European Journal of Health Economics最新文献

Background: This study investigates the excess costs of dementia from healthcare, social care services, and prescription drugs 3 years before to 6 years after diagnosis. Further, sociodemographic cost differences are explored.

Methods: Using Swedish register data from 2013 to 2016 to compare individuals diagnosed with dementia (n = 15,339) with population controls, the excess formal care costs for people with a dementia diagnosis are obtained with longitudinal regression analysis.

Results: People with dementia incur higher formal care costs for all years studied compared to people without dementia. The excess costs vary from €3400 3 years before diagnosis to €49,700 6 years after diagnosis. The costs are mainly driven by institutional care, and solitary living is a strong predictor of high excess costs.

Conclusion: The results show that the formal care costs of individuals with dementia are substantial, and that the economic burden of dementia in Sweden is larger than previously estimated.

Nearly all empirical studies that estimate the coefficients of a risk equalization formula present the value of the statistical measure R². The R²-value is often (implicitly) interpreted as a measure of the extent to which the risk equalization payments remove the regulation-induced predictable profits and losses on the insured, with a higher R²-value indicating a better performance. In many cases, however, we do not know whether a model with R² = 0.30 reduces the predictable profits and losses more than a model with R² = 0.20. In this paper we argue that in the context of risk equalization R² is hard to interpret as a measure of selection incentives, can lead to wrong and misleading conclusions when used as a measure of selection incentives, and is therefore not useful for measuring selection incentives. The same is true for related statistical measures such as the Mean Absolute Prediction Error (MAPE), Cumming's Prediction Measure (CPM) and the Payment System Fit (PSF). There are some exceptions where the R² can be useful. Our recommendation is to either present the R² with a clear, valid, and relevant interpretation or not to present the R². The same holds for the related statistical measures MAPE, CPM and PSF.

Objectives: Multiple studies suggest that the EQ-5D may overestimate health-related quality of life (HRQoL) in patients with coeliac disease (CD). We aimed to develop and psychometrically test potentially relevant bolt-on dimensions to improve the measurement performance of the EQ-5D-5L in CD patients.

Methods: The development and selection of bolt-ons were informed by a literature review on HRQoL in CD, expert and patient input. A cross-sectional online survey was conducted amongst 312 adult CD patients. Respondents completed the EQ-5D-5L, two condition-specific bolt-ons newly-developed for the present study [dining (DI) and gastrointestinal problems (GI)] and three existing bolt-ons [cognition (CO), sleep (SL) and tiredness (TI)]. The following psychometric properties were tested: ceiling, informativity, convergent and known-group validity, and dimensionality (confirmatory factor analysis).

Results: Adding the TI, SL, GI, DI and CO individual bolt-ons reduced the ceiling of the EQ-5D-5L (39%) to 17%, 23%, 24%, 26% and 37%, respectively. GI excelled with strong convergent validity with the Gastrointestinal Symptom Rating Scale total score (r_s=0.71) and improved the discriminatory power for all known-groups. GI was the only bolt-on loading on a different factor from the five core dimensions, whereas the other four bolt-ons loaded onto the same 'psychosocial health' factor as the EQ-5D-5L anxiety/depression dimension.

Conclusion: The DI, GI, SL and TI bolt-ons, especially the GI, enhance the validity of EQ-5D-5L in patients with CD, suggesting their value in capturing important HRQoL aspects potentially missed by the five core dimensions. These bolt-ons can be used in sensitivity analyses supporting health technology assessments and subsequent resource allocation decisions.

Aims: This study aims to evaluate the cost effectiveness of genetic and genomic testing strategies for the diagnosis of rare developmental disorders in NHS Scotland.

Methods: Six genetic and genomic testing strategies were evaluated using a decision tree model. First-line, second-line and last-resort trio genome sequencing (GS), and second-line and last-resort trio exome sequencing (ES) were compared with standard genetic testing. The cost effectiveness of each strategy was expressed in terms of incremental cost per additional diagnosis. The impact of uncertainty on cost-effectiveness results was explored using deterministic and probabilistic sensitivity analysis.

Results: 2nd-line ES was a cost-saving option, increasing diagnostic yield by 13.9% and decreasing cost by £1027 per trio compared to standard genetic testing. Compared to ES, strategies involving GS increased costs significantly, with only a moderate or zero improvement in diagnostic yield. Sensitivity analysis indicated that significant reductions in cost or improvements in diagnostic yield are required before 1st-line GS becomes cost effective.

