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Background: Randomised clinical trials are key to advancing medical knowledge and to enhancing patient care, but major barriers to their conduct exist. The present paper presents some of these barriers.Methods: We performed systematic literature searches and internal European Clinical Research Infrastructure Network (ECRIN) communications during face-to-face meetings and telephone conferences from 2013 to 2017 within the context of the ECRIN Integrating Activity (ECRIN-IA) project.Results: The following barriers to randomised clinical trials were identified: inadequate knowledge of clinical research and trial methodology; lack of funding; excessive monitoring; restrictive privacy law and lack of transparency; complex regulatory requirements; and inadequate infrastructures. There is a need for more pragmatic randomised clinical trials conducted with low risks of systematic and random errors, and multinational cooperation is essential.Conclusions: The present paper presents major barriers to randomised clinical trials. It also underlines the value of using a pan-European-distributed infrastructure to help investigators overcome barriers for multi-country trials in any disease area. [ABSTRACT FROM AUTHOR]

Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP's is realised. [ABSTRACT FROM AUTHOR]

Background: This paper reports the methods and findings from a systematic review of economic evaluations conducted in the community aged care sector between 2000 and 2016.Methods: Online databases searched were PubMed, Medline, Scopus, and web of science, CINAHL and informit. Studies were included if they 1) were full economic evaluations that compared both the costs and outcomes of two or more interventions 2) in study population of people aged 65 years and over 3) dependent older people living in the community 4) alternatives being compared were care models or service delivery interventions in the community aged care sector (a group of programs that have been established as a support system to allow older people to remain living in their own homes for as long as possible, as an alternative to institutional or residential care) and 5) published in the English language between 2000 and November 2016.Results: Eleven studies reporting upon economic evaluations of service delivery interventions in community aged care were identified; the majority of which were undertaken in Europe. Critical appraisal of the identified studies highlighted the methodological rigour in these evaluations.Conclusion: This systematic review highlights the paucity of economic evaluation studies conducted to date in the community aged care sector. The findings highlight the importance of cost utility analysis methodology as it allows for a uniform outcome measure, that facilitates the comparison of different interventions. In addition, multi-attribute utility measures that represent those quality of life domains that are most important to older people should be used and attention must be paid to the inclusion of informal care costs and outcomes as this is a key resource in community aged care service delivery. [ABSTRACT FROM AUTHOR]

Background: Many low-and middle-income countries (LMIC) of the World Health Organization (WHO) European Region have introduced social health insurance payroll taxes after the political transition in the late 1980s, combined with budget transfers to allow for exempting specific population groups from paying contributions, such as those outside formal sector work and in particular vulnerable groups. This paper assesses the institutional design aspects of such financing arrangements and their performance with respect to universal health coverage progress in LMIC of the European region. Methods: The study is based on a literature review and review of secondary databases for the performance assessment. Results: Such financing arrangements currently exist in 13 LMIC of that region, with strong commonalities in institutional design: This includes a wide range of different eligible population groups, mostly mandatory membership, integrated pools for both the exempted and contributors, and relatively comprehensive benefit packages. Performance is more varied. Enrolment rates range from about 65 % to above 95 %, and access to care and financial protection has improved in several countries. Yet, inequities between income quintiles persist. Conclusions: Budget transfers to health insurance arrangements have helped to deepen UHC or maintain achievements with respect to UHC in these European LMICs by covering those outside formal sector work, and in particular vulnerable population groups. However, challenges remain: a comprehensive benefit package on paper is not enough as long as supply side constraints and quality gaps as well as informal payments prevail. A key policy question is how to reach those so far uncovered. [ABSTRACT FROM AUTHOR]

Background: The association between income inequality and societal performance has been intensely debated in recent decades. This paper reports how unmet need for medical care has changed in Europe during The Great Recession, and investigates whether countries with smaller income differences have been more successful than inegalitarian countries in protecting access to medical care during an economic crisis. Methods: Six waves of EU-SILC surveys (2008-2013) from 30 European countries were analyzed. Foregone medical care, defined as self-reported unmet need for medical care due to costs, waiting lists, or travel difficulties, was examined among respondents aged 30-59 years (N= 1.24 million). Countries' macro-economic situation was measured by Real Gross Domestic Product (GDP) per capita. The S80/S20 ratio indicated the country's level of income inequality. Equity issues were highlighted by separate analyses of disadvantaged respondents with limited economic resources and relatively poor health. Cross-tabulations and multilevel linear probability regression models were utilized. Results: Foregone medical care increased 2008-2013 in the majority of the 30 countries, especially among the disadvantaged parts of the population. For the disadvantaged, unmet need for medical care tended to be higher in countries with larger income inequalities, regardless of the average economic standard in terms of GDP per capita. Both for disadvantaged and for other parts of the samples, a decline in GDP had more severe effects on access in inegalitarian countries than in countries with less income inequality. Conclusions: During The Great Recession, unmet need for medical care increased in Europe, and social inequalities in foregone medical care widened. Overall, countries with a more egalitarian income distribution have been more able to protect their populations, and especially disadvantaged groups, against deteriorated access to medical care when the country is confronted with an economic crisis. [ABSTRACT FROM AUTHOR]

