The European Journal of Health Economics最新文献

Purpose

Generic preferenced-based measures, such as EQ-5D-3L, that are used to estimate quality adjusted life years (QALYs) for economic evaluation are not always available in clinical trials. Predicting EQ-5D-3L values from the commonly used Patient Health Questionnaire 9 (PHQ-9) and Generalised Anxiety Disorder-7 (GAD-7) would allow estimation of QALYs from such trials. The aim was to provide mapping functions to estimate EQ-5D-3L from PHQ-9 and GAD-7 to facilitate economic evaluation.

Methods

Data was drawn from four trials of patients with symptoms of depression testing collaborative care or computerised cognitive behavioural therapy. Patients completed PHQ-9, GAD-7, and EQ-5D-3L at different timepoints. Mapping was undertaken using adjusted limited dependent variable mixture models (ALDVMM), ordinary least squares (OLS), and Tobit models based on PHQ-9, GAD-7 scores or questions, and age to predict EQ-5D-3L utilities. Models were selected based on mean error (ME), mean absolute error (MAE), root mean squared error (RMSE), model goodness of fit, and visual inspection of the predictions.

Results

There were 5583 and 3942 observations for EQ-5D-3L combined with PHQ-9 and GAD-7 respectively. ALDVMM models had low ME ( ≤|0.0018|) and MAE ranging from 0.189 to 0.192, while RMSE was from 0.251 to 0.254 and had better predictions than OLS and Tobit models. ALDVMM models with four components based on PHQ-9 and GAD-7 scores are recommended for estimating EQ-5D-3L utilities.

Conclusions

Recommended mapping functions provide users with an approach to estimate EQ-5D-3L utilities for economic evaluation using PHQ-9, GAD-7, or both scores where they have been used together.

Introduction

Abemaciclib is an oral inhibitor of cyclin-dependent kinases 4 and 6 (CDK4/6). Data from the clinical trial monarchE (2023) showed improved survival from invasive disease. The aim of the present article was to conduct an economic assessment of adjuvant treatment with abemaciclib in women with luminal, HER2- and node-positive breast cancer.

Methods

A Markov model was constructed with four mutually exclusive health states (disease-free, local recurrence, distal recurrence and death). Analyses were based on the clinical trial monarchE which compared an intervention group (abemaciclib + hormone therapy [HT]) with HT alone. The effectiveness measure used was quality-adjusted life years (QALY), with unit costs and utilities being obtained from existing literature. The incremental cost–utility ratio (ICUR) was used to compare the two treatment strategies.

Results

Total costs were €98,765 and €17,935 for the abemaciclib plus HT group and the HT alone group, respectively. The health outcome was 10.076QALY for the intervention group and 9.495QALY for the control group, with the ICUR being€139,173/QALY.

Conclusion

Despite the significant gains of abemaciclib as adjuvant treatment in terms of progression-free survival, this treatment is not cost-effective for the Spanish National Health System at published prices. It may be cost-effective with an appropriate discount on the official price.

Background

Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder. Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions.

Objectives

To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA.

Methods

Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007–2018), matched to a reference cohort grouped into four SMA types (1, 2, 3, unspecified adult onset [UAO]). HCRU included inpatient admissions, outpatient visits, procedures, and dispensed medications. Direct medical costs were estimated by multiplying HCRU by respective unit costs. Average annual HCRU and medical costs were modelled for SMA versus reference cohorts to estimate differences attributable to the disease (i.e., average treatment effect estimand). The trajectory of direct costs over time were assessed using synthetic cohorts.

Results

We identified 290 SMA patients. Annualised HCRU was higher in SMA patients compared with reference cohorts. Highest risk ratios were observed for inpatient overnight stays for type 1 (risk ratio [RR]: 29.2; 95% confidence interval [CI]: 16.0, 53.5) and type 2 (RR: 23.3; 95% CI: 16.4,33.1). Mean annual direct medical costs per patient for each year since first diagnosis were greatest for type 1 (€114,185 and SMA-attributable: €113,380), type 2 (€61,876 and SMA-attributable: €61,237), type 3 (€45,518 and SMA-attributable: €44,556), and UAO (€4046 and SMA-attributable: €2098). Costs were greatest in the 2–3 years after the first diagnosis for all types.

