Nancy Devlin

Valuing health-related quality of life: An EQ-5D-5L value set for England

Abstract A new version of the EQ‐5D, the EQ‐5D‐5L, is available. The aim of this study is to produce a value set to support use of EQ‐5D‐5L data in decision‐making. The study design followed an international research protocol. Randomly selected members of the English general public completed 10 time trade‐off and 7 discrete choice experiment tasks in face‐to‐face interviews. A 20‐parameter hybrid model was used to combine time trade‐off and discrete choice experiment data to generate values for the 3,125 EQ‐5D‐5L health states. Valuation data are available for 996 respondents. Face validity of the data has been demonstrated, with more severe health states generally given lower values. Problems with pain/discomfort and anxiety/depression received the greatest weight. Compared to the existing EQ‐5D‐3L value set, there are considerably fewer “worse than dead” states (5.1%, compared with over one third), and the minimum value is higher. Values range from −0.285 (extreme problems on all dimensions) to 0.950 (for health states 11211 and 21111). Results have important implications for users of the EQ‐5D‐5L both in England and internationally. Quality‐adjusted life year gains from interventions seeking to improve very poor health may be smaller using this value set and may previously have been overestimated.

A program of methodological research to arrive at the new international eq-5d-5l valuation protocol

OBJECTIVES To describe the research that has been undertaken by the EuroQol Group to improve current methods for health state valuation, to summarize the results of an extensive international pilot program, and to outline the key elements of the five-level EuroQol five-dimensional (EQ-5D-5L) questionnaire valuation protocol, which is the culmination of that work. METHODS To improve on methods of health state valuation for the EQ-5D-5L questionnaire, we investigated the performance of different variants of time trade-off and discrete choice tasks in a multinational setting. We also investigated the effect of three modes of administration on health state valuation: group interviews, online self-completion, and face-to-face interviews. RESULTS The research program provided the basis for the EQ-5D-5L questionnaire valuation protocol. Two different types of tasks are included to derive preferences: a newly developed composite time trade-off task and a forced-choice paired comparisons discrete choice task. Furthermore, standardized blocked designs for the selection of the states to be valued by participants were created and implemented together with all other elements of the valuation protocol in a digital aid, the EuroQol Valuation Technology, which was developed in conjunction with the protocol. CONCLUSIONS The EuroQol Group has developed a standard protocol, with accompanying digital aid and interviewer training materials, that can be used to create value sets for the EQ-5D-5L questionnaire. The use of a well-described, consistent protocol across all countries enhances the comparability of value sets between countries, and allows the exploration of the influence of cultural and other factors on health state values.

The development of new research methods for the valuation of EQ-5D-5L

The EQ-5D is arguably now the most well-known and commonly used generic measure of health status internationally. It is available in 169 languages, with applications in clinical, cost-effectiveness and population health studies, as well as (more recently) its routine use by health-care systems. A key feature of the EQ-5D is the availability of ‘value sets’ to weight the EQ-5D health states reported by patients and populations. These value sets provide, for each of the 243 health states described by the EQ-5D, a value (‘utility’) on a scale anchored at 1 (full health) and 0 (dead), reflecting the preferences of the general public, which can be used to estimate quality-adjusted life years (QALYs). These value sets are widely used in the analysis of EQ-5D data and inform a wide range of resource allocation decisions. Value sets for the 3-level version of the EQ-5D (3L) are available

The Influence of Cost‐Effectiveness and Other Factors on Nice Decisions

The National Institute for Health and Care Excellence (NICE) emphasises that cost-effectiveness is not the only consideration in health technology appraisal and is increasingly explicit about other factors considered relevant but not the weight attached to each. The objective of this study is to investigate the influence of cost-effectiveness and other factors on NICE decisions and whether NICEs decision-making has changed over time. We model NICEs decisions as binary choices for or against a health care technology in a specific patient group. Independent variables comprised of the following: clinical and economic evidence; characteristics of patients, disease or treatment; and contextual factors potentially affecting decision-making. Data on all NICE decisions published by December 2011 were obtained from HTAinSite [www.htainsite.com]. Cost-effectiveness alone correctly predicted 82% of decisions; few other variables were significant and alternative model specifications had similar performance. There was no evidence that the threshold has changed significantly over time. The model with highest prediction accuracy suggested that technologies costing £40 000 per quality-adjusted life-year (QALY) have a 50% chance of NICE rejection (75% at £52 000/QALY; 25% at £27 000/QALY). Past NICE decisions appear to have been based on a higher threshold than £20 000-£30 000/QALY. However, this may reflect consideration of other factors that cannot be easily quantified.

