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Background: Market authorization does not guarantee patient access to any given drug. This is particularly true for costly orphan drugs because access depends primarily on co-payments, reimbursement policies and prices. The objective of this article is to identify differences in the availability of orphan drugs and in patient access to them in 11 pharmaceutical markets: Australia, Canada, England, France, Germany, Hungary, the Netherlands, Poland, Slovakia, Switzerland and the US.Methods: Four rare diseases were selected for analysis: pulmonary arterial hypertension (PAH), Fabry disease (FD), hereditary angioedema (HAE) and chronic myeloid leukaemia (CML). Indicators for availability were defined as (i) the indications for which orphan drugs had been authorized in the treatment of these diseases; (ii) the application date; and (iii) the date upon which these drugs received market authorization in each country. Indicators of patient access were defined as (i) the outcomes of technology appraisals; (ii) the extent of coverage provided by healthcare payers; and (iii) the price of the drugs in each country. For PAH we analysed bosentan, iloprost, sildenafil, treprostinil (intravenous and inhaled) as well as sitaxentan and ambrisentan; for FD we analysed agalsidase alfa and agalsidase beta; for HAE we analysed icatibant, ecallantide and two complement C1s inhibitors; for CML we analysed imatinib, dasatinib and nilotinib.Results: Most drugs included in this study had received market authorization in all countries, but the range of indications for which they had been authorized differed by country. The broadest range of indications was found in Australia, and the largest variations in indications were found for PAH drugs. Authorization process speed (the time between application and market authorization) was fastest in the US, with an average of 362 days, followed by the EU (394 days). The highest prices for the included drugs were found in Germany and the US, and the lowest in Canada, Australia and England. Although the prices of all of the included drugs were high compared with those of most non-orphan drugs, most of the insurance plans in our country sample provided coverage for authorized drugs after a certain threshold.Conclusions: Availability of and access to orphan drugs play a key role in determining whether patients will receive adequate and efficient treatment. Although the present study showed some variations between countries in selected indicators of availability and access to orphan drugs, virtually all of the drugs in question were available and accessible in our sample. However, substantial co-payments in the US and Canada represent important barriers to patient access, especially in the case of expensive treatments such as those analysed in this study. Market exclusivity is a strong instrument for fostering orphan drug development and drug availability. However, despite the positive effect of this instrument, the conditions under which market exclusivity is granted should be reconsidered in cases where the costs of developing an orphan drug have already been amortized through the use of the drug's active ingredient for the treatment of a common indication. [ABSTRACT FROM AUTHOR]

Objective: To explore variation in rates of acute care utilization for mental health conditions, including hospitalizations and emergency department (ED) visits, across high-income countries before and during the COVID-19 pandemic., Data Sources and Study Setting: Administrative patient-level data between 2017 and 2020 of eight high-income countries: Canada, England, Finland, France, New Zealand, Spain, Switzerland, and the United States (US)., Study Design: Multi-country retrospective observational study using a federated data approach that evaluated age-sex standardized rates of hospitalizations and ED visits for mental health conditions., Principal Findings: There was significant variation in rates of acute mental health care utilization across countries. Among the subset of four countries with both hospitalization and ED data, the US had the highest pre-COVID-19 combined average annual acute care rate of 1613 episodes/100,000 people (95% CI: 1428, 1797). Finland had the lowest rate of 776 (686, 866). When examining hospitalization rates only, France had the highest rate of inpatient hospitalizations of 988/100,000 (95% CI 858, 1118) while Spain had the lowest at 87/100,000 (95% CI 76, 99). For ED rates for mental health conditions, the US had the highest rate of 958/100,000 (95% CI 861, 1055) while France had the lowest rate with 241/100,000 (95% CI 216, 265). Notable shifts coinciding with the onset of the COVID-19 pandemic were observed including a substitution of care setting in the US from ED to inpatient care, and overall declines in acute care utilization in Canada and France., Conclusion: The study underscores the importance of understanding and addressing variation in acute care utilization for mental health conditions, including the differential effect of COVID-19, across different health care systems. Further research is needed to elucidate the extent to which factors such as workforce capacity, access barriers, financial incentives, COVID-19 preparedness, and community-based care may contribute to these variations., What Is Known on This Topic: Approximately one billion people globally live with a mental health condition, with significant consequences for individuals and societies. Rates of mental health diagnoses vary across high-income countries, with substantial differences in access to effective care. The COVID-19 pandemic has exacerbated mental health challenges globally, with varying impacts across countries., What This Study Adds: This study provides a comprehensive international comparison of hospitalization and emergency department visit rates for mental health conditions across eight high-income countries. It highlights significant variations in acute care utilization patterns, particularly in countries that are more likely to care for people with mental health conditions in emergency departments rather than inpatient facilities The study identifies temporal and cross-country differences in acute care management of mental health conditions coinciding with the onset of the COVID-19 pandemic., (© 2024 The Author(s). Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Ageism in healthcare has received increased attention in recent years, but literature focusing on how it affects individuals living with rare diseases remains scant. The rare disease population already faces obstacles when navigating health systems, and ageism has the potential to exacerbate existing health inequities. We conducted a systematic review of peer-reviewed and gray literature on health inequities in rare disease populations, seeking to identify publications that reported primary or secondary data on the equitable or inequitable treatment of these populations, or that discussed related regulatory, moral, or philosophical issues. Our aims were to understand how health inequities in these populations arise, how they are justified from societal points of view, how they manifest themselves in laws and regulations, and what effects they have on health care access and health outcomes. We retrieved information from 63 publications, which we inductively synthesized into five categories: ethical discussions, societal preferences, regulations, access to care, and health outcomes. Integrating insights from these categories, we developed an Ethical Spectrum and Resource Allocation Framework, which explains the emergence of equity issues and how they are manifested in health systems. By providing a better understanding of the root causes of health inequities, particularly among older adults, the framework can inform health policymaking, improving access to care and health outcomes for rare disease patients., Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.)

