European Journal of Health Economics最新文献

Alternative payment models (APMs) aim to improve efficiency and fairness in healthcare by shifting financial responsibility from payers to providers. Given their prospective nature, APMs require effective risk adjustment (RA) to prevent risk-selection incentives. RA design comes with complex trade-offs between risk selection, cost control and gaming. In the light of these trade-offs, thorough ex-ante evaluation of RA models is crucial. Traditionally, RA-model evaluation in the context of APMs has heavily relied on statistical metrics like R-squared. While useful for assessing model fit, these metrics often fail to capture the full spectrum of relevant incentives. This study therefore addresses the question: "What do meaningful incentive metrics for ex-ante evaluation of RA models look like in the context of prospective APMs for healthcare providers?" We conducted a literature review and consulted experts to synthesize existing work on RA evaluation. This informed the development of a conceptual framework for defining incentive metrics, distinguishing among risk-selection, cost-control, and gaming incentives. We applied our framework in a simulation of prospective payments to primary care practices (PCPs) in the Netherlands, using 2019 claims data from 346 PCPs (N = 1.4 M patients). The analysis focused on selection incentives, comparing traditional statistical metrics with metrics derived from our framework. Results show that statistical metrics like R-squared fall short in assessing selection incentives compared to our incentive metrics. This highlights the need for tailored incentive metrics for the ex-ante evaluation of RA models that are grounded in a thorough understanding of relevant provider behaviors in the light of APM goals.

Over the past decades, most European countries switched emergency contraceptive availability from prescription to over-the-counter (OTC) status. However, the effect of OTC switches on emergency and regular contraceptive use, on demographic indicators (birth rate, abortion rate) and health outcomes (sexually transmitted diseases) varies depending on the study setting. Only Poland switched emergency contraceptives back to prescription (Rx) status after previously loosening restrictions and causal analyses of an Rx switch are lacking entirely. We employ multiple (staggered/synthetic) difference-in-differences models to evaluate the effect of (a) an OTC switch, exploiting variation in the timing of the OTC switch among several European countries and (b) a Rx switch at the example of Poland. We find that the use of emergency contraceptives increased (decreased) after OTC (Rx) switches. The use of regular hormonal contraceptives, the rate of births and abortions and the incidence of various sexually transmitted diseases remained essentially unaffected or - if found - effects did not withstand robustness checks. Our results suggest that increased use of emergency contraceptives due to liberalized over-the-counter access may be reversed by a return to prescription bound access.

Background: Transthyretin amyloid cardiomyopathy (ATTR-CA) is a progressive, fatal disease that often presents as heart failure with preserved ejection fraction (HFpEF). To date, scarce evidence about the economic burden of ATTR-CA has been published. This study compared the economic impact of HFpEF in patients with and without ATTR-CA in Spain.

Methods: The PRACTICA study was a cross-sectional, multicenter, nationwide study in twenty sites in Spain. A total of 387 consecutive ambulatory or hospitalized patients aged ≥ 50 years with HFpEF and LVH ≥ 12 mm were included and screened for ATTR-CA. Healthcare resource utilization (HRU), except specific pharmacologic treatments, non-HRU and its associated average cost per-patient per-year were assessed in year 2021 for ATTR-CA and non-ATTR-CA patients.

Results: Patients were classified as: ATTR-CA (n = 65), non-ATTR-CA (n = 306) and inconclusive (n = 16). Mean total cost per-patient per-year was higher in ATTR-CA patients: €3,407 (Min-Max: €1,067-€6,473) in ATTR-CA, €3,203 (€1,168-€5,646) in non-ATTR-CA and €2,920 (€1,165-€5,080) in inconclusive patients (p < 0.001, Kruskal-Wallis). Determinants of differential cost favoring ATTR-CA were hospitalizations, genetic testing, implantable cardiac defibrillator, and scintigraphy. No statistical differences were observed between patients with and without ATTR-CA in non-healthcare resources paid by patients themselves, although inconclusive patients showed significantly higher home adaptation (shower, bed rail) and crutch utilization (p = 0.031, Kruskal-Wallis).

Conclusions: This is the first multicenter nationwide study assessing the economic impact on Spanish society of ATTR-CA patients with HFpEF and LVH ≥ 12 mm. The cost was substantial, with negative implications particularly for the Spanish National Health System.

Objective: To assess the cost-effectiveness of enzyme replacement therapy (ERT) compared with best supportive care (BSC) for late-onset Pompe disease (LOPD) in a UK NHS setting.

