Centralizing and decentralizing governance in the COVID-19 pandemic: The politics of credit and blame.

COVID-19 led to significant and dynamic shifts in power relations within and between governments, teaching us how governments make health policies and how health crises affect government. We focus on centralization and decentralization within and between governments: within government, meaning the extent to which the head of government controls policy; and between governments, meaning the extent to which the central government pre-empts or controls local and regional government. Political science literature suggests that shifting patterns of centralization and decentralization can be explained by leading politicians' efforts to gain credit for popular actions and outcomes and deflect blame for unpopular ones. We test this hypothesis in two ways: by coding the Health Systems Response Monitor's data on government responses, and through case studies of the governance of COVID-19 in Austria, Czechia and France. We find that credit and blame do substantially explain the timing and direction of changes in centralization and decentralization. In the first wave, spring 2020, heads of government centralized and raised their profile in order to gain credit for decisive action, but they subsequently tried to decentralize in order to avoid blame for repeated restrictions on life or surges of infection. These findings should shape advice on governance for pandemic response.

Regulatory niches: Diagnostic reform as a process of fragmented expansion. Evidence from the UK 1990-2018.

This paper analyses the politics of regulatory expansion within the diagnostics sector. Since 1990, an informal, clinician-led process of diagnostic innovation within the UK NHS has been challenged by new mechanisms for the evaluation of diagnostics. We describe these diagnostic reforms as a process of fragmented regulatory expansion. New governance mechanisms function as regulatory niches: discrete spaces within an overarching sociotechnical regime. The boundaries of regulatory niches are organisational and epistemological. Organisational boundaries map onto established communities of practice that constitute the regulatory target; epistemological boundaries are defined by distinctive evaluation frameworks. Niches are also distinguished by their outcomes (rate of positive decisions) and their origins. Niche formation was triggered by five drivers: public scandal; technological change; marketisation; institutional isomorphism; and transnational policy transfer. Each niche was triggered by a unique confluence of these drivers, but common to all were historic shifts in healthcare politics, as the rise of evidence-based medicine intersected with the centralising impulse of the regulatory state, which encroached on clinical autonomy in a contest for power that is increasingly mediated by influential non-governmental organisations.

Health Technology Assessment and Health Care Reimbursement in the European Union: Permissive Dissensus and the Limits of Harmonization through the Backdoor.

Member states have consistently limited the European Union's competences in the area of health care reimbursement. Despite these efforts, there has been a slow but steady tendency toward harmonization of a key tool in reimbursement decision-making: health technology assessment (HTA), a multidisciplinary evaluation of "value for money" of medicines, devices, diagnostics, and interventions, which provides expert advice for reimbursement decisions. This article examines the origins of this paradoxical appetite for harmonization as well as of the dissensus that has, at the moment, somewhat stalled further integration in HTA. It finds that the prointegration neofunctionalist "permissive dissensus" is still present in decision making on HTA but potentially offset by dissensus or outright opposition from key actors, including member states and the medical device industry. These actors are able to decipher the potential consequences of highly technical issues, such as HTA, for national systems of social protection. Despite that, they have little interest in politicizing the issue, potentially opening the door to integrative policy solutions in the future.

Half a Century of Wilson & Jungner: Reflections on the Governance of Population Screening.

Background: In their landmark report on the "Principles and Practice of Screening for Disease" (1968), Wilson and Jungner noted that the practice of screening is just as important for securing beneficial outcomes and avoiding harms as the formulation of principles. Many jurisdictions have since established various kinds of "screening governance organizations" to provide oversight of screening practice. Yet to date there has been relatively little reflection on the nature and organization of screening governance itself, or on how different governance arrangements affect the way screening is implemented and perceived and the balance of benefits and harms it delivers. Methods: An international expert policy workshop convened by Sturdy, Miller and Hogarth. Results: While effective governance is essential to promote beneficial screening practices and avoid attendant harms, screening governance organizations face enduring challenges. These challenges are social and ethical as much as technical. Evidence-based adjudication of the benefits and harms of population screening must take account of factors that inform the production and interpretation of evidence, including the divergent professional, financial and personal commitments of stakeholders. Similarly, when planning and overseeing organized screening programs, screening governance organizations must persuade or compel multiple stakeholders to work together to a common end. Screening governance organizations in different jurisdictions vary widely in how they are constituted, how they relate to other interested organizations and actors, and what powers and authority they wield. Yet we know little about how these differences affect the way screening is implemented, and with what consequences. Conclusions: Systematic research into how screening governance is organized in different jurisdictions would facilitate policy learning to address enduring challenges. Even without such research, informal exchange and sharing of experiences between screening governance organizations can deliver invaluable insights into the social as well as the technical aspects of governance.