Newborn screening is a public health measure to diagnose rare diseases at birth, thereby minimising negative effects of late treatment. Genomic technologies promise an unprecedented expansion of screened diseases at low cost and with transformative potential for newborn screening programmes. However, barriers to the public funding of genomic newborn screening are poorly understood, particularly in light of the heterogenous European newborn screening landscape. This study therefore aims to understand whether international newborn screening experts share a common understanding of the barriers to fund genomic newborn screening. For this purpose, we convened 21 European newborn screening experts across a range of professions and national backgrounds in a Delphi study. Stable consensus, determined via the Wilcoxon matched-pairs signed-ranks test, was found via three consecutive survey rounds for all presented barriers. Experts generally judged the scenario of genomic newborn screening being available to every newborn in seven years to be unlikely, identifying treatability and the absence of counselling and a skilled workforce as the most significant barriers to public funding. We identify value re-definition for rare disease treatments, centralisation of genomic expertise, and international research consortia as avenues for pan-European actions which build on the consensus achieved by our Delphi panel., Competing Interests: Declaration of competing interest Anne-Sophie Chalandon and Alicia Granados are employed by Sanofi S.A. The views presented here are those of the authors and not necessarily those of Sanofi S.A., its directors, officers, or staff. Julian Nam is employed by F. Hoffmann-La Roche Ltd. The views presented here are those of the authors and not necessarily those of F. Hoffmann-La Roche Ltd., its directors, officers, or staff. Andreas Clemens is employed by Novartis AG. The views presented here are those of the authors and not necessarily those of Novartis AG, its directors, officers, or staff. All other authors declare that they have no conflict of interest., (Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.)

Countries with small and/or less-resourced regulatory authorities that operate outside of a larger medical product regulatory system face a regulatory strategy dilemma. These countries may rely on foreign well-resourced regulators by recognising the regulatory decisions of large systems and following suit (regulatory reliance); alternatively, such countries may extend formal decision recognition to regulators in multiple other jurisdictions with similar oversight and public health goals, following a system which we call regulatory pluralism. In this policy comment, we discuss three potential limitations to regulatory pluralism: (i) regulatory escape, in which manufacturers exploit regulatory variation and choose the lowest regulatory threshold for their product; (ii) increased fragmentation and complexity for countries adopting this approach, which may, in turn, lead to inconsistent processes; and (iii) loss of international bargaining power in developing regulatory policies. We argue that regulatory pluralism has important long-term implications, which may not be readily apparent to policy makers opting for such an approach. We advocate for the long-term value of an alternative approach relying on greater collaboration between regulatory authorities, which may relieve administrative pressures on countries with small or less-resourced regulatory authorities, regardless of whether countries pursue a strategy of domestic regulation or regulatory pluralism., Competing Interests: Conflict of interest None., (Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.)

One Health is an important approach to addressing health threats and promoting health through interdisciplinary health, policy, legislation and leadership research to achieve better human and animal health and better outcomes for the planet. The Covid-19 pandemic has triggered an urgent awareness of the need to develop innovative integrative solutions to address root causes of such threats to health, which requires collaboration across disciplines and amongst different sectors and communities. We explore how achieving the Quadripartite Organizations' One Health Joint Plan of Action can be supported by the concepts of 'One Health literacy' and 'One Health governance' and promote both academic and policy dialogue. We show how One Health literacy and One Health governance influence and reinforce each other, while an interdisciplinary systems leadership approach acts as a catalyst and mechanism for understanding and enacting change. Based on our understanding of how these elements influence the implementation of the One Health approach, we describe a model for considering how external triggering events such as the Covid-19 pandemic may prompt a virtuous circle whereby exposure to and exploration of One Health issues may lead to improved One Health literacy and to better governance. We close with recommendations to international organisations, national governments and to leaders in policy, research and practice to enhance their influence on society, the planetary environment, health and well-being., Competing Interests: Declarations of competing interest None., (Copyright © 2024 The Author(s). Published by Elsevier B.V. All rights reserved.)

Background: The delay in diagnosis for rare disease (RD) patients is often longer than for patients with common diseases. Machine learning (ML) technologies have the potential to speed up and increase the precision of diagnosis in this population group. We aim to explore the expectations and experiences of the members of the European Reference Networks (ERNs) for RDs with those technologies and their potential for application., Methods: We used a mixed-methods approach with an online survey followed by a focus group discussion. Our study targeted primarily medical professionals but also other individuals affiliated with any of the 24 ERNs., Results: The online survey yielded 423 responses from ERN members. Participants reported a limited degree of knowledge of and experience with ML technologies. They considered improved diagnostic accuracy the most important potential benefit, closely followed by the synthesis of clinical information, and indicated the lack of training in these new technologies, which hinders adoption and implementation in routine care. Most respondents supported the option that ML should be an optional but recommended part of the diagnostic process for RDs. Most ERN members saw the use of ML limited to specialised units only in the next 5 years, where those technologies should be funded by public sources. Focus group discussions concluded that the potential of ML technologies is substantial and confirmed that the technologies will have an important impact on healthcare and RDs in particular. As ML technologies are not the core competency of health care professionals, participants deemed a close collaboration with developers necessary to ensure that results are valid and reliable. However, based on our results, we call for more research to understand other stakeholders' opinions and expectations, including the views of patient organisations., Conclusions: We found enthusiasm to implement and apply ML technologies, especially diagnostic tools in the field of RDs, despite the perceived lack of experience. Early dialogue and collaboration between health care professionals, developers, industry, policymakers, and patient associations seem to be crucial to building trust, improving performance, and ultimately increasing the willingness to accept diagnostics based on ML technologies., (© 2024. The Author(s).)

Since 72% of rare diseases are genetic in origin and mostly paediatrics, genetic newborn screening represents a diagnostic "window of opportunity". Therefore, many gNBS initiatives started in different European countries. Screen4Care is a research project, which resulted of a joint effort between the European Union Commission and the European Federation of Pharmaceutical Industries and Associations. It focuses on genetic newborn screening and artificial intelligence-based tools which will be applied to a large European population of about 25.000 infants. The neonatal screening strategy will be based on targeted sequencing, while whole genome sequencing will be offered to all enrolled infants who may show early symptoms but have resulted negative at the targeted sequencing-based newborn screening. We will leverage artificial intelligence-based algorithms to identify patients using Electronic Health Records (EHR) and to build a repository "symptom checkers" for patients and healthcare providers. S4C will design an equitable, ethical, and sustainable framework for genetic newborn screening and new digital tools, corroborated by a large workout where legal, ethical, and social complexities will be addressed with the intent of making the framework highly and flexibly translatable into the diverse European health systems., Competing Interests: none., (Copyright: © 2023 Garnier et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.)

Objective: Development of an aggregate quality index to evaluate hospital performance in cardiovascular events treatment., Methods: We applied a two-stage regression approach using an accelerated failure time model based on variance weights to estimate hospital quality over four cardiovascular interventions: elective coronary bypass graft, elective cardiac resynchronization therapy, and emergency treatment for acute myocardial infarction. Mortality and readmissions were used as outcomes. For the estimation we used data from a statutory health insurer in Germany from 2005 to 2016., Results: The precision-based weights calculated in the first stage were higher for mortality than for readmissions. In general, teaching hospitals performed better in our ranking of hospital quality compared to non-teaching hospitals, as did private not-for-profit hospitals compared to hospitals with public or private for-profit ownership., Discussion: The proposed approach is a new method to aggregate single hospital quality outcomes using objective, precision-based weights. Likelihood-based accelerated failure time models make use of existing data more efficiently compared to widely used models relying on dichotomized data. The main advantage of the variance-based weights approach is that the extent to which an indicator contributes to the aggregate index depends on the amount of its variance., (© 2022. The Author(s).)

