European Journal of Health Economics最新文献

Background: Evidence on the cost effectiveness of deprescribing in multimorbidity is limited.

Objective: To investigate the cost effectiveness of a general practitioner (GP) delivered, individualised medication review to reduce polypharmacy and potentially inappropriate prescribing in older patients with multimorbidity in Irish primary care.

Methods: Within trial economic evaluation, from a healthcare perspective and based on a cluster randomised controlled trial with a 6 month follow up and 403 patients (208 Intervention and 195 Control) recruited between April 2017 and December 2019. Intervention GPs used the SPPiRE website which contained educational materials and a template to support a web-based individualised medication review. Control GPs delivered usual care. Incremental costs, quality adjusted life years (QALYs) generated using the EQ-5D-5L instrument, and expected cost effectiveness were estimated using multilevel modelling and multiple imputation techniques. Uncertainty was explored using parametric, deterministic and probabilistic methods.

Results: On average, the SPPiRE intervention was dominant over usual care, with non-statistically significant mean cost savings of €410 (95% confidence interval (CI): - 2211, 1409) and mean health gains of 0.014 QALYs (95% CI - 0.011, 0.039). At cost effectiveness threshold values of €20,000 and €45,000 per QALY, the probability of SPPiRE being cost effective was 0.993 and 0.988. Results were sensitive to missing data and data collection period.

Conclusions: The study observed a pattern towards dominance for the SPPiRE intervention, with high expected cost effectiveness. Notably, observed differences in costs and outcomes were consistent with chance, and missing data and related uncertainty was non trivial. The cost effectiveness evidence may be considered promising but equivocal.

Trial registration: ISRCTN: 12752680, 20th October 2016.

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.

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: 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.

The aims of this paper are to estimate the monetary value of informal care for people with Alzheimer's disease (AD) in Spain, to compare results with those obtained in 2008 and to analyse the main determinants of the time of the value of informal care. The Survey on Disabilities, Autonomy and Dependency carried out in Spain in 2020/21 was used to obtain information about disabled individuals with AD and their informal caregivers. Assessment of informal care time was carried out using two alternative approaches: the replacement method, and the contingent valuation method (willingness to pay & willingness to accept). The number of people with AD residing in Spanish households and receiving informal care rose to more than 200,000, representing an increase of 43% compared with 2008. The average number of hours of informal care per week ranged from 86 to 101 h, with an estimated value of between €31,584 - €37,019 per year per caregiver (willingness to accept) or €71,653 - €83,984 per year (replacement). The annual total number of caregiving hours ranged between 896 and 1,061 million hours, representing between 0.52 and 0.62 of GDP in 2021 (willingness to accept) or 1.19-1.40 of GDP (replacement). The level of care needs plays a central role in explaining heterogeneity in estimates. These results should be taken into account by decision-makers for long-term care planning in the coming years.

Objectives: To assess the feasibility of estimating an EQ-5D-5L value set using a small study design in cancer patients and to compare the EQ-5D-5L values based on the preferences of cancer patients with those of the general public.

Methods: Patients with clinically diagnosed cancers were recruited from two hospitals in Shanghai, China. In face-to-face interviews using the EQ-PVT survey, health states were valued by cancer patients using both cTTO and DCE methods. cTTO data was modelled alone or jointly with DCE data. Forty-eight models using different model specifications (cross-attribute level effect [CALE] and additive models), random/fixed effects model assumptions, data heteroscedasticity and censoring were estimated. The best performed model was identified in terms of monotonicity of estimated model coefficients and out-of-sample prediction accuracy.

Results: Data collected from 221 cancer patients who participated in the study were included. The hybrid CALE model using both TTO and DCE data performed best in terms of prediction accuracy (Lin's concordance coefficient = 0.989; root mean squared error = 0.058) and suggested that pain/discomfort and anxiety/depression were the most undesirable health problems. Compared to values based on general Chinese public's health preferences, the values based on cancer patients' preferences were much higher and lower for health states characterized by extreme mobility problems and severe/extreme pain or discomfort, respectively.

Conclusion: This study demonstrated the feasibility of using a small design to develop EQ-5D-5L value sets based on cancer patients' health preferences. Since there were signs of differences between preferences of patients and general population, it may be valuable to develop patient-specific value sets and use them in clinical decision making and economic evaluations.

Background: Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.

Objectives: This study reviews the FDA's five special designations for drug development regarding their safety, efficacy/clinical benefit, clinical trials, innovation, economic incentives, development timelines, and price.

Methods: We conducted a keyword search to identify studies analyzing the impact of the FDA's special designations (orphan, fast track, accelerated approval, priority review, and breakthrough therapy) on the safety, efficacy/clinical benefit, trials, innovativeness, economic incentives, development times, and pricing of new drugs. Results were summarized in a narrative overview.

