Systematic Review on the Cost Effectiveness of Prostate Cancer Screening in Europe.

BACKGROUND AND OBJECTIVE: In Europe, prostate cancer (PCa) is the most common cancer in men. Screening may therefore be crucial to lower health care costs, morbidity, and mortality. This systematic review aimed to provide a contemporary overview of the costs and benefits of PCa screening programmes. METHODS: A peer-reviewed literature search was conducted, using the PICO method. A detailed search strategy was developed in four databases based on the following key search terms: "PCa", "screening", and "cost effectiveness". Any type of economic evaluation was included. The search strategy was restricted to European countries, but no restrictions were set on the year of publication. KEY FINDINGS AND LIMITATIONS: A total of 7484 studies were identified initially. Of these, 19 studies described the cost effectiveness of PCa screening in Europe. Among the studies using an initially healthy study population, most focussed on risk- and/or age- and/or magnetic resonance imaging (MRI)-based screening in addition to prostate-specific antigen (PSA) testing and compared this with no screening. Incremental cost ratios (ICERs) varied from €5872 per quality-adjusted life year (QALY) to €372 948/QALY, with a median of €56 487/QALY. Risk-based screening followed by MRI testing seemed to be a more cost-effective strategy than no screening. CONCLUSIONS AND CLINICAL IMPLICATIONS: This systematic review indicates that screening programmes incorporating a risk-based approach and MRI have the potential to be cost effective. PATIENT SUMMARY: In this review, we looked at the cost effectiveness of prostate cancer screening in Europe. We found that a risk-based approach and incorporation of magnetic resonance imaging has the potential to be cost effective. However, there remains a knowledge gap regarding cost effectiveness of prostate cancer screening. Therefore, determinants of cost effectiveness require further investigation.

The Disease Burden and Economic Burden of Cancer in 9 Countries in the Middle East and Africa.

OBJECTIVES: The objective of this study was to characterize the epidemiological development of cancer in the Middle East and Africa since 2000 and to quantify its current economic impact. METHODS: Nine countries were studied: Algeria, Egypt, Jordan, Kuwait, Lebanon, Morocco, Saudi Arabia, South Africa, and the United Arab Emirates. Information on causes of death and disability-adjusted life-years (DALYs) was obtained from the World Health Organization. Information on cancer incidence was collected from local cancer registries and estimations by the World Health Organization. The economic burden of cancer was estimated from local health expenditure data and from age-specific mortality data. RESULTS: Between 2000 and 2019, cancer went from third-leading to second-leading cause of death (10% to 13% of all deaths) across these 9 countries. It also climbed from the sixth-leading to third-leading cause of DALYs (6% to 8% of all DALYs). New cancer cases per 100 000 inhabitants increased by 10% to 100% between 2000 and 2019, whereas future increases until 2040 range from 27% in Egypt to 208% in the United Arab Emirates, solely because of expected demographic changes. The economic burden of cancer ranged from around USD 15 per capita in the 4 African countries to USD 79 in Kuwait in 2019. CONCLUSIONS: Cancer is becoming one of the leading causes of disease burden in the Middle East and Africa. Patient numbers are expected to rise strongly in the coming decades. Increasing healthcare expenditure on appropriate cancer care is important to improve patient outcomes and can attenuate the economic impact of cancer on society.

The Allocation of the Economic Value of Second-Generation Antipsychotics Over the Product Life Cycle: The Case of Risperidone in Sweden and the United Kingdom.

OBJECTIVE: This article estimates the life-cycle value of risperidone as representative of second-generation antipsychotics (SGA) relative to haloperidol (first-generation antipsychotics). METHODS: We estimated the number of patients treated with risperidone in Sweden and the United Kingdom, from 1994 to 2017, using data of usage and volume sales. We collected data from the literature on the effectiveness (quality-adjusted life-years per patient per year), direct costs (health services), and indirect costs (productivity) of risperidone and haloperidol. We proxied the incremental value added by the new class (SGA) using a comparator from the inferior class. Next, we modeled the life-cycle uptake of risperidone to estimate the life-cycle incremental cost (ie, direct, indirect, and medicine costs), incremental quality-adjusted life-years, and net monetary benefit of risperidone. We also assessed the life-cycle distribution of the social surplus between the payer (consumer surplus) and the innovator (producer surplus). RESULTS: For the United Kingdom, consumer surplus represents around 72% of the total surplus before patent expiration and around 95% after patent expiration. For Sweden, the consumer surplus represents around 94% of the total surplus before patent expiration and around 99% after generic competition. CONCLUSION: These results suggest that the value added by SGAs to the system is higher than the expected value estimated using cost-effectiveness analysis at launch. Pricing and reimbursement decisions could recognize the full life cycle of value of innovative medicines. This not only presents a challenge of estimation but also of assessing the appropriate division of shares of social value.

