34 Eurohealth Systems and Policies

UNIVERSAL HEALTH COVERAGE AND THE ROLE OF EVIDENCE-BASED APPROACHES IN BENEFIT BASKET DECISIONS

By: Juliane Winkelmann, Dimitra Panteli, Miriam Blümel and Reinhard Busse

Summary: The extension of universal health coverage along its three dimensions – population coverage, benefit coverage and financial protection – has dominated health policy agendas in recent years. However, decisions on the benefits covered by publicly financed schemes have only recently received increased attention, being supported by evidence-based approaches such as health technology assessment (HTA) to ensure quality and “value for money” of care. Yet, new developments in the area of high-cost speciality have highlighted the limitations of HTA in guiding the optimal allocation of finite resources, posing a challenge to “universality” of coverage and requiring increased efforts towards aligned HTA in .

Keywords: Universal Health Coverage, Health Basket, Innovations, Pharmaceuticals, Health Technology Assessment

Introduction of health coverage, encompassing a mandatory public and a voluntary All health care systems are confronted private component. with the question of which treatments and pharmaceuticals to pay for publicly The rationale behind covering certain as resources for health are limited, thus Juliane Winkelmann is Research benefits while excluding others varies competing with other sectors within the Fellow; Dimitra Panteli is Research between jurisdictions, reflecting both Fellow; Miriam Blümel is Research public budget. Despite health needs and societal norms and system characteristics. Fellow at the Department of desires, it is not possible for a health Health Care , Public benefit “baskets” or packages system to afford to pay for all available University of Technology, . are usually defined more broadly at the Reinhard Busse is Co-Director of health care benefits for everyone, even legislative level with a stipulation of the the European Observatory on Health under universal coverage aspirations. areas of care to be covered. They are Systems and Policies and Therefore, trade-offs arise in coverage and Head of the Department then regulated more concretely, centrally decisions when priorities have to be set for Health Care Management, or regionally and usually within each Berlin University of Technology, between different benefits and cost- area of care, resulting in more or less Berlin, Germany. Email: juliane. sharing levels as well as the population [email protected] explicit benefit baskets. Especially in the groups covered. As a consequence, most realm of coverage decisions for health countries opt for two-tiered models

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Figure 1: The three dimensions of universal health coverage population coverage and financial protection. While both dimensions offer little scope for policy variation if the fundamental values of universality and Total health expenditure Height: solidarity are not to be contradicted, the What range of services covered by publicly proportion financed schemes constitutes a playing of the costs 8 is covered? field in health policy for decision-making.

Indeed, there is a lot of variation in the level of explicitness and the approaches Cost sharing Other services countries use to define their priorities and benefit packages. They range from very detailed (positive) lists of all goods and services available through statutory coverage to a vaguely formulated and Public expenditure implicit benefit package with reference to Uninsured broad categories of services (e.g. primary on health Depth: 4 7 9 Which benefits care, pharmaceuticals). For example, are covered? UK legislation defines very broad categories of health care services, Breadth: Who is insured? considering services necessary within ‘reasonable limits’, while leaving providers with the possibility to establish positive Source: 7 lists. 6 8 At the same time, an institution tasked with identifying necessary, technologies, evidence-based approaches demonstrating their commitment to appropriate and cost-effective care, the have been increasingly employed to ensure achieving health care for all. Today it is National Institute for Health and Care quality and efficiency of care, or “value one of the most prominent global health Excellence (NICE) provides very clear for money”, in the composition of the policies, most notably retained in the guidance on whether a new benefit package. Sustainable Development Goals (SDGs) should be made available to NHS patients in 2015. The UHC concept encompasses who meet particular criteria. 8 Health In recent years, benefit baskets in many three dimensions: coverage for everyone benefit baskets can also be defined European countries have been expanded (breadth), type and number of needed negatively by excluding certain benefits. by costly innovations in medicines health services covered (depth) and For example, Italy and use positive and devices leading to rising health the proportion of total health service and negative lists and have a structured expenditures. In a context of already costs that are publicly funded and not and detailed minimum benefit baskets that constrained health budgets, formal subject to cost sharing (height), also can be further adapted by regional health structures to support evidence-based referred to as financial risk protection, authorities. 3 9 Israel is probably the only decision-making in a multitude of and is best reflected in the UHC cube country in the world with one detailed list countries have been established to identify (see Figure 1). The UHC cube was first of all benefits across all sectors covered (non-) cost-effective services. At the same conceived in mid-2000 2 3 and was further under the National Health Insurance Act; time, the fundamental values of universal developed for the framework behind the the list is updated once a year. 10 health coverage (UHC) and solidarity have European Observatory’s Health Systems come under threat; this became evident in Transition reports. 4 It was most Over the last two decades, there has been particularly during the economic crisis prominently used in the World Health a general trend to make positive lists more when countries had to decide between Reports 2008 and 2010 5 6 and has since explicit, both in tax-funded countries restricting the number of people covered become known as the coverage cube. (where benefits were previously left to the (most visibly in Greece), the services Today, it is used worldwide to illustrate discretion of providers) as well as those included the benefit basket (see Box 1) and UHC and supports related analyses. with Social Health Insurance (where lists the extent of the cost to be borne privately used to be merely fee schedules), and to for services in the benefit basket. 1 expand the range of services in the benefit Defining the health benefit basket is 3 7 still challenging baskets. However, the opposite can Achieving UHC along the also be observed, in particular during ‘coverage cube’ Despite the importance of the range the economic crisis when services were of benefits covered, the focus in the removed from the benefits package In the last 20 years, UHC has substantially discussion on UHC to date has been (see Box 1). gained importance with governments dominated by the two dimensions of