Conclusion: 2nd-line ES (after chromosomal microarray; replacing gene panel testing) for the diagnosis of developmental disorders is a cost-saving option for the Scottish NHS. Ongoing economic evaluation is required to monitor the evolving cost and diagnostic yield of GS and ES over time.

Objective: Hydroxychloroquine (HCQ) effectively treats autoimmune diseases but prolonged use may lead to retinopathy and subsequent vision loss. Guidelines suggest annual follow-up after 5 years for low-risk and 1 year for high-risk patients. This study evaluates the cost-effectiveness of current screening guidelines and a reduced regimen in the Netherlands from a societal perspective.

Methods: A Markov model assessed costs and quality-adjusted life-years (QALYs) for current and reduced screening regimens. The model included 359 HCQ-treated patients from Radboud University Medical Center. Cost-effectiveness was examined in the general population and patients using < 5.0 mg/kg, 5.0-6.0 mg/kg, or > 6.0 mg/kg HCQ per day for several reduced regimens.

Results: Compared to no screening, the current screening guideline saves costs (i.e., €210 per patient), while gaining QALYs (i.e., 0.79 QALY per patient) over a lifetime in the Netherlands. However, in patients receiving < 5.0 mg/kg HCQ per day, a biennial screening regimen after 10 years using SD-OCT was more cost-effective. For those with 5.0-6.0 mg/kg and > 6.0 mg/kg per day, initiating annual screening with an SD-OCT after 5 years was more cost-effective than the current guideline.

Conclusions: Screening for HCQ retinopathy is cost-effective, but delayed initiation and a reduced frequency, using solely an SD-OCT, are more cost-effective. We recommend screening with an SD-OCT and a biennial regimen after 10 years for low-risk patients, an annual regimen after 5 years for intermediate- and high-risk patients.

Background: In Jordan, no national value set is available for any preference-accompanied health utility measure.

Objective: This study aims to develop a value set for EQ-5D-3L based on the preferences of the Jordanian general population.

Methods: A representative sample of the Jordanian general population was obtained through quota sampling involving age, gender, and region. Participants aged above 18 years were interviewed via videoconferencing using the EuroQol Valuation Technology 2.1 protocol. Participants completed ten composite time trade-offs (cTTO) and ten discrete choice experiments (DCE) tasks. cTTO and DCE data were analyzed using linear and logistic regression models, respectively, and hybrid models were applied to the combined DCE and cTTO data.

Results: A total of 301 participants with complete data were included in the analysis. The sample was representative of the general population regarding region, age, and gender. All model types applied, that is, random intercept model, random intercept Tobit, linear model with correction for heteroskedasticity, Tobit with correction for heteroskedasticity, and all hybrid models, were statistically significant. They showed logical consistency in terms of higher utility decrements with more severe levels. The hybrid model corrected for heteroskedasticity was selected to construct the Jordanian EQ-5D-3L value set as it showed the best fit and lowest mean absolute error. The predicted value for the most severe health state (33333) was - 0.563. Utility decrements due to mobility had the largest weight, followed by anxiety/depression, while usual activities had the smallest weight.

Conclusion: This study provides the first EQ-5D-3L value set in the Middle East. The Jordanian EQ-5D-3L value set can now be used in health technology assessments for health policy planning by the Jordanian health sector's decision-makers.

Objectives: Respiratory infectious diseases like COVID-19 profoundly impacts the health of children and adolescents, but validated instruments to measure their impacts on health-related quality of life (HRQoL) are lacking. The EQ-5D-Y-3L, widely used for youth HRQoL, now features a Chinese value set. The experimental EQ-TIPS addresses HRQoL assessment for toddlers and infants. This study tested the psychometric properties of both instruments in paediatric COVID-19 patients, and compared the performance of self-complete and proxy EQ-5D-Y-3L.

Methods: This longitudinal study recruited 861 COVID-19 patients aged 0-18 years and their parental caregivers, with 311 dyads completing the follow-up. Digital administration included the EQ-TIPS, the EQ-5D-Y-3L, and Overall Health Assessment (OHA). Controls comprised 231 healthy children. Analysis encompassed known-group validity, child-parent agreement, and responsiveness to change in disease severity and OHA.