Results: Expedited approval reduces new drugs' time to market. However, faster drug development and regulatory review are associated with more unrecognized adverse events and post-marketing safety revisions. Clinical trials supporting special FDA approvals frequently use small, non-randomized, open-label designs. Required post-approval trials to monitor unknown adverse events are often delayed or not even initiated. Evidence suggests that drugs approved under special review pathways, marketed as "breakthroughs", are more innovative and deliver a higher clinical benefit than those receiving standard FDA approval. Special designations are an economically viable strategy for investors and pharmaceutical companies to develop drugs for rare diseases with unmet medical needs, due to financial incentives, expedited development timelines, higher clinical trial success rates, alongside greater prices. Nonetheless, patients, physicians, and insurers are concerned about spending money on drugs without a proven benefit or even on drugs that turn out to be ineffective. While European countries established performance- and financial-based managed entry agreements to account for this uncertainty in clinical trial evidence and cost-effectiveness, the pricing and reimbursement of these drugs remain largely unregulated in the US.

Conclusion: Special FDA designations shorten clinical development and FDA approval times for new drugs treating rare and severe diseases with unmet medical needs. Special-designated drugs offer a greater clinical benefit to patients. However, physicians, patients, and insurers must be aware that special-designated drugs are often approved based on non-robust trials, associated with more unrecognized side effects, and sold for higher prices.

Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe. This paper elicited the opinions of European stakeholders on how HTA can be improved to facilitate access.

Methods: A scoping review identified HTA features that influence access to medicines within markets and areas for improvement, while three access dimensions were identified (availability, affordability, timeliness). Using the Delphi method, we elicited the opinions of European stakeholders to validate the literature findings.

Results: Nineteen participants from 14 countries participated in the Delphi panel. Thirteen HTA features that could be improved to optimise access to medicines in Europe were identified. Of these, 11 recorded a positive impact on at least one of the three access dimensions. HTA features had mostly a positive impact on timeliness and a less clear impact on affordability. 'Early scientific advice' and 'clarity in evidentiary requirements' showed a positive impact on all access dimensions. 'Established ways to deal with uncertainty during HTA' could improve medicines' availability and timeliness, while more 'reliance on real-world evidence' could expedite time to market access.

Conclusions: Our results reiterate that increased transparency during HTA and the decision-making processes is essential; the use of and reliance on new evidence generation such as real-world evidence can optimise the availability of medicines; and better collaborations between regulatory institutions within and between countries are paramount for better access to medicines.

Healthcare knowledge management systems can mitigate hospitals' operational inefficiency. As a healthcare information technology, the electronic health record (EHR) receives much attention from medical institutions due to its considerable impact on operational cost performance. This paper focuses on EHR systems to address operational inefficiency by which patients pay more for health care services, and many U.S. hospitals are filing for bankruptcy. From the theoretical perspective of the practice-based view, this paper introduces a path to implement EHR systems for improving cost performance. The empirical investigation is archival data of 200 hospitals collected from the U.S. healthcare agencies. Findings contribute to prior work by hypothesizing moderating and mediating roles in EHR systems implementation. This paper introduces absorptive capacity and monitoring mechanisms as enablers of implementing EHR systems. The results showed that hospital monitoring strengthens the relationship between absorptive capacity and electronic health record systems implementation, which results in better operational cost performance. Theoretically, this study supports the long-term potential benefits of EHR adoption, and its findings are consistent with optimizing efficiency through data standardization and interoperability. From a practical perspective, this study supports hospitals' investments in evolving healthcare information technology systems through the development of a knowledge-based system employing EHR, particularly when hospitals are merging or need a financial strategic plan to control expenses.

Supplier-induced demand (SID) is a long-standing concern in health economics and health system studies; however, empirical evidence on SID-critical to the development of health policy-is difficult to obtain, especially from China. This study, therefore, aims to add new evidence on SID in China's public tertiary hospitals and facilitates the development of evidence-based health policies in China and other countries with similar healthcare systems. For this study, we used patient-level electronic medical records (EMRs) collected from the information systems of tertiary hospitals in a western province in China. From 11 tertiary hospitals, we collected 274,811 hospitalization records dated between 15 February and 30 November 2019. Total expenditure on hospitalization and length of admission of each patient were the primary metrics for measuring SID. We constructed a character indicator to measure the high-season or off-season status of hospitals, and log-linear estimations were applied to estimate the "off-season effect" on hospitalized expenditures and length of admission. We find that the cost of hospitalization is indeed higher in the off-season in China's public tertiary hospitals; specifically, expenditures for patients admitted in the off-season increased by an average of 5.3-7.9% compared to patients admitted in the peak season, while the length of admission in the hospital increased by an average of 6.8% to 10.2%. We also checked the robustness of our findings by performing subgroup analyses of EMRs in the city-level hospitals and surgical group. We name this phenomenon the "hospital off-season effect" and suggest that the main reason for it is inappropriate financial incentives combined with a Fee-For-Services payment method. We suggest that China should work to reform inappropriate financial incentives in public hospitals to eliminate SID by changing its payment and financing compensation system.