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AIMS: The goal of this study was to review the economic evaluations of health technologies in multiple myeloma (MM) and provide guidance and recommendations for future health economic analyses. MATERIALS AND METHODS: A systemic literature review (SLR) was conducted on original economic assessment studies and structured review papers focusing on the studies in MM. The search was limited to English language papers published from 1 January 2000 onwards. Publications not applying any type of modelling methodology to describe disease progression and patient pathways over a specific time horizon were excluded. RESULTS: A total of 2,643 publications were initially identified, of which 148 were eligible to be included in the full-text review phase. From these, 49 publications were included in the final analysis. Most published health economic analyses supported by models came from high-income countries. Evaluations from middle-income countries were rarely published. Diagnostic technologies were rarely modelled and integrated care had not been modelled. Very few models investigated MM treatments from a societal perspective and there was a relative lack of evaluations regarding minimal residual disease (MRD). LIMITATIONS: Limitations of the publications included differences between trial populations and modelled populations, justification of methods, lack of confounder analyses, and small trial populations. Limitations of our study included the infeasibility of comparing MM economic evaluations due to the significant variance in modelled therapeutic lines and indications, and the relative scarcity of published economic evaluations from non-high-income countries. CONCLUSIONS: As published economic models lacked many of the elements of the complex and heterogeneous patient pathways in MM and they focused on single decision problems, a thorough, open-source economic whole disease modelling framework is needed to assess the economic value of a wide range of technologies across countries with various income levels with a more detailed view on MM, by including patient-centric and societal aspects.

Achieving high vaccination rates is important for overcoming an epidemic. This study investigates the association between religious faith and intentions to become vaccinated against COVID-19 in Israel and Japan. Most of Israel’s population is monotheistic, whereas most Japanese are unaffiliated with any religion. Therefore, our findings might be applicable to various countries that differ in their religions and levels of religiosity. We conducted almost identical large-scale surveys four times in Israel and five times in Japan from March to June 2020 to obtain panel data. We found that intentions of getting vaccinated depend on people’s level of religiosity in a non-linear way. Those who have strong religious beliefs are less likely to become vaccinated than those who say they are less religious. Two other factors that play a role in this relationship are religious denomination in Israel and identifying with a religion in Japan. Supplementary Information The online version contains supplementary material available at 10.1007/s10198-021-01389-8.

Objectives EQ-5D-Y-3L health states are valued by adults taking the perspective of a 10-year-old child. Compared to valuation of adult EQ-5D instruments, this entails two changes to the perspective: (i) child health states are valued instead of adult health states and: (ii) health states are valued for someone else instead of for oneself. Although earlier work has shown that these combined changes yield different values for child and adult health states that are otherwise equal, it currently remains unclear why. Hence, we aimed to disentangle the effects of both changes. Methods A sample of 205 students (mean age: 19.48) was surveyed. Each respondent completed visual analogue scale (VAS) and time trade-off (TTO) tasks for five EQ-5D-Y-3L states, using four randomly ordered perspectives: (i) self-adult (themselves), (ii) other-adult (someone their age), (iii) self-child (themselves as a 10-year-old), (iv) other-child (a child of 10 years old). We compared how each perspective impacted outcomes, precision and quality of EQ-5D-Y-3L valuation. Results Overall, differences between perspectives were consistent, with their direction being dependent on the health states and respondents. For VAS, the effect on outcomes of valuation depended on severity, but variance was higher in valuation with child perspectives. For TTO, we observed that EQ-5D-Y-3L states valued on behalf of others (i.e., children or adults) received higher valuations, but lower variances. Conclusion The use of a different perspective appears to yield systematic differences in EQ-5D-Y-3L valuation, with considerable heterogeneity between health states and respondents. This may explain mixed findings in earlier work.

Objective Large reductions in inpatient length of stay and inpatient bed supply have occurred across health systems in recent years. However, the direction of causation between length of stay and bed supply is often overlooked. This study examines the impact of changes to inpatient bed supply, as a result of recession-induced healthcare expenditure changes, on emergency inpatient length of stay in Ireland between 2010 and 2015. Study design We analyse all public hospital emergency inpatient discharges in Ireland from 2010 to 2015 using the administrative Hospital In-Patient Enquiry dataset. We use changes to inpatient bed supply across hospitals over time to examine the impact of bed supply on length of stay. Linear, negative binomial, and hospital–month-level fixed effects models are estimated. Results U-shaped trends are observed for both average length of stay and inpatient bed supply between 2010 and 2015. A consistently large positive relationship is found between bed supply and length of stay across all regression analyses. Between 2010 and 2012 while length of stay fell by 6.4%, our analyses estimate that approximately 42% (2.7% points) of this reduction was associated with declines in bed supply. Conclusion Changes in emergency inpatient length of stay in Ireland between 2010 and 2015 were closely related to changes in bed supply during those years. The use of length of stay as an efficiency measure should be understood in the contextual basis of other health system changes. Lower length of stay may be indicative of the lack of resources or available bed supply as opposed to reduced demand for care or the shifting of care to other settings.

The COVID-19 has been a worldwide pandemic and it needs for studies related to effect on people's demand for insurance during the pandemic which is an important way to transfer risk. However, there is a lack of research linking COVID-19 and people's demand for insurance. The objective of this paper is to investigate the impact of COVID-19 pandemic on issuance demand, using data covering 241 cities on confirmed COVID-19 cases and insurance company revenue in China. The empirical results show that more confirmed COVID-19 cases are associated with greater per capita insurance revenue and the results are robust when considering endogeneity concern. Economically, the per capita insurance revenue increases by 0.896 Yuan for each more confirmed case. In terms of insurance type, the greatest increased insurance revenue is for life insurance, followed by health insurance. We further consider the heterogeneity of regions and find that the impact of COVID-19 on insurance revenue only exists in regions with worse medical treatment conditions or higher medical burden.

