Skip to main content
Top
Gepubliceerd in: Quality of Life Research 4/2015

01-04-2015

Multiple imputation to deal with missing EQ-5D-3L data: Should we impute individual domains or the actual index?

Auteurs: Claire L. Simons, Oliver Rivero-Arias, Ly-Mee Yu, Judit Simon

Gepubliceerd in: Quality of Life Research | Uitgave 4/2015

Log in om toegang te krijgen
share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

Abstract

Purpose

Missing data are a well-known and widely documented problem in cost-effectiveness analyses alongside clinical trials using individual patient-level data. Current methodological research recommends multiple imputation (MI) to deal with missing health outcome data, but there is little guidance on whether MI for multi-attribute questionnaires, such as the EQ-5D-3L, should be carried out at domain or at summary score level. In this paper, we evaluated the impact of imputing individual domains versus imputing index values to deal with missing EQ-5D-3L data using a simulation study and developed recommendations for future practice.

Methods

We simulated missing data in a patient-level dataset with complete EQ-5D-3L data at one point in time from a large multinational clinical trial (n = 1,814). Different proportions of missing data were generated using a missing at random (MAR) mechanism and three different scenarios were studied. The performance of using each method was evaluated using root mean squared error and mean absolute error of the actual versus predicted EQ-5D-3L indices.

Results

In large sample sizes (n > 500) and a missing data pattern that follows mainly unit non-response, imputing domains or the index produced similar results. However, domain imputation became more accurate than index imputation with pattern of missingness following an item non-response. For smaller sample sizes (n < 100), index imputation was more accurate. When MI models were misspecified, both domain and index imputations were inaccurate for any proportion of missing data.

Conclusions

The decision between imputing the domains or the EQ-5D-3L index scores depends on the observed missing data pattern and the sample size available for analysis. Analysts conducting this type of exercises should also evaluate the sensitivity of the analysis to the MAR assumption and whether the imputation model is correctly specified.
Bijlagen
Alleen toegankelijk voor geautoriseerde gebruikers
Voetnoten
1
Missing data due to dropouts for informative reasons are cases when the participant fails to complete a questionnaire as a result of their severity of their illness, death or other known reason. Non-responders occur when the participant does not respond to a questionnaire at one or multiple time points, which creates different missing data patterns in a dataset. In cross-sectional studies, the main missing data patterns are unit non-response when the participant fails to complete all the items within a questionnaire, and item non-response when the participant fails to complete some of the items within a questionnaire. In longitudinal studies, the participant may drop out before the end of the study and do not return creating a monotone missing data pattern.
 