Multiple Criteria Decision Analysis for Health Care Decision Making—Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force

Health care decisions are complex and involve confronting trade-offs between multiple, often conflicting objectives. Using structured, explicit approaches to decisions involving multiple criteria can improve the quality of decision making. A set of techniques, known under the collective heading, multiple criteria decision analysis (MCDA), are useful for this purpose. In 2014, ISPOR established an Emerging Good Practices Task Force. The task forces first report defined MCDA, provided examples of its use in health care, described the key steps, and provided an overview of the principal methods of MCDA. This second task force report provides emerging good-practice guidance on the implementation of MCDA to support health care decisions. The report includes: a checklist to support the design, implementation and review of an MCDA; guidance to support the implementation of the checklist; the order in which the steps should be implemented; illustrates how to incorporate budget constraints into an MCDA; provides an overview of the skills and resources, including available software, required to implement MCDA; and future research directions.

Protocols for Time Tradeoff Valuations of Health States Worse than Dead: A Literature Review

Background. The time tradeoff (TTO) method of preference elicitation allows respondents to value a state as worse than dead, generally either through the Torrance protocol or the Measurement and Valuation of Health (MVH) protocol. Both of these protocols have significant weaknesses: Valuations for states worse than dead (SWD) are elicited through procedures different from those for states better than dead (SBD), and negative values can be extremely negative. Purpose. To provide an account of the different TTO designs for SWD, to identify any alternatives to the MVH and Torrance approaches, and to consider the merits of the approaches identified. Methods. Medline was searched to identify all health state valuation studies employing TTO. The ways in which SWD were handled were recorded. Furthermore, to ensure that there are no unpublished but feasible TTO variants, the authors developed a theoretical framework for identifying all potential variants. Results. The search produced 593 hits, of which 218 were excluded. Of the remaining 375 articles, only 29 included protocols for SWD. Of these, 23 used the MVH protocol and 4 used the Torrance protocol. The other 2 used 1 protocol for SBD and SWD, one making use of lead time and the other using a 2-stage procedure with chaining. The systematic framework did not identify any alternatives to the Torrance and MVH protocols that were superior to the lead time approach. Conclusions. Few studies elicit values for SWD. The lead time approach is a potential alternative to the Torrance and MVH protocols. Key words: QALY; states worse than dead; health state valuation; preference elicitation.

Operationalizing Value-Based Pricing of Medicines

The UK Government is proposing a novel form of price regulation for branded medicines, which it has dubbed ‘value-based pricing’ (VBP). The specifics of how VBP will work are unclear. We provide an account of the possible means by which VBP of medicines might be operationalized, and a taxonomy to describe and categorize the various approaches. We begin with a brief discussion of the UK Government’s proposal for VBP and proceed to define a taxonomy of approaches to VBP. The taxonomy has five main dimensions: (1) what is identified as being of value, (2) how each element is measured, (3) how it is valued, (4) how the different elements of value are aggregated, and (5) how the result is then used to determine the price of a medicine. We take as our starting point that VBP will include a measure of health gain and that, as proposed by the UK Government, this will be built on the QALY. Our principal interest is in the way criteria other than QALYs are taken into account, including severity of illness, the extent of unmet need, and wider societal considerations such as impacts on carers. We set out to: (1) identify and describe the full range of alternative means by which ‘value’ might be measured and valued, (2) identify and describe the options available for aggregating the different components of value to establish a maximum price, and (3) note the challenges and relative advantages associated with these approaches. We review the means by which aspects of VBP are currently operationalized in a selection of countries and place these, and proposals for the UK, in the context of our taxonomy. Finally, we give an initial assessment of the challenges, pros and cons of each approach. We conclude that identifying where VBP should lie on each of the five dimensions entails value judgements: there are no simple ‘right or wrong’ solutions. If a wider definition of value than incremental QALYs gained is adopted, as is desirable, then a pragmatic way to aggregate the different elements of value, including both QALYs and benefits unrelated to QALYs, is to use a multi-criteria decision analysis (MCDA) approach. All approaches to VBP ultimately require the conversion of value, however assessed, into a monetary price. This requires assessment of the marginal values of all types of benefit, not just of QALYs. All stages of the VBP process are subject to uncertainty and margins of error. Consequently, the assessment of overall value can provide bounds to a price negotiation but cannot be expected to identify a precise value-based price.