Discussion and conclusion

The economic burden attributable to SMA is significant. Further research is needed to understand the burden in other European countries and the impact of new treatments.

Background

There is a dearth of research on the cost-effectiveness of intensive home treatment (IHT), an alternative to psychiatric hospitalisation for patients experiencing psychiatric crises. We therefore present a health economic evaluation alongside a pre-randomised controlled trial of IHT compared to care as usual (CAU).

Method

Patients were pre-randomised to IHT or CAU using a double-consent open-label Zelen design. For the cost-utility analysis, the EuroQol 5-dimensional instrument was used. The cost-effectiveness was assessed using the Brief Psychiatric Rating Scale (BPRS).

Results

Data of 198 patients showed that each additional QALY gained from offering IHT instead of CAU was on average associated with an extra cost of €48,003. There is a 38% likelihood that IHT will lead to more QALYs at lower costs compared to CAU. An improvement of one additional point on the BPRS by offering IHT instead of CAU was associated with an extra cost of €19,203. There is a 38% likelihood that IHT will lead to higher BPRS score improvements at lower costs. Based on the NICE willingness-to-pay threshold of £30,000 (€35,000) per QALY, IHT could potentially be considered cost-effective with a likelihood of 55–60% when viewed from a societal perspective, and > 75% from a health care perspective.

Conclusions

IHT appears slightly more attractive in terms of cost-utility and cost-effectiveness than CAU, although differences in both costs and effects are small especially when viewed from the societal costs perspective. From the health care sector costs perspective, IHT has a higher probability of being cost-effective compared to CAU.

Trials registration

Netherlands Trial Register: NTR6151.

Purpose

The recently developed EQ Health and Wellbeing Instrument (EQ-HWB) is a broad, generic measure of quality-of-life designed to be suitable for caregivers. The aim of this study was to investigate performance and validity of the 9-item version (EQ-HWB-S) for caregivers where families had experienced adverse-life-events.

Methods

Using survey data from caregivers of children aged 0–8 years attending a community-health centre in 2021–2022, the general performance, feasibility, convergent and known-group validity, responsiveness-to-change, and test–retest reliability of the EQ-HWB-S was assessed. Twelve semi-structured interviews were conducted with survey respondents to assess acceptability and content validity.

Results

The sample included 234 caregivers at baseline (81% female, mean age 36-years, 38% Australian-born) and 190 at 6-months follow-up. Most EQ-HWB-S item responses were evenly spread, except for ‘Mobility’. The instrument showed good convergent validity with psychological distress (Kessler 6 (K6)) and personal-wellbeing (PWI-A) scales. EQ-HWB-S level sum-scores and preference-weighted scores were significantly different in all known-group analyses, in expected directions, and the instrument was responsive to change. For test–retest reliability, Intraclass Correlation Coefficients were excellent and individual item Kappa scores were moderate. The instrument was well received by interviewees who found the questions clear and relevant. The items were appropriate for parents experiencing adversity and carers of children with additional needs.

Conclusion

The EQ-HWB-S appeared valid, responsive to change, feasible, and well accepted by caregivers. By demonstrating the validity of the EQ-HWB-S in this hard-to-reach population of caregivers in families experiencing adverse events, this study adds to existing international evidence supporting its use.

In the generalized context of general practitioner shortages and transitions towards team-based primary care, we investigated how the different practising models relate to general practitioners’ labour supply. More precisely, we analysed the association between practice models—solo, groups of general practitioners, and multiprofessional groups—and their reported labour supply and level of satisfaction with work-life balance. We used a French cross-sectional survey from 2018 that surveyed a representative national sample of 3,032 self-employed general practitioners about their working conditions. We found that the model of practice was significantly associated with differences in physician labour supply at the intensive margin and that group practice appeared to be positively associated with general practitioners’ reported satisfaction with work-life balance. In terms of weekly working time, only practice in groups of general practitioners was associated with a significantly lower labour supply. However, general practitioners in groups–whether groups of general practitioners or multiprofessional groups–reported more annual leave and seemed more willing to diversify their activity by devoting more time to secondary activities, including salaried activities. Consistently, general practitioners working in groups were also more likely than solo practitioners to report being satisfied with their work-life balance. Although group practice, whether multiprofessionnal or not, seems to be well suited to meeting the new aspirations of general practitioners, those working in multiprofessional groups are associated with a higher level of weekly work supply, which might justify special attention from the public authorities.