Methods: A discrete event simulation model was developed using data from a Bayesian network meta-analysis, trials and extension studies, and long-term observational cohorts. Costings were derived from NICE assessments of LOPD treatments. Disease progression was modelled using forced vital capacity (FVC) % predicted and six-minute walk distance (6MWD).

Results: ERT was associated with an incremental cost of £3.26 million and 1.64 additional QALYs compared to BSC, yielding an incremental cost-effectiveness ratio (ICER) of £2 million per QALY gained, and generating a net health effect (NHE) exceeding -100 QALYs per treated patient. Scenario analyses confirmed that ERT remained cost-ineffective under all plausible assumptions. Discounts of ~ 92.3% and ~ 89% on the list price of ERT would be required to achieve cost-effectiveness at thresholds of £30,000 and £100,000 per QALY gained, respectively.

Conclusions: While ERT provides modest long-term health benefits relative to BSC, it is structurally highly cost-ineffective, generating substantial negative NHE for the NHS population, even under the most optimistic assumptions. These results are primarily driven by very high acquisition costs of ERT. The historic commissioning of ERT without reference to the UK's value-based pricing framework has significantly impacted NHS spending and distorted NICE decision-making. Without reform to the appraisal process, the NHS faces affordability challenges that may hinder access to innovative therapies and result in recommendations that displace more health than they generate.

Background: Despite its increasing prevalence, the economic impact of eosinophilic esophagitis (EoE) in Europe is understudied and direct economic costs remain unknown.

Objective: To assess contemporaneous direct medical costs during the first year after EoE diagnosis in Spain to identify key cost determinants and assess variables that could affect these costs.

Methods: Observational study of Spanish participants in the EUREOS EoE CONNECT registry with incident EoE in 2017-2018. We assessed average costs/year in Euros for endoscopic procedures, out-patient visits, medication, emergency room visits and hospitalizations, 2018 being our base year. Official regional tariffs were used to estimate direct costs from a healthcare provider perspective; official price lists were used for drug therapies. Cost differences and determinants were assessed with multivariate models.

Results: The mean annual direct cost of EoE per person among 302 patients (76.5% male, 77.5% adults) was €1,842.07 (SD €795.69). Endoscopic procedures accounted for 68% of this, followed by outpatient visits (15.9%) and drug prescriptions (10.8%). Cost was higher for pediatric compared to adult patients (€2,101.51 vs. €1,784.29, p < 0.01), for those with higher EREFS score (€1,979.36 vs. €1,794.49; p < 0.01), and in high-volume hospitals (€1,941.48 vs. €1,723.92; p = 0.02). Early response to first-line treatment reduced annual costs by -€368.95 (p < 0.01), by avoiding further medical procedures.

Conclusion: First year after EoE diagnosis produces significant direct costs in Spain, mainly due to endoscopic procedures, which are reduced in early responders to first-line therapy. Research for non-invasive methods for EoE assessment and identifying predictors of response are, therefore, key.

Background: The EQ-TIPS (v2.0) is an experimental instrument to measure and value the HRQoL in children aged 0-3 years. This study consulted experts and caregivers to (a) review the instrument's wording and content for global relevance; (b) assess the need for additional dimensions; (c) identify potential challenges in measuring HRQoL in young children.

Methods: Participants included 26 HRQoL experts, 28 child health and development experts, and 65 caregivers of young children, recruited through snowballing and international networks. Participants completed online surveys, with analyses involving thematic assessment of open-ended responses and frequency reporting of closed-ended responses.

Results: EQ-TIPS v2.0 was positively received with participants recognising its potential value in clinical practice, health research and policymaking. There was limited consensus amongst experts regarding the use of the term 'age-appropriate' within the EQ-TIPS dimensions. Concern was highlighted regarding the perceived overlap between social interaction and communication; and whether in their currently worded forms they are distinct enough to warrant being separate dimensions. Furthermore, respondents questioned whether the dimensions are sensitive enough to account for the nuances of a child's abilities across the age range. It was suggested that introducing examples across all dimensions would decrease this subjectivity of proxy reporting and reduce the global differences in perceptions of dimensions. Additional dimensions suggested for further investigation at the multinational level were sleep and emotional functioning.

Conclusion: Further multinational development of the EQ-TIPS will be guided by expert and caregiver input, with proposed descriptive system modifications to be qualitatively tested in subsequent research.

Objective: Time preference is increasingly considered in health state valuation. As its inclusion impacts the produced value sets, it is important to account for it using the correct specification. Various discounting functions have been used, and both directions of time preference have been identified. I aimed to extend the composite time-trade off task (composite TTO, cTTO) by follow-up questions (TTOFUs) to identify the discounting function and direction for individual respondents.