Policymakers often suggest that expansion of care in community settings may ease increasing pressures on hospital services. Substitution may lower overall health system costs, but complementarity due to previously unidentified needs might raise them. We used new national data on community and primary medical care services in England to undertake system-level analyses of whether activity in the community acts as a complement or a substitute for activity provided in hospitals. We used two-way fixed effects regression to relate monthly counts of community care and primary medical care contacts to emergency department attendances, outpatient visits and admissions for 242 hospitals between November 2017 and September 2019. We then used national unit costs to estimate the effects of increasing community activity on overall system expenditure. The findings show community care contacts to be weak substitutes with all types of hospital activity and primary care contacts are weak substitutes for emergency hospital attendances and admissions. Our estimates ranged from 28 [95% CI 21, 45] to 517 [95% CI 291, 7265] community care contacts and from 34 [95% CI 17, 1283] to 1655 [95% CI − 1995, 70,145] GP appointments to reduce one hospital service visit. Primary care and planned hospital services are complements. Increases in community services and primary care activity are both associated with increased overall system expenditure of £34 [95% CI £156, £54] per visit for community care and £41 [95% CI £78, £74] per appointment in general practice. Expansion of community-based services may not generate reductions in hospital activity and expenditure. Supplementary Information The online version contains supplementary material available at 10.1007/s10198-021-01329-6.

Objectives Cancer diagnoses at later stages are associated with a decrease in health-related quality of life (HRQOL). Health state utility values (HSUVs) reflect preference-based HRQOL and can vary based on cancer type, stage, treatment, and disease progression. Detecting and treating cancer at earlier stages may lead to improved HRQOL, which is important for value assessments. We describe published HSUVs by cancer type and stage. Methods A systematic review was conducted using Embase, MEDLINE®, EconLit, and gray literature to identify studies published from January 1999 to September 2019 that reported HSUVs by cancer type and stage. Disutility values were calculated from differences in reported HSUVs across cancer stages. Results From 13,872 publications, 27 were eligible for evidence synthesis. The most frequent cancer types were breast (n = 9), lung (n = 5), colorectal (n = 4), and cervical cancer (n = 3). Mean HSUVs decreased with increased cancer stage, with consistently lower values seen in stage IV or later-stage cancer across studies (e.g., − 0.74, − 0.44, and − 0.51 for breast, colorectal, and cervical cancer, respectively). Disutility values were highest between later-stage (metastatic or stage IV) cancers compared to earlier-stage (localized or stage I–III) cancers. Conclusions This study provides a summary of HSUVs across different cancer types and stages that can inform economic evaluations. Despite the large variation in HSUVs overall, a consistent decline in HSUVs can be seen in the later stages, including stage IV. These findings indicate substantial impairment on individuals’ quality of life and suggest value in early detection and intervention.

Background Cancer patients have one of the highest health care expenditures (HCE) at the end of life. However, the growth of HCE at the end of life remains poorly documented in the literature. Objective To describe monthly reimbursed expenditure during the last year of life among cancer patients, by performing detailed analysis according to type of expenditure and the person’s age. Method Data were derived from the Système national des données en santé (SNDS) [national health data system], which comprises information on ambulatory and hospital care. Analyses focused on general scheme beneficiaries (77% of the French population) treated for cancer who died in 2015. Results Average reimbursed expenditure during the last year of life was €34,300 per person in 2015, including €21,100 (62%) for hospital expenditure. "Short-stays hospital" and "rehabilitation units" stays expenditure were €14,700 and €2000, respectively. Monthly expenditure increased regularly towards the end of life, increasing from 12 months before death €2000 to €5200 1 month before death. The highest levels of expenditure did not concern the oldest people, as average reimbursed expenditure was €50,300 for people 18–59 years versus €25,600 for people 80–90 years. Out-of-pocket payments varied only slightly according to age, but increased towards the end of life. Conclusion A marked growth of HCE was observed during the last 4 months of life, mainly driven by hospital expenditure, with a more marked growth for younger people.

This paper asks whether marriage decisions of unmarried mature couples are driven by the prospect of financial advantages for the later widowed after one partner has suffered a serious health shock. We hypothesize that, in contrast to traditional marriage models, such health shocks may induce unmarried couples to obtain economic benefits, such as survivors’ pensions in particular, through marriage in advance of one partner’s death. This question has not yet been studied empirically. Hazard models capturing unobserved effects are applied to longitudinal data of the German Socioeconomic Panel. It turns out that the probability of marriage after male partners’ health shocks can increase significantly depending on the amount of expected survivors’ pensions for the (likely) surviving female partners. In contrast, an increased probability of marriage after health shocks to women (depending on the expected financial benefits to men) was not found. These findings are supported by various robustness checks. Economic and political implications are discussed and the results are placed in an international context.