Across-Country Variations of Real-World Data and Evidence for Drugs: A 5-European-Country Study.

OBJECTIVES: This study aimed to describe the role of real-world data (RWD) and real-world evidence (RWE) in health technology assessment (HTA) in 5 European countries and to identify the hurdles to the acceptance of RWE and suggest directions toward its more effective use. METHODS: Authors from France, Germany, Italy, and Sweden used a common template to extract evidence. For England, the Cancer Drugs Fund was described and analyzed as a particular model for the use of RWD to provide evidence for coverage decisions and managed entry agreements. RESULTS: In all countries except Germany, HTA bodies acknowledged the relevance of RWD/RWE to address postlaunch uncertainties. In Germany, evidence from randomized controlled trials remains the gold standard, and evidence based on RWD is generally rejected. Multiple sources of RWD exist, but the quality, the immediate relevance of existing sources, and their interoperability limit their adaptation to the specifics of a given drug. This leads to skepticism about the validity of the evidence. Timing is also a key issue: the production of evidence may not be synchronized with the HTA and pricing bodies' agendas. The Cancer Drugs Fund case emphasizes that a strong partnership among all stakeholders and a pragmatic use of existing data, alongside clinical evidence provided by companies, are key success factors. CONCLUSIONS: A continuous investment in national health information systems is a key issue for providing valid RWE. Processes and aids to guide the acceptability and usage of RWE derived from pairing between sources and questions are essential.

Motivation 2 Quit (M2Q): A cluster randomized controlled trial evaluating the effectiveness of Tobacco Cessation on Prescription in Swedish primary healthcare.

OBJECTIVE: To evaluate the effectiveness of Tobacco Cessation on Prescription (TCP) compared to standard treatment in socioeconomically disadvantaged areas in Swedish primary healthcare (PHC). STUDY DESIGN: A pragmatic cluster randomized controlled trial, where randomization was conducted at the PHC center level using a computer-generated random allocation sequence. SETTING: 18 PHC centers in socioeconomically disadvantaged areas in Stockholm. PARTICIPANTS: 250 adult daily tobacco users (56% female, 41% foreign born) with Swedish social security numbers and permanent resident permits, fluent in Swedish or Arabic, of which 140 responded to the follow-up at 6 months and 139 to the follow-up at 12 months. No blinding was applied. INTERVENTIONS: TCP (tobacco cessation counseling for ≥10 minutes, an individualized prescription for tobacco cessation treatment and follow-up on ≥1 occasion) compared to standard treatment. PRIMARY AND SECONDARY OUTCOME MEASURES: The primary outcome was self-reported 7-day abstinence at 6 months and the secondary outcomes included self-reported 7-day abstinence at 12 months and 3-month continued abstinence at 6 and 12 months follow-up. RESULTS: PHC centers were randomized to the intervention group (n = 8) and control group (n = 10). At the PHC centers, 250 patients (TCP n = 188, standard treatment n = 62) were recruited. There was a statistically significant effect of TCP compared to standard treatment for the outcomes 7-day abstinence at 6 months (OR adjusted 5.4, 95% CI 1.57 to 18.93) and 3-month continued abstinence at 6 (OR adjusted 6.4, 95% CI 1.30 to 31.27) and 12 months follow-up (OR adjusted 7.8, 95% CI 1.25 to 48.82). CONCLUSIONS: TCP may be effective in achieving abstinence from tobacco use compared to standard treatment in the given setting but due to several limitations, resulting in high attrition rates and a low statistical power in the study, more research is needed to evaluate this. TRIAL REGISTRATION: ISRCTN 11498135.

A Proposal of a Cost-Effectiveness Modeling Approach for Heart Failure Treatment Assessment: Considering the Short- and Long-Term Impact of Hospitalization on Event Rates.

