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Neurodegenerative diseases are one of the most important contributors to morbidity and mortality in the elderly. In Europe, over 14 million people are currently living with dementia, at a cost of over 400 billion EUR annually. Recent advances in diagnostics and approval for new pharmaceutical treatments for Alzheimer's disease (AD), the most common etiology of dementia, heralds the beginning of precision medicine in this field. However, their implementation will challenge an already over-burdened healthcare systems. There is a need for innovative digital solutions that can drive the related clinical pathways and optimize and personalize care delivery. Public-private partnerships are ideal vehicles to tackle these challenges. Here we describe the Innovative Health Initiative (IHI) public-private partnership project PROMINENT that has been initiated by connecting leading dementia researchers, medical professionals, dementia patients and their care partners with the latest innovative health technologies using a precision medicine based digital platform. The project builds upon the knowledge and already implemented digital tools from several collaborative initiatives that address new models for early detection, diagnosis, and monitoring of AD and other neurodegenerative disorders. The project aims to provide support to improvement efforts to each aspect of the care pathway including diagnosis, prognosis, treatment, and data collection for real world evidence and cost effectiveness studies. Ultimately the PROMINENT project is expected to lead to cost-effective care and improved health outcomes. [ABSTRACT FROM AUTHOR]

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. 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. 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. 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. • The total cost of cancer was €199 billion in Europe in 2018. • Cancer caused nearly equally large costs within and outside the health-care system. • The total costs of cancer differed greatly between countries. • Health spending on cancer has been increasing faster than cancer incidence. • The productivity loss from premature mortality has been decreasing over time. [ABSTRACT FROM AUTHOR]

The objective of this observational study in nine European countries was to establish the current cost of MS at different levels of disease severity. The study used a prevalence-based bottom-up to estimate costs from a societal perspective. Patients were recruited via patient organizations and neurology centres and asked by mail to complete a detailed questionnaire concerning their resource consumption, work capacity, quality of life and current disease status. Resources included all health-care and social services, personal expenditures, informal care and temporary or definitive work absence. Quality of life was collected as utility and disease information collected related to functional capacity, relapses and fatigue. The data were analyzed separately for each country, using local unit costs but a fully standardized methodology. A total of 13 186 patients at all levels of disease severity participated in the survey, with an average response rate of around 40% (range 20–75%). This paper describes the methodology used for the study, while results are presented in the individual country reports. [ABSTRACT FROM AUTHOR]

The article focuses on the costs and utility in patients with Parkinson's disease (PD) in Europe. The study was performed as part of the larger study on the cost of disorders of the brain in Europe, undertaken by the European Brain Council. It shows a coherent picture of increasing costs with disease progression although the costs are of different magnitudes. Preventing disease progression could thus be a way of not only improving the situation for the patient, but also decreasing these costs. These studies show that PD is associated with high costs, and this is likely to be true for the other European countries as well.

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., (Copyright © 2024. Published by Elsevier B.V.)