The Emerging Social Science Literature on Health Technology Assessment: A Narrative Review.

BACKGROUND: Social scientists have paid increasing attention to health technology assessment (HTA). This paper provides an overview of existing social scientific literature on HTA, with a focus on sociology and political science and their subfields. METHODS: Narrative review of key pieces in English. RESULTS: Three broad themes recur in the emerging social science literature on HTA: the drivers of the establishment and concrete institutional designs of HTA bodies; the effects of institutionalized HTA on pricing and reimbursement systems and the broader society; and the social and political influences on HTA decisions. CONCLUSION: Social scientists bring a focus on institutions and social actors involved in HTA, using primarily small-N research designs and qualitative methods. They provide valuable critical perspectives on HTA, at times challenging its otherwise unquestioned assumptions. However, they often leave aside questions important to the HTA practitioner community, including the role of culture and values. Closer collaboration could be beneficial to tackle new relevant questions pertaining to HTA.

Shedding light on the HTA consultancy market: Insights from Poland.

Research on health technology assessment (HTA) from a policy perspective typically examines public HTA bodies, with little attention devoted to how manufacturers develop their evidence submissions. Taking Poland as a crucial case, we explored the market of HTA consultancy firms which assist drug manufacturers in developing these submissions, called HTA reports. We reviewed 318 HTA reports from 2012 to 2015, data from the Polish National Company Registry, the content of HTA consulting firms' websites, and appraisal reports developed by the Polish HTA body. We identified HTA consultancy firms which developed 96-98% HTA reports. We found that the transparency of information about the authors of HTA reports provided by the HTA body had improved between 2012 and 2015. Six companies with market shares from 10 to 30% dominated the market. The market size was estimated to be 5-6 million EUR annually. HTA consultancies had a broad service portfolio related to preparation of HTA reports. Over 90% of HTA reports did not meet the official minimum quality requirements, and only half of the resubmissions took into account remarks made by the HTA body. Our study provides insights into the structure, evolution and role of the for-profit HTA consultancy market as a crucial part of the public HTA system. This raises important policy points about transparency and regulation at the intersection of public and private sectors in HTA.

Patterns of alternative access: Unpacking the Slovak extraordinary drug reimbursement regime 2012-2016.

Many countries employ "alternative access schemes" (e.g. compassionate use, early access programs, off-label use) that seek to provide patients with access to drugs not included on a positive drug list. These schemes offer flexibility to policy-makers but often lack transparency and clear rules. This ambiguity allows for dynamic responses to weaknesses in the main drug approval and reimbursement systems, but also opportunistic use by the health professionals, industry or patients. Yet, most descriptions of these schemes focus on the de jure rather than the de facto situation, presenting a potentially misleading picture. We describe one such scheme in practice: the Slovak "extraordinary reimbursement regime" (ERR), using semi-structured interviews with 18 experts and a new dataset of ERR drugs. The ERR expanded rapidly, doubling between 2012 and 2016. It combined features of four reimbursement schemes: (1) a backdoor market access for expensive drugs; (2) a compassionate use scheme for investigational drugs combined with a "legacy drugs" scheme for older unlicensed drugs; (3) a disease-specific scheme for cancer and orphan drugs; and (4) a scheme for off-label and "off-indication" drugs. These four features reflect broader challenges facing the Slovak reimbursement system. We conclude that detailed study of the type, size and evolution over time of alternative access schemes can serve as indicators of health policy objectives neglected by standard reimbursement systems.