Results: COVID-19 children exhibited lower HRQoL than non-infected peers. The EQ-TIPS and the EQ-5D-Y-3L distinguished groups by disease presence, severity and symptoms, showing moderate to good known-group validity (ESs: 0.45-1.39 for EQ-TIPS, 0.44-1.91 for self-complete EQ-5D-Y-3L, and 0.32-1.67 for proxy EQ-5D-Y-3L). Child-parent agreement was moderate to good for EQ-5D-Y-3L (ICC: 0.653-0.823; Gwet's AC1: 0.470-0.738), and responsiveness was good for both EQ-TIPS Level Sum Score (LSS) (ESs: 1.21-1.39) and EQ-5D-Y-3L index scores (ESs: 1.00-1.16).

Conclusions: This study demonstrates the reliability, validity, and responsiveness of the experimental EQ-TIPS and the EQ-5D-Y-3L in paediatric COVID-19 patients. It is the first evidence of the EQ-TIPS' responsiveness, supporting its use in assessing the impact of COVID-19 on paediatric HRQoL.

Objectives: The aim of this study is to analyse the trends in technology appraisals for non-small cell lung cancer (NSCLC) treatments performed by the National Institute for Health and Care Excellence (NICE) over the last ten years.

Methods: A systematic search was conducted for single technology appraisals of NSCLC drugs in the online NICE database from 2012 to 2022. Search terms used were 'non small cell lung cancer', and 'NSCLC'. Appraisals that were under development or terminated as well as multiple technology appraisals were considered out of scope.

Results: In the 30 included appraisals for targeted therapies and immunotherapies within NSCLC, a total of 53 different comparators were included by NICE for 41 assorted indications or subgroups. Partitioned survival models were most frequently used, often including three health states and time horizons of up to 30 years. Throughout the decade the use of indirect comparisons was high and became more established and complex over time. Of all appraisals, 90% positively recommended the treatment for use in the UK.

Conclusion: Technology appraisals became more complex over time due to the emergence of targeted therapies and immunotherapies, leading to multiple different indications, subpopulations and comparators that needed to be included in appraisals. Partitioned Survival Analysis (PartSA) models became the cornerstone within NSCLC, with time horizons up to 30 years and over time methods for indirect treatment comparisons became more established. The majority of the appraisals resulted in a positive recommendation for reimbursement.

This paper analyses the structure of and variability in taxation and prescription drug distribution policies and quantifies the impact of such policies on the cost of prescription drugs to health systems in 35 countries. Taxes on prescription drugs remain highly prevalent (83% of the sample) although 63% of the sample countries implement a lower than standard VAT rate. Three remuneration types of the wholesale and retail distribution chain have been identified. Wholesale and retail distributors are remunerated on a regressive mark-up basis, which is price-dependent, although fixed fees and fixed percentages, which are non-price dependent, are also highly prevalent. Price component analysis for three groups of products classed as high-, medium- and low-priced suggests that mark-ups plus taxes varied significantly across countries and products, and ranged from 5% to 187% of ex-factory prices. Average margins also vary significantly by countries and products ranging 5-65% of retail prices. The cost of distribution and taxation contributes significantly to prescription drug costs for health systems. Although distribution chain remuneration raises efficiency and overall affordability questions, these need to be considered together with the regulatory framework shaping market structure of the distribution chain, as well as any prevailing horizontal and vertical integration policies. The overall cost of prescription drugs could be reduced immediately by eliminating taxation; this could go some way to alleviate fiscal pressures on health budgets, whilst avoiding resource re-allocation from health to other sectors.

Objectives: This study aimed to evaluate the psychometric properties of four candidate cognition bolt-on items and their combinations to the EQ-5D-5L.

Methods: Four cognition items (concentration, memory, calculation, and learning) were developed as separate questionnaire items, and were administered with the standard EQ-5D-5L to 640 individuals in a general population survey in China. From the 4 items, 11 compound items were constructed, and the 'worse level counts' rule was used to calculate a compound item score. Psychometric performance of the cognition bolt-ons was assessed in terms of informativity, convergent validity, explanatory power, and discriminatory power.

Results: The tested four cognition bolt-on items improved the informativity, convergent validity, explanatory power, and discriminatory power of EQ-5D-5L, with calculation and learning yielding better psychometric performance. The compound bolt-on items that coverd a range of cognitive functions demonstrated superior psychometric performance compared to single-aspect bolt-on items, with those items covering calculation and learning resulting in better psychometric performance.

Conclusion: This study confirmed the validity of the tested cognition bolt-ons in a general Chinese population. It supported the use of a compound bolt-on item covering a range of cognitive functions such as the ability to calculate and learn.