BACKGROUND: The rate of events such as recurrent heart failure (HF) hospitalization and death are known to dramatically increase directly after HF hospitalization. Furthermore, the number of HF hospitalizations is associated with irreversible long-term disease progression, which is in turn associated with increased event rates. However, cost-effectiveness models of HF treatments commonly fail to capture both the short- and long-term association between HF hospitalization and events. OBJECTIVE: The aim of this study was to provide a decision-analytic model that reflects the short- and long-term association between HF hospitalization and event rates. Furthermore, we assess the impact of omitting these associations. METHODS: We developed a life-time Markov cohort model to evaluate HF treatments, and modeled the short-term impact of HF hospitalization on event rates via a sequence of tunnel states, with transition probabilities following a parametric survival curve. The corresponding long-term impact was modeled via hazard ratios per HF hospitalization. We obtained baseline event rates and utilities from published literature. Subsequently, we assessed, for a hypothetical HF treatment, how omitting the modeled associations (through a simple two-state model) affects incremental quality-adjusted life-years (QALYs). RESULTS: We developed a model that incorporates both short- and long-term impacts of HF hospitalizations. Based on an assumed treatment effect of a 20% risk reduction for HF hospitalization (and associated reductions in all-cause mortality of 15%), omitting the short-term, the long-term, or both associations resulted in a 5%, 1%, and 22% decrease in QALYs gained, respectively. CONCLUSION: For both modeling components, i.e., the short- and long-term implications of HF hospitalization, the impact on incremental outcomes associated with treatment was substantial. Considering these aspects as proposed within this modeling approach better reflects the natural course of this progressive condition and will enhance the evaluation of future HF treatments.

Cost-effectiveness of proprotein convertase subtilisin/kexin type 9 inhibition with evolocumab in patients with a history of myocardial infarction in Sweden.

AIMS: To assess the cost-effectiveness of proprotein convertase subtilisin/kexin type 9 inhibition with evolocumab added to standard-of-care lipid-lowering treatment [maximum tolerated dose (MTD) of statin and ezetimibe] in Swedish patients with a history of myocardial infarction (MI). METHODS AND RESULTS: Cost-effectiveness was evaluated using a Markov model based on Swedish observational data on cardiovascular event rates and efficacy from the FOURIER trial. Three risk profiles were considered: recent MI in the previous year; history of MI with a risk factor; and history of MI with a second event within 2 years. For each population, three minimum baseline low-density lipoprotein cholesterol (LDL-C) levels were considered: 2.5 mmol/L (≈100 mg/dL), based on the current reimbursement recommendation in Sweden; 1.8 mmol/L (≈70 mg/dL), based on 2016 ESC/EAS guidelines; and 1.4 mmol/L (≈55 mg/dL), or 1.0 mmol/L (≈40 mg/dL) for MI with a second event, based on 2019 ESC/EAS guidelines. Proprotein convertase subtilisin/kexin type 9 inhibition with evolocumab was associated with increased quality-adjusted life-years and costs vs. standard-of-care therapy. Incremental cost-effectiveness ratios (ICERs) were below SEK700 000 (∼€66 500), the generally accepted willingness-to-pay threshold in Sweden, for minimum LDL-C levels of 2.3 (recent MI), 1.7 (MI with a risk factor), and 1.7 mmol/L (MI with a second event). Sensitivity analyses demonstrated that base-case results were robust to changes in model parameters. CONCLUSION: Proprotein convertase subtilisin/kexin type 9 inhibition with evolocumab added to MTD of statin and ezetimibe may be considered cost-effective at its list price for minimum LDL-C levels of 1.7-2.3 mmol/L, depending on risk profile, with ICERs below the accepted willingness-to-pay threshold in Sweden.

Value appropriation in hepatitis C.

BACKGROUND: In 2015, the Swedish government in an unprecedented move decided to allocate 150 million € to provide funding for new drugs for hepatitis C. This was triggered by the introduction of the first second generation of direct-acting antivirals (DAAs) promising higher cure rates and reduced side effects. The drugs were cost-effective but had a prohibitive budget impact. Subsequently, additional products have entered the market leading to reduction in prices and expansions of the eligible patient base. METHODS: We estimated the social surplus generated by the new DAAs in Stockholm, Sweden, for the years 2014-2019. The actual use and cost of the drugs was based on registry data. Effects on future health care costs, indirect costs and QALY gains were estimated using a Markov model based primarily on Swedish data and using previous generations of interferon-based therapies as the counterfactual. RESULTS: A considerable social surplus was generated, 15% of which was appropriated by the producers whose share fell rapidly over time as prices fell. Most of the consumer surplus was generated by QALY gains, although 10% was from reduced indirect costs. QALY gains increased less rapidly than the number of treated patients as the eligibility criteria was loosened. CONCLUSIONS: The transfer of funds from the government to the regions helped generate substantial surplus for both consumers and producers with indirect costs playing an important role. The funding model may serve as a model for the financing of innovative treatments in the future.

