From Health to Well-Being: Toward a Monetary Valuation of a Well-Being-Adjusted Life-Year.

OBJECTIVES: Economic evaluations using broader measures to capture benefits beyond improved health can inform policy making, but only if the monetary value of gains measured using these instruments is understood. This study explored contingent valuation as a method to estimate the monetary value of a well-being-adjusted life-year (WALY) as measured by ICEpop Capability Measure for Adults (ICECAP-A). METHODS: In a large online survey of representative samples from 7 European countries, participants valued a change in the ICECAP-A from their current health state to a randomly assigned hypothetical state. Participants were instructed that an unspecified treatment could avoid a loss or produce a gain in well-being and were asked for their willingness to pay (WTP) for this treatment. WTP per WALY was calculated using an aggregated approach that used ICECAP-A tariffs from the United Kingdom. RESULTS: We analyzed a sample of 7428 observations, focusing on avoided losses (n = 6002) because the results for gains were not theoretically valid. Different cutoff points for a marginal change were explored. Depending on the definition of a marginal change, WTP per WALY averaged between €13 323.28 and €61 375.63 for avoided losses between [0, 0.5] and [0, 0.1], respectively, for 1 month. Mean WTP per WALY varied across the countries as follows: Denmark (€17 867.93-€88 634.14), France (€10 278.35-€45 581.28), Germany (€12 119.39-€54 566.56), Italy (€11 753.69-€52 161.25), The Netherlands (€14 612.88-€58 951.74), Spain (€11 904.12-€57 909.17), and United Kingdom (€13 133.75-€68 455.85). CONCLUSION: Despite the inherent limitations of our study, it offers valuable insights into methods for eliciting the WTP for changes in capability well-being as measured with ICECAP-A.

Construct Validity, Reliability, and Responsiveness of the 10-Item Well-Being Instrument for Use in Economic Evaluation Studies.

OBJECTIVES: Economic evaluations of interventions in health and social care require outcome measures that capture their full benefits, including those beyond health. This study aimed to assess construct validity, test-retest reliability, and responsiveness of the newly developed 10-item Well-being instrument (WiX). METHODS: Data were gathered via an online survey in a representative sample of the adult general population in The Netherlands (N = 1045). Construct validity was assessed by inspecting convergent, structural, and discriminant validity, following the COnsensus-based Standards for the selection of health status Measurement INstruments methodology. Regression analyses of the WiX and its items on other validated measures of well-being were performed to assess the convergent validity of the instrument and the relevance of its items. Dimensionality of the WiX was assessed using exploratory factor analysis. To assess discriminant validity, several hypotheses in terms of well-being differences were assessed. Finally, a second survey was sent out 2 weeks after the initial survey (n = 563; 53.9% response rate) to assess the test-retest reliability and responsiveness of the WiX. RESULTS: The WiX showed to be correlated with alternative well-being measures as expected and able to sufficiently differentiate between relevant subgroups in the population. Moreover, the dimensionality analysis indicated that the WiX captures a broad array of elements relevant to well-being, including physical and mental health. The test-retest reliability was good, with an intraclass correlation coefficient of 0.82. CONCLUSIONS: The results regarding the WiX are favorable and indicate that this new instrument may be a promising alternative for existing measures of well-being for evaluating interventions in health and social care.

The cost-effectiveness of seizure dogs for persons living with severe refractory epilepsy: results from the EPISODE study.

OBJECTIVES: The EPISODE study was commissioned by the Dutch Ministry of Health, Welfare and Sports to inform a reimbursement decision on seizure dogs. The randomized trial found that seizure dogs reduce seizure frequency and improve health-related quality of life of persons with severe refractory epilepsy (PSREs). This paper examines the cost-effectiveness of adding seizure dogs to usual care for PSREs in the Netherlands. METHODS: A microsimulation model was developed, informed by generalized linear mixed models using patient level trial data from the EPISODE study. The model adopted a 10-year time horizon and took a societal perspective. Seizure frequency was predicted as a function of time with the seizure dog. Patient utilities, caregiver utilities and costs were predicted as a function of seizure frequency and time with the seizure dog. RESULTS: Quality-adjusted life years (QALYs) of PSREs with a seizure dog and usual care alone were estimated at 6.28 and 5.65 respectively (Δ 0.63). For caregivers, estimated QALYs were 6.94 and 6.52, respectively (Δ 0.42). Total costs were respectively €228,691 and €226,261 (Δ €2,430). Intervention costs were largely offset by savings in informal care and healthcare. The incremental cost-effectiveness ratio (ICER) was €2,314/QALY. Probabilistic sensitivity analysis indicated a 91% probability of seizure dogs being cost-effective at the €50,000/QALY threshold. The ICER fell well below this threshold in scenario analyses. CONCLUSIONS: Seizure dogs are likely to be a cost-effective addition to usual care for PSREs in the Netherlands.