Literatuur
1.
go back to reference Briggs, A., Clark, T., Wolstenholme, J., & Clarke, P. (2003). Missing....presumed at random: Cost-analysis of incomplete data. Health Economics, 12(5), 377–392.CrossRefPubMed Briggs, A., Clark, T., Wolstenholme, J., & Clarke, P. (2003). Missing....presumed at random: Cost-analysis of incomplete data. Health Economics, 12(5), 377–392.CrossRefPubMed
2.
go back to reference Manca, A., & Palmer, S. (2005). Handling missing data in patient-level cost-effectiveness analysis alongside randomised clinical trials. Applied Health Economics and Health Policy, 4(2), 65–75.CrossRefPubMed Manca, A., & Palmer, S. (2005). Handling missing data in patient-level cost-effectiveness analysis alongside randomised clinical trials. Applied Health Economics and Health Policy, 4(2), 65–75.CrossRefPubMed
3.
go back to reference Burton, A., Billingham, L. J., & Bryan, S. (2007). Cost-effectiveness in clinical trials: Using multiple imputation to deal with incomplete cost data. Clinical Trials, 4(2), 154–161.CrossRefPubMed Burton, A., Billingham, L. J., & Bryan, S. (2007). Cost-effectiveness in clinical trials: Using multiple imputation to deal with incomplete cost data. Clinical Trials, 4(2), 154–161.CrossRefPubMed
4.
go back to reference Grieve, R., Cairns, J., & Thompson, S. G. (2010). Improving costing methods in multicentre economic evaluation: The use of multiple imputation for unit costs. Health Economics, 19(8), 939–954.CrossRefPubMed Grieve, R., Cairns, J., & Thompson, S. G. (2010). Improving costing methods in multicentre economic evaluation: The use of multiple imputation for unit costs. Health Economics, 19(8), 939–954.CrossRefPubMed
5.
go back to reference Oostenbrink, J. B., & Al, M. J. (2005). The analysis of incomplete cost data due to dropout. Health Economics, 14(8), 763–776.CrossRefPubMed Oostenbrink, J. B., & Al, M. J. (2005). The analysis of incomplete cost data due to dropout. Health Economics, 14(8), 763–776.CrossRefPubMed
6.
go back to reference Yu, L. M., Burton, A., & Rivero-Arias, O. (2007). Evaluation of software for multiple imputation of semi-continuous data. Statistical Methods in Medical Research, 16(3), 243–258.CrossRefPubMed Yu, L. M., Burton, A., & Rivero-Arias, O. (2007). Evaluation of software for multiple imputation of semi-continuous data. Statistical Methods in Medical Research, 16(3), 243–258.CrossRefPubMed
7.
go back to reference Faria, R., Gomes, M., Epstein, D., & White, I. R. (2014). A guide to handling missing data in cost-effectiveness analysis conducted within randomised controlled trials. PharmacoEconomics. doi:10.1007/s40273-014-0193-3. Faria, R., Gomes, M., Epstein, D., & White, I. R. (2014). A guide to handling missing data in cost-effectiveness analysis conducted within randomised controlled trials. PharmacoEconomics. doi:10.​1007/​s40273-014-0193-3.
8.
go back to reference Little, R. J. & D. B. Rubin. (2002). Statistical analysis with missing data. 2nd ed. Wiley Series in Probability and Statistics. Hoboken, NJ: Wiley. Little, R. J. & D. B. Rubin. (2002). Statistical analysis with missing data. 2nd ed. Wiley Series in Probability and Statistics. Hoboken, NJ: Wiley.
9.
go back to reference Noble, S. M., Hollingworth, W., & Tilling, K. (2012). Missing data in trial-based cost-effectiveness analysis: The current state of play. Health Economics, 21(2), 187–200.CrossRefPubMed Noble, S. M., Hollingworth, W., & Tilling, K. (2012). Missing data in trial-based cost-effectiveness analysis: The current state of play. Health Economics, 21(2), 187–200.CrossRefPubMed
10.
go back to reference Wood, A. M., White, I. R., & Thompson, S. G. (2004). Are missing outcome data adequately handled? A review of published randomized controlled trials in major medical journals. Clinical Trials, 1(4), 368–376.CrossRefPubMed Wood, A. M., White, I. R., & Thompson, S. G. (2004). Are missing outcome data adequately handled? A review of published randomized controlled trials in major medical journals. Clinical Trials, 1(4), 368–376.CrossRefPubMed
11.