Healthcare pathways and resource use: mapping consequences of ambulance assessment for direct care with alternative healthcare providers.

BACKGROUND: A decision system in the ambulance allowing alternative pathways to alternate healthcare providers has been developed for older patients in Stockholm, Sweden. However, subsequent healthcare resource use resulting from these pathways has not yet been addressed. The aim of this study was therefore to describe patient pathways, healthcare utilisation and costs following ambulance transportation to alternative healthcare providers. METHODS: The design of this study was descriptive and observational. Data from a previous RCT, where a decision system in the ambulance enabled alternative healthcare pathways to alternate healthcare providers were linked to register data. The receiving providers were: primary acute care centre or secondary geriatric ward, both located at the same community hospital, or the conventional pathway to the emergency department at an acute hospital. Resource use over 10 days, subsequent to assessment with the decision system, was mapped in terms of healthcare pathways, utilisation and costs for the 98 included cases. RESULTS: Almost 90% were transported to the acute care centre or geriatric ward. The vast majority arriving to the geriatric ward stayed there until the end of follow-up or until discharged, whereas patients conveyed to the acute care centre to a large extent were admitted to hospital. The median patient had 6 hospital days, 2 outpatient visits and costed roughly 4000 euros over the 10-day period. Arrival destination geriatric ward indicated the longest hospital stay and the emergency department the shortest. However, the cost for the 10-day period was lower for cases arriving to the geriatric ward than for those arriving to the emergency department. CONCLUSIONS: The findings support the appropriateness of admittance directly to secondary geriatric care for older adults. However, patients conveyed to the acute care centre ought to be studied in more detail with regards to appropriate level of care.

Costs of diabetes complications: hospital-based care and absence from work for 392,200 people with type 2 diabetes and matched control participants in Sweden.

AIMS/HYPOTHESIS: The risk of complications and medical consequences of type 2 diabetes are well known. Hospital costs have been identified as a key driver of total costs in studies of the economic burden of type 2 diabetes. Less evidence has been generated on the impact of individual diabetic complications on the overall societal burden. The objective of this study was to analyse costs of hospital-based healthcare (inpatient and outpatient care) and work absence related to individual macrovascular and microvascular complications of type 2 diabetes in Sweden in 2016. METHODS: Data for 2016 were retrieved from a Swedish national retrospective observational database cross-linking individual-level data for 1997-2016. The database contained information from population-based health, social insurance and socioeconomic registers for 392,200 people with type 2 diabetes and matched control participants (5:1). Presence of type 2 diabetes and of diabetes complications were derived using all years, 1997-2016. Costs of hospital-based care and of absence from work due to diabetes complications were estimated for the year 2016. Regression analysis was used for comparison with control participants to attribute absence from work to individual complications, and to account for joint presence of complications. RESULTS: Use of hospital care for complications was higher in type 2 diabetes compared with control participants in 2016: 26% vs 12% had ≥1 hospital contact; there were 86,104 vs 24,608 outpatient visits per 100,000 people; and there were 9894 vs 2546 inpatient admissions per 100,000 people (all p < 0.001). The corresponding total costs of hospital-based care for complications were €919 vs €232 per person (p < 0.001), and 74.7% of costs were then directly attributed to diabetes (€687 per person). Regression analyses distributed the costs of days absent from work across diabetes complications per se, basic type 2 diabetes effect and unattributed causes. Diabetes complications amounted to €1317 per person in 2016, accounting for possible complex interactions (25% of total costs of days absent). Key drivers of costs were the macrovascular complications angina pectoris, heart failure and stroke; and the microvascular complications eye diseases, including retinopathy, kidney disease and neuropathy. Early mortality in working ages cost an additional €579 per person and medications used in risk-factor treatment amounted to €418 per person. CONCLUSIONS/INTERPRETATION: The economic burden of complications in type 2 diabetes is substantial. Costs of absence from work in this study were found to be greater than of hospital-based care, highlighting the need for considering treatment consequences in a societal perspective in research and policy. Graphical abstract.

