33.CrossRefPubMed
54.
Zurück zum Zitat Schulz KF, Altman DG, Moher D, CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c332.CrossRefPubMedPubMedCentral Schulz KF, Altman DG, Moher D, CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ. 2010;340:c332.CrossRefPubMedPubMedCentral
55.
Zurück zum Zitat Sylvestre Y. CONSORT: missing missing data, guidelines, the effects on HTA monograph reporting. Trials. 2011;12(Suppl 1):A61.CrossRefPubMedCentral Sylvestre Y. CONSORT: missing missing data, guidelines, the effects on HTA monograph reporting. Trials. 2011;12(Suppl 1):A61.CrossRefPubMedCentral
56.
Zurück zum Zitat Husereau D, Drummond M, Petrou S, Carswell C, Moher D, Greenberg D, et al. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement. Pharmacoeconomics. 2013;31(5):361–7.CrossRefPubMed Husereau D, Drummond M, Petrou S, Carswell C, Moher D, Greenberg D, et al. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement. Pharmacoeconomics. 2013;31(5):361–7.CrossRefPubMed
57.
Zurück zum Zitat Wood AM, White IR, Thompson SG. Are missing outcome data adequately handled? A review of published randomized controlled trials in major medical journals. Clin Trials. 2004;1(4):368–76.CrossRefPubMed Wood AM, White IR, Thompson SG. Are missing outcome data adequately handled? A review of published randomized controlled trials in major medical journals. Clin Trials. 2004;1(4):368–76.CrossRefPubMed
58.
Zurück zum Zitat Little RJA, Rubin DB. Statistical analysis with missing data. 2nd ed. Hoboken: Wiley; 2002. Little RJA, Rubin DB. Statistical analysis with missing data. 2nd ed. Hoboken: Wiley; 2002.
59.
Zurück zum Zitat Little RJA. Missing data adjustments in large surveys. J Bus Econ Stat. 1988;6(3):287–96. Little RJA. Missing data adjustments in large surveys. J Bus Econ Stat. 1988;6(3):287–96.
60.
Zurück zum Zitat Sinharay S, Stern HS, Russell D. The use of multiple imputation for the analysis of missing data. Psychol Methods. 2001;6(4):317–29.CrossRefPubMed Sinharay S, Stern HS, Russell D. The use of multiple imputation for the analysis of missing data. Psychol Methods. 2001;6(4):317–29.CrossRefPubMed
61.
Zurück zum Zitat Marshall A, Altman DG, Holder RL, Royston P. Combining estimates of interest in prognostic modelling studies after multiple imputation: current practice and guidelines. BMC Med Res Methodol. 2009;9:57.CrossRefPubMedPubMedCentral Marshall A, Altman DG, Holder RL, Royston P. Combining estimates of interest in prognostic modelling studies after multiple imputation: current practice and guidelines. BMC Med Res Methodol. 2009;9:57.CrossRefPubMedPubMedCentral
62.
Zurück zum Zitat Wayman J. Multiple imputation for missing data: what is it and how can I use it? Paper presented at the proceedings of the annual meeting of the American educational research association. Chicago; 2003. Wayman J. Multiple imputation for missing data: what is it and how can I use it? Paper presented at the proceedings of the annual meeting of the American educational research association. Chicago; 2003.
63.
Zurück zum Zitat Heckman J. Sample selection bias as a specification error. Econometrica. 1979;47(1):153–61.CrossRef Heckman J. Sample selection bias as a specification error. Econometrica. 1979;47(1):153–61.CrossRef
64.
Zurück zum Zitat Little R. Pattern-mixture models for multivariate incomplete data. J Am Stat Assoc. 1993;88(421):125–34. Little R. Pattern-mixture models for multivariate incomplete data. J Am Stat Assoc. 1993;88(421):125–34.
65.
Zurück zum Zitat Carpenter JR, Kenward MG, White IR. Sensitivity analysis after multiple imputation under missing at random: a weighting approach. Stat Methods Med Res. 2007;16(3):259–75.CrossRefPubMed Carpenter JR, Kenward MG, White IR. Sensitivity analysis after multiple imputation under missing at random: a weighting approach. Stat Methods Med Res. 2007;16(3):259–75.CrossRefPubMed
66.
Zurück zum Zitat Rubin DB, Stern HS, Vehovar V. Handling, “don’t know” survey responses: the case of the Slovenian plebiscite. J Am Stat Assoc. 1995;90:822–8. Rubin DB, Stern HS, Vehovar V. Handling, “don’t know” survey responses: the case of the Slovenian plebiscite. J Am Stat Assoc. 1995;90:822–8.
67.
Zurück zum Zitat Schafer JL, Graham JW. Missing data: our view of the state of the art. Psychol Methods. 2002;7(2):147–77.CrossRefPubMed Schafer JL, Graham JW. Missing data: our view of the state of the art. Psychol Methods. 2002;7(2):147–77.CrossRefPubMed
68.
Zurück zum Zitat Shroufi A, Powles JW. Adherence and chemoprevention in major cardiovascular disease: a simulation study of the benefits of additional use of statins. J Epidemiol Community Health. 2010;64(2):109–13.CrossRefPubMed Shroufi A, Powles JW. Adherence and chemoprevention in major cardiovascular disease: a simulation study of the benefits of additional use of statins. J Epidemiol Community Health. 2010;64(2):109–13.CrossRefPubMed
69.
Zurück zum Zitat Kadambi A, Leipold RJ, Kansal AR, Sorensen S, Getsios D. Inclusion of compliance and persistence in economic models: past, present and future. Appl Health Econ Health Policy. 2012;10(6):365–79.CrossRefPubMed Kadambi A, Leipold RJ, Kansal AR, Sorensen S, Getsios D. Inclusion of compliance and persistence in economic models: past, present and future. Appl Health Econ Health Policy. 2012;10(6):365–79.CrossRefPubMed
70.