Alternative access schemes for pharmaceuticals in Europe: Towards an emerging typology.

European governments employ sophisticated health technology assessment and regulatory procedures to identify which pharmaceuticals to fund publicly. However, there are persisting demands from patients for those drugs excluded from positive reimbursement lists, leading to the emergence of what are here termed "alternative access schemes". This paper presents a purposive review of these schemes based on available scholarly and grey literature, illustrated with real-world examples from recent practice. It puts forward an original typology of alternative access schemes based on their marketing authorization (regulation) and reimbursement (redistribution) status. We describe the complex, multidimensional policy trade-offs between the principles of patient freedom of choice, clinical autonomy, encouragement of innovation, evidence-informed decisions on safety and quality, access to treatment, and financial sustainability, involved in marketing authorization and reimbursement decisions. We discuss the ways in which alternative access schemes differ and conclude that our typology can illuminate salient policy dilemmas raised by alternative access schemes in national drug reimbursement systems.

Transparency in practice: Evidence from 'verification analyses' issued by the Polish Agency for Health Technology Assessment in 2012-2015.

Transparency is recognised to be a key underpinning of the work of health technology assessment (HTA) agencies, yet it has only recently become a subject of systematic inquiry. We contribute to this research field by considering the Polish Agency for Health Technology Assessment (AHTAPol). We situate the AHTAPol in a broader context by comparing it with the National Institute for Health and Care Excellence (NICE) in England. To this end, we analyse all 332 assessment reports, called verification analyses, that the AHTAPol issued from 2012 to 2015, and a stratified sample of 22 Evidence Review Group reports published by NICE in the same period. Overall, by increasingly presenting its key conclusions in assessment reports, the AHTAPol has reached the transparency standards set out by NICE in transparency of HTA outputs. The AHTAPol is more transparent than NICE in certain aspects of the HTA process, such as providing rationales for redacting assessment reports and providing summaries of expert opinions. Nevertheless, it is less transparent in other areas of the HTA process, such as including information on expert conflicts of interest. Our findings have important implications for understanding HTA in Poland and more broadly. We use them to formulate recommendations for policymakers.

When health technology assessment is confidential and experts have no power: the case of Hungary.

Health technology assessment (HTA) is not simply a mechanistic technical exercise as it takes place within a specific institutional context. Yet, we know little about how this context influences the operation of HTA and its ability to influence policy and practice. We seek to demonstrate the importance of considering institutional context, using a case study of Hungary, a country that has pioneered HTA in Central and Eastern Europe. We conducted 26 in-depth, semi-structured interviews with public- and private-sector stakeholders. We found that while the HTA Department, the Hungarian HTA organisation, fulfilled its formal role envisaged in the legislation, its potential for supporting evidence-based decision-making was not fully realised given the low levels of transparency and stakeholder engagement. Further, the Department's practical influence throughout the reimbursement process was perceived as being constrained by the payer and policy-makers, as well as its own limited organisational capacity. There was also scepticism as to whether the current operational form of the HTA process delivered 'good value for money'. Nevertheless, it still had a positive impact on the development of a broader institutional HTA infrastructure in Hungary. Our findings highlight the importance of considering institutional context in analysing the HTA function within health systems.

Epistemic communities and experts in health policy-making.

The role of evidence and expertise in policy-making has been of interest to public health professionals and political scientists alike. The public health community often sees its efforts as part of a linear knowledge transfer process and tends to blame itself for inadequate communication or translation of its arguments to policy-makers' language when its efforts fail. Political science, especially theories of the policy process, offer alternative perspectives to explain the success or failure of experts' preferred policy goals. This paper focuses on the concept of epistemic communities (groups of experts with a common policy goal derived from their shared knowledge) in policy-making, drawing on examples from the field of health technology assessment in Europe. By combining the parsimony and the central focus on experts of the linear knowledge transfer model with the recognition of complexity of political science, the epistemic communities concept provides a useful structure for the public health community to analyze its efforts to influence policy.

Rationalizing the introduction and use of pharmaceutical products: The role of managed entry agreements in Central and Eastern European countries.