Time-driven activity-based costing for patients with multiple chronic conditions: a mixed-method study to cost care in a multidisciplinary and integrated care delivery centre at a university-affiliated tertiary teaching hospital in Stockholm, Sweden.

OBJECTIVE: This study can be applied to cost the complex non-standardised processes used to treat patients with multiple chronic conditions. DESIGN: A mixed-method approach to cost analysis, following a modified healthcare-specific version of the seven-step Time-Driven Activity-Based Costing (TDABC) approach. SETTING: A multidisciplinary integrated and person-centred care delivery centre at a university-affiliated tertiary teaching hospital in Stockholm, Sweden, designed to improve care coordination for patients with multiple chronic conditions, specifically diabetes, cardiovascular disease and kidney disease. PARTICIPANTS: 314 patients (248 men and 66 women) fit inclusion criteria. Average age was 80 years. RESULTS: This modified TDABC analysis costed outpatient care for patients with multiple chronic conditions. The approach accounted for the difficulty of conceptualising care cycles. The estimated total cost, stratified by resources, can be reviewed together with existing managerial accounting statements to inform management decisions regarding the multidisciplinary centre. CONCLUSIONS: This article demonstrates that the healthcare-specific seven-step approach to TDABC can be applied to cost care for patients with multiple chronic conditions, where pathways are not yet discernable. It became clear that there was a need for slight methodological adaptations for this particular patient group to make it possible to cost these pathways, stratified by activity and resource. The value of this approach can be discerned from the way management incorporated the results of this analysis into the development of their hospital strategy. In the absence of integrated data infrastructures that can link patients and resources across financial, clinical and process data sets, the scalability of this method will be difficult.

The cost of cancer in Europe 2018.

BACKGROUND: Cancer care is evolving rapidly, and costs and value of new treatments are frequently debated. Up-to-date evidence on the total cost of cancer is needed to inform policy decisions. This study estimates the cost of cancer in Europe in 2018 and extends a previous analysis for 1995-2014. METHODS: Cancer-specific health expenditure were derived from national estimates. Data on cancer drug sales were obtained from IQVIA. The productivity loss from premature mortality was estimated from data from Eurostat and the World Health Organization. Estimates of the productivity loss from morbidity and informal care costs were based on previous studies. FINDINGS: The total cost of cancer was €199 billion in Europe (EU-27 plus Iceland, Norway, Switzerland, and the United Kingdom) in 2018. Total costs ranged from €160 per capita in Romania to €578 in Switzerland (after adjustment for price differentials). Health expenditure on cancer care were €103 billion, of which €32 billion were spent on cancer drugs. Informal care costs were €26 billion. The total productivity loss was €70 billion, composed of €50 billion from premature mortality and €20 billion from morbidity. INTERPRETATION: Health expenditure on cancer care were of a similar magnitude as the sum of non-health-care costs in 2018. Over the last two decades, health spending on cancer has increased faster than the increase in cancer incidence. The productivity loss from premature mortality has decreased because of reductions in mortality in the working-age population. Trends in informal care costs and productivity loss from morbidity are uncertain because of lack of comparable data.

Risk sharing in managed entry agreements-A review of the Swedish experience.

BACKGROUND: In 2014 managed entry agreements (MEA) between Swedish health care payers (county councils), the reimbursement authority (the Dental and Pharmaceutical Benefits Agency (TLV)) and pharmaceutical companies were introduced to facilitate early and equal access to treatments. MEAs complement the value-based pricing system for out-patient drugs and enables stake-holders to negotiate risk-sharing agreements. AIM: The aim of the study was to review existing agreements, describe the type of uncertainty dealt with, and to analyze incentives created using the literature on performance-based reimbursement schemes in Sweden. METHOD: A document review of all agreements made in the period January 2015 to August 2019 was conducted, classifying agreements by the type of uncertainty dealt with. RESULTS: Agreements were made for 56 products during the studied time period (45 ongoing), mainly in the fields of hepatitis C (n = 10) and oncology (n = 14). Uncertainties addressed in ongoing agreements included size of treated population (10), treatment duration (13), and effectiveness (9). The mechanism for risk-sharing was limited to refunds based on patient numbers, duration or just flat-rate refunds. The estimated refund in 2018 was 50 % of total sales. DISCUSSION: This review show that the main driver behind risk sharing in Sweden so far have seem to be affordability rather than managing uncertainty.