Background: Dissemination bias occurs when only some results emerging from clinical research reach their intended audience in the knowledge translation process. Given that coverage decisions increasingly rely on evidence, it is important to explore the types of evidence considered. This paper aimed to examine the evidence base used by regulatory institutions involved in pricing and reimbursement of pharmaceuticals in a broad range of European countries, as well as their awareness of and approach towards dissemination bias.Methods: A mixed methods approach was adopted. Regulatory documents and published literature were identified in systematic searches and relevant documents were analysed. An online survey was carried out to verify and expand insights.Results: Forty-two relevant regulatory documents and 10 publications were included. The survey had a 35% response rate, yielding valid responses for 13 countries. A fragmented impression was obtained for most countries indicating a general lack of transparency regarding both processes of decision-making and approaches towards unpublished information. Dissemination bias was rarely consistently considered. Practices for the identification and inclusion of all available evidence varied considerably, as did the influence of missing evidence on decision-making. Differences were often attributable to the regulatory context and/or institutional principles.Conclusions: Best practice is difficult to generalize given the identified variations. Individual exemplary practices support the necessity for institutional exchange at international level. Increased institutional commitment to transparency of methods and processes should be advocated. [ABSTRACT FROM AUTHOR]

Background Chronic pain is common in Europe and elsewhere and its under treatment confers a substantial burden on individuals, employers, healthcare systems and society in general. Indeed, the personal and socioeconomic impact of chronic pain is as great as, or greater, than that of other established healthcare priorities. In light of review of recently published data confirming its clinical and socioeconomic impact, this paper argues that chronic pain should be ranked alongside other conditions of established priority in Europe. We outline strategies to help overcome barriers to effective pain care resulting in particular from deficiencies in education and access to interdisciplinary pain management services, We also address the confusion that exists between proper clinical and scientific uses of opioid medications and their potential for misuse and diversion, as reflected in international variations in the access to, and availability of, these agents. Discussion As the economic costs are driven in part by the costs of lost productivity, absenteeism and early retirement, pain management should aim to fully rehabilitate patients, rather than merely to relieve pain. Accredited education of physicians and allied health professionals regarding state-of-the-art pain management is crucial. Some progress has been made in this area, but further provision and incentivization is required. We support a tiered approach to pain management, whereby patients with pain uncontrolled by non-specialists are able to consult a physician with a pain competency or a specialist in pain medicine, who in turn can recruit the services of other professionals on a case-by-case basis. A fully integrated interdisciplinary pain service should ideally be available to patients with refractory pain. Governments and healthcare systems should ensure that their policies on controlled medications are balanced, safeguarding public health without undue restrictions that compromise patient care, and that physician education programmes support these aims. Summary Strategic prioritization and co-ordinated actions are required nationally and internationally to address the unacceptable and unnecessary burden of uncontrolled chronic pain that plagues European communities and economies. An appreciation of the 'return on investment' in pain management services will require policymakers to adopt a long-term, cross-budgetary approach. [ABSTRACT FROM AUTHOR]

Introduction: The aim of this paper was to investigate the association between health, social position, social participation and the welfare state. Extending recent research on the social consequences of poor health, we asked whether and how welfare generosity is related to the risk of social exclusion associated with combinations of poor health, low education and economic inactivity. Methods: Our analyses are based on data from the European Social Survey, round 3 (2006/7), comprising between 21,205 and 21,397 individuals, aged 25--59 years, within 21 European welfare states. The analyses were conducted by means of multilevel logistic regression analysis in STATA 12. Results: The results demonstrated that the risk of non-participation in social networks decreased as welfare generosity increased. The risk of social exclusion, i.e. non-participation in social networks among disadvantaged groups, seldom differed from the overall association, and in absolute terms it was invariably smaller in more generous welfare state contexts. Conclusions: The results showed that there were no indications of higher levels of non-participation among disadvantaged groups in more generous welfare states. On the contrary, resources made available by the welfare state seemed to matter to all individuals in terms of overall lower levels of non-participation. As such, these results demonstrate the importance of linking health related social exclusion to the social policy context. [ABSTRACT FROM AUTHOR]