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(most commonly following an application to existing alternatives. 16 17 However, they for inclusion in the benefit basket by the do require evaluation and investment of Box 1: UHC and the economic crisis manufacturer or a request by relevant HTA-related resources. decision-makers), scientific evidence In response to budget pressures is collected and evaluated (evidence New medicines based on novel during the economic crisis, many assessment) and subsequently appraised mechanisms, such as gene and cell countries redefined benefit baskets in context (evidence appraisal). therapies, have started entering the and some tried to remove non-cost- market with extremely high price effective services from coverage. These formal assessment mechanisms tags (e.g. Novartis´ immunocellular In a study jointly carried out by the are most frequently in place for therapy against leukaemia was priced European Observatory and the WHO pharmaceuticals. In Europe, at $475 000 per infusion for the US Regional Office for Europe in 2014, pharmaceuticals have historically market). Viewed against a backdrop of 15 countries represented one of the largest expenditure a per capita pharmaceutical expenditure reported trying to restrict or redefine items in health care spending with of US$ PPP 553 (OECD country average the publicly financed benefit basket costs predominantly being covered in 2015 14 ), it becomes clear that health between 2008 and 2013. Of these, by statutory funds. 14 To bring a new systems will be unable to bear such costs only four countries incorporated medicine to market, demonstration of in a routine manner as part of the benefit HTA in decision-making while eleven safety and clinical “efficacy” are usually package. A new discussion on the effect countries restricted benefits on an sufficient. These are demonstrated within of these medicines on the “universality” ad hoc basis. Disinvestment mostly randomised controlled trials, with selected of coverage in European health systems is involved medicines, followed by cash patients (e.g. excluding multimorbid warranted. Indeed, the Dutch Presidency benefits for temporary sickness leave ones) and using placebo as control. It of the European Council in 2016 placed and dental care, but also primary is the role of the subsequent HTA to the spotlight on the imbalances in the care visits (e.g. a cap was introduced determine whether – at least in principle current system of development, pricing on the number of general practice – the therapeutic benefit is meaningful to and reimbursement of medicines and visits covered in Romania) and patients compared to alternatives in real raised questions about its sustainability preventive services (the Netherlands world conditions – and therefore whether, for Europe and Europeans. and Bulgaria). 1 11 12 to what extent and/or at what price new medicines will be covered publicly. To Looking forward ensure that they are subsequently used The importance of HTA for coverage appropriately is mainly the domain of Decision-makers are increasingly decisions has grown clinical guidelines. 15 confronted with difficult coverage decisions due to budget constraints and Tools supporting evidence-based decision- new and costly health technologies. making are increasingly incorporated Expensive innovations have big Over the last two decades numerous in formal decision-making structures, implications for coverage decisions techniques have been applied to guide as mentioned above, especially in the New developments in the output portfolio the decision-making process and to direct realm of coverage decisions for health of the pharmaceutical industry have the optimal allocation of finite resources. technologies (i.e. pharmaceuticals, medical highlighted the limitations of traditional The desire to maximise the value for devices, procedures or interventions). HTA-based systems in guiding the optimal money of health services and to ensure The concept of technology assessment as allocation of finite resources. The market the long-term sustainability of access to a policy-informing tool to guide decision- entry of breakthrough therapies with technologies, have been met by increased making for coverage in health care was large target populations and steep price use of evidence-based approaches. In first introduced in the United States tags (such as the pharmaceuticals against this context, the application of HTA has in 1975. The evaluation model of the Hepatitis C in 2014) served as a wake- received increased attention in health Office of Technology Assessment (OTA) up call for policymakers, who were policy in most European countries and included elements of safety, effectiveness suddenly confronted with unmanageable will continue to play an important role, and cost, as well as socioeconomic and budget impacts and a lack of suitable thus requiring enhanced collaboration ethical implications of adopting (new) management levers. The number of and knowledge exchange. Indeed, the technologies in health care. It was new high-cost specialty medicines and has been promoting subsequently adapted by national health so-called “niche-busters” (aimed at very related research and collaborative activities technology assessment programmes in a narrowly defined patient sub-populations) for more than 15 years, culminating in number of European countries. 13 has increased substantially over the last the establishment of an HTA network in two decades. At the same time, evidence Directive 2011/24/EU. The scientific and The exact scope and configuration of HTA suggests that a substantial majority of technical cooperation of the network has are country-specific and heterogeneous. these new pharmaceuticals do not provide been the responsibility of the EUnet HTA However, HTA is generally applied substantial patient benefit gains compared Joint Actions. following marketing authorisation. After selection of the technologies to evaluate