Introduction: The aim of this study was to evaluate the effects of a non-invasive telemonitoring intervention on mortality, healthcare costs, and hospital and pharmaceutical utilisation in patients with chronic heart failure (CHF) of a large statutory health insurer in Germany., Methods: In a retrospective observational cohort study using real-world data, we assessed differences between 635 patients who received a telemonitoring intervention versus 635 receiving usual care covering 36 months after intervention. We used propensity score matching on a set of 102 parameters collected in the 24-month pre-intervention period to correct for observed differences, as well as difference-in-difference (DiD) estimators to account for unobserved differences. We analysed the effect of the intervention for up to three years on (i) all-cause mortality; (ii) costs (i.e. inpatient stays, ambulatory care, pharmaceuticals, and medical aids and appliances); and (iii) healthcare utilisation (i.e. length and number of hospital stays, number of prescriptions)., Results: DiD estimates suggest lower inpatient costs of the telemonitoring group of up to €1160 (95% confidence interval (CI): -2253 to -69) in year three. Ambulatory care costs increased significantly in all three years up to €316 (95% CI: 1267 to 505) per year. Telemonitoring had a positive effect on survival (hazard ratio = 0.71; 95% CI: 0.51 to 0.99) and increased the number of prescriptions for diuretics. Effects were more prominent for patients with severe CHF., Discussion: The study suggests that the telemonitoring intervention led to a significant decrease in mortality and a shift in costs from the inpatient to the ambulatory care sector 36 months after intervention.

One of the most pressing challenges facing most health care systems is rising costs. As the population ages and the demand for health care services grows, there is a growing need to understand the drivers of these costs across systems. This paper attempts to address this gap by examining utilization and spending of the course of a year for two specific high-need high-cost patient types: a frail older person with a hip fracture and an older person with congestive heart failure and diabetes. Data on utilization and expenditure is collected across five health care settings (hospital, post-acute rehabilitation, primary care, outpatient specialty and drugs), in six countries (Canada (Ontario), France, Germany, Spain (Aragon), Sweden and the United States (fee for service Medicare) and used to construct treatment episode Purchasing Power Parities (PPPs) that compare prices using baskets of goods from the different care settings. The treatment episode PPPs suggest other countries have more similar volumes of care to the US as compared to other standardization approaches, suggesting that US prices account for more of the differential in US health care expenditures. The US also differs with regards to the share of expenditures across care settings, with post-acute rehab and outpatient speciality expenditures accounting for a larger share of the total relative to comparators., Competing Interests: Declarations of Competing Interest None., (Copyright © 2022 The Author(s). Published by Elsevier B.V. All rights reserved.)

Following a series of major scandals over the past two decades, the medical device industry is now undergoing a major regulatory change: the implementation of the European Union Medical Device Regulation (MDR) of 2017. After a transitional period of four years, the MDR became fully binding in May 2021. Although the medium- and long-term impacts of the MDR are still unclear, a range of scholars, health care professionals, and industry representatives have begun to publish early evidence and publicize their views on how the new regulation may impact free trade and patient safety. Even a cursory view of their output reveals hypotheses and opinions that could hardly be more divergent. The reasons for this disagreement are unclear and may extend beyond the different interests of the various stakeholder groups. To find out why this might be the case, we examine the MDR and seek to improve scholarly understanding of this most extensive regulation of medical devices since the early 1990s. We offer policymakers suggestions to help them improve future regulation. Based on theoretical considerations, we conclude that while the MDR is highly likely to strengthen the internal market of the EU, its impact on patient safety will remain unclear as long as there are no comprehensive studies on this topic that are based on empirical data., Competing Interests: Declaration of Competing Interest None declared., (Copyright © 2022. Published by Elsevier B.V.)

Health economists have written extensively on the design and implementation of coverage with evidence development (CED) schemes and have proposed theoretical frameworks based on cost-effectiveness modeling and value of information analysis. CED may aid decision-makers when there is uncertainty about the (cost-)effectiveness of a new health technology at the time of reimbursement. Medical devices are potential candidates for CED schemes, as regulatory regimes do not usually require the same level of efficacy and safety data normally needed for pharmaceuticals. The purpose of this research is to assess whether the actual practice of CED for medical devices in Europe meets the theoretical principles proposed by health economists and whether theory and practice can be more closely aligned. Based on decision-makers' perceptions of the challenges associated with CED schemes, plus examples from the schemes themselves, we discuss a series of proposals for assessing the desirability of schemes, their design, implementation, and evaluation. These proposals, while reflecting the practical challenges with developing CED programs, embody many of the principles suggested by economists and should support decision-makers in dealing with uncertainty about the real-world performance of devices., (© 2022 The Authors. Health Economics published by John Wiley & Sons Ltd.)

Health economic modeling of novel technology at the early stages of a product lifecycle has been used to identify technologies that are likely to be cost-effective. Such early assessments are challenging due to the potentially limited amount of data. Modelers typically conduct uncertainty analyses to evaluate their effect on decision-relevant outcomes. Current approaches, however, are limited in their scope of application and imposes an unverifiable assumption, that is, uncertainty can be precisely represented by a probability distribution. In the absence of reliable data, an approach that uses the fewest number of assumptions is desirable. This study introduces a generalized approach for quantifying parameter uncertainty, that is, probability bound analysis (PBA), that does not require a precise specification of a probability distribution in the context of early-stage health economic modeling. We introduce the concept of a probability box (p-box) as a measure of uncertainty without necessitating a precise probability distribution. We provide formulas for a p-box given data on summary statistics of a parameter. We describe an approach to propagate p-boxes into a model and provide step-by-step guidance on how to implement PBA. We conduct a case and examine the differences between the status-quo and PBA approaches and their potential implications on decision-making., (© 2022 John Wiley & Sons Ltd.)

