Use of Bayesian Markov Chain Monte Carlo Methods to Estimate EQ-5D Utility Scores from EORTC QLQ Data in Myeloma for Use in Cost-Effectiveness Analysis

Background. Patient-reported outcome measures are an important component of the evidence for health technology appraisal. Their incorporation into cost-effectiveness analyses (CEAs) requires conversion of descriptive information into utilities. This can be done by using bespoke utility algorithms. Otherwise, investigators will often estimate indirect utility models for the patient-reported outcome measures using off-the-shelf utility data such as the EQ-5D or SF-6D. Numerous modeling strategies are reported; however, to date, there has been limited utilization of Bayesian methods in this context. In this article, we examine the relative advantage of the Bayesian methods in relation to dealing with missing data, relaxing the assumption of equal variances and characterizing the uncertainty in the model predictions. Methods. Data from a large myeloma trial were used to examine the relationship between scores in each of the 19 domains of the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30/QLQ-MY20 and the EQ-5D utility. Data from 1839 patients were divided 75%/25% between derivation and validation sets. A conventional ordinary least squares model assuming equal variance and a Bayesian model allowing unequal variance were estimated on complete cases. Two further models were estimated using conventional and Bayesian multiple imputation, respectively, using the full data set. Models were compared in terms of data fit, accuracy in model prediction, and characterization of uncertainty in model predictions. Conclusions. Mean EQ-5D utility weights can be estimated from the EORTC QLQ-C30/QLQ-MY20 for use in CEAs. Frequentist and Bayesian methods produced effectively identical models. However, the Bayesian models provide distributions describing the uncertainty surrounding the estimated utility values and are thus more suited informing analyses for probabilistic CEAs.

[1]  David B. Dunson,et al.  Bayesian Data Analysis , 2010 .

[2]  Benjamin M Craig,et al.  Deriving a preference-based measure for cancer using the EORTC QLQ-C30. , 2011, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[3]  K Claxton,et al.  Informing a decision framework for when NICE should recommend the use of health technologies only in the context of an appropriately designed programme of evidence development. , 2012, Health technology assessment.

[4]  J. Robins,et al.  Inference for imputation estimators , 2000 .

[5]  S. Kaasa,et al.  Development of an EORTC questionnaire module to be used in health‐related quality‐of‐life assessment for patients with multiple myeloma , 1999, British journal of haematology.

[6]  R. Kass,et al.  Reference Bayesian Methods for Generalized Linear Mixed Models , 2000 .

[7]  Leonie Segal,et al.  Health and Quality of Life Outcomes , 2006 .

[8]  Nick Kontodimopoulos,et al.  Mapping the cancer-specific EORTC QLQ-C30 to the preference-based EQ-5D, SF-6D, and 15D instruments. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[9]  David R. Jones,et al.  An introduction to bayesian methods in health technology assessment , 1999, BMJ.

[10]  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.

[11]  J Lipscomb,et al.  Predicting the Cost of Illness , 1998, Medical decision making : an international journal of the Society for Medical Decision Making.

[12]  William F Lawrence,et al.  Predicting EuroQoL EQ-5D Preference Scores from the SF-12 Health Survey in a Nationally Representative Sample , 2004, Medical decision making : an international journal of the Society for Medical Decision Making.

[13]  J. Brazier,et al.  Deriving preference-based single indices from non-preference based condition-specific instruments: converting AQLQ into EQ5D indices , 2002 .

[14]  Ling-Hsiang Chuang,et al.  Converting the SF-12 into the EQ-5D , 2012, PharmacoEconomics.

[15]  Roger A. Sugden,et al.  Multiple Imputation for Nonresponse in Surveys , 1988 .

[16]  D. Rubin,et al.  Statistical Analysis with Missing Data , 1988 .

[17]  M. Sculpher,et al.  Probabilistic sensitivity analysis for NICE technology assessment: not an optional extra. , 2005, Health economics.

[18]  Roderick J. A. Little,et al.  Statistical Analysis with Missing Data: Little/Statistical Analysis with Missing Data , 2002 .

[19]  David R. Jones,et al.  Bayesian methods in health technology assessment: a review. , 2000, Health technology assessment.

[20]  Ross D Crosby,et al.  Estimating a preference-based single index for the Impact of Weight on Quality of Life-Lite (IWQOL-Lite) instrument from the SF-6D. , 2004, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[21]  Joseph L Schafer,et al.  Analysis of Incomplete Multivariate Data , 1997 .

[22]  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.

[23]  Andrea Manca,et al.  Regression Estimators for Generic Health-Related Quality of Life and Quality-Adjusted Life Years , 2012, Medical decision making : an international journal of the Society for Medical Decision Making.

[24]  James M. Robins,et al.  Large-sample theory for parametric multiple imputation procedures , 1998 .

[25]  A O'Hagan,et al.  Bayesian cost‐effectiveness analysis from clinical trial data , 2001, Statistics in medicine.