Background Uncontrolled hyperphosphatemia in chronic kidney disease (CKD) patients commonly results in vascular calcification leading to increased risk of cardiovascular disease. Phosphate binders (PBs) are used for hyperphosphatemia and can be calcium-based (CBPBs) or non-calcium-based (NCBPBs), the latter being more expensive than CBPBs. In this study, we used meta-analysis approaches to assess the cost-utility of PBs for hyperphosphatemia in CKD patients. Methods Relevant studies published prior to June 2019 were identified from PubMed, Scopus, the Cochrane Library, the National Health Service Economic Evaluation Database, and the Cost-Effectiveness Analysis Registry. Studies were eligible if they included CKD patients with hyperphosphatemia, compared any PBs and reported economic outcomes. Meta-analysis was applied to pool incremental net benefit (INB) across studies stratified by country income. Results A total of 25 studies encompassing 32 comparisons were eligible. Lanthanum carbonate, a NCBPB, was a more cost-effective option than CBPBs in high-income countries (HICs), with a pooled INB of $3984.4 (599.5–7369.4), especially in pre-dialysis patients and used as a second-line option with INBs of $4860.2 (641.5–9078.8), $4011.0 (533.7–7488.3), respectively. Sevelamer, also a NCBPB, was not more cost-effective as a first-line option compared to CBPBs with a pooled INB of $6045.8 (− 23,453.0 to 35,522.6) and $34,168.9 (− 638.0 to 68,975.7) in HICs and upper middle-income countries, respectively. Conclusions Lanthanum carbonate was significantly more cost-effective than CBPBs as a second-line option for hyperphosphatemia in pre-dialysis patients in HICs. However, the use of sevelamer is not more cost-effective as a first-line option compared to CBPBs.

The capability approach is potentially valuable for economic evaluation at the end of life because of its conceptualization of wellbeing as freedom and the potential for capturing outcomes for those at end of life and those close to persons at the end of life. For decision making, however, this information needs to be integrated into current evaluation paradigms. This research explored weights for an integrated economic evaluation framework using a deliberative approach. Twelve focus groups were held (38 members of the public, 29 “policy makers,” seven hospice volunteers); budget pie tasks were completed to generate weights. Constant comparison was used to analyze qualitative data, exploring principles behind individuals' weightings. Average weights elicited from members of the general population and policy makers for the importance that should be given to close persons (vs. patients) were very similar, at around 30%. A “sliding scale” of weights between health gain and the capability for a good death resulted from the policy maker and volunteer groups, with increasing weight given to the capability for a good death as the trajectory got closer to death. These weights can be used in developing a more comprehensive framework for economic evaluation at end of life.

Results from economic evaluations of long-term outcomes are strongly dependent on the chosen discount rate. A recent review of national guidelines for evaluation of healthcare interventions finds that “the level of currently used discount rates seems relatively high in many countries”. However, this conclusion comes from a comparison to rates derived or observed for investments in safe assets, while rate of return requirements are typically considerably higher when investment involves risk. This paper reviews recent literature on how to account for project-specific risk in determination of the social rate of discount and discusses implications for economic evaluation of healthcare interventions. It concludes that the available empirical evidence strongly suggests that the demand for and consumer value of health and healthcare is co-variant with income, which therefore implies that there is a non-diversifiable risk component of health-related investment.

ObjectiveTo determine the association between income inequality and COVID-19 cases and deaths per million in OECD countries.MethodsCross-sectional regression methods are used to model the relationship between income inequality, as measured by the Gini coefficient, and COVID-19 reported cases and deaths per-million.ResultsThe results demonstrate a significant positive association between income inequality and COVID-19 cases and death per million in all estimated models. A 1% increase in the Gini coefficient is associated with an approximately 4% increase in cases per-million and an approximately 5% increase in deaths per-million.ConclusionsThe results demonstrate that countries with high levels of income inequality have performed significantly worse when dealing with the COVID-19 outbreak in terms cases and deaths. Income inequality is a proxy for many elements of socioeconomic disadvantage that may contribute to the spread of, and deaths from, COVID-19. These include poor housing, smoking, obesity and pollution.Policy ImplicationsThe findings suggest the importance of closing the gap in income inequality and improving the health and incomes of the poorest and most vulnerable groups.

IntroductionThe majority of tuberculosis (TB) cases in Sweden occur among migrants from endemic countries through activation of latent tuberculosis infection (LTBI). Sweden has LTBI-screening policies for migrants that have not been previously evaluated. This study aimed to assess the cost-effectiveness of the current screening strategy in Stockholm.MethodsA Markov model was developed to predict the costs and effects of the current LTBI-screening program compared to a scenario of no LTBI screening over a 50-year time horizon. Epidemiological and cost data were obtained from local sources when available. The primary outcomes were incremental cost-effectiveness ratio (ICER) in terms of societal cost per quality-adjusted life year (QALY).ResultsScreening migrants in the age group 13–19 years had the lowest ICER, 300,082 Swedish Kronor (SEK)/QALY, which is considered cost-effective in Sweden. In the age group 20–34, ICER was 714,527 SEK/QALY (moderately cost-effectives) and in all age groups above 34 ICERs were above 1,000,000 SEK/QALY (not cost-effective). ICER decreased with increasing TB incidence in country of origin.ConclusionScreening is cost-effective for young cohorts, mainly between 13 and 19, while cost-effectiveness in age group 20–34 years could be enhanced by focusing on migrants from highest incidence countries and/or by increasing the LTBI treatment initiation rate. Screening is not cost-effective in older cohorts regardless of the country of origin.

Background The prevalence of diagnosed chronic hepatitis C virus (HCV) infection among pregnant women in the Netherlands is 0.26%, yet many cases remain undiagnosed. HCV screening and treatment of pregnant HCV carriers could reduce the burden of disease and limit vertical transmission from mother to child. We assessed the impact of HCV screening and subsequent treatment with new direct-acting antivirals (DAAs) among pregnant women in the Netherlands. Methods An HCV natural history Markov transition state model was developed, to evaluate the public-health and economic impact of HCV screening and treatment. Besides all 179,000 pregnant women in the Netherlands (cohort 1), we modelled 3 further cohorts: all 79,000 first-time pregnant women (cohort 2), 33,000 pregnant migrant women (cohort 3) and 16,000 first-time pregnant migrant women (cohort 4). Each cohort was analyzed in various scenarios: i no intervention, i.e., the current practice, ii screen-and-treat, i.e., the most extensive approach involving treatment of all individuals found HCV-positive, and iii screen-and-treat/monitor, i.e., a strategy involving treatment of symptomatic (F1–F4) patients and follow-up of asymptomatic (F0) HCV carriers with subsequent treatment only at progression. Results For all cohorts, comparison between scenarios (ii) and (i) resulted in ICERs between €9,306 and €10,173 per QALY gained and 5 year budget impacts varying between €6,283,830 and €19,220,405. For all cohorts, comparison between scenarios (iii) and (i) resulted in ICERs between €1,739 and €2,749 per QALY gained and budget impacts varying between €1,468,670 and €5,607,556. For all cohorts, the ICERs (scenario iii versus ii) involved in delayed treatment of asymptomatic (F0) HCV carriers varied between €56,607 and €56,892, well above the willingness-to-pay (WTP) threshold of €20,000 per QALY gained and even above a threshold of €50,000 per QALY gained. Conclusion Universal screening for HCV among all pregnant women in the Netherlands is cost-effective. However, it would be reasonable to consider smaller risk groups in view of the budget impact of the intervention.