go back to reference Eekhout, I., de Boer, R. M., Twisk, J. W. R., de Vet, H. C. W., & Heymans, M. W. (2012). Missing data: A systematic review of how they are reported and handled. Epidemiology, 23(5), 729–732.CrossRefPubMed Eekhout, I., de Boer, R. M., Twisk, J. W. R., de Vet, H. C. W., & Heymans, M. W. (2012). Missing data: A systematic review of how they are reported and handled. Epidemiology, 23(5), 729–732.CrossRefPubMed
12.
go back to reference EuroQol, G. (1990). EuroQol—a new facility for the measurement of health-related quality of life. Health Policy, 16, 199–208.CrossRef EuroQol, G. (1990). EuroQol—a new facility for the measurement of health-related quality of life. Health Policy, 16, 199–208.CrossRef
14.
go back to reference Horsman, J., Furlong, W., Feeny, D., & Torrance, G. (2003). The health utilities index (HUI): Concepts, measurement properties and applications. Health and Quality of Life Outcomes, 1, 54.CrossRefPubMedCentralPubMed Horsman, J., Furlong, W., Feeny, D., & Torrance, G. (2003). The health utilities index (HUI): Concepts, measurement properties and applications. Health and Quality of Life Outcomes, 1, 54.CrossRefPubMedCentralPubMed
15.
go back to reference National Institute for Health and Care Excellence. (2013). Guide to the methods of technology appraisal. London: National Institute for Health and Care Excellence. National Institute for Health and Care Excellence. (2013). Guide to the methods of technology appraisal. London: National Institute for Health and Care Excellence.
16.
go back to reference Ratcliffe, J., Young, T., Longworth, L., & Buxton, M. (2005). An assessment of the impact of informative dropout and nonresponse in measuring health-related quality of life using the EuroQol (EQ-5D) descriptive system. Value Health, 8(1), 53–58.CrossRefPubMed Ratcliffe, J., Young, T., Longworth, L., & Buxton, M. (2005). An assessment of the impact of informative dropout and nonresponse in measuring health-related quality of life using the EuroQol (EQ-5D) descriptive system. Value Health, 8(1), 53–58.CrossRefPubMed
17.
go back to reference Blough, D. K., Ramsey, S., Sullivan, S. D., & Yusen, R. (2009). The impact of using different imputation methods for missing quality of life scores on the estimation of the cost-effectiveness of lung-volume-reduction surgery. Health Economics, 18(1), 91–101.CrossRefPubMed Blough, D. K., Ramsey, S., Sullivan, S. D., & Yusen, R. (2009). The impact of using different imputation methods for missing quality of life scores on the estimation of the cost-effectiveness of lung-volume-reduction surgery. Health Economics, 18(1), 91–101.CrossRefPubMed
18.
go back to reference Szende, A., M. Oppe, & N. Devlin.(2007). EQ-5D value sets: Inventory, comparative review and user guide. A. Szende, M. Oppe, and N. Devlin. (Eds.) Dordrecht: Springer. Szende, A., M. Oppe, & N. Devlin.(2007). EQ-5D value sets: Inventory, comparative review and user guide. A. Szende, M. Oppe, and N. Devlin. (Eds.) Dordrecht: Springer.
19.
go back to reference StataCorp. Stata Statistical Software. (2011). Stata Press: College Station. TX: StataCorp LP. StataCorp. Stata Statistical Software. (2011). Stata Press: College Station. TX: StataCorp LP.
20.
go back to reference Molyneux, A., Kerr, R., Stratton, I., Sandercock, P., Clarke, M., Shrimpton, J., et al. (2002). International Subarachnoid Aneurysm Trial (ISAT) of neurosurgical clipping versus endovascular coiling in 2143 patients with ruptured intracranial aneurysms: A randomised trial. Lancet, 360(9342), 1267–1274.CrossRefPubMed Molyneux, A., Kerr, R., Stratton, I., Sandercock, P., Clarke, M., Shrimpton, J., et al. (2002). International Subarachnoid Aneurysm Trial (ISAT) of neurosurgical clipping versus endovascular coiling in 2143 patients with ruptured intracranial aneurysms: A randomised trial. Lancet, 360(9342), 1267–1274.CrossRefPubMed
21.
go back to reference Jennett, B., & Bond, M. (1975). Assessment of outcome after severe brain damage. Lancet, 1(7905), 480–484.CrossRefPubMed Jennett, B., & Bond, M. (1975). Assessment of outcome after severe brain damage. Lancet, 1(7905), 480–484.CrossRefPubMed
22.
go back to reference van Swieten, J. C., Koudstaal, P. J., Visser, M. C., Schouten, H. J., & van Gijn, J. (1988). Interobserver agreement for the assessment of handicap in stroke patients. Stroke, 19(5), 604–607.CrossRefPubMed van Swieten, J. C., Koudstaal, P. J., Visser, M. C., Schouten, H. J., & van Gijn, J. (1988). Interobserver agreement for the assessment of handicap in stroke patients. Stroke, 19(5), 604–607.CrossRefPubMed