This paper aims to provide an overview of the rationalization strategies for the introduction and use of pharmaceuticals, focusing on the role of managed entry agreements (MEA) in Central and Eastern European (CEE) countries, namely Bulgaria, the Czech Republic, Croatia, Hungary, Poland and Romania. We developed a conceptual framework on MEAs that was used as the basis for a standardized assessment questionnaire sent to country experts to capture their perceptions on their countries' rationalization strategies and MEAs. Our study shows that the main role of MEAs and other related policies embedded in the health care system is to limit the budget impact of drugs in all examined 6 countries. Uncertainty about outcomes and appropriate utilization seem to be of lower priority. Finance-based MEAs are used by all countries. Performance-based MEAs are scarce and used to a limited extent by Hungary and Poland. The overall transparency of the existence and details of MEAs is limited. Expansion of the use and increased transparency of MEAs is recommended. Still, the informational infrastructure and competencies in implementing MEA's need to be developed further.

Who's afraid of institutionalizing health technology assessment (HTA)?: Interests and policy positions on HTA in the Czech Republic.

This article identifies the interests and policy positions of key health policy stakeholders regarding the creation of a health technology assessment (HTA) agency in the Czech Republic, and what considerations influenced them. Vested interests have been suggested as a factor mitigating the diffusion of HTA bodies internationally. The Czech Republic recently considered and discarded establishing an HTA agency, making it a good case for studying actors' policy positions throughout the policy debates. Findings are based on in-depth, semi-structured expert and elite interviews with 34 key Czech health policy actors, supported by document analysis and extensive triangulation. Findings show that the HTA epistemic community of 'aspiring agents' was the only actor strongly in favor of an HTA body. Payers and the medical device and diagnostics industry were against it; patients and clinicians had no clear preferences. Original decision-makers were in favor but a new minister of health opted for a simpler policy alternative to solve his need for expertise. Existing institutions, policy alternatives and the institutional design of a future HTA body influence domestic actors' preferences for or against an HTA agency. Domestic and international proponents of HTA should give serious thought to their concerns when advocating for HTA bodies.

What has health technology assessment ever done for us?

Health technology assessment (HTA) has over the past three decades become a well-established part of decisions about allocation of resources in many countries. Despite this, little is known about HTA's impact on health systems. Few studies have evaluated the benefits of HTA for health outcomes, for access to care or for public budgets. In contrast, HTA has relatively clear upfront costs, which could potentially discourage policy-makers from establishing HTA agencies, especially in low income countries with restricted resources. It may be premature, though, to dismiss this approach altogether, as less tangible modernizing goals are still significant.

The Implementation of Managed Entry Agreements in Central and Eastern Europe: Findings and Implications.

BACKGROUND: Managed entry agreements (MEAs) are a set of instruments to facilitate access to new medicines. This study surveyed the implementation of MEAs in Central and Eastern Europe (CEE) where limited comparative information is currently available. METHOD: We conducted a survey on the implementation of MEAs in CEE between January and March 2017. RESULTS: Sixteen countries participated in this study. Across five countries with available data on the number of different MEA instruments implemented, the most common MEAs implemented were confidential discounts (n = 495, 73%), followed by paybacks (n = 92, 14%), price-volume agreements (n = 37, 5%), free doses (n = 25, 4%), bundle and other agreements (n = 19, 3%), and payment by result (n = 10, >1%). Across seven countries with data on MEAs by therapeutic group, the highest number of brand names associated with one or more MEA instruments belonged to the Anatomical Therapeutic Chemical (ATC)-L group, antineoplastic and immunomodulating agents (n = 201, 31%). The second most frequent therapeutic group for MEA implementation was ATC-A, alimentary tract and metabolism (n = 87, 13%), followed by medicines for neurological conditions (n = 83, 13%). CONCLUSIONS: Experience in implementing MEAs varied substantially across the region and there is considerable scope for greater transparency, sharing experiences and mutual learning. European citizens, authorities and industry should ask themselves whether, within publicly funded health systems, confidential discounts can still be tolerated, particularly when it is not clear which country and party they are really benefiting. Furthermore, if MEAs are to improve access, countries should establish clear objectives for their implementation and a monitoring framework to measure their performance, as well as the burden of implementation.