Vaccinations are crucial to fighting SARS-CoV-2, and high coverage rates can in most countries probably only be achieved with the involvement of primary care physicians (PCPs). We aimed to explore how SARS-CoV-2 vaccination payment schemes in 43 countries differ with regard to the (i) type of payment scheme, (ii) amount paid, (iii) degree of bundling, and (iv) use of pay-for-performance elements. We collected information on payments and health system characteristics, such as PCP income and employment status, in all EU and OECD countries over time. We regressed the payment amount on the income of PCPs for countries with activity-dependent schemes using a linear regression (OLS), and we interpreted the residuals of this regression as a vaccination payment index. The majority of countries (30/43) had chosen payment schemes that reward PCPs for the activity they perform. Seventeen countries paid less per vaccination than the income-adjusted average, whereas 13 countries paid more. Twelve countries used pay-for-performance elements., (Copyright © 2022 The Author(s). Published by Elsevier B.V. All rights reserved.)

Objective: This study explores differences in spending and utilization of health care services for an older person with frailty before and after a hip fracture., Data Sources: We used individual-level patient data from five care settings., Study Design: We compared utilization and spending of an older person aged older than 65 years for 365 days before and after a hip fracture across 11 countries and five domains of care as follows: acute hospital care, primary care, outpatient specialty care, post-acute rehabilitative care, and outpatient drugs. Utilization and spending were age and sex standardized.., Data Collection/extraction Methods: The data were compiled by the International Collaborative on Costs, Outcomes, and Needs in Care (ICCONIC) across 11 countries as follows: Australia, Canada, England, France, Germany, the Netherlands, New Zealand, Spain, Sweden, Switzerland, and the United States., Principal Findings: The sample ranged from 1859 patients in Spain to 42,849 in France. Mean age ranged from 81.2 in Switzerland to 84.7 in Australia. The majority of patients across countries were female. Relative to other countries, the United States had the lowest inpatient length of stay (11.3), but the highest number of days were spent in post-acute care rehab (100.7) and, on average, had more visits to specialist providers (6.8 per year) than primary care providers (4.0 per year). Across almost all sectors, the United States spent more per person than other countries per unit ($13,622 per hospitalization, $233 per primary care visit, $386 per MD specialist visit). Patients also had high expenditures in the year prior to the hip fracture, mostly concentrated in the inpatient setting., Conclusion: Across 11 high-income countries, there is substantial variation in health care spending and utilization for an older person with frailty, both before and after a hip fracture. The United States is the most expensive country due to high prices and above average utilization of post-acute rehab care., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Objective: To compare within-country variation of health care utilization and spending of patients with chronic heart failure (CHF) and diabetes across countries., Data Sources: Patient-level linked data sources compiled by the International Collaborative on Costs, Outcomes, and Needs in Care across nine countries: Australia, Canada, England, France, Germany, New Zealand, Spain, Switzerland, and the United States., Data Collection Methods: Patients were identified in routine hospital data with a primary diagnosis of CHF and a secondary diagnosis of diabetes in 2015/2016., Study Design: We calculated the care consumption of patients after a hospital admission over a year across the care pathway-ranging from primary care to home health nursing care. To compare the distribution of care consumption in each country, we use Gini coefficients, Lorenz curves, and female-male ratios for eight utilization and spending measures., Principal Findings: In all countries, rehabilitation and home nursing care were highly concentrated in the top decile of patients, while the number of drug prescriptions were more uniformly distributed. On average, the Gini coefficient for drug consumption is about 0.30 (95% confidence interval (CI): 0.27-0.36), while it is, 0.50 (0.45-0.56) for primary care visits, and more than 0.75 (0.81-0.92) for rehabilitation use and nurse visits at home (0.78; 0.62-0.9). Variations in spending were more pronounced than in utilization. Compared to men, women spend more days at initial hospital admission (+5%, 1.01-1.06), have a higher number of prescriptions (+7%, 1.05-1.09), and substantially more rehabilitation and home care (+20% to 35%, 0.79-1.6, 0.99-1.64), but have fewer visits to specialists (-10%; 0.84-0.97)., Conclusions: Distribution of health care consumption in different settings varies within countries, but there are also some common treatment patterns across all countries. Clinicians and policy makers need to look into these differences in care utilization by sex and care setting to determine whether they are justified or indicate suboptimal care., (© 2021 Health Research and Educational Trust.)