Vaccinations, lockdowns and testing strategies are three potential elements of an effective anti-coronavirus, and in particular Covid-19, health policy. The following analysis considers - within a simple model - the potentially crucial role of a Corona testing approach in combination with a quarantine approach which is shown herein to be a substitute for broad lockdown measures. The cost of lockdowns/shutdowns are rather high so that – beyond progress in terms of a broad vaccination program – a rational testing strategy should also be carefully considered. Testing has to be organized on the basis of an adequate testing infrastructure which could largely be implemented in firms, schools, universities and public administration settings. As regards the cost of a systematic broad Covid-19 testing strategy, these could come close to 0.5% of national income if there are no vaccinations. The Testing & Quarantine approach suggested here – with tests for symptomatic as well as asymptomatic people - is based on a random sampling and would require rather broad and frequent testing; possibly one test per person every 7–10 days. At the same time, one should consider that the cost of further lockdowns/shutdowns of a duration of 1 month could be very high, such that a standard cost benefit analysis supports the testing approach suggested herein. Also, an optimal policy mix could be designed where both vaccinations and testing play a crucial role. As of late January 2021, no further lockdowns in Germany and other OECD countries would be necessary if a broad testing infrastructure can be established rather quickly. This in turn will reinforce economic optimism and help to jumpstart economic growth in Europe, the US and Asia in a solid way. The basic logic of the testing approach pointed out here for industrialized countries could also be applied in developing countries. The approach presented is complementary to the IMF analysis of Cherif/Hasanov.

In this paper, we examine the variation in the outbreak of COVID-19 across departments in continental France. We use information on the cumulated number of deaths, discharged patients and infections from COVID-19 at the department level, and study how these relate to income inequality, controlling for other factors. We find that unfortunately, inequality kills: departments with higher income inequality face more deaths, more discharged (gravely ill) patients and more infections. While other papers have studied the impact of the level of income on the severity of COVID-19, we find that it is in fact the dispersion across incomes within the same department that drives the results. Our results suggest that individuals in relatively more precarious conditions deserve dedicated policies, to avoid that temporary shocks such as COVID-19 lead to permanent increases in inequality.

Background The CULPRIT-SHOCK trial compared two treatment strategies for patients with acute myocardial infarction and multivessel coronary artery disease complicated by cardiogenic shock: (a) culprit vessel only percutaneous coronary intervention (CO-PCI), with additional staged revascularisation if indicated, and (b) immediate multivessel PCI (MV-PCI). Methods A German societal and national health service perspective was considered for three different analyses. The cost utility analysis (CUA) estimated costs and quality adjusted life years (QALYs) based on a pre-trial decision analytic model taking a lifelong time horizon. In addition, a within trial CUA estimated QALYs and costs for 1 year. Finally, the cost effectiveness analysis (CEA) used the composite primary outcome, mortality and renal failure at 30-day follow-up, and the within trial costs. Econometric and survival analysis on the trial data was used for the estimation of the model parameters. Subgroup analysis was performed following an economic protocol. Results The lifelong CUA showed an incremental cost effectiveness ratio (ICER), CO-PCI vs. MV-PCI, of €7010 per QALY and a probability of CO-PCI being the most cost-effective strategy > 64% at a €30,000 threshold. The ICER for the within trial CUA was €14,600 and the incremental cost per case of death/renal failure avoided at 30-day follow-up was €9010. Cost-effectiveness improved with patient age and for those without diabetes. Conclusions The estimates of cost-effectiveness for CO-PCI vs. MV-PCI have been shown to change depending on the time horizon and type of economic evaluation performed. The results favoured a long-term horizon analysis for avoiding underestimation of QALY gains from the CO-PCI arm.

Background Health economic evaluations require cost data as key inputs. Many countries do not have standardized reference costs so costs used often vary between studies, thereby reducing transparency and transferability. The present review provided a comprehensive overview of cost sources and suggested unit costs for France, Germany and Italy, to support health economic evaluations in these countries, particularly in the field of diabetes. Methods A literature review was conducted across multiple databases to identify published unit costs and cost data sources for resource items commonly used in health economic evaluations of antidiabetic therapies. The quality of unit cost reporting was assessed with regard to comprehensiveness of cost reporting and referencing as well as accessibility of cost sources from published cost-effectiveness analyses (CEA) of antidiabetic medications. Results An overview of cost sources, including tariff and fee schedules as well as published estimates, was developed for France, Germany and Italy, covering primary and specialist outpatient care, emergency care, hospital treatment, pharmacy costs and lost productivity. Based on these sources, unit cost datasets were suggested for each country. The assessment of unit cost reporting showed that only 60% and 40% of CEAs reported unit costs and referenced them for all pharmacy items, respectively. Less than 20% of CEAs obtained all pharmacy costs from publicly available sources. Conclusions This review provides a comprehensive account of available costs and cost sources in France, Germany and Italy to support health economists and increase transparency in health economic evaluations in diabetes.