Methods: Respondents completed cTTO tasks for 6 EQ-5D-5L health states. If utility exceeded 0.1, three TTOFUs (Q1-Q3) were posed. Each TTOFU modified the indifference-yielding alternative by halving durations (Q1) or adding a 5-year lead-time in full health (Q2) or a 5-year lag-time in the valued state (Q3). As shown in the paper, the pattern of Q1-Q3 answers unambiguously discerns the discounting function (exponential, power, or hyperbolic) and direction.

Results: Data from 148 respondents, 16 health states, and 577 respondent-state pairs were analysed. Only 20.4% of pairs yielded theoretically-plausible patterns, and among those 60% imply no time preference. For Q2 and Q3, responses mostly implied negative ([Formula: see text]) and positive ([Formula: see text]) time preference, respectively. Majority-rule pooling within respondents produced a pattern not matching the considered discounting models but that can arise when quality-of-life and longevity are combined additively, not multiplicatively as in the quality-adjusted life years (QALY) model.

Conclusions: Standard discounting models poorly fit observed choices in TTO-like questions. The discounting parameters measured in other studies may be driven by other phenomena, including departures from the QALY model. Hence, careful interpretation of estimated parameters is warranted, and future studies are recommended.

Introduction: Treatment landscape in metastatic lung cancer has progressed quickly over the last decade, mainly due to immunotherapies and targeted therapies. This study aimed to describe changes in costs associated with metastatic lung cancer in France.

Materials and methods: A cohort of patients identified between 2013 and 2021 with lung cancer and a marker of metastases (ICD-10 code or reimbursement for Bevacizumab or Pemetrexed) was built from the French claims database. Healthcare resource use was described each year. The trend in total mean monthly costs (MMC) expressed in gross price over the study period was studied using Joinpoint software.

Results: Between 2013 and 2021, 147,760 metastatic lung cancer patients were identified (men :66.5%, median age: 66 years). The annual cost for all patients increased from 611,074,408€ in 2013 (N = 24,595) to 1,308,745,922€ in 2021 (N = 40,321). The MMC per patient decreased from 2013 to 2015 (from 5,853€ to 4,895€), by 9.37% per year (95%CI: -17.30; -0.69), then stabilized (0.90%; 95%CI: -0.84; 2.66). An increase in drug acquisition MMC in the same proportions as the decrease in full hospitalization MMC was observed, excluding 2020.

Conclusion: Although the global cost of metastatic lung cancer management has increased, the MMC per patient has not notably surged between 2013 and 2021. Drug acquisition MMC increase was offset by reduced full hospitalization MMC (excluding 2020), resulting in no significant uptrend. These results suggest that the global management cost increase is mainly driven by epidemiological and demographic factors. This highlights the importance of continued investment in prevention and in therapeutic innovations that may improve outcomes.

Objective: The objective of this study was to create a mapping algorithm by utilizing traditional regression analyses and a machine learning approach to estimate EQ-5D-5 L values based on EORTC QLQ-LC43 data in the absence of direct EQ-5D-5 L measurements.

Methods: Data for EQ-5D-5 L and EORTC QLQ-LC43 were collected from patients with lung cancer at the Departments of Thoracic Surgery, Medical Oncology, and Radiation Oncology at Sichuan Cancer Hospital. Mapping algorithms were applied using the ordinary least squares model (OLS), Tobit model, Beta mixture regression (BM), the adjusted limited dependent variable mixture model (ALDVMM), and ridge regression (RR) as a machine learning model to map QLQ-LC43 results based on EQ-5D-5 L scores. To develop these models, dimension scores, squared items, and interaction items were incorporated. Performance metrics, including R², root mean square error (RMSE), and mean absolute error (MAE), were used to identify the optimal model. The stability of the models was assessed using five-fold cross-validation (CV).

Results: The Beta mixture regression model (BETAMIX M1A), incorporating all dimensions of QLQ-C30 and QLQ-LC13 as covariates, exhibited the best mapping performance. The final prediction metrics were R²=0.816, RMSE = 0.125, MAE = 0.083, AIC=-717.810, and BIC=-482.609. The BM model has good explanatory ability and low prediction error. Five-fold cross-validation (CV) results also demonstrated that the BM model had the best mapping power.

Conclusions: This study developed an optimized mapping algorithm to predict the utility index from the QLQ-LC43 to the EQ-5D-5 L, offering an effective alternative for estimating EQ-5D-5 L values when preference-based health utility data are unavailable.