Objective: To compare countries' health care needs by segmenting populations into a set of needs-based health states., Data Sources: We used seven waves of the Survey of Health, Aging and Retirement in Europe (SHARE) panel survey data., Study Design: We developed the Cross-Country Simple Segmentation Tool (CCSST), a validated clinician-administered instrument for categorizing older individuals by distinct, homogeneous health and related social service needs. Using clinical indicators, self-reported physician diagnosis of chronic disease, and performance-based tests conducted during the survey interview, individuals were assigned to 1-5 global impressions (GI) segments and assessed for having any of the four identifiable complicating factors (CFs). We used Cox proportional hazard models to estimate the risk of mortality by segment. First, we show the segmentation cross-sectionally to assess cross-country differences in the fraction of individuals with different levels of medical needs. Second, we compare the differences in the rate at which individuals transition between those levels and death., Data Collection/extraction Methods: We segmented 270,208 observations (from Austria, Belgium, Czech Republic, Denmark, France, Germany, Greece, Israel, Italy, the Netherlands, Poland, Spain, Sweden, and Switzerland) from 96,396 individuals into GI and CF categories., Principal Findings: The CCSST is a valid tool for segmenting populations into needs-based states, showing Switzerland with the lowest fraction of individuals in high medical needs segments, followed by Denmark and Sweden, and Poland with the highest fraction, followed by Italy and Israel. Comparing hazard ratios of transitioning between health states may help identify country-specific areas for analysis of ecological and cultural risk factors., Conclusions: The CCSST is an innovative tool for aggregate cross-country comparisons of both health needs and transitions between them. A cross-country comparison gives policy makers an effective means of comparing national health system performance and provides targeted guidance on how to identify strategies for curbing the rise of high-need, high-cost patients., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Objective: To establish a methodological approach to compare two high-need, high-cost (HNHC) patient personas internationally., Data Sources: Linked individual-level administrative data from the inpatient and outpatient sectors compiled by the International Collaborative on Costs, Outcomes, and Needs in Care (ICCONIC) across 11 countries: Australia, Canada, England, France, Germany, the Netherlands, New Zealand, Spain, Sweden, Switzerland, and the United States., Study Design: We outline a methodological approach to identify HNHC patient types for international comparisons that reflect complex, priority populations defined by the National Academy of Medicine. We define two patient profiles using accessible patient-level datasets linked across different domains of care-hospital care, primary care, outpatient specialty care, post-acute rehabilitative care, long-term care, home-health care, and outpatient drugs. The personas include a frail older adult with a hip fracture with subsequent hip replacement and an older person with complex multimorbidity, including heart failure and diabetes. We demonstrate their comparability by examining the characteristics and clinical diagnoses captured across countries., Data Collection/extraction Methods: Data collected by ICCONIC partners., Principal Findings: Across 11 countries, the identification of HNHC patient personas was feasible to examine variations in healthcare utilization, spending, and patient outcomes. The ability of countries to examine linked, individual-level data varied, with the Netherlands, Canada, and Germany able to comprehensively examine care across all seven domains, whereas other countries such as England, Switzerland, and New Zealand were more limited. All countries were able to identify a hip fracture persona and a heart failure persona. Patient characteristics were reassuringly similar across countries., Conclusion: Although there are cross-country differences in the availability and structure of data sources, countries had the ability to effectively identify comparable HNHC personas for international study. This work serves as the methodological paper for six accompanying papers examining differences in spending, utilization, and outcomes for these personas across countries., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Objective: This study explores variations in outcomes of care for two types of patient personas-an older frail person recovering from a hip fracture and a multimorbid older patient with congestive heart failure (CHF) and diabetes., Data Sources: We used individual-level patient data from 11 health systems., Study Design: We compared inpatient mortality, mortality, and readmission rates at 30, 90, and 365 days. For the hip fracture persona, we also calculated time to surgery. Outcomes were standardized by age and sex., Data Collection/extraction Methods: Data was compiled by the International Collaborative on Costs, Outcomes and Needs in Care across 11 countries for the years 2016-2017 (or nearest): Australia, Canada, England, France, Germany, the Netherlands, New Zealand, Spain, Sweden, Switzerland, and the United States., Principal Findings: The hip sample across ranged from 1859 patients in Aragon, Spain, to 42,849 in France. Mean age ranged from 81.2 in Switzerland to 84.7 in Australia, and the majority of hip patients across countries were female. The congestive heart failure (CHF) sample ranged from 742 patients in England to 21,803 in the United States. Mean age ranged from 77.2 in the United States to 80.3 in Sweden, and the majority of CHF patients were males. Average in-hospital mortality across countries was 4.1%. for the hip persona and 6.3% for the CHF persona. At the year mark, the mean mortality across all countries was 25.3% for the hip persona and 32.7% for CHF persona. Across both patient types, England reported the highest mortality at 1 year followed by the United States. Readmission rates for all periods were higher for the CHF persona than the hip persona. At 30 days, the average readmission rate for the hip persona was 13.8% and 27.6% for the CHF persona., Conclusion: Across 11 countries, there are meaningful differences in health system outcomes for two types of patients., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Objective: The objectives of this study are to compare the relative use of different postacute care settings in different countries and to compare three important outcomes as follows: total expenditure, total days of care in different care settings, and overall longevity over a 1-year period following a hip fracture., Data Sources: We used administrative data from hospitals, institutional and home-based long-term care (LTC), physician visits, and medications compiled by the International Collaborative on Costs, Outcomes, and Needs in Care (ICCONIC) from five countries as follows: Canada, France, Germany, the Netherlands, and Sweden., Data Extraction Methods: Data were extracted from existing administrative data systems in each participating country., Study Design: This is a retrospective cohort study of all individuals admitted to acute care for hip fracture. Descriptive comparisons were used to examine aggregate institutional and home-based postacute care. Care trajectories were created to track sequential care settings after acute-care discharge through institutional and community-based care in three countries where detailed information allowed. Comparisons in patient characteristics, utilization, and costs were made across these trajectories and countries., Principal Findings: Across five countries with complete LTC data, we found notable variations with Germany having the highest days of home-based services with relatively low costs, while Sweden incurred the highest overall expenditures. Comparisons of trajectories found that France had the highest use of inpatient rehabilitation. Germany was most likely to discharge hip fracture patients to home. Over 365 days, France averaged the highest number of days in institution with 104, Canada followed at 94, and Germany had just 87 days of institutional care on average., Conclusion: In this comparison of LTC services following a hip fracture, we found international differences in total use of institutional and noninstitutional care, longevity, and total expenditures. There exist opportunities to organize postacute care differently to maximize independence and mitigate costs., (© 2021 Health Research and Educational Trust.)

Objective: To identify and explore differences in spending and utilization of key health services at the end of life among hip fracture patients across seven developed countries., Data Sources: Individual-level claims data from the inpatient and outpatient health care sectors compiled by the International Collaborative on Costs, Outcomes, and Needs in Care (ICCONIC)., Study Design: We retrospectively analyzed utilization and spending from acute hospital care, emergency department, outpatient primary care and specialty physician visits, and outpatient drugs. Patterns of spending and utilization were compared in the last 30, 90, and 180 days across Australia, Canada, England, Germany, New Zealand, Spain, and the United States. We employed linear regression models to measure age- and sex-specific effects within and across countries. In addition, we analyzed hospital-centricity, that is, the days spent in hospital and site of death., Data Collection/extraction Methods: We identified patients who sustained a hip fracture in 2016 and died within 12 months from date of admission., Principal Findings: Resource use, costs, and the proportion of deaths in hospital showed large variability being high in England and Spain, while low in New Zealand. Days in hospital significantly decreased with increasing age in Canada, Germany, Spain, and the United States. Hospital spending near date of death was significantly lower for women in Canada, Germany, and the United States. The age gradient and the sex effect were less pronounced in utilization and spending of emergency care, outpatient care, and drugs., Conclusions: Across seven countries, we find important variations in end-of-life care for patients who sustained a hip fracture, with some differences explained by sex and age. Our work sheds important insights that may help ongoing health policy discussions on equity, efficiency, and reimbursement in health care systems., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Objective: The objective of this study was to explore cross-country differences in spending and utilization across different domains of care for a multimorbid persona with heart failure and diabetes., Data Sources: We used individual-level administrative claims or registry data from inpatient and outpatient health care sectors compiled by the International Collaborative on Costs, Outcomes, and Needs in Care (ICCONIC) across 11 countries: Australia, Canada, England, France, Germany, the Netherlands, New Zealand, Spain, Sweden, Switzerland, and the United States (US)., Data Collection/extraction Methods: Data collected by ICCONIC partners., Study Design: We retrospectively analyzed age-sex standardized utilization and spending of an older person (65-90 years) hospitalized with a heart failure exacerbation and a secondary diagnosis of diabetes across five domains of care: hospital care, primary care, outpatient specialty care, post-acute rehabilitative care, and outpatient drugs., Principal Findings: Sample sizes ranged from n = 1270 in Spain to n = 21,803 in the United States. Mean age (standard deviation [SD]) ranged from 76.2 (5.6) in the Netherlands to 80.3 (6.8) in Sweden. We observed substantial variation in spending and utilization across care settings. On average, England spent $10,956 per person in hospital care while the United States spent $30,877. The United States had a shorter length of stay over the year (18.9 days) compared to France (32.9) and Germany (33.4). The United States spent more days in facility-based rehabilitative care than other countries. Australia spent $421 per person in primary care, while Spain (Aragon) spent $1557. The United States and Canada had proportionately more visits to specialist providers than primary care providers. Across almost all sectors, the United States spent more than other countries, suggesting higher prices per unit., Conclusion: Across 11 countries, there is substantial variation in health care spending and utilization for a complex multimorbid persona with heart failure and diabetes. Drivers of spending vary across countries, with the United States being the most expensive country due to high prices and higher use of facility-based rehabilitative care., (© 2021 The Authors. Health Services Research published by Wiley Periodicals LLC on behalf of Health Research and Educational Trust.)