Updated Cost-effectiveness Analysis of Evolocumab in Patients With Very High-risk Atherosclerotic Cardiovascular Disease.

Importance: In October 2018, evolocumab was made available at a reduced annual list price of $5850 in the United States. This 60% reduction was aimed at improving patient access by lowering patient copays. Shortly thereafter, the 2018 American College of Cardiology/American Heart Association cholesterol management guideline was released. An updated cost-effectiveness analysis of evolocumab in the United States may be therefore of interest to payers and prescribers. Objective: To present an updated cost-effectiveness analysis of evolocumab added to standard background therapy compared with standard background therapy alone in patients with very high-risk atherosclerotic cardiovascular disease, reflecting the 2018 ACC/AHA guideline definition and using the new evolocumab list price. Design, Setting, and Participants: This study used the Markov model originally used in a previous study by Fonarow et al in 2017. A US societal perspective was considered, and a range of baseline cardiovascular event rates were modeled to reflect varying risk profiles in clinical practice within patients with very high-risk atherosclerotic cardiovascular disease. Exposures: Addition of evolocumab to standard background therapy, including maximally tolerated statin therapy (ie, the maximum intensity of statin therapy a patient can safely receive), with or without ezetimibe. Main Outcomes and Measures: Major cardiovascular events (myocardial infarction, ischemic stroke, and cardiovascular death), costs, quality-adjusted life-years, and incremental cost-effectiveness ratios. Results: Evolocumab was associated with both increased costs and improved outcomes when added to standard background therapy. Incremental costs ranged from $22 228 to $3411, depending on the varying level of risk within the defined population. Incremental quality-adjusted life years ranged from 0.39 to 0.44. Incremental cost-effectiveness ratios ranged from $56 655 to $7667 per quality-adjusted life-year gained. For a range of baseline cardiovascular event rates in patients with very high-risk atherosclerotic cardiovascular disease, incremental cost-effectiveness ratios were below the generally accepted willingness-to-pay thresholds. Moreover, the ratios were below the threshold of $50 000 per quality-adjusted life-years gained for any baseline rate of 6.9 or more events per 100 patient-years. Conclusions and Relevance: At its current list price, the addition of evolocumab to standard background therapy meets accepted cost-effectiveness thresholds across a range of baseline cardiovascular event rates in patients with very high-risk atherosclerotic cardiovascular disease as defined by the 2018 ACC/AHA guideline.

Exploring staff experience of economic efficiency requirements in health care: A mixed method study.

BACKGROUND: Economic resources are limited in health care, and governance strategies are used to push provider organizations to use resources efficiently. Although studies show that hybrid managers are successful in reconciling economic efficiency requirements with professional values to meet patient needs, surprisingly few studies focus on staff. The aim of this study is to explore staff members' experience of economic efficiency requirements. METHODS: A mixed method design was applied, targeting multi-professional staff in the Department of Rehabilitation Medicine in a Swedish university hospital. Survey data was collected (n = 93), followed by focus-group interviews to support the understanding of the quantitative findings. FINDINGS: The findings show that health care staff is knowledgeable and intrinsically motivated to consider efficiency requirements, albeit it should not dominate clinical decisions. However, staff experiences little influence over resource allocation and identifies limitations in the system's abilities to meet patient needs. Staff experience incorporates a local unit and a system perspective. CONCLUSION: Staff members are aware of economic efficiency requirements and will behave accordingly if patients are not at risk. However, their engagement seems to rely on how economic efficiency requirements are handled at multiple system levels and their trust in the system to fairly support patient needs.

Standardized Questionnaire for the Measurement, Valuation, and Estimation of Costs of Informal Care Based on the Opportunity Cost and Proxy Good Method.

Costs of informal care account for a significant component of total societal costs for many chronic and disabling illnesses. Yet, costs associated with the provision of informal care is seldom included in economic evaluations of new health technologies, increasing the risk of suboptimal decisions on the allocation of resources from the perspective of society. Our objective was to propose a standardized questionnaire for the measurement, valuation, and estimation of caregiver indirect (productivity) and informal care costs as separate mutually exclusive subsets of total costs in cost-of-illness studies and as an input to economic evaluations from the societal perspective. We developed a questionnaire for data collection and step-by-step analysis procedures for resource valuation and cost estimation. Data concerning absenteeism from work and time devoted to informal care were recorded using the recall method. Indirect (productivity) and paid informal care costs were valued and estimated according to the human-capital approach as the loss of production. Unpaid informal care costs were valued and estimated as the loss of leisure time quantified using the opportunity cost and proxy good method. The new questionnaire, titled the Caregiver Indirect and Informal Care Cost Assessment Questionnaire, contains 13 questions regarding caregiver current and previous work status, productivity, and the provision of informal care (stratified by time devoted to household activities, personal care, practical support, and emotional support). The proposed questionnaire should be helpful to inform the design, implementation, and execution of future cost-of-illness studies and economic evaluations from the perspective of society.

