Tails from the Peak District: adjusted censored mixture models of EQ-5D health state utility values

Health state utility data generated using the EQ-5D instrument are typically right bounded at one with a substantial gap to the next set of observations, left bounded by some negative value, and are multi modal. These features present challenges to the estimation of the e¤ect of clinical and socioeconomic characteristics on health utilities. We present an adjusted censored model and then use this in a flexible, mixture modelling framework to address these issues. We demonstrate superior performance of this model compared to linear regression and Tobit censored regression using a dataset from repeated observations of patients with rheumatoid arthritis. We �nd that three latent classes are appropriate in estimating EQ-5D from function, pain and sociodemographic factors. Analysis of utility data should apply methods that recognise the distributional features of the data.

[1]  Paul Kind,et al.  A social tariff for EuroQol: results from a UK general population survey , 1995 .

[2]  John Brazier,et al.  Mapping SF-36 onto the EQ-5D index: how reliable is the relationship? , 2009, Health and quality of life outcomes.

[3]  Rachelle Buchbinder,et al.  Functional Status and Health-related Quality of Life Assessment in Patients with Rheumatoid Arthritis , 2001 .

[4]  Aki Tsuchiya,et al.  Using the health assessment questionnaire to estimate preference-based single indices in patients with rheumatoid arthritis. , 2007, Arthritis and rheumatism.

[5]  Alan Brennan,et al.  Biologic drugs for rheumatoid arthritis in the Medicare program: a cost-effectiveness analysis. , 2008, Arthritis and rheumatism.

[6]  Andrew Briggs,et al.  Mapping the QLQ-C30 quality of life cancer questionnaire to EQ-5D patient preferences , 2010, The European Journal of Health Economics.

[7]  A. Brennan,et al.  Cost effectiveness of adalimumab in the treatment of patients with moderate to severe rheumatoid arthritis in Sweden , 2004, Annals of the rheumatic diseases.

[8]  Martin J Buxton,et al.  Mapping of the EQ-5D index from clinical outcome measures and demographic variables in patients with coronary heart disease , 2010, Health and quality of life outcomes.

[9]  Geoffrey J. McLachlan,et al.  Finite Mixture Models , 2019, Annual Review of Statistics and Its Application.

[10]  J. Tobin Estimation of Relationships for Limited Dependent Variables , 1958 .

[11]  A. Silman,et al.  The prevalence of rheumatoid arthritis in the United Kingdom: new estimates for a new century. , 2002, Rheumatology.

[12]  P. Kind,et al.  Measuring health-related quality of life in rheumatoid arthritis: validity, responsiveness and reliability of EuroQol (EQ-5D). , 1997, British journal of rheumatology.

[13]  Peter Lindgren,et al.  Modeling the progression of rheumatoid arthritis: a two-country model to estimate costs and consequences of rheumatoid arthritis. , 2002, Arthritis and rheumatism.

[14]  P Tugwell,et al.  Reporting disease activity in clinical trials of patients with rheumatoid arthritis: EULAR/ACR collaborative recommendations. , 2008, Arthritis and rheumatism.

[15]  Allan Wailoo,et al.  Multilevel modelling of cost data: an application to thrombolysis and primary angioplasty in the UK NHS , 2010 .

[16]  Peter Lindgren,et al.  Modeling the cost-effectiveness of treatment of rheumatoid arthritis with rituximab using registry data from Southern Sweden , 2009, International Journal of Technology Assessment in Health Care.

[17]  Peter C Austin,et al.  A comparison of methods for analyzing health-related quality-of-life measures. , 2002, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[18]  S A Marion,et al.  Not all "quality-adjusted life years" are equal. , 2007, Journal of clinical epidemiology.

[19]  Liang Li,et al.  Some methodological issues with the analysis of preference-based EQ-5D index score , 2009, Health Services and Outcomes Research Methodology.

[20]  Wojciech W. Charemza,et al.  NEW DIRECTIONS IN ECONOMETRIC PRACTICE , 1992 .

[21]  P Barton,et al.  A systematic review of the effectiveness of adalimumab, etanercept and infliximab for the treatment of rheumatoid arthritis in adults and an economic evaluation of their cost-effectiveness. , 2006, Health technology assessment.

[22]  William L. Goffe,et al.  SIMANN: FORTRAN module to perform Global Optimization of Statistical Functions with Simulated Annealing , 1992 .

[23]  B. Muthén,et al.  Deciding on the Number of Classes in Latent Class Analysis and Growth Mixture Modeling: A Monte Carlo Simulation Study , 2007 .

[24]  M. Escobar,et al.  The use of the Tobit model for analyzing measures of health status , 2004, Quality of Life Research.

[25]  V. Farewell,et al.  Factorial randomised controlled trial of glucocorticoids and combination disease modifying drugs in early rheumatoid arthritis , 2007, Annals of the rheumatic diseases.

[26]  R. Goeree,et al.  Analysis of health utility data when some subjects attain the upper bound of 1: are Tobit and CLAD models appropriate? , 2010, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[27]  Stephen Joel Coons,et al.  US Valuation of the EQ-5D Health States: Development and Testing of the D1 Valuation Model , 2005, Medical care.

[28]  N. Cooper Economic burden of rheumatoid arthritis: a systematic review. , 2000, Rheumatology.

[29]  Sandro Ridella,et al.  Minimizing multimodal functions of continuous variables with the “simulated annealing” algorithmCorrigenda for this article is available here , 1987, TOMS.

[30]  Richard Grieve,et al.  Addressing the issues that arise in analysing multicentre cost data, with application to a multinational study. , 2006, Journal of health economics.

[31]  Albert W Wu,et al.  Addressing ceiling effects in health status measures: a comparison of techniques applied to measures for people with HIV disease. , 2007, Health services research.

[32]  Aki Tsuchiya,et al.  A review of studies mapping (or cross walking) non-preference based measures of health to generic preference-based measures , 2010, The European Journal of Health Economics.

[33]  Mónica Hernández. Alava,et al.  Growth dynamics : an empirical investigation of output growth using international data , 2002 .