Introduction: Specific guidance and examples for health technology assessment (HTA) of medical devices are scarce in medical device development. A more intense dialogue of competent authorities, HTA agencies, and manufactures may improve evidence base on clinical and cost-effectiveness. Especially as the new Medical Device Regulation requires more clinical evidence., Methods: We explore the perceptions of manufacturers, competent authorities, and HTA agencies towards such dialogues and investigate how they should be designed to accelerate the translational process from development to patient access using semi-structured interviews. We synthesized the evidence from manufacturers, competent authorities, and HTA agencies from 14 different jurisdictions across Europe., Results: Eleven HTA agencies, four competent authorities, and eight manufacturers of high-risk devices expressed perceptions on the current situation and the expected development of three types of early dialogues., Discussion: The MDR has to be taken into account when designing the early dialogue processes. Transferring insights from medicinal product regulation is limited as the regulatory pathways differ substantially., Conclusion: Early dialogues promise to accelerate the translational process and to provide faster access to innovative medical devices. However, health policy-makers should promote and fully establish regulatory and HTA early dialogues before introducing parallel early dialogues of regulatory, HTA agencies, and manufacturers. For initiating change, the legislator must create the legal basis and set the appropriate incentives for manufacturers., Competing Interests: Declaration of Competing Interest The authors declare that they have no financial/personal interest or belief that could affect their objectivity other than the stated funding source., (Copyright © 2021 The Author(s). Published by Elsevier B.V. All rights reserved.)

Background: Kaizen-a management technique increasingly employed in health care-enables employees, regardless of their hierarchy level, to contribute to the improvement of their organization. The approach puts special emphasis on frontline employees because it represents one of their main opportunities to participate directly in decision making. In this study, we aimed to (1) understand the experiences of nurses in two hospitals that had recently implemented kaizen, and (2) identify factors affecting the implementation of the technique., Methods: By means of purposeful sampling, we selected 30 nurses from different units in two private acute care hospitals in Switzerland in May 2018. We used the Organizational Transformation Model to conduct semi-structured interviews and perform qualitative content analysis. Lastly, originating from Herzberg's motivation theory, we suggest two types of factor influencing the implementation of kaizen-hygiene factors that may prevent nurses from getting demotivated, and motivational factors that may boost their motivation., Results: Nurses generally experienced kaizen as a positive practice that enabled them to discuss work-related activities in a more comprehensive manner. In some cases, however, a lack of visible improvement in the workplace lowered nurses' motivation to make suggestions. Nurses' attitudes towards kaizen differed across both hospitals depending on the available managerial support, resources such as infrastructure and staffing levels., Conclusions: From our findings, we derived several coping strategies to help health practitioners implement kaizen for the benefit of their organization and employees: Strong managerial support, appropriate use of kaizen tools, and a greater sense of team cohesion, among other factors, can influence how effectively hospital teams implement kaizen. To reap the benefits of kaizen, hospital managers should promote the exchange of opinions across hierarchy levels, allocate the necessary resources in terms of personnel and infrastructure, and show nurses how the technique can help them improve their workplace., Competing Interests: All authors of this manuscript have read the journal’s policy and have the following competing interests: KS, CP and RB declare that they have no conflicts of interest. KH, BT, and AG are employed by Hislanden AG, the hospital organization that was examined in this study. The commercial affiliation of the authors KH, BT, and AG does not alter our adherence to PLOS ONE policies on sharing data and materials. Hislanden AG does not profit financially or otherwise from the study results. All authors declare that no other competing interests exist.

Objectives: Methodological challenges in the evaluation of medical devices (MDs) may be different for early and late technology adopter countries, as well as the potential health technology assessment (HTA) solutions to tackle them. This study aims to provide guidance to Central and Eastern European (CEE) countries on how to address key challenges of HTA for MDs with special focus on the transferability of scientific evidence. Methods: As part of the COMED Horizon 2020 project, a comprehensive list of issues related to MD HTA were identified based on a targeted literature review. Health technology assessment issues which pose a greater challenge or require different solutions in late technology adopter countries were selected. Draught recommendations to address these issues were developed and discussed in a focus group. The recommendations were then validated with a wider group of experts, including HTA and reimbursement decision makers from CEE countries in May and June 2020. Results: A consolidated list of 11 recommendations were developed in 3 major areas: (1) clinical value assessment, focusing on the use of joint EU work, relying on real-world evidence, use of coverage with evidence development schemes, transferring evidence from foreign countries and addressing the challenges of learning curve and centre effect; (2) economic value assessment, covering cost calculation of complex medical devices and transferability of economic evaluations of MDs; (3) HTA processes, related to the frequent product modifications and various indications of MDs. Conclusions: Central and Eastern European countries with limited resources for conducting HTA, can benefit from HTA methods and evidence generated in early technology adopter countries. Considering the appropriate reuse of international HTA materials, late technology adopter countries can still implement HTA, even for MDs, which have a more limited evidence base compared with pharmaceuticals., Competing Interests: RD-B, SK, AZ, and ZK were employed by Syreon Research Institute. MH is self-employed in HTA/EBM Consulting Centre and has not received any remuneration of funding from the COMED project, nor has any commercial or financial relationship that could be construed as a potential conflict of interest. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2021 Daubner-Bendes, Kovács, Niewada, Huic, Drummond, Ciani, Blankart, Mandrik, Torbica, Yfantopoulos, Petrova, Holownia-Voloskova, Taylor, Al, Piniazhko, Lorenzovici, Tarricone, Zemplényi and Kaló.)