The role of budget impact and its relationship with cost-effectiveness in reimbursement decisions on health technologies in the Netherlands.

Health authorities using cost-effectiveness analysis (CEA) for informing reimbursement decisions on health technologies increasingly require economic evaluations encompassing both CEA and budget impact analysis (BIA). Good Research Practices advocate that the economic and clinical assumptions underlying these analyses are aligned and consistently applied. Nonetheless, CEAs and BIAs often are stand-alone analyses used in different stages of the decision-making process. This article used policy reports and Ministerial correspondence to discuss and elucidate the role of budget impact and its relationship with cost-effectiveness in reimbursement decisions in the Netherlands. The results indicate that CEAs and BIAs are both considered important for informing these decisions. While the requirements regarding CEAs-and application of the associated decision rule-are consistent across the different stages, the same does not hold for BIAs. Importantly, the definition of and evidence on budget impact differs between stages. Some important aspects (e.g. substitution and saving effects) typically are considered in the assessment and appraisal stages but are seemingly not considered in price negotiations and the final reimbursement decision. Further research is warranted to better understand why BIAs are not aligned with CEAs (e.g. in terms of underlying assumptions), vary in form and importance between stages, and do not have a clear relationship with the results of CEAs in the decision-making framework. Improving the understanding of the circumstances under which decision-makers attach a relatively larger or smaller weight to (different aspects of) budget impact may contribute to increasing the transparency, consistency, and optimality of reimbursement decisions in the Netherlands.

On spillovers in economic evaluations: definition, mapping review and research agenda.

An important issue in economic evaluations is determining whether all relevant impacts are considered, given the perspective chosen for the analysis. Acknowledging that patients are not isolated individuals has important implications in this context. Increasingly, the term "spillovers" is used to label consequences of health interventions on others. However, a clear definition of spillovers is lacking, and as a result, the scope of the concept remains unclear. In this study, we aim to clarify the concept of spillovers by proposing a definition applicable in health economic evaluations. To illustrate the implications of this definition, we highlight the diversity of potential spillovers through an expanded impact inventory and conduct a mapping review that outlines the evidence base for the different types of spillovers. In the context of economic evaluations of health interventions, we define spillovers as all impacts from an intervention on all parties or entities other than the users of the intervention under evaluation. This definition encompasses a broader range of potential costs and effects, beyond informal caregivers and family members. The expanded impact inventory enables a systematic approach to identifying broader impacts of health interventions. The mapping review shows that the relevance of different types of spillovers is context-specific. Some spillovers are regularly included in economic evaluations, although not always recognised as such, while others are not. A consistent use of the term "spillovers", improved measurement of these costs and effects, and increased transparency in reporting them are still necessary. To that end, we propose a research agenda.

Examining the Effect of Depicting a Patient Affected by a Negative Reimbursement Decision in Healthcare on Public Disagreement with the Decision.

BACKGROUND: The availability of increasingly advanced and expensive new health technologies puts considerable pressure on publicly financed healthcare systems. Decisions to not-or no longer-reimburse a health technology from public funding may become inevitable. Nonetheless, policymakers are often pressured to amend or revoke negative reimbursement decisions due to the public disagreement that typically follows such decisions. Public disagreement may be reinforced by the publication of pictures of individual patients in the media. Our aim was to assess the effect of depicting a patient affected by a negative reimbursement decision on public disagreement with the decision. METHODS: We conducted a discrete choice experiment in a representative sample of the public (n = 1008) in the Netherlands and assessed the likelihood of respondents' disagreement with policymakers' decision to not reimburse a new pharmaceutical for one of two patient groups. We presented a picture of one of the patients affected by the decision for one patient group and "no picture available" for the other group. The groups were described on the basis of patients' age, health-related quality of life (HRQOL) and life expectancy (LE) before treatment, and HRQOL and LE gains from treatment. We applied random-intercept logit regression models to analyze the data. RESULTS: Our results indicate that respondents were more likely to disagree with the negative reimbursement decision when a picture of an affected patient was presented. Consistent with findings from other empirical studies, respondents were also more likely to disagree with the decision when patients were relatively young, had high levels of HRQOL and LE before treatment, and large LE gains from treatment. CONCLUSIONS: This study provides evidence for the effect of depicting individual, affected patients on public disagreement with negative reimbursement decisions in healthcare. Policymakers would do well to be aware of this effect so that they can anticipate it and implement policies to mitigate associated risks.