This paper forecasts the life-cycle treatment effects on health of a high-quality early childhood program. Our predictions combine microsimulation using non-experimental data with experimental data from a midlife long-term follow-up. The follow-up incorporated a full epidemiological exam. The program mainly benefits males and significantly reduces the prevalence of heart disease, stroke, cancer, and mortality across the life-cycle. For men, we estimate an average reduction of 3.8 disability-adjusted years (DALYs). The reduction in DALYs is relatively small for women. The gain in quality-adjusted life years (QALYs) is almost enough to offset all of the costs associated with program implementation for males and half of program costs for women.

Background Discrete choice experiments (DCEs) are widely used to elicit health state preferences. However, additional information is required to transform values to a scale with dead valued at 0 and full health valued at 1. This paper presents DCE-VAS, an understandable and easy anchoring method with low participant burden based on the visual analogue scale (VAS). Methods Responses from 1450 members of the UK general public to a discrete choice experiment (DCE) were analysed using mixed logit models. Latent scale valuations were anchored to a full health = 1, dead = 0 scale using participants’ VAS ratings of three states including the dead. The robustness of results was examined. This included a filtering procedure with the influence each individual respondent had on valuation being calculated, and those whose influence was more than two standard deviations away from the mean excluded. Results Coefficients in all models were in the expected direction and statistically significant. Excluding respondents who self-reported not understanding the VAS task did not significantly influence valuation, but excluding a small number who valued 33333 extremely low did. However, after eight respondents were removed via the filtering procedure, valuations were robust to removing other participants. Conclusion DCE-VAS is a feasible way of anchoring DCE results to a 0–1 anchored scale with low additional respondent burden.

Outcomes in economic evaluations, such as health utilities and costs, are products of multiple variables, often requiring complete item responses to questionnaires. Therefore, missing data are very common in cost-effectiveness analyses. Multiple imputations (MI) are predominately recommended and could be made either for individual items or at the aggregate level. We, therefore, aimed to assess the precision of both MI approaches (the item imputation vs. aggregate imputation) on the cost-effectiveness results. The original data set came from a cluster-randomized, controlled trial and was used to describe the missing data pattern and compare the differences in the cost-effectiveness results between the two imputation approaches. A simulation study with different missing data scenarios generated based on a complete data set was used to assess the precision of both imputation approaches. For health utility and cost, patients more often had a partial (9% vs. 23%, respectively) rather than complete missing (4% vs. 0%). The imputation approaches differed in the cost-effectiveness results (the item imputation: − 61,079€/QALY vs. the aggregate imputation: 15,399€/QALY). Within the simulation study mean relative bias (

ObjectivesPreterm birth occurs in more than 10% of U.S. births and is the leading cause of U.S. neonatal deaths, with estimated annual costs exceeding $25 billion USD. Using real-world data, we modeled the potential clinical and economic utility of a prematurity-reduction program comprising screening in a racially and ethnically diverse population with a validated proteomic biomarker risk predictor, followed by case management with or without pharmacological treatment.MethodsThe ACCORDANT microsimulation model used individual patient data from a prespecified, randomly selected sub-cohort (N=847) of a multicenter, observational study of U.S. subjects receiving standard obstetric care with masked risk predictor assessment (TREETOP; NCT02787213). All subjects were included in three arms across 500 simulated trials: standard of care (SoC, control); risk predictor/case management comprising increased outreach, education and specialist care (RP-CM, active); and risk predictor/case management with pharmacological treatment (RP-MM, active). In the active arms, only subjects stratified as higher-risk by the predictor were modeled as receiving the intervention, whereas lower-risk subjects received standard care. Higher-risk subjects’ gestational ages at birth were shifted based on published efficacies, and dependent outcomes, calibrated using national datasets, were changed accordingly. Subjects otherwise retained their original TREETOP outcomes. Arms were compared using survival analysis for neonatal and maternal hospital length of stay, bootstrap intervals for neonatal cost, and Fisher’s exact test for neonatal morbidity/mortality (significance, pResultsThe model predicted improvements for all outcomes. RP-CM decreased neonatal and maternal hospital stay by 19% (p=0.029) and 8.5% (p=0.001), respectively; neonatal costs’ point estimate by 16% (p=0.098); and moderate-to-severe neonatal morbidity/mortality by 29% (p=0.025). RP-MM strengthened observed reductions and significance. Point estimates of benefit did not differ by race/ethnicity.ConclusionsModeled evaluation of a biomarker-based test-and-treat strategy in a diverse population predicts clinically and economically meaningful improvements in neonatal and maternal outcomes.Plain language summaryPreterm birth, defined as delivery before 37 weeks’ gestation, is the leading cause of illness and death in newborns. In the United States, more than 10% of infants is born prematurely, and this rate is substantially higher in lower-income, inner-city and Black populations. Prematurity associates with substantially increased risk of short- and long-term medical complications and can generate significant costs throughout the lives of affected children. Annual U.S. health care costs to manage short- and long-term prematurity complications are estimated to exceed $25 billion.Clinical interventions, including case management (increased patient outreach, education and specialist care), pharmacological treatment and their combination, can provide benefit to pregnancies at higher risk for preterm birth. Early and sensitive risk detection, however, remains a challenge.We have developed and validated a proteomic biomarker risk predictor for early identification of pregnancies at increased risk of preterm birth. The ACCORDANT study modeled treatments with real-world patient data from a racially and ethnically diverse U.S. population to compare the benefits of risk predictor testing plus clinical intervention for higher-risk pregnancies versus no testing and standard care. Measured outcomes included neonatal and maternal length of hospital stay, associated costs and neonatal morbidity and mortality. The model projected improved outcomes and reduced costs across all subjects, including ethnic and racial populations, when predicted higher-risk pregnancies were treated using case management with or without pharmacological treatment. The biomarker risk predictor shows high potential to be a clinically important component of risk stratification for pregnant women, leading to tangible gains in reducing the impact of preterm birth.