Psychometric properties of the Zarit Caregiver Burden Interview administered to caregivers to patients with Duchenne muscular dystrophy: a Rasch analysis.

PURPOSE: To explore the psychometric properties of the full 22-item English (UK and US) version of the Zarit Caregiver Burden Interview administered to caregivers to patients with Duchenne muscular dystrophy. MATERIALS AND METHODS: Caregivers to patients with Duchenne muscular dystrophy from the United Kingdom and the United States, recruited through the TREAT-NMD network, completed the Zarit Caregiver Burden Interview online. The psychometric properties of the Zarit Caregiver Burden Interview were examined using Rasch analysis. RESULTS: A total of 475 caregivers completed the Zarit Caregiver Burden Interview. Model misfit was identified for 9 of 22 items (mean item fit residual 0.061, SD: 2.736) and 13 of 22 items displayed disordered thresholds. The overall item-trait interaction chi-square value was 499 (198 degrees of freedom, p < 0.001). The mean person fit residual was estimated at -0.213 (SD: 1.235). The Person Separation Index and Cronbach's α were estimated at 0.902 and 0.914, respectively. Item dependency was low and we found no significant differential item functioning by country or sex. CONCLUSION: Our Rasch analysis shows that the Zarit Caregiver Burden Interview fails to fully operationalize a quantitative conceptualization of caregiver burden among caregivers to patients with Duchenne muscular dystrophy from the United Kingdom and the United States. Further research is needed to understand the psychometric properties of the Zarit Caregiver Burden Interview in other populations and settings. Implications for Rehabilitation Duchenne muscular dystrophy is a terminal disease characterized by progressive muscle degeneration resulting in substantial disability and a significant burden on family caregivers. The Zarit Caregiver Burden Interview is one of the most widely applied measures of caregiver burden. Our Rasch analysis suggests that the Zarit Caregiver Burden Interview is not fit for purpose to measure burden in UK and US caregivers to patients with Duchenne muscular dystrophy. Clinicians and decision-makers should interpret Zarit Caregiver Burden Interview data from these populations with caution.

The development and validation of a scale to explore staff experience of governance of economic efficiency and quality (GOV-EQ) of health care.

BACKGROUND: In publicly funded health care systems, governance models are developed to push public service providers to use tax payers' money more efficiently and maintain a high quality of service. Although this implies change in staff behaviors, evaluation studies commonly focus on organizational outputs. Unintended consequences for staff have been observed in case studies, but theoretical and methodological development is necessary to enable studies of staff experience in larger populations across various settings. The aim of the study is to develop a self-assessment scale of staff experience of the governance of economic efficiency and quality of health care and to assess its psychometric properties. METHODS: Factors relevant to staff members' experience of economic efficiency and quality requirements of health care were identified in the literature and through interviews with practitioners, and then compared to a theoretical model of behavior change. Relevant experiences were developed into sub-factors and items. The scale was tested in collaboration with the Department of Rehabilitation Medicine at a university hospital. 93 staff members participated. The scale's psychometric properties were assessed using exploratory factor analysis, analysis of internal consistency and criterion-related validity. RESULTS: The analysis revealed an eight factor structure (including sub-factors knowledge and awareness, opportunity to influence, motivation, impact on professional autonomy and organizational alignment), and items showed strong factor loadings and high internal consistency within sub-factors. Sub-factors were interrelated and contributed to the prediction of impact on clinical behavior (criterion). CONCLUSIONS: The scale clearly distinguishes between various experiences regarding economic efficiency and quality requirements among health care staff, and shows satisfactory psychometric quality. The scale has broad applications for research and practice, as it serves as a tool for capturing staff members' perspectives when evaluating and improving health care governance. The scale could also be useful for understanding the underlying processes of changes in provider performance and for adapting management strategies to engage staff in driving change that contributes to increased economic efficiency and quality, for the benefit of health care systems, patients and staff.