Zurück zum Zitat Hughes DA, Bagust A, Haycox A, Walley T. Accounting for noncompliance in pharmacoeconomic evaluations. Pharmacoeconomics. 2001;19(12):1185–97.CrossRefPubMed Hughes DA, Bagust A, Haycox A, Walley T. Accounting for noncompliance in pharmacoeconomic evaluations. Pharmacoeconomics. 2001;19(12):1185–97.CrossRefPubMed
71.
Zurück zum Zitat Hughes D, Cowell W, Koncz T, Cramer J, International Society for Pharmacoeconomics & Outcomes Research Economics of Medication Compliance Working Group. Methods for integrating medication compliance and persistence in pharmacoeconomic evaluations. Value Health. 2007;10(6):498–509.CrossRefPubMed Hughes D, Cowell W, Koncz T, Cramer J, International Society for Pharmacoeconomics & Outcomes Research Economics of Medication Compliance Working Group. Methods for integrating medication compliance and persistence in pharmacoeconomic evaluations. Value Health. 2007;10(6):498–509.CrossRefPubMed
72.
Zurück zum Zitat Fischer K, Goetghebeur E, Vrijens B, White IR. A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments. Biostatistics. 2011;12(2):247–57.CrossRefPubMedPubMedCentral Fischer K, Goetghebeur E, Vrijens B, White IR. A structural mean model to allow for noncompliance in a randomized trial comparing 2 active treatments. Biostatistics. 2011;12(2):247–57.CrossRefPubMedPubMedCentral
73.
Zurück zum Zitat Hughes DA, Walley T. Predicting, “real world” effectiveness by integrating adherence with pharmacodynamic modeling. Clin Pharmacol Ther. 2003;74(1):1–8.CrossRefPubMed Hughes DA, Walley T. Predicting, “real world” effectiveness by integrating adherence with pharmacodynamic modeling. Clin Pharmacol Ther. 2003;74(1):1–8.CrossRefPubMed
74.
Zurück zum Zitat Reed SD, Anstrom KJ, Bakhai A, Briggs AH, Califf RM, Cohen DJ, et al. Conducting economic evaluations alongside multinational clinical trials: toward a research consensus. Am Heart J. 2005;149(3):434–43.CrossRefPubMed Reed SD, Anstrom KJ, Bakhai A, Briggs AH, Califf RM, Cohen DJ, et al. Conducting economic evaluations alongside multinational clinical trials: toward a research consensus. Am Heart J. 2005;149(3):434–43.CrossRefPubMed
75.
Zurück zum Zitat Sculpher MJ, Pang FS, Manca A, Drummond MF, Golder S, Urdahl H, et al. Generalisability in economic evaluation studies in healthcare: a review and case studies. Health Technol Assess. 2004;8(49):iii–iv, 1–192. Sculpher MJ, Pang FS, Manca A, Drummond MF, Golder S, Urdahl H, et al. Generalisability in economic evaluation studies in healthcare: a review and case studies. Health Technol Assess. 2004;8(49):iii–iv, 1–192.
76.
Zurück zum Zitat Drummond M, Barbieri M, Cook J, Glick HA, Lis J, Malik F, et al. Transferability of economic evaluations across jurisdictions: ISPOR Good Research Practices Task Force report. Value Health. 2009;12(4):409–18.CrossRefPubMed Drummond M, Barbieri M, Cook J, Glick HA, Lis J, Malik F, et al. Transferability of economic evaluations across jurisdictions: ISPOR Good Research Practices Task Force report. Value Health. 2009;12(4):409–18.CrossRefPubMed
77.
Zurück zum Zitat Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, et al. Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of the ISPOR Task Force for Translation and Cultural Adaptation. Value Health. 2005;8(2):94–104.CrossRefPubMed Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, et al. Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of the ISPOR Task Force for Translation and Cultural Adaptation. Value Health. 2005;8(2):94–104.CrossRefPubMed
78.
Zurück zum Zitat Manca A, Sculpher MJ, Goeree R. The analysis of multinational cost-effectiveness data for reimbursement decisions: a critical appraisal of recent methodological developments. Pharmacoeconomics. 2010;28(12):1079–96.CrossRefPubMed Manca A, Sculpher MJ, Goeree R. The analysis of multinational cost-effectiveness data for reimbursement decisions: a critical appraisal of recent methodological developments. Pharmacoeconomics. 2010;28(12):1079–96.CrossRefPubMed
79.
Zurück zum Zitat Keeling MJ, Rohani P. Modeling infectious diseases in humans and animals. 1st ed. Princeton: Princeton University Press; 2007. Keeling MJ, Rohani P. Modeling infectious diseases in humans and animals. 1st ed. Princeton: Princeton University Press; 2007.
Metadaten
Titel
Conducting Economic Evaluations Alongside Randomised Trials: Current Methodological Issues and Novel Approaches
verfasst von
Dyfrig Hughes
Joanna Charles
Dalia Dawoud
Rhiannon Tudor Edwards
Emily Holmes
Carys Jones
Paul Parham
Catrin Plumpton
Colin Ridyard
Huw Lloyd-Williams
Eifiona Wood
Seow Tien Yeo
Publikationsdatum
01.05.2016
Verlag
Springer International Publishing
Erschienen in
PharmacoEconomics / Ausgabe 5/2016
Print ISSN: 1170-7690
Elektronische ISSN: 1179-2027
DOI
https://doi.org/10.1007/s40273-015-0371-y

Weitere Artikel der Ausgabe 5/2016

PharmacoEconomics 5/2016 Zur Ausgabe