In trial-based cost-effectiveness analysis baseline mean utility values are invariably imbalanced between treatment arms. A patient's baseline utility is likely to be highly correlated with their quality-adjusted life-years (QALYs) over the follow-up period, not least because it typically contributes to the QALY calculation. Therefore, imbalance in baseline utility needs to be accounted for in the estimation of mean differential QALYs, and failure to control for this imbalance can result in a misleading incremental cost-effectiveness ratio. This paper discusses the approaches that have been used in the cost-effectiveness literature to estimate absolute and differential mean QALYs alongside randomised trials, and illustrates the implications of baseline mean utility imbalance for QALY calculation. Using data from a recently conducted trial-based cost-effectiveness study and a micro-simulation exercise, the relative performance of alternative estimators is compared, showing that widely used methods to calculate differential QALYs provide incorrect results in the presence of baseline mean utility imbalance regardless of whether these differences are formally statistically significant. It is demonstrated that multiple regression methods can be usefully applied to generate appropriate estimates of differential mean QALYs and an associated measure of sampling variability, while controlling for differences in baseline mean utility between treatment arms in the trial.
Non-UK purchasers will have to pay a small fee for post and packing. For European countries the cost is £2 per monograph and for the rest of the world £3 per monograph.You can order HTA monographs from our Despatch Agents:-fax (with credit card or official purchase order) -post (with credit card or official purchase order or cheque) -phone during office hours (credit card only).Additionally the HTA website allows you either to pay securely by credit card or to print out your order and then post or fax it. NHS libraries can subscribe free of charge. Public libraries can subscribe at a very reduced cost of £100 for each volume (normally comprising 30-40 titles). The commercial subscription rate is £300 per volume. Please see our website for details. Subscriptions can only be purchased for the current or forthcoming volume. Contact details are as follows: Payment methods Paying by chequeIf you pay by cheque, the cheque must be in pounds sterling, made payable to Direct Mail Works Ltd and drawn on a bank with a UK address. Paying by credit cardThe following cards are accepted by phone, fax, post or via the website ordering pages: Delta, Eurocard, Mastercard, Solo, Switch and Visa. We advise against sending credit card details in a plain email. Paying by official purchase orderYou can post or fax these, but they must be from public bodies (i.e. NHS or universities) within the UK.We cannot at present accept purchase orders from commercial companies or from outside the UK. How do I get a copy of HTA on CD?Please use the form on the HTA website (www.hta.ac.uk/htacd.htm). Or contact Direct Mail Works (see contact details above) by email, post, fax or phone. HTA on CD is currently free of charge worldwide.The website also provides information about the HTA Programme and lists the membership of the various committees. HTA NHS R&D HTA ProgrammeT he research findings from the NHS R&D Health Technology Assessment (HTA) Programme directly influence key decision-making bodies such as the National Institute for Clinical Excellence (NICE) and the National Screening Committee (NSC) who rely on HTA outputs to help raise standards of care. HTA findings also help to improve the quality of the service in the NHS indirectly in that they form a key component of the 'National Knowledge Service' that is being developed to improve the evidence of clinical practice throughout the NHS.The HTA Programme was set up in 1993. Its role is to ensure that high-quality research information on the costs, effectiveness and broader impact of health technologies is produced in the most efficient way for those who use, manage and provide care in the NHS. 'Health technologies' are broadly defined to include all interventions used to promote health, prevent and treat disease, and improve rehabilitation and long-term care, rather than settings of care.The HTA programme commissions research only on topics where it has identified key gaps in the evidence needed by the NHS. Suggestions for topics are actively sought from people working in the NHS, the public,...
ACKNOWLEDGMENTSThe individual contributions of Sorrel Wolowacz and Richard Willke are gratefully acknowledged. We thank all reviewers who commented during our forums at the ISPOR Milan and Amsterdam European Congresses. We especially thank the following individuals who reviewed drafts of the report and submitted written comments. Their feedback has both improved the manuscript and made it an expert consensus ISPOR task force report. Many thanks to Tony Ades, René Allard, Gouri Shankar Bhattacharyya, Lance Brannman, Michael Carter, David Cella, Akriti Chahar, Stephanie Yanjing Chen, Shiva Devarakonda, Salah Ghabri, Linda Gore Martin, Inigo Gorostiza, Thomas Grochtdreis, Michael Hagan, Nadine Hillock, Cynthia Holmes, Shrividya Iyer, Steve Kay, Jeanette Kusel, Ramanath KV, Dawn Lee, Joanna Le niowska, Sophia E. Marsh, Alan Martin, Nicholas Mitsakakis, Sharanya Murty, Nneka Onwudiwe, Guilhem Pietri, G.M. Rabiul Islam, Ghabri Salah, Mihail Samnaliev, Carsten Schousboe, Sarah Shingler, Fatema Turkistani, and Uday Venkat. Finally, many thanks to Theresa Tesoro for her assistance in developing this task force report. 2 AbstractEconomic evaluation conducted in terms of cost per Quality Adjusted Life Year (QALY) provides information that decision makers find useful in many parts of the world. Ideally, clinical studies designed to assess the effectiveness of health technologies would include outcome measures that are directly linked to health utility in order to calculate QALYs. Often this does not happen and, even where it does, clinical studies may be insufficient for a cost-utility assessment. Mapping can solve this problem. It uses an additional dataset to estimate the relationship between outcomes measured in clinical studies and health utility. This bridges the evidence gap between available evidence on the effect of a health technology in one metric and the requirement for decision makers to express it in a different one (QALYs). In 2014, ISPOR established a Good Practices for Outcome Research Task Force for mapping studies. This Task Force Report provides recommendations to analysts undertaking mapping studies, those that use the results in cost utility analysis, and those that need to critically review such studies. The recommendations cover all areas of mapping practice: the selection of datasets for the mapping estimation, model selection and performance assessment, reporting standards, and the use of results including the appropriate reflection of variability and uncertainty. This report is unique because it takes an international perspective, is comprehensive in its coverage of the aspects of mapping practice, and reflects the current state of the art.