24.
go back to reference Van Buuren, S., Brand, J. P. L., Groothuis-Oudshoorn, C. G. M., & Rubin, D. B. (2006). Fully conditional specification in multivariate imputation. Journal of Statistical Computation and Simulation, 76(12), 1049–1064.CrossRef Van Buuren, S., Brand, J. P. L., Groothuis-Oudshoorn, C. G. M., & Rubin, D. B. (2006). Fully conditional specification in multivariate imputation. Journal of Statistical Computation and Simulation, 76(12), 1049–1064.CrossRef
25.
go back to reference Fairbank, J., Frost, H., Wilson-MacDonald, J., Yu, L. M., Barker, K., & Collins, R. (2005). Spine stabilisation trial. Randomised controlled trial to compare surgical stabilisation of the lumbar spine with an intensive rehabilitation programme for patients with chronic low back pain: The MRC spine stabilisation trial. BMJ, 330(7502), 1233.CrossRefPubMedCentralPubMed Fairbank, J., Frost, H., Wilson-MacDonald, J., Yu, L. M., Barker, K., & Collins, R. (2005). Spine stabilisation trial. Randomised controlled trial to compare surgical stabilisation of the lumbar spine with an intensive rehabilitation programme for patients with chronic low back pain: The MRC spine stabilisation trial. BMJ, 330(7502), 1233.CrossRefPubMedCentralPubMed
26.
go back to reference Trial Group, K. A. T. (2009). The Knee Arthroplasty Trial (KAT) design features, baseline characteristics, and two-year functional outcomes after alternative approaches to knee replacement. The Journal of Bone and Joint Surgery, 91(1), 134–141.CrossRef Trial Group, K. A. T. (2009). The Knee Arthroplasty Trial (KAT) design features, baseline characteristics, and two-year functional outcomes after alternative approaches to knee replacement. The Journal of Bone and Joint Surgery, 91(1), 134–141.CrossRef
27.
go back to reference Rivero-Arias, O., Gray, A., Frost, H., Lamb, S. E., & Stewart-Brown, S. (2006). Cost-utility analysis of physiotherapy treatment compared with physiotherapy advice in low back pain. Spine, 31(12), 1381–1387.CrossRefPubMed Rivero-Arias, O., Gray, A., Frost, H., Lamb, S. E., & Stewart-Brown, S. (2006). Cost-utility analysis of physiotherapy treatment compared with physiotherapy advice in low back pain. Spine, 31(12), 1381–1387.CrossRefPubMed
28.
go back to reference Kendrick, T., Simons, L., Mynors-Wallis, L., Gray, A., Lathlean, J., Pickering, R., et al. (2006). Cost-effectiveness of referral for generic care or problem-solving treatment from community mental health nurses, compared with usual general practitioner care for common mental disorders: Randomised controlled trial. British Journal of Psychiatry, 189, 50–59.CrossRefPubMed Kendrick, T., Simons, L., Mynors-Wallis, L., Gray, A., Lathlean, J., Pickering, R., et al. (2006). Cost-effectiveness of referral for generic care or problem-solving treatment from community mental health nurses, compared with usual general practitioner care for common mental disorders: Randomised controlled trial. British Journal of Psychiatry, 189, 50–59.CrossRefPubMed
29.
go back to reference Horton, N. J., & Kleinman, K. P. (2007). Much ado about nothing: A comparison of missing data methods and software to fit incomplete data regression models. American Statistician, 61(1), 79–90.CrossRefPubMedCentralPubMed Horton, N. J., & Kleinman, K. P. (2007). Much ado about nothing: A comparison of missing data methods and software to fit incomplete data regression models. American Statistician, 61(1), 79–90.CrossRefPubMedCentralPubMed
30.
go back to reference Harel, O., & Zhou, X. H. (2007). Multiple imputation: Review of theory, implementation and software. Statistics in Medicine, 26(16), 3057–3077.CrossRefPubMed Harel, O., & Zhou, X. H. (2007). Multiple imputation: Review of theory, implementation and software. Statistics in Medicine, 26(16), 3057–3077.CrossRefPubMed
31.
go back to reference White, I. R., Royston, P., & Wood, A. M. (2011). Multiple imputation using chained equations: Issues and guidance for practice. Statistics in Medicine, 30(4), 377–399.CrossRefPubMed White, I. R., Royston, P., & Wood, A. M. (2011). Multiple imputation using chained equations: Issues and guidance for practice. Statistics in Medicine, 30(4), 377–399.CrossRefPubMed