A new decision rule based on net benefit per capita is proposed and exemplified with the aim of assisting policymakers in deciding whether to lockdown or reopen an economy—fully or partially—amidst a pandemic. Bayesian econometric models using Markov chain Monte Carlo algorithms are used to quantify this rule, which is illustrated via several sensitivity analyses. While we use COVID-19 data from the United States to demonstrate the ideas, our approach is invariant to the choice of pandemic and/or country. The actions suggested by our decision rule are consistent with the closing and reopening of the economies made by policymakers in Florida, Texas, and New York, these states were selected to exemplify the methodology since they capture the broad spectrum of COVID-19 outcomes in the U.S.

Objectives Transfusion-dependent β-thalassemia (TDT) is a genetic disease that affects production of red blood cells. Conventional treatment involves regular red blood cell transfusions and iron chelation, which has a substantial impact on quality of life. While potentially curative, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is associated with risk of complications, including graft-versus-host disease (GvHD). Gene addition therapy, a novel treatment approach, involves autologous transplantation of the patient’s own genetically modified hematopoietic stem cells. The purpose of this study was to estimate utilities associated with treatment approaches for TDT. Methods General population respondents in England valued eight health state vignettes (developed with clinician, patient, and parent input) in time trade-off interviews. Results A total of 207 participants completed interviews (49.8% female; mean age = 43.2 years). Mean (SD) utilities for the pre-transplant health states were 0.73 (0.25) with oral chelation and 0.63 (0.32) with subcutaneous chelation. Mean utilities for the transplant year were 0.62 (0.35) for gene addition therapy, 0.47 (0.39) for allo-HSCT, and 0.39 (0.39) for allo-HSCT with acute GvHD. Post-transplant utilities were 0.93 (0.15) for transfusion independent, 0.75 (0.25) for 60% transfusion reduction, and 0.51 (0.38) for chronic GvHD. Acute and chronic GvHD were associated with significant disutility (acute = − 0.09, p p Conclusions Utilities followed expected patterns, with logical differences between treatment options for TDT and substantially greater utility for transfusion independence than for ongoing treatment involving transfusion and chelation. These utilities may be useful in cost-utility models estimating the value of treatments for TDT.

Although the study of the association between interventions in primary care and health outcomes continues to produce mixed findings, programs designed to promote the greater compliance of General Practitioners and their diabetic patients with treatment guidelines have been increasingly introduced worldwide, in an attempt to achieve better quality diabetes care through the enhanced standardisation of patient supervision. In this study we use clinical data taken from the Diabetes Register of one Local Health Authority (LHAs) in Italy’s Emilia-Romagna Region for the period 2014-2015. Firstly, we test to see whether the monitoring activities prescribed for diabetics by regional diabetes guidelines, actually have a positive impact on patients’ health outcomes and increase appropriateness in health care utilization. Secondly, we investigate whether GPs’ participation in the local Diabetes Management Program (DMP) leads to improved patient compliance with regional guidelines. Our results show that such a program, which aims to increase GPs’ involvement and cooperation in following regional guidelines for best practices, achieves its goal of improved patient compliance with the prescribed actions. In turn, through the implementation of the DMP and the greater involvement of physicians, regional policies have succeeded in promoting better health outcomes and the improved appropriateness of health care utilization.

Background To date, a multi-country review evaluating the cost-of-illness (COI) studies from the Central and Eastern European (CEE) region has not yet been published. Our main objective was to provide a general description about published COI studies from CEE. Methods A systematic search was performed between 1 January 2006 and 1 June 2017 in Medline, EMBASE, The Cochrane Library, CINAHL, and Web of Science to identify all relevant COI studies from nine CEE countries. COI studies reporting costs without any restrictions by age, co-morbidities, or treatment were included. Methodology, publication standards, and cost results were analysed. Results We identified 58 studies providing 83 country-specific COI results: Austria (n = 9), Bulgaria (n = 16), Croatia (n = 3), the Czech Republic (n = 10), Hungary (n = 24), Poland (n = 11), Romania (n = 3), Slovakia (n = 3), and Slovenia (n = 4). Endocrine, nutritional, and metabolic diseases (18%), neoplasms (12%), infections (11%), and neurological disorders (11%) were the most frequently studied clinical areas, and multiple sclerosis was the most commonly studied disease. Overall, 57 (98%) of the studies explicitly stated the source of resource use data, 45 (78%) the study perspective, 34 (64%) the costing method, and 24 (58%) reported at least one unit costs. Regardless of methodological differences, a positive relationship was observed between costs of diseases and countries’ per capita GDP. Conclusions Cost-of-illness studies varied considerably in terms of methodology, publication practice, and clinical areas. Due to these heterogeneities, transferability of the COI results is limited across Central and Eastern European countries. Electronic supplementary material The online version of this article (10.1007/s10198-019-01066-x) contains supplementary material, which is available to authorized users.