Operationalising Value Based Pricing of Medicines: A Taxonomy of Approaches

The purpose of this paper is to provide an account of the full set of possible means by which value based pricing (VBP) might be operationalised; to describe and categorise them by developing a taxonomy of approaches; to give an initial assessment of the challenges, pros and cons that each of the principal types of approach implies. To achieve this, we review the elements of value that could be taken into account, how they might be measured and valued, how the different elements could be combined into an overall assessment of a medicine’s value, and how that then could be linked to the maximum price the health service is willing to reimburse.The UK Department of Health’s consultation document regarding the introduction of VBP (DH, 2010a) outlines one possible approach to these steps – but others are possible. We begin with a brief discussion of value in economics and theoretical frameworks from economics relevant to the normative question of which attributes of medicines should be taken into account in VBP. We proceed to outline a taxonomy of approaches to VBP, taking as our starting point that VBP will include a measure of health gain and that this will be built on the QALY. Our principal interest is in the way criteria other than QALYs are taken into account. We set out to: (i) identify and describe the full range of alternative means by which “value” might be measured and valued, (ii) identify and describe the options available for aggregating the different components of value to establish a maximum price, and (iii) note the challenges and relative advantages associated with these approaches. Finally, we review the means by which VBP is currently operationalised in a selection of countries and place these, and proposals for the UK, in the context of our taxonomy.

Binary Choice Health State Valuation and Mode of Administration: Head-to-Head Comparison of Online and CAPI

Background Health state valuation exercises can be conducted online, but the quality of data generated is unclear. Objective To investigate whether responses to binary choice health state valuation questions differ by administration mode: online versus face to face. Methods Identical surveys including demographic, self-reported health status, and seven types of binary choice valuation questions were administered in online and computer-assisted personal interview (CAPI) settings. Samples were recruited following procedures employed in typical online or CAPI studies. Analysis included descriptive comparisons of the distribution of responses across the binary options and probit regression to explain the propensity to choose one option across modes of administration, controlling for background characteristics. Results Overall, 422 (221 online; 201 CAPI) respondents completed a survey. There were no overall age or sex differences. Online respondents were educated to a higher level than were the CAPI sample and general population, and employment status differed. CAPI respondents reported significantly better general health and health/life satisfaction. CAPI took significantly longer to complete. There was no effect of the mode of administration on responses to the valuation questions, and this was replicated when demographic differences were controlled. Conclusions The findings suggest that both modes may be equally valid for health state valuation studies using binary choice methods (e.g., discrete choice experiments). There are some differences between the observable characteristics of the samples, and the groups may differ further in terms of unobservable characteristics. When designing health state valuation studies, the advantages and disadvantages of both approaches must be considered.

Multinational evidence of the applicability and robustness of discrete choice modeling for deriving EQ-5D-5L health-state values.

Aims:To investigate the feasibility of discrete choice experiments for valuing EQ-5D-5L states using computer-based data collection, the consistency of the estimated regression coefficients produced after modeling the preference data, and to examine the similarity of the values derived across countries. Methods:Data were collected in Canada, England, The Netherlands, and the United States (US). Interactive software was developed to standardize the format of the choice tasks across countries, except for face-to-face interviewing in England. The choice task required respondents to choose between 2 suboptimal health states. A Bayesian design was used to generate 200 pairs of states that were randomly grouped into 20 blocks. Each respondent completed 1 block of 10 pairs. A main-effects probit model was used to estimate regression coefficients and to derive values. Results:Approximately 400 respondents participated from each country. The mean time to perform 1 choice task was between 29.2 (US) and 45.2 (England) seconds. All regression coefficients were statistically significant, except level 2 for Usual Activities in The Netherlands (P=0.51). Predictions for the complete set of 3125 EQ-5D-5L health states were similar for the 4 countries. Intraclass correlation coefficients between the countries were high: from 0.80 (England vs. US) through 0.98 (Canada vs. US). Conclusions:Derivation of value sets from the general population using computer-based choice tasks for the EQ-5D-5L is feasible. Parameter estimates were generally consistent and logical, and health-state values were similar across the 4 countries.