People with schizophrenia die prematurely, yet regional differences are unclear. PRISMA 2020-compliant systematic review/random-effects meta-analysis of cohort studies assessing mortality relative risk (RR) versus any control group, and moderators, in people with ICD/DSM-defined schizophrenia, comparing countries and continents. We conducted subgroup, meta-regression analyses, and quality assessment. The primary outcome was all-cause mortality. Secondary outcomes were suicide-, /natural-cause- and other-cause-related mortality. We included 135 studies from Europe (n = 70), North-America (n = 29), Asia (n = 33), Oceania (n = 2), Africa (n = 1). In incident plus prevalent schizophrenia, differences across continents emerged for all-cause mortality (highest in Africa, RR=5.98, 95 %C.I.=4.09-8.74, k = 1, lowest in North-America, RR=2.14, 95 %C.I.=1.92-2.38, k = 16), suicide (highest in Oceania, RR=13.5, 95 %C.I.=10.08-18.07, k = 1, lowest in North-America, RR=4.4, 95 %C.I.=4.07-4.76, k = 6), but not for natural-cause mortality. Europe had the largest association between antipsychotics and lower all-cause mortality/suicide (Asia had the smallest or no significant association, respectively), without differences for natural-cause mortality. Higher country socio-demographic index significantly moderated larger suicide-related and smaller natural-cause-related mortality risk in incident schizophrenia, with reversed associations in prevalent schizophrenia. Antipsychotics had a larger/smaller protective association in incident/prevalent schizophrenia regarding all-cause mortality, and smaller protective association for suicide-related mortality in prevalent schizophrenia. Additional regional differences emerged in incident schizophrenia, across countries, and secondary outcomes. Significant regional differences emerged for all-cause, cause-specific and suicide-related mortality. Natural-cause death was homogeneously increased globally. Moderators differed across countries. Global initiatives are needed to improve physical health in people with schizophrenia, local studies to identify actionable moderators., Competing Interests: Declaration of Competing Interest MS has been a consultant and/or advisor to or have received honoraria from Angelini, Lundbeck, Otsuka. EV has received grants and served as consultant, advisor or CME speaker for the following entities: Abbott, Allergan, Angelini, Dainippon Sumitomo Pharma, Janssen, Lundbeck, Novartis, Otsuka, Raffo, Richter, Sage, Sanofi-Aventis, and Takeda, unrelated to the present work. PFP has received advisory consultancy fees from Lundbeck outside of this work. CUC has been a consultant and/or advisor to or has received honoraria from: AbbVie, Acadia, Alkermes, Allergan, Angelini, Aristo, Axsome, Boehringer-Ingelheim, Cardio Diagnostics, Cerevel, CNX Therapeutics, Compass Pathways, Damitsa, Gedeon Richter, Hikma, Holmusk, IntraCellular Therapies, Janssen/J&J, Karuna, LB Pharma, Lundbeck, MedAvante-ProPhase, MedInCell, Medscape, Merck, Mindpax, Mitsubishi Tanabe Pharma, Mylan, Neurocrine, Noven, Otsuka, Pfizer, Pharmabrain, PPD Biotech, Recordati, Relmada, Reviva, Rovi, Seqirus, Servier, SK Life Science, Sumitomo Dainippon, Sunovion, Sun Pharma, Supernus, Takeda, Teva, and Viatris. He provided expert testimony for Janssen and Otsuka. He served on a Data Safety Monitoring Board for Lundbeck, Relmada, Reviva, Rovi, and Teva. He has received grant support from Janssen and Takeda. He received royalties from UpToDate and is also a stock option holder of Cardio Diagnostics, Mindpax, and LB Pharma. IB reports grants from the EU to the Semmelweise University; royalties from Oxford University for a published book (editor); consulting fees from Gedeon Richter, Janssen, Janssen Cilag; speaker fees from Hikma Janssen, Janssen Cilag, Gedeon Richter, Medichem Pharmaceuticals by Unilab, and Mitsubishi Tanabe Pharma Signapure; and leadership or fiduciary roles with European College of Neuropsychopharmacology, the European Psychiatry Association, and Clincal Pharmacological Ethics Committee and Medical Research Council (Hungary). PG received fees for presentations at congresses or participation in scientific boards from Alcediag-Alcen, Angelini, GSK, Janssen, Lundbeck, Otsuka, SAGE, and Servier. HT reports personal fees from Janssen-Cilag and Otsuka and has participated in research projects funded by grants from Janssen Cilag and Eli Lilly to her employing institution. JT has participated in research projects funded by grants from Janssen Cilag and Eli Lilly to his employing institution. Dr. Tiihonen reports personal fees from the European Medicines Agency, Eli Lilly, Janssen Cilag, Lundbeck, Otsuka, Evidera, Mediuutiset, Sidera, and Sunovion. EDR has served as a speaker for Shire Sweden AB outside the submitted work. CAH has received speaker's honoraria for Medice. PL reports receiving grants from Novartis AG for the work submitted and grants from AstraZeneca, MSD, EFPIA, and IPHA outside the submitted work. MM received honoraria/has been a consultant for Angelini and Lundbeck outside of the present work. Henrik Larsson reports receiving grants from Shire Pharmaceuticals; personal fees from and serving as a speaker for Medice, Shire/Takeda Pharmaceuticals and Evolan Pharma AB; all outside the submitted work. Henrik Larsson is editor-in-chief of JCPP Advances. All other authors have nothing to disclose., (Copyright © 2023. Published by Elsevier B.V.)