Competing Interests: Conflicts of Interest: All authors have completed the ICMJE uniform disclosure form (available at http://dx.doi.org/10.21037/cdt.2020.03.06). The series "Arts and Medicine" was commissioned by the editorial office without any funding or sponsorship. YvK serves as the unpaid editorial board member of Cardiovascular Diagnosis and Treatment from February 2018 to January 2020. The authors have no other conflicts of interest to declare.

Objective: To evaluate the effect of partisan political control on financial performance, structure, and outcomes of for-profit and not-for-profit US nursing homes., Data Sources/study Setting: Nineteen-year panel (1996-2014) of state election outcomes, financial performance data from nursing home cost reports, operational and aggregate resident characteristics from OSCAR of 13 737 nursing homes., Study Design: A linear panel model was estimated to identify the effect of Democratic and Republican political control on next year's outcomes. Nursing home outcomes were defined as yearly facility revenues, expenses, and profits; the number of Medicaid, Medicare, and private-pay residents; staffing levels; and selected resident outcomes., Principal Findings: Democratic political control leads to an increase in financial flows to for-profit nursing homes, boosting profits without producing observable improvements in resident outcomes. Republican political control leads to lower revenues and profits of for-profit nursing homes. A shift from Medicaid to more profitable private-pay residents following Republican political control is observed for all nursing homes. Financial performance of not-for-profit nursing homes is not significantly affected by changes in political control., Conclusion: Political control of the two legislative chambers-but not of the governorship-shapes the structure of the nursing home industry as seen in provider behavior., (© 2018 The Authors. Health Services Research published by Wiley Periodicals, Inc. on behalf of Health Research and Educational Trust.)

Health care providers are under pressure to improve both efficiency and quality. The two objectives are not always mutually consistent, because achieving higher levels of quality may require additional resources. The aim of this study is to demonstrate how the nonparametric conditional approach can be used to integrate quality into the analysis of efficiency and to investigate the mechanisms through which quality enters the production process. Additionally, we explain how the conditional approach relates to other nonparametric methods that allow integrating quality into efficiency analysis and provide guidance on the selection of an appropriate methodology. We use data from 178 departments of interventional cardiology and consider three different measures of quality: patient satisfaction, standardized mortality ratio, and patient radiation exposure. Our results refute the existence of a clear trade-off between efficiency and quality. In fact, the impact of quality on the production process differs according to the utilized quality measure. Patient satisfaction does not affect the attainable frontier but does have an inverted U-shaped effect on the distribution of inefficiencies; mortality ratio negatively impacts the attainable frontier when the observed mortality more than doubles the predicted mortality; and patient radiation exposure is not associated with the production process.

In recent years, the expiration of patents for large drug classes has increased the importance of post-patent drug markets. However, previous research has focused solely on patent drug markets. In this study, the authors evaluate the influence of preferred supplier contracts, the German approach to tendering, in post-patent drug markets using a hierarchical market share attraction model. The authors find that preferred supplier contracts are a powerful strategic instrument for generic manufacturers in a highly competitive environment. They quantify the effects of signing a preferred supplier contract and show that brand-name manufacturers are vulnerable to tendering. Therefore, brand-name manufacturers should readjust their strategies and consider including preferred supplier contracts in their marketing mix. In addition, the authors employ a simulation to demonstrate that a first-mover advantage might be gained from signing a preferred supplier contract. Furthermore, their results can be used as a blueprint for decision makers in the pharmaceutical industry to assess the market share effects of different contracting strategies regarding preferred supplier contracts.

Traditional nonparametric frontier techniques to measure hospital efficiency have been criticized for their deterministic nature and the inability to incorporate external factors into the analysis. Moreover, efficiency estimates represent a relative measure meaning that the implications from a hospital efficiency analysis based on a single-country dataset are limited by the availability of suitable benchmarks. Our first objective is to demonstrate the application of advanced nonparametric methods that overcome the limitations of the traditional nonparametric frontier techniques. Our second objective is to provide guidance on how an international comparison of hospital efficiency can be conducted using the example of two countries: Italy and Germany. We rely on a partial frontier of order-m to obtain efficiency estimates robust to outliers and extreme values. We use the conditional approach to incorporate hospital and regional characteristics into the estimation of efficiency. The obtained conditional efficiency estimates may deviate from the traditional unconditional efficiency estimates, which do not account for the potential influence of operational environment on the production possibilities. We nonparametrically regress the ratios of conditional to unconditional efficiency estimates to examine the relation of hospital and regional characteristics with the efficiency performance. We show that the two countries can be compared against a common frontier when the challenges of international data compatibility are successfully overcome. The results indicate that there are significant differences in the production possibilities of Italian and German hospitals. Moreover, hospital characteristics, particularly bed-size category, ownership status, and specialization, are significantly related to differences in efficiency performance across the analyzed hospitals.

Background: Studies have often stated that individual-level determinants are important drivers for the adoption of medical devices. Empirical evidence supporting this claim is, however, scarce. At the individual level, physicians' adoption motivation was often considered important in the context of adoption decisions, but a clear notion of its dimensions and corresponding measurement scales is not available., Objectives: To develop and subsequently validate a scale to measure the motivation to adopt medical devices of hospital-based physicians., Methods: The development and validation of the physician-motivation-adoption (PMA) scale were based on a literature search, internal expert meetings, a pilot study with physicians, and a three-stage online survey. The data collected in the online survey were analyzed using exploratory factor analysis (EFA), and the PMA scale was revised according to the results. Confirmatory factor analysis (CFA) was conducted to test the results from the EFA in the third stage. Reliability and validity tests and subgroup analyses were also conducted., Results: Overall, 457 questionnaires were completed by medical personnel of the National Health Service England. The EFA favored a six-factor solution to appropriately describe physicians' motivation. The CFA confirmed the results from the EFA. Our tests indicated good reliability and validity of the PMA scale., Conclusions: This is the first reliable and valid scale to measure physicians' adoption motivation. Future adoption studies assessing the individual level should include the PMA scale to obtain more information about the role of physicians' motivation in the broader adoption context., (Copyright © 2017 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Studies of determinants of adoption of new medical technology have failed to coalesce into coherent knowledge. A flaw obscuring strong patterns may be a common habit of treating a wide range of health care innovations as a generic technology. We postulate three decisional systems that apply to different medical technologies with distinctive expertise, interest, and authority: medical-individualistic, fiscal-managerial, and strategic-institutional decisional systems. This review aims to examine the determinants of the adoption of medical technologies based on the corresponding decision-making system. We included quantitative and qualitative studies that analyzed factors facilitating or inhibiting the adoption of medical technologies. In total, 65 studies published between 1974 and 2014 met our inclusion criteria. These studies contained 688 occurrences of variables that were used to examine the adoption decisions, and we subsequently condensed these variables to 62 determinants in four main categories: organizational, individual, environmental, and innovation-related. The determinants and their empirical association with adoption were grouped and analyzed by the three decision-making systems. Although we did not identify substantial differences across the decision-making systems in terms of the direction of the determinants' influence on adoption, a clear pattern emerged in terms of the categories of determinants that were targeted in different decision-making systems., (Copyright © 2017 Elsevier B.V. All rights reserved.)