32.
go back to reference Benjamini, Y., & Hochberg, Y. (1995). Controlling the False Discovery Rate: A Practical and Powerful Approach to Multiple Testing. Journal of the Royal Statistical Society. Series B (Methodological), 57(1), 289–300. Benjamini, Y., & Hochberg, Y. (1995). Controlling the False Discovery Rate: A Practical and Powerful Approach to Multiple Testing. Journal of the Royal Statistical Society. Series B (Methodological), 57(1), 289–300.
33.
go back to reference Efron, B. (1979). The 1977 rietz lecture—bootstrap methods—another look at the Jackknife. The annals of Statistics, 7(1), 1–26. Efron, B. (1979). The 1977 rietz lecture—bootstrap methods—another look at the Jackknife. The annals of Statistics, 7(1), 1–26.
34.
go back to reference Kind, P., Hardman, G., & Macran, S. (1999). UK population norms for EQ-5D. UK: Centre for Health Economics, University of York. Kind, P., Hardman, G., & Macran, S. (1999). UK population norms for EQ-5D. UK: Centre for Health Economics, University of York.
35.
go back to reference Janssen, M. F., Pickard, A. S., Golicki, D., Gudex, C., Niewada, M., Scalone, L., et al. (2013). Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: A multi-country study. Quality of Life Research, 22(7), 1717–1727.CrossRefPubMedCentralPubMed Janssen, M. F., Pickard, A. S., Golicki, D., Gudex, C., Niewada, M., Scalone, L., et al. (2013). Measurement properties of the EQ-5D-5L compared to the EQ-5D-3L across eight patient groups: A multi-country study. Quality of Life Research, 22(7), 1717–1727.CrossRefPubMedCentralPubMed
36.
go back to reference Konig, H. H., Born, A., Gunther, O., Matschinger, H., Heinrich, S., Riedel-Heller, S. G., et al. (2010). Validity and responsiveness of the EQ-5D in assessing and valuing health status in patients with anxiety disorders. Health and Quality of Life Outcomes, 8, 47.CrossRefPubMedCentralPubMed Konig, H. H., Born, A., Gunther, O., Matschinger, H., Heinrich, S., Riedel-Heller, S. G., et al. (2010). Validity and responsiveness of the EQ-5D in assessing and valuing health status in patients with anxiety disorders. Health and Quality of Life Outcomes, 8, 47.CrossRefPubMedCentralPubMed
37.
go back to reference Long, J. S. (1997). Regression models for categorical and limited dependent variables. London: Sage. Long, J. S. (1997). Regression models for categorical and limited dependent variables. London: Sage.
38.
go back to reference Ramsey, J. B. (1969). Tests for specification errors in classical linear least-squares regression analysis. Journal of the Royal Statistical Society. Series B-Statistical Methodology, 31(2), 350–371. Ramsey, J. B. (1969). Tests for specification errors in classical linear least-squares regression analysis. Journal of the Royal Statistical Society. Series B-Statistical Methodology, 31(2), 350–371.
39.
go back to reference Carpenter, J. R., Kenward, M. G., & White, I. R. (2007). Sensitivity analysis after multiple imputation under missing at random: A weighting approach. Statistical Methods in Medical Research, 16(3), 259–275.CrossRefPubMed Carpenter, J. R., Kenward, M. G., & White, I. R. (2007). Sensitivity analysis after multiple imputation under missing at random: A weighting approach. Statistical Methods in Medical Research, 16(3), 259–275.CrossRefPubMed
40.
go back to reference Herdman, M., Gudex, C., Lloyd, A., Janssen, M., Kind, P., Parkin, D., et al. (2011). Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Quality of Life Research, 20(10), 1727–1736.CrossRefPubMedCentralPubMed Herdman, M., Gudex, C., Lloyd, A., Janssen, M., Kind, P., Parkin, D., et al. (2011). Development and preliminary testing of the new five-level version of EQ-5D (EQ-5D-5L). Quality of Life Research, 20(10), 1727–1736.CrossRefPubMedCentralPubMed
Metagegevens
Titel
Multiple imputation to deal with missing EQ-5D-3L data: Should we impute individual domains or the actual index?
Auteurs
Claire L. Simons
Oliver Rivero-Arias
Ly-Mee Yu
Judit Simon
Publicatiedatum
01-04-2015
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 4/2015
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-014-0837-y

Andere artikelen Uitgave 4/2015

Quality of Life Research 4/2015 Naar de uitgave