Three worlds of health technology assessment: explaining patterns of diffusion of HTA agencies in Europe.

In the past two decades, setting up independent health technology assessment (HTA) agencies has become a popular tool to inform reimbursement decision-making in health care, spreading from Northern European countries across Western Europe but much less so to post-communist countries. Structural political science explanations leave gaps in clarifying this diffusion pattern. This paper proposes a theoretical model focusing on the influence of domestic epistemic communities mitigating policy diffusion. Based on a review of HTA institutions in the EU, it proposes a chronological taxonomy of HTA agencies in Europe (the forerunners, the mainstreamers and the non-adopters) and asks why there is such an important East-West divide. The paper discusses theoretical explanations from different literatures, finding unsatisfactory many traditional political science answers such as the degree of centralization of a country's health system, its financial organization (Bismarckian or Beveridgian), the attitude toward independent regulatory bodies in general, the influence of international actors, or lack of resources. Finally, it suggests cases for empirical testing of the domestic epistemic communities model.

Patients' perceptions of information and education for renal replacement therapy: an independent survey by the European Kidney Patients' Federation on information and support on renal replacement therapy.

BACKGROUND: Selection of an appropriate renal replacement modality is of utmost importance for patients with end stage renal disease. Previous studies showed provision of information to and free modality choice by patients to be suboptimal. Therefore, the European Kidney Patients' Federation (CEAPIR) explored European patients' perceptions regarding information, education and involvement on the modality selection process. METHODS: CEAPIR developed a survey, which was disseminated by the national kidney patient organisations in Europe. RESULTS: In total, 3867 patients from 36 countries completed the survey. Respondents were either on in-centre haemodialysis (53%) or had a functioning graft (38%) at the time of survey. The majority (78%) evaluated the general information about kidney disease and treatment as helpful, but 39% did not recall being told about alternative treatment options than their current one. Respondents were more often satisfied with information provided on in-centre haemodialysis (90%) and transplantation (87%) than with information provided on peritoneal dialysis (79%) or home haemodialysis (61%), and were more satisfied with information from health care professionals vs other sources such as social media. Most (75%) felt they had been involved in treatment selection, 29% perceived they had no free choice. Involvement in modality selection was associated with enhanced satisfaction with treatment (OR 3.13; 95% CI 2.72-3.60). Many respondents (64%) could not remember receiving education on how to manage their kidney disease in daily life. Perceptions on information seem to differ between countries. CONCLUSIONS: Kidney patients reported to be overall satisfied with the information they received on their disease and treatment, although information seemed mostly to have been focused on one modality. Patients involved in modality selection were more satisfied with their treatment. However, in the perception of the patients, the freedom to choose an alternative modality showed room for improvement.

Health technology assessment in Poland, the Czech Republic, Hungary, Romania and Bulgaria.

This paper describes and discusses the development and use of health technology assessment (HTA) in five Central and Eastern European countries (CEE): Poland, the Czech Republic, Hungary, Romania and Bulgaria. It provides a general snapshot of HTA policies in the selected CEE countries to date by focusing on country case-studies based on document analysis and expert opinion. It offers an overview of similarities and differences between the individual CEE countries and discusses in detail the role of HTA by assessing its formalization and institutionalization, standardization of methodology, the use of HTA in practice and the degree of professionalization of HTA in the region. It finds that HTA has been to some extent implemented in all five countries studied, with methodologies in accordance with international standards, but that challenges remain when it comes to the role of HTA in health care decision-making as well as to human resource capacities of the countries. This paper suggests that coming years will show whether CEE countries develop adequate national analytical capacity to assess and appraise technologies in the context of local need and affordability, instead of using HTA as a mere administrative procedure to fulfill (inter)national requirements. Finally, suggestions are provided to strengthen HTA in CEE countries through cooperation, mutual learning, a common accreditation of HTA bodies and increased network building among CEE HTA experts.