Objectives:Health technology assessment (HTA) is increasingly an international activity, and HTA agencies collaborate to avoid unnecessary duplication of effort. However, the sharing of the results from HTAs raises questions about their generalizability; namely, are the results of an HTA undertaken in one country relevant to another?Methods:This study presents recommendations for increasing the generalizability of economic evaluations. They represent an important component of HTAs and are commonly thought to have limited generalizability.Results:Recommendations are given for studies using patient-level data (i.e., evaluations conducted alongside clinical trials) and for studies using decision analytic modeling.Conclusions:If implemented, the recommendations would increase the value for investments in HTA.
Economic evaluations alongside randomised controlled trials (RCTs) are increasingly being designed to prospectively collect patient-specific resource use and preference-based health status (utility) data. This paper examines the ways in which preference-based health status (utility) data are used to generate quality adjusted life years (QALYs). A literature review was carried out which identified 23 published cost utility analyses suitable for inclusion. The methodology employed to calculate QALYs was not always consistent, as well as being poorly reported. The use of different methodologies in the calculation of QALYs may influence the magnitude and direction of results of evaluations. Analysts need to be consistent and fully transparent in the methodology chosen to calculate QALYs.
Non-UK purchasers will have to pay a small fee for post and packing. For European countries the cost is £2 per monograph and for the rest of the world £3 per monograph.You can order HTA monographs from our Despatch Agents:-fax (with credit card or official purchase order) -post (with credit card or official purchase order or cheque) -phone during office hours (credit card only).Additionally the HTA website allows you either to pay securely by credit card or to print out your order and then post or fax it. NHS libraries can subscribe free of charge. Public libraries can subscribe at a very reduced cost of £100 for each volume (normally comprising 30-40 titles). The commercial subscription rate is £300 per volume. Please see our website for details. Subscriptions can only be purchased for the current or forthcoming volume. Contact details are as follows: Payment methods Paying by chequeIf you pay by cheque, the cheque must be in pounds sterling, made payable to Direct Mail Works Ltd and drawn on a bank with a UK address. Paying by credit cardThe following cards are accepted by phone, fax, post or via the website ordering pages: Delta, Eurocard, Mastercard, Solo, Switch and Visa. We advise against sending credit card details in a plain email. Paying by official purchase orderYou can post or fax these, but they must be from public bodies (i.e. NHS or universities) within the UK. We cannot at present accept purchase orders from commercial companies or from outside the UK. How do I get a copy of HTA on CD?Please use the form on the HTA website (www.hta.ac.uk/htacd.htm). Or contact Direct Mail Works (see contact details above) by email, post, fax or phone. HTA on CD is currently free of charge worldwide.The website also provides information about the HTA Programme and lists the membership of the various committees. HTA NHS R&D HTA ProgrammeT he research findings from the NHS R&D Health Technology Assessment (HTA) Programme directly influence key decision-making bodies such as the National Institute for Clinical Excellence (NICE) and the National Screening Committee (NSC) who rely on HTA outputs to help raise standards of care. HTA findings also help to improve the quality of the service in the NHS indirectly in that they form a key component of the 'National Knowledge Service' that is being developed to improve the evidence of clinical practice throughout the NHS.The HTA Programme was set up in 1993. Its role is to ensure that high-quality research information on the costs, effectiveness and broader impact of health technologies is produced in the most efficient way for those who use, manage and provide care in the NHS. 'Health technologies' are broadly defined to include all interventions used to promote health, prevent and treat disease, and improve rehabilitation and long-term care, rather than settings of care.The HTA programme commissions research only on topics where it has identified key gaps in the evidence needed by the NHS. Suggestions for topics are actively sought from people working in the NHS, the public...
Purpose To develop regression models for outcomes with truncated supports, such as health-related quality of life (HRQoL) data, and account for features typical of such data such as a skewed distribution, spikes at one or zero, and heteroskedasticity. Methods Regression estimators based on features of the Beta distribution. Firstly, we present both a single equation and a two-part model, along with estimation algorithms based on maximum-likelihood, quasi-likelihood and Bayesian Markov-chain Monte Carlo methods. A novel Bayesian quasi-likelihood estimator is proposed. Secondly, we present a simulation exercise to assess the performance of the proposed estimators against ordinary least squares (OLS) regression for a variety of HRQoL distributions that are encountered in practice. Finally, we assess the performance of the proposed estimators by using them to quantify the treatment effect on QALYs in the EVALUATE hysterectomy trial. Overall model fit is studied using several goodness-of-fit tests such as Pearson’s correlation test, Link and Reset tests and a modified Hosmer-Lemeshow test. Results Our simulation results indicate that the proposed methods are more robust in estimating covariate effects than OLS, especially when the effects are large or the HRQoL distribution has a large spike at one. Quasi-likelihood techniques are more robust than maximum likelihood estimators. When applied to the EVALUATE trial, all but the maximum likelihood estimators produce unbiased estimates of the treatment effect. Conclusion One and two-part Beta regression models provide flexible approaches to regress the outcomes with truncated supports, such as HRQoL, on covariates, after accounting for many idiosyncratic features of the outcomes distribution. This work will provide applied researchers with a practical set of tools to model outcomes in cost-effectiveness analysis.
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