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A further promising step towards 3 Schreyögg J, Stargardt T, Velasco-Garrido M, 11 Thomson S, Evetovits E, Kluge H. Universal aligned and centralised HTA in the EU Busse R. Defining the “Health Benefit Basket” in nine health coverage and the economic crisis in Europe. was made on 31 January 2018 when European countries. Evidence from the European Eurohealth 2016;22(2):18 – 22. Union Health BASKET Project. European Journal 12 Thomson S. Changes to health coverage. In the European Commission issued a Health 2005;6(Suppl. 1):2–10. proposal for regulation building on the Thomson S, Figueras J, Evetovits T, et al. (eds.) 4 exeprience of EU Member States in the Rechel B, Thomson S, van Ginneken E. Health Economic Crisis, Health Systems and Health Systems in Transition: template for authors. in Europe. Impact and implications for policy. area of HTA and related collaboration Copenhagen: WHO Regional Office for Europe, Maidenhead: Open University Press, 2015. and mandating joint assessments of on behalf of the European Observatory on Health 13 Velasco-Garrido M, Børlum Kristensen F, clinical elements (effectiveness and Systems and Policies, 2010. Palmhøj Nielsen C, Busse R (eds.) Health technology safety) of new medicines and certain new 5 World Health Organization. World Health Report: assessment and health policy-making in Europe – medical devices. Although the proposal primary health care – no more than ever. Geneva: Current status, challenges and potential. Copenhagen: has been criticised for various reasons World Health Organization, 2008. WHO Regional Office for Europe on behalf of the European Observatory on Health Systems and (e.g. manufacturers are not mandated to 6 World Health Organization. World Health Report: Policies, 2008. provide full trial data but are afforded health systems financing: the path to universal the possibility to comment on assessment coverage. Geneva: World Health Organization, 2010. 14 OECD. Health at a Glance 2017. OECD Indicators. Paris: OECD Publishing, 2017. Available at: http:// drafts and specify which information is 7 Busse R, Schlette S. Focus on Prevention, Health dx.doi.org/10.1787/health_glance-2017-en not to be made publicly available), more and Ageing and Human Resources. Gütersloh: Verlag collaboration in the evaluation of new Bertelsmann Stiftung, 2007. 15 Legido-Quigley H, Panteli D, Car J, McKee M, Busse R. Clinical guidelines for chronic conditions medicines is a welcome concept on the 8 Smith P, Chalkidou K. Should Countries Set an in the European Union. Copenhagen: World Health path to ensuring that new technologies Explicit Health Benefits Package? The Case of the Organization, on behalf of the European Observatory English National Health Service. Value in Health with true patient benefit are identified on Health Systems and Policies, 2013. early and evaluated for inclusion in the 2017;20(1):60 – 6. 16 Salas-Vega S, Iliopoulos O, benefit basket at affordable costs. 9 Auraaen A, Fujisawa R, de Lagasnerie G, Paris V, Mossialos E. Assessment of overall survival, quality et al. How OECD health systems define the range of life, and safety benefits associated with new cancer of good and services to be financed collectively. medicines. JAMA Oncology 2017;3(3):382 – 90. References OECD Health Working Papers, No. 90. Paris: OECD Publishing, 2016. 17 Davis C,Huseyin N, Evrim G, Elita P, Ashlyn P, 1 Thomson S, Figueras J, Evetovits T, et al. Ajay A. Availability of evidence of benefits on overall Economic crisis, health systems and health in Europe: 10 Brammli-Greenberg S, Waitzberg R, Medina- survival and quality of life of cancer drugs approved impact and implications for policy. Policy Summary, Artom T, Adijes-Toren A. Low-budget policy tool by European Medicines Agency: retrospective Copenhagen: WHO/European Observatory on Health to empower Israeli insureds to demand their cohort study of drug approvals 2009 – 13. BMJ Systems and Policies, 2014. rights in the healthcare system. Health Policy 2017;359:j4530. 2014;118(3):279 – 84. 2 Busse R, Schreyögg J, Velasco- Garrido M. HealthBASKET: Synthesis Report. Brussels: EHMA, 2006.