Estimating a Preference-Based Value Set for the Mental Health Quality of Life Questionnaire (MHQoL).

BACKGROUND: Health economic evaluations using common health-related quality of life measures may fall short in adequately measuring and valuing the benefits of mental health care interventions. The Mental Health Quality of Life questionnaire (MHQoL) is a standardized, self-administered mental health-related quality of life instrument covering 7 dimensions known to be relevant across and valued highly by people with mental health problems. The aim of this study was to derive a Dutch value set for the MHQoL to facilitate its use in cost-utility analyses. METHODS: The value set was estimated using a discrete choice experiment (DCE) with duration that accommodated nonlinear time preferences. The DCE was embedded in a web-based self-complete survey and administered to a representative sample (N = 1,308) of the Dutch adult population. The matched pairwise choice tasks were created using a Bayesian heterogeneous D-efficient design. The overall DCE design comprised 10 different subdesigns, with each subdesign containing 15 matched pairwise choice tasks. Each participant was asked to complete 1 of the subdesigns to which they were randomly assigned. RESULTS: The obtained coefficients indicated that "physical health,""mood," and "relationships" were the most important dimensions. All coefficients were in the expected direction and reflected the monotonic structure of the MHQoL, except for level 2 of the dimension "future." The predicted values for the MHQoL ranged from -0.741 for the worst state to 1 for the best state. CONCLUSIONS: This study derived a Dutch value set for the recently introduced MHQoL. This value set allows for the generation of an index value for all MHQoL states on a QALY scale and may hence be used in Dutch cost-utility analyses of mental healthcare interventions. HIGHLIGHTS: A discrete choice experiment was used to derive a Dutch value set for the MHQoL.This allows the use of the MHQoL in Dutch cost-utility analyses.The dimensions physical health, mood, and relationships were the most important.The utility values range from -0.741 for the worst state to 1 for the best state.

Effectiveness of Seizure Dogs for People With Severe Refractory Epilepsy: Results From the EPISODE Study.

BACKGROUND AND OBJECTIVES: The aim of this study was to evaluate whether people living with severe medically refractory epilepsy (PSRE) benefit from a seizure dog. METHODS: An individual-level stepped-wedge randomized controlled trial was conducted. The study was conducted in the Netherlands among adults with daily to weekly seizures. All participants were included simultaneously (on June 1, 2019) while receiving usual care. Then, during the 36-month follow-up, they received a seizure dog in a randomized sequence. Participants kept a seizure diary and completed 3-monthly surveys. Seizure frequency was the primary outcome. Secondary outcomes included seizure-free days, seizure severity, health-related quality of life (HRQoL), and well-being. Data were analyzed using generalized linear mixed modeling (GLMM). The models assumed a delayed intervention effect, starting when the seizure dog reached an advanced stage of training. Effects were calculated as changes per 28-day period with the intervention. RESULTS: Data were collected from 25 participants, of whom 20 crossed over to the intervention condition. The median follow-up was 19 months with usual care and 12 months with the intervention. On average, participants experienced 115 (SD 164) seizures per 28-day period in the usual care condition and 73 (SD 131) seizures in the intervention condition. Seven participants achieved a reduction of 50% or more at the end of follow-up. GLMM indicated a 3.1% decrease in seizure frequency for each consecutive 28-day period with the intervention (0.969, 95% CI 0.960-0.977). Furthermore, an increase in the number of seizure-free days was observed (1.012, 95% CI 1.009, 1.015), but no effect on seizure severity measured with the NHS3. Generic HRQoL scores improved, as reflected in the decrease in EQ-5D-5L utility decrement (0.975, 95% CI 0.954-0.997). Smaller improvements were observed on overall self-rated HRQoL, epilepsy-specific HRQoL, and well-being, measured with the EQ VAS, QOLIE-31-P, and ICECAP-A, respectively. DISCUSSION: Seizure dogs reduce seizure frequency, increase the number of seizure-free days, and improve the quality of life of PSRE. The magnitude of the effect on generic HRQoL indicates that seizure dogs benefit PSRE beyond the impact on seizure frequency alone. Early discontinuation of seizure dog partnerships suggests that this intervention is not suitable for all PSRE and requires further study. TRIAL REGISTRATION INFORMATION: This study was registered in the Dutch Trial Register (NL6682) on November 28, 2017. Participants were enrolled on June 1, 2019. CLASSIFICATION OF EVIDENCE: This study provides Class III evidence that seizure dogs are associated with a decrease in seizure frequency in adult patients with medically refractory epilepsy.