Abstract

Background

Policymakers use clinical and cost-effectiveness evidence to support decisions about health service commissioning. In England, the National Institute for Health and Care Excellence (NICE) recommend that in cost-effectiveness analyses “effectiveness” is measured as quality-adjusted life years (QALYs), derived from health utility values. The impact of perinatal death (stillbirth/neonatal death) on parents’ health utility is currently unknown. This knowledge would improve the robustness of cost-effectiveness evidence for policymakers.

Objective

This study aimed to estimate the impact of perinatal death on parents’ health utility.

Methods

An online survey conducted with mothers and fathers in England who experienced a perinatal death. Participants reported how long ago their baby died and whether they/their partner subsequently became pregnant again. They were asked to rate their health on the EQ-5D-5L instrument (generic health measure). EQ-5D-5L responses were used to calculate health utility values. These were compared with age-matched values for the general population to estimate a utility shortfall (i.e. health loss) associated with perinatal death.

Results

There were 256 survey respondents with a median age of 40 years (IQR 26–40). Median time since death was 27 months (IQR 8–71). The mean utility value of the sample was 0.774 (95% CI 0.752–0.796). Utility values in the sample were 13% lower than general population values (p < 0.05). Over 10 years, this equated to a loss of 1.1 QALYs. This reduction in health utility was driven by anxiety and depression.

Conclusions

Perinatal death has important and long-lasting health impacts on parents. Mental health support following perinatal bereavement is especially important.

Objectives

Health state valuation assumes that respondents trade off between all aspects of choice tasks and maximize their utility. Yet, respondents may use heuristic valuation processes, i.e., strategies to simplify or avoid the trade-offs that are core to health state valuation. The objective of this study is to explore if heuristic valuation processes are more prevalent for valuation from a 10-year-old child’s perspective compared to the use of an adult perspective.

Methods

We reused existing data in which EQ-5D health states were valued from adult and child perspectives with composite time trade-off (cTTO) and discrete choice experiment (DCE) tasks. Our analyses focused on comparing completion time and responding patterns across both perspectives. We also explored how reflective of a set of heuristic strategies respondents’ choices were in both perspectives.

Results

We found no evidence for systematic differences in completion time across perspectives. Generally, we find different responding patterns in child perspectives, e.g., more speeding, dominance violations, and clustering of utilities at 1.0, 0.8, and 0. Very few heuristic strategies provide a coherent explanation for the observed DCE responses.

Conclusion

Our results provide some, albeit indirect, evidence for differences in heuristic valuation processes between perspectives, although not across all data sources. Potential effects of heuristic valuation processes, such as transfer of responsibility, may be identified through studying responding patterns in cTTO and DCE responses.

Purpose

The calculation of aggregated composite measures is a widely used strategy to reduce the amount of data on hospital report cards. Therefore, this study aims to elicit and compare preferences of both patients as well as referring physicians regarding publicly available hospital quality information

Methods

Based on systematic literature reviews as well as qualitative analysis, two discrete choice experiments (DCEs) were applied to elicit patients’ and referring physicians’ preferences. The DCEs were conducted using a fractional factorial design. Statistical data analysis was performed using multinomial logit models

Results

Apart from five identical attributes, one specific attribute was identified for each study group, respectively. Overall, 322 patients (mean age 68.99) and 187 referring physicians (mean age 53.60) were included. Our models displayed significant coefficients for all attributes (p < 0.001 each). Among patients, “Postoperative complication rate” (20.6%; level range of 1.164) was rated highest, followed by “Mobility at hospital discharge” (19.9%; level range of 1.127), and ‘‘The number of cases treated” (18.5%; level range of 1.045). In contrast, referring physicians valued most the ‘‘One-year revision surgery rate’’ (30.4%; level range of 1.989), followed by “The number of cases treated” (21.0%; level range of 1.372), and “Postoperative complication rate” (17.2%; level range of 1.123)

Conclusion

We determined considerable differences between both study groups when calculating the relative value of publicly available hospital quality information. This may have an impact when calculating aggregated composite measures based on consumer-based weighting.