Related Observatory publications:

Economic crisis, health systems Clinical guidelines for chronic Health technology assessment and health and health in Europe: impact and conditions in the European Union (2013) policy-making in Europe – Current implications for policy (2014) https://goo.gl/Fh4kCj status, challenges and potential (2008) https://goo.gl/vB5Wp8 https://goo.gl/zNu1gj

on Health Systems and Policies Series European Observatory

Economic Crisis,Europe Health Systems and Health in Impact and implications for policy stem threat to health and health sy nd Economic shocks pose a eople’s need for health care anded performance by increasing p difficult – a situation compourvices. making access to care moreon health and other socialy public se HealthSystems Economic Crisis, by cuts in public spendingcts can be avoided by timel tside But these negative effe ant public policy levers lieor ou fiscal Health EconomicCrisis, policy action. While importands of those responsible f is and the health sector, in the h the response Health Systems policy and social protection, critical. ted to w health systems in Europe reac at began in in Europe This book looks at ho nancial and economic crisistries, th the Health Europe pressure created by the fi xperience of over 45 coun in 2008. Drawing on the e olicy and authors: ponses to the crisis in three p rage; Impact and implications for policy • analyse health system resr the health system; health cove areas: public funding fo purchasing and delivery nd and health service planning, sponses on health systems a Thomson,Figueras, Evetovits,Jowett, Mladovsky, Maresso, • assess the impact of these re Cylus, Karanikolos, Kluge population health of ely to sustain the performance • identify policies most lik ncial pressure health systems facing fina in a omy of implementing reforms • explore the political econ crisis for anyone who wants to d the The book is essential readingilable to policy-makers – anr sustain Written by understand the choices avaprotect population health oic and other implications of failing toance – in the face of econom Sarah Thomson, health system perform Josep Figueras forms of shock. pe and the WHO Regional Office for Euro Josep Tamás Evetovits Sarah Thomson ics and Political Science. London School of Econom ealth Systems and Policies. rope. Matthew Matthew Jowett Figueras European Observatory on H WHO Regional Office for EuEuropean Tamás Evetovits Philipa Mladovsky London Philipa Mladovsky Jowett WHO (Geneva). Systems and PoliciesAnna and theMaresso Anna Maresso Observatory on Health litical Science. . Jonathan School of Economics and PoHealth Systems and Policies Jonathan Cylus lth Systems and Policies. European Observatory on alth Systems European Observatory on Hea Marina Karanikolos Cylus European Observatory on He ope. Marina Karanikolos WHO Regional Office for Eur Hans Kluge Hans Kluge and Policies.

Eurohealth — Vol.24 | No.2 | 2018