Background We aimed to determine the acceptability of non-perfect health states with age using the EQ VAS and analyse the influencing factors. Methods We conducted a cross-sectional survey on a convenience sample from the general population (N = 200). Respondents were asked to indicate on the EQ VAS the health states that are still acceptable for ages between 30 and 80 years in 10-year intervals (VAS acceptable health curve, AHCvas). We recorded respondents’ current health, health-related lifestyle, demographic background and explored the reference person they imagined when evaluating acceptable health states. We evaluated the AHCvas by estimating linear multilevel models including a random intercept (estimated at age 30) and a random slope for age. Results AHCvas scores were available for 194 respondents (mean age = 42.8 years, range 19–93, 58% female). For ages of 30, 40, 50, 60, 70 and 80 years, mean AHCvas scores were 93, 87, 80, 73, 65 and 57, respectively. The decline of AHCvas was linear with age. Respondents’ age, health status, lifestyle and health-related experiences, as well as their reference point taken (e.g. imagining themselves, others or both during the valuation task) influenced significantly the acceptability of health problems. Conclusions When measured with the EQ VAS, health problems were increasingly acceptable with age. Capturing well the individual variability in the assessment of acceptable health states at different ages, the EQ VAS is a useful addition to EQ-5D-3L descriptive system-based measures of acceptable health. Electronic supplementary material The online version of this article (10.1007/s10198-019-01060-3) contains supplementary material, which is available to authorized users.

Background EQ-5D-3L population data are available only from Hungary, Poland and Slovenia in Central and Eastern Europe (CEE). We aimed to compare the accessible studies and estimate a regional EQ-5D-3L population norm for CEE. Methods A combined dataset using patient-level data of 8850 respondents was created. Based on the European Census of 2011, regional population norm estimates were calibrated by gender, age and education for the joint citizenry of 11 CEE countries. Results EQ-5D-3L health states were available for 6926 and EQ VAS scores for 6569 respondents. Demographic characteristics of the samples reflected the recruitment methods (Hungary: online; Slovenia: postal survey, Poland: personal interviews). Occurrence of problems differed significantly by educational level in all the five dimensions (p

Background The recent update of the European Union’s (EU) regulation on public procurement has created new opportunity for progress in the purchasing of medical devices by shifting towards focus on value from one purely on price. Patient-reported outcome measures (PROMs) may serve as additional tools for manufacturers to demonstrate value beyond traditional metrics of safety and performance and to differentiate their products in a market of increasing competition. The aim of our study was to investigate the extent to which PROMs are included in registered device studies in the EU and interpret the results in the context of the purchasing of medical devices. Methods Twelve device groups were searched in clinical trial registries to determine the frequency distribution of PROMs in related studies. Results Results indicate that clinical studies of the selected device categories are done predominately in the western EU nations and are increasingly including PROMs. In the United Kingdom 121 (65%) study, out of 186 included PROMs, and in Germany, 92 (52%) out of 178 between 1998 and 2018. Few device studies were done in the Central and Eastern European region, and out of 76 studies 27 (35%) included PROMs. Since there is no requirement to include PROMs in device studies for regulatory purposes, it seems probable that their increasing use is driven by competitive market pressures. Conclusion The trend of increasing use of PROMs might be driven by the demand of purchasers to demonstrate value of devices, but is manifested at different levels in various regions of the EU.

Background The nine-item Shared Decision Making Questionnaire (SDM-Q-9) is one of the most frequently applied instruments for assessing patients’ involvement in medical decision-making. Our objectives were to develop a Hungarian version of SDM-Q-9, to evaluate its psychometric properties and to compare its performance between primary and specialised care settings. Methods In 2019, a sample of adults (n = 537) representative of the Hungarian general population in terms of age, gender and geographic region completed an online survey with respect to a recent health-related decision. Outcome measures included SDM-Q-9 and Control Preferences Scale-post (CPSpost). Item characteristics, internal consistency reliability and the factor structure of SDM-Q-9 were determined. Results The overall ceiling and floor effects for SDM-Q-9 total scores were 12.3% and 2.2%, respectively. An excellent internal consistency reliability (Cronbach’s alpha 0.925) was demonstrated. Exploratory factor analysis resulted in a one-factor model explaining 63.5% of the variance of SDM-Q-9. A confirmatory factor analysis supported the acceptability of this model. Known-groups validity was confirmed with CPSpost categories; mean SDM-Q-9 total scores were higher in the ‘Shared decision’ category (72.6) compared to both ‘Physician decided’ (55.1, p = 0.0002) and ‘Patient decided’ (57.2, p = 0.0086) categories. In most aspects of validity and reliability, there was no statistically significant difference between primary and specialised care. Conclusions The overall good measurement properties of the Hungarian SDM-Q-9 make the questionnaire suitable for use in both primary and specialised care settings. SDM-Q-9 may be useful for health policies targeting the implementation of shared decision-making and aiming to improve efficiency and quality of care in Hungary.

In 2010–2012, new outpatient service locations were established in poor Hungarian micro-regions. We exploit this quasi-experiment to estimate the extent of substitution between outpatient and inpatient care. Fixed-effects Poisson models on individual-level panel data for years 2008–2015 show that the number of outpatient visits increased by 19% and the number of inpatient stays decreased by 1.6% as a result, driven by a marked reduction of potentially avoidable hospitalization (PAH) (5%). In our dynamic specification, PAH effects occur in the year after the treatment, whereas non-PAH only decreases with a multi-year lag. The instrumental variable estimates suggest that a one euro increase in outpatient care expenditures produces a 0.6 euro decrease in inpatient care expenditures. Our results (1) strengthen the claim that bringing outpatient care closer to a previously underserved population yields considerable health benefits, and (2) suggest that there is a strong substitution element between outpatient and inpatient care.