Background and Aims: Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality. Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective., Methods: The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). The survey was distributed at participating clinics and included questions on e.g. symptoms (including the Gross Motor Function Classification System, GMFCS, and cognitive impairment), health care use, medication, ability to work, caregiving, and impact on health-related quality-of-life (HRQoL) using the EQ-5D-5L., Results: The estimated total mean societal cost per patient and year was £63,775 (SD: £49,944). The cost varied significantly with both mobility impairment (from £49,809 for GMFCS level 1 to £103,639 for GMFCS levels 3-5) and cognitive impairment (from £43,860 for mild level to £99,162 for severe level). The mean utility score on the EQ-5D-5L for patients was 0.498 (SD: 0.352). The utility score also varied significantly with both mobility impairment (from 0.783 for GMFCS level 1 to 0.153 for GMFCS level 3-5) and cognitive impairment (from 0.738 for mild level to 0.364 for severe level)., Conclusions: Similar to other studies of rare diseases, the study is based on a limited number of observations. However, the sample appear to be reasonably representative when comparing to previous studies of ARG1-D. This study shows that ARG1-D is associated with a high societal cost and significant impact on HRQoL. Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.

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., (Copyright © 2023 International Society for Pharmacoeconomics and Outcomes Research, Inc. Published by Elsevier Inc. All rights reserved.)