[26]  D. Brennan,et al.  Mapping oral health related quality of life to generic health state values , 2006, BMC Health Services Research.

[27]  Christopher McCabeRichard Constructing Indirect Utility Models: Some Observations on the Principles and Practice of Mapping to Obtain Health State Utilities , 2013 .

[28]  J. Brazier,et al.  Deriving a preference-based single index from the UK SF-36 Health Survey. , 1998, Journal of clinical epidemiology.

[29]  Bradley P. Carlin,et al.  Markov Chain Monte Carlo conver-gence diagnostics: a comparative review , 1996 .

[30]  R. Osborne,et al.  The Assessment of Quality of Life (AQoL) instrument: a psychometric measure of Health-Related Quality of Life , 1999, Quality of Life Research.

[31]  Christopher McCabe,et al.  Constructing Indirect Utility Models: Some Observations on the Principles and Practice of Mapping to Obtain Health State Utilities , 2013, PharmacoEconomics.

[32]  M. van der Pol,et al.  Mapping the EORTC QLQ C-30 onto the EQ-5D instrument: the potential to estimate QALYs without generic preference data. , 2009, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[33]  Andrew Briggs,et al.  Missing... presumed at random: cost-analysis of incomplete data. , 2003, Health economics.

[34]  Karl Claxton,et al.  Probabilistic analysis and computationally expensive models: Necessary and required? , 2006, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[35]  P. Dolan Modelling valuations for health states , 1997 .

[36]  Matt Stevenson,et al.  Monte Carlo probabilistic sensitivity analysis for patient level simulation models: efficient estimation of mean and variance using ANOVA. , 2007, Health economics.

[37]  David J. Spiegelhalter,et al.  WinBUGS user manual version 1.4 , 2003 .

[38]  Allan Wailoo,et al.  A Comparison of Direct and Indirect Methods for the Estimation of Health Utilities from Clinical Outcomes , 2014, Medical decision making : an international journal of the Society for Medical Decision Making.

[39]  Michael G Kenward,et al.  Multiple Imputation Methods for Handling Missing Data in Cost-effectiveness Analyses That Use Data from Hierarchical Studies , 2013, Medical decision making : an international journal of the Society for Medical Decision Making.

[40]  Peter C Austin,et al.  Bayesian Extensions of the Tobit Model for Analyzing Measures of Health Status , 2002, Medical decision making : an international journal of the Society for Medical Decision Making.

[41]  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.

[42]  D. Rubin,et al.  Inference from Iterative Simulation Using Multiple Sequences , 1992 .

[43]  Dennis G. Fryback,et al.  Predicting the EuroQol Group’s EQ-5D Index from CDC’s “Healthy Days” in a US Sample , 2011, Medical decision making : an international journal of the Society for Medical Decision Making.

[44]  Andrew Booth,et al.  A Review of the Use of Health Status Measures in Economic Evaluation , 1999, Journal of health services research & policy.

[45]  Ron Goeree,et al.  Probabilistic Analysis of Cost-Effectiveness Models: Choosing between Treatment Strategies for Gastroesophageal Reflux Disease , 2002, Medical decision making : an international journal of the Society for Medical Decision Making.

[46]  A. Williams EuroQol : a new facility for the measurement of health-related quality of life , 1990 .

[47]  R. Brooks EuroQol: the current state of play. , 1996, Health policy.

[48]  David Feeny,et al.  Multi-Attribute Preference Functions , 1995, PharmacoEconomics.

[49]  Andrew Bottomley,et al.  International perspective on health-related quality-of-life research in cancer clinical trials: the European Organisation for Research and Treatment of Cancer experience. , 2007, Journal of clinical oncology : official journal of the American Society of Clinical Oncology.

[50]  Oliver Rivero-Arias,et al.  Mapping the Modified Rankin Scale (mRS) Measurement into the Generic EuroQol (EQ-5D) Health Outcome , 2010, Medical decision making : an international journal of the Society for Medical Decision Making.

[51]  George Tomlinson,et al.  Predicting EQ-5D Utility Scores from the Seattle Angina Questionnaire in Coronary Artery Disease , 2011, Medical decision making : an international journal of the Society for Medical Decision Making.

[52]  Sylvia Richardson,et al.  Markov Chain Monte Carlo in Practice , 1997 .

[53]  B. McNeil,et al.  Probabilistic Sensitivity Analysis Using Monte Carlo Simulation , 1985, Medical decision making : an international journal of the Society for Medical Decision Making.

[54]  Anthony J. Culyer,et al.  Health Utilities Index , 2014 .

[55]  D. Osoba,et al.  The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. , 1993, Journal of the National Cancer Institute.

[56]  G Parmigiani,et al.  Measuring uncertainty in complex decision analysis models , 2002, Statistical methods in medical research.

[57]  Jason N Doctor,et al.  Probabilistic Mapping of Descriptive Health Status Responses Onto Health State Utilities Using Bayesian Networks: An Empirical Analysis Converting SF-12 Into EQ-5D Utility Index in a National US Sample , 2011, Medical care.