Background Health care expenditures (HCE) are known to steepen with increasing age, but the contributions of biological age, morbidity, or proximity to death as cost drivers are debated. Age-associated HCE growth can be studied across two dimensions: within fixed groups of persons with the same birth year followed over time (birth cohort), or the same age classes (e.g. 66 to 70 year olds) at different time points (age-class analysis). Using health insurance claims data including morbidity and mortality information, HCE growth was analyzed in Swiss mandatory health insurance for the years 1996 to 2011 and compared across the two age dimensions. Results Deflated HCE were analyzed for 104,000 persons from three birth cohorts (1921-25, 1926-30, 1931-35). Two-part regression models were adjusted for proximity-to-death (death within same or next calendar year) and morbidity indicators (hospitalization, high drug expenditures, and pharmaceutical cost groups from 2006 onwards). When analyzing HCE growth within birth cohorts, controlling for survival and morbidity status decreased age-associated HCE estimates by 31% to 51% compared to crude age averages. The total HCE volume share of decedents rose from 19% to 31% in the 1931-35 birth cohort and from 28% to 51% for the 1921-25 birth cohort. The analysis of same age classes (e.g. 71-75 year olds) over different years revealed no HCE growth (steepening) in excess of deflation for groups aged 75 years or less, and only moderate HCE growth for those ≥76 years. For the 76+ age classes, the population fraction of decedents decreased by -3% (age 76-80) and -15% (age 81-85) over time, whilst the total HCE volume share of decedent-associated HCE increased by +16% and +9%, with an HCE growth of +3.2% and +2.5% per year. Conclusions HCE growth was dominated by end-of-life HCE, but residual age-associated HCE growth remained pertinent, the extent of which however depended on morbidity indicator definitions. A better understanding of shifts in chronic disease prevalence with rising age, as well as associated HCE and survival impacts of treatment will be key for further refining future HCE projections. Electronic supplementary material The online version of this article (10.1186/s13561-019-0224-z) contains supplementary material, which is available to authorized users.

In response to the Covid-19 outbreak, among other previous ``non-pharmaceutical interventions99, on March 22, 2020 the Italian Government imposed an economic lockdown and ordered the closing of all non-essential economic activities. This paper estimates the causal effect of this measure on mortality by Covid-19 and on mobility patterns. The identification of the causal effect exploits the variation in the number of active workers across municipalities induced by the economic lockdown. The difference-in-difference empirical design compares outcomes in municipalities above and below the median variation in the share of active population before and after the lockdown within a province, also controlling for municipality-specific dynamics, daily-shocks at the provincial level and municipal unobserved characteristics. Our results show that the intensity of the economic lockdown is associated to a statistically significant reduction in mortality by Covid-19 and, in particular, for age groups between 40-64 and older. Back of the envelope calculations indicate that 4,793 deaths were avoided, in the 26 days between April 5 to April 30, in the 3,518 municipalities which experienced a more intense lockdown. Assuming linearity, a 1 percentage point reduction in the share of active population caused a 1.32 percentage points reduction in mortality by Covid-19. We also find that the economic lockdown, as expected, led to a reduction in human mobility. Several robustness checks corroborate our empirical findings.

Background This paper attempts to evaluate the primary health care system by evaluating health centres in Greece. Methods Malmquist Index Data Envelopment Analysis is applied to study the total productivity of 155 health centres in Greece during 2016–2018. The data were collected from the Ministry of Health and submitted to quality tests to ensure validity and avoid bias. Results This paper measures the productivity of each of the 155 health centres in Greece and how it shifted during 2016–2018. In addition, the overall productivity change of the 155 health centres over time is calculated and analysed as being due to technical efficiency or technological efficiency. The analysis of the mean values showed a decrease of 0.9% in the overall productivity factor from 2016 to 2017 and a decrease of 5.2% from 2017 to 2018. The overall decrease in the productivity of the 155 health centres was 3.1%. From 2016 to 2018, 59 health centres changed their productivity mainly due to technological change and 91 changed mainly due to technical efficiency change. One health centre showed regression to its total productivity due to equal regression of its technical efficiency and technology. Conclusions The method used is nonparametric data envelopment analysis along with the Malmquist index to include panel data in the analysis. Meaningful results were extracted by indicating the number of health centres that improved their productivity, regressed in productivity, or remained constant through the period 2016–2018. This paper may contribute to improving health centres’ efficiency and productivity. Furthermore, valuable results can be extracted for the National Health Care System to match available resources that correspond to each health centre’s needs, as well as for manager planners and stakeholders in primary health care.

Subjective well-being measures are gaining recognition as important determinants of health outcomes. This paper examines whether life satisfaction matters for healthcare usage in the older population and, if so, what might help explain this relationship. To that end, we develop a mediation framework and test whether lifestyle choices and social capital are pathways through which life satisfaction at baseline influences subsequent healthcare usage. Using Heckman's approach to correct for sample selection bias, we find that those high in life satisfaction may need less outpatient care. We also show that this effect is explained by increased physical activity.

We study the effect of unemployment on birth outcomes by exploiting geographical variation in the unemployment rate across local areas in England, and comparing siblings born to the same mother via family fixed effects. Using rich individual data from hospital administrative records between 2003 and 2012, babies' health is found to be strongly pro-cyclical. A one-percentage point increase in the unemployment rate leads to an increase in low birth weight and preterm babies of respectively 1.3 and 1.4%, and a 0.1% decrease in foetal growth. We find heterogenous responses: unemployment has an effect on babies' health which varies from strongly adverse in the most deprived areas, to mildly favourable in the most prosperous areas. We provide evidence of three channels that can explain the overall negative effect of unemployment on new-born health: maternal stress; unhealthy behaviours - namely excessive alcohol consumption and smoking; and delays in the take-up of prenatal services. While the heterogenous effects of unemployment by area of deprivation seem to be explained by maternal behaviour. Most importantly, we also show for the first time that selection into fertility is the main driver for the previously observed, opposite counter-cyclical results, e.g., Dehejia and Lleras-Muney (2004). Our results are robust to internal migration, different geographical aggregation of the unemployment rate, the use of gender-specific unemployment rates, and potential endogeneity of the unemployment rate which we control for by using a shift-share instrumental variable approach.