Background: Systemic anti-cancer therapy (SACT) is the recommended treatment modality in patients with advanced non-small cell lung cancer (aNSCLC) in clinical guidelines. SACT options in aNSCLC have multiplied in recent years with the introduction of immunotherapy and targeted therapy. This article presents findings from the first comparative analysis of SACT patterns in Europe., Methods: SACT rates in aNSCLC were estimated as the ratio between the number of patients treated with SACT (chemotherapy, immunotherapy, targeted therapy) and the number of potentially eligible patients for SACT in 11 countries (Belgium, Bulgaria, Finland, Hungary, Ireland, Netherlands, Norway, Poland, Portugal, Romania, UK) between 2014 and 2020. Treated patients were estimated by combining national sales volume data of cancer drugs and average drug use per patient based on clinical trials. Potentially eligible patients were estimated from national epidemiological data., Results: SACT rates in aNSCLC differed greatly, ranging from around 30 % in Hungary, Poland, and the UK to almost 60 % in Ireland, Norway, and Portugal in 2014. SACT rates seemed to increase over time in most countries, but differences were still large by 2020, ranging from around 40 % in the UK to 75 % or more in Belgium, Norway, and Portugal. Even in countries with the highest SACT rates, far from all patients seemed to receive guideline-recommended SACT options, as underuse of immunotherapy and targeted therapy was common., Conclusion: Up to 35 % of eligible patients with aNSCLC receives no SACT in certain European countries, although improvements have been achieved over time. The use of immunotherapy and targeted therapy is suboptimal even in countries with high SACT rates, indicating room to improve the quality of care and patient outcomes., Policy Summary: Measuring if and what kind of therapy cancer patients have access to is vital to assess quality of care. The care of aNSCLC patients seems to be suboptimal in Europe, due to factors such as exclusion of patients with moderate performance status from SACT, limited resources for diagnostic testing, long reimbursement timelines and slow adoption of new medicines in clinical practice., Competing Interests: Declaration of Competing Interest TH and PL are employed at IHE, an independent research organization receiving funding from both public and private sector organizations. TH and PL report institutional fees from MSD to IHE. NW reports personal fees from AstraZeneca, Bayer, Daichii Sankyo, Incyte, Janssen, MSD, Novartis, Oasmia, and Pierre Fabre for participation in advisory boards and educational activities outside the submitted work., (Copyright © 2022 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Background: There is limited information on drivers of utilities in patients with chronic heart failure (CHF)., Objectives: To analyze determinants of utility in CHF and drivers of change over 1 year in a large sample from clinical practice., Methods: We included 5334 patients from the Swedish Heart Failure Registry with EuroQol five-dimensional questionnaire information available following inpatient or outpatient care during 2008 to 2010; 3495 had 1-year follow-up data. Utilities based on Swedish and UK value sets were derived. We applied ordinary least squares (OLS) and two-part models for utility at inclusion and OLS regression for change over 1 year, all with robust standard errors. We assessed the predictive accuracy of both models using cross-validation., Results: Patients' mean age was 73 years, 65% were men, 19% had a left ventricular ejection fraction of 50% or more, 23% had 40% to 49%, 27% had 30% to 39%, and 31% had less than 30%. For both models and value sets, utility at inclusion was affected by sex, age, New York Heart Association class, ejection fraction, hemoglobin, blood pressure, lung disease, diabetes, angiotensin-converting enzyme inhibitors/angiotensin receptor blockers, nitrates, antiplatelets, and diuretics. The OLS model performed slightly better than did the two-part model on a population level and for capturing utility ranges. Change in utility over 1 year was influenced by age, sex, and (measured at inclusion) disease duration, New York Heart Association class, blood pressure, ischemic heart disease, lung disease, angiotensin-converting enzyme inhibitors/angiotensin receptor blockers, and antiplatelets., Conclusions: Utilities in CHF and their change over time are influenced by diverse demographic and clinical factors. Our findings can be used to target clinical interventions and for economic evaluations of new therapies., (Copyright © 2015. Published by Elsevier Inc.)

Objective: To investigate the cost-utility of eprosartan versus enalapril (primary prevention) and versus nitrendipine (secondary prevention) on the basis of head-to-head evidence from randomized controlled trials., Methods: The HEALTH model (Health Economic Assessment of Life with Teveten for Hypertension) is an object-oriented probabilistic Monte Carlo simulation model. It combines a Framingham-based risk calculation with a systolic blood pressure approach to estimate the relative risk reduction of cardiovascular and cerebrovascular events based on recent meta-analyses. In secondary prevention, an additional risk reduction is modeled for eprosartan according to the results of the MOSES study ("Morbidity and Mortality after Stroke--Eprosartan Compared to Nitrendipine for Secondary Prevention"). Costs and utilities were derived from published estimates considering European country-specific health-care payer perspectives., Results: Comparing eprosartan to enalapril in a primary prevention setting the mean costs per quality adjusted life year (QALY) gained were highest in Germany (Euro 24,036) followed by Belgium (Euro 17,863), the UK (Euro 16,364), Norway (Euro 13,834), Sweden (Euro 11,691) and Spain (Euro 7918). In a secondary prevention setting (eprosartan vs. nitrendipine) the highest costs per QALY gained have been observed in Germany (Euro 9136) followed by the UK (Euro 6008), Norway (Euro 1695), Sweden (Euro 907), Spain (Euro -2054) and Belgium (Euro -5767)., Conclusions: Considering a Euro 30,000 willingness-to-pay threshold per QALY gained, eprosartan is cost-effective as compared to enalapril in primary prevention (patients >or=50 years old and a systolic blood pressure >or=160 mm Hg) and cost-effective as compared to nitrendipine in secondary prevention (all investigated patients).