Decisions to adopt medical devices at the hospital level have consequences for health technology assessment (HTA) on system level and are therefore important to decision makers. Our aim was to investigate the characteristics of organizations and individuals that are more inclined to adopt and utilize cardiovascular devices based on a comprehensive analysis of environmental, organizational, individual, and technological factors and to identify corresponding implications for HTA. Seven random intercept hurdle models were estimated using the data obtained from 1249 surveys completed by members of the European Society of Cardiology. The major findings were that better manufacturer support increased the adoption probability of 'new' devices (i.e. in terms of CE mark approval dates), and that budget pressure increased the adoption probability of 'old' devices. Based on our findings, we suggest investigating the role of manufacturer support in more detail to identify diffusion patterns relevant to HTA on system level, to verify whether it functions as a substitute for medical evidence of new devices, and to receive new insights about its relationship with clinical effectiveness and cost-effectiveness. © 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd., (© 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd.)

Changes in performance due to learning may dynamically influence the results of a technology evaluation through the change in effectiveness and costs. In this study, we estimate the effect of learning using the example of two minimally invasive treatments of abdominal aortic aneurysms: endovascular aneurysm repair (EVAR) and fenestrated EVAR (fEVAR). The analysis is based on the administrative data of over 40,000 patients admitted with unruptured abdominal aortic aneurysm to more than 500 different hospitals over the years 2006 to 2013. We examine two patient outcomes, namely, in-hospital mortality and length of stay using hierarchical regression models with random effects at the hospital level. The estimated models control for patient and hospital characteristics and take learning interdependency between EVAR and fEVAR into account. In case of EVAR, we observe a significant decrease both in the in-hospital mortality and length of stay with experience accumulated at the hospital level; however, the learning curve for fEVAR in both outcomes is effectively flat. To foster the consideration of learning in health technology assessments of medical devices, a general framework for estimating learning effects is derived from the analysis. © 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd., (© 2017 The Authors. Health Economics published by John Wiley & Sons, Ltd.)

Background: Guidelines summarize medical evidence, they identify the most efficient therapy under study conditions and recommend this therapy for use. The physician now has the challenge to translate a therapy that is efficient under laboratory conditions to a patient who is an individual person. To accomplish this task the physician has to make sure that (I) the ideal typical therapy is applicable and effective in this individual patient taking the special features into consideration, that (II) therapy is compliant with the norm including guidelines, laws and ethical requirements (conformity) and that (III) the therapy meets the patient's needs., Objective: How can physicians together with the patients translate the medical evidence into an individually optimized therapy?, Material and Methods: At the German Aortic Center in Hamburg we use I‑SWOT as an instrument to identify such individually optimized therapy. With I‑SWOT, we present an instrument with which we have developed an (I) efficient, (II) conform and (III) needs-oriented therapeutic strategy for individual patients., Results: I-SWOT cross-tabulates strengths (S) and weaknesses (W) related to therapy with opportunities (O) and threats (T) related to individual patients. This I‑SWOT matrix identifies four fundamental types of strategy, which comprise "SO" maximizing strengths and opportunities, "WT" minimizing weaknesses and threats, "WO" minimizing weaknesses and maximizing opportunities and "ST" maximizing strengths and minimizing threats. We discuss the case of a patient with asymptomatic thoracoabdominal aneurysm to show how I‑SWOT is used to identify an individually optimized therapy strategy.

Introduction: Marfan syndrome is a rare multisystem disease of the connective tissue, which affects multiple organ systems. advances in healthcare have doubled the life-expectancy of patients over the past three decades. to date, there is no comprehensive review that consolidates economic considerations and care for marfan patients. Areas covered: Present research suggests that there may be a link between treatment pattern, disease progression and economic costs of Marfan syndrome. It indicates that an early detection of the disease and preventive interventions achieve a dual aim. From a patient perspective, it may reduce the amount of emergency surgery or intervention, and inpatient stays. In addition, it slows disease progression, lowers lifestyle restrictions, reduces psychological stress, and improves health-related quality of life. Expert commentary: Early detection and preventive measures are likely to achieve a dual aim by simultaneously containing costs and reducing the number and length of inpatient stays.

Background: Chronic lymphocytic leukemia (CLL) is the most prevalent type of leukemia in the Western hemisphere. The disease affects quality of life (QOL) and poses an economic burden on patients, payers, and society. The objective of this review was to quantify the economic burden and quality-of-life effects and identify the gaps that should be addressed by future research., Methods: Free-text and subject heading searches in MEDLINE, EMBASE, the Cochrane Library, the University of York Centre for Reviews and Dissemination Database, and the Web of Science Core Collection database were conducted to identify observational and interventional studies reporting costs and/or quality-of-life effects published up to 2 October 2015. Studies were included irrespective of whether they were conducted prospectively or retrospectively. The focus population consisted of adult patients aged 18 years or older affected by any stage of CLL. Studies were included regardless of whether the underlying population was treated at baseline or not. Risk of bias was assessed using a quality checklist developed by the Effective Public Health Practice Project for (randomized) controlled trials, cohort studies, and cross-sectional studies. Economic evaluations were rated using a checklist developed by Stuhldreher et al. (Int J Eat Disord 45:476-91, 2012)., Results: From 2451 records identified, 27 studies were found to be eligible for inclusion. Studies were heterogeneous with respect to methodology, perspective, and data used. Annual direct costs per person ranged from US$4491 in Germany to US$43,913 in the USA. The share of costs attributable to drug treatment varied between 26.2 and 79 %. Indirect costs amounted to US$4208. Severity of disease was a predictor for quality of life, whereas differences by age and sex were mainly present in subdomains. Comparisons of treated and untreated populations resulted in an increase of quality of life in favor of treated populations in the long-term perspective. Differences between treatments were small. Consequently, cost effectiveness in decision-analytic models did not depend on whether quality of life or survival are used to describe the benefits of treatment., Conclusions: Although the quantity and the quality of health economic and quality-of-life evidence have substantially increased, there is still a need for studies that take a patient or societal perspective. Factors that influence costs and the quality of life of patients seem to be well-established, while longitudinal lifetime cost studies at the population level are still scarce.