Evidence Quality and Health Technology Assessment Outcomes in Reappraisals of Drugs for Rare Diseases in Germany.

OBJECTIVES: Evidence on reappraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs), the Federal Joint Committee follows different processes (limited or regular), depending on whether an annual revenue threshold has been exceeded. Our objective is to better understand (re)appraisal processes and their outcomes for RDTs in Germany. METHODS: We analyzed appraisal documents of 55 RDT indications for which an initial appraisal and a reappraisal were conducted between 2011 and 2023. We extracted information for the type of evidence, the risk of bias, the availability of additional evidence, and the change in the maturity of survival data as proxies for evidence quality. Specifically, we reviewed the reasons for conducting reappraisals, examined how evidence quality and the clinical benefit rating (CBR) differed between initial appraisals and reappraisals, and explored the association between evidence quality and (1) the CBR and (2) the change in the CBR after reappraisal. RESULTS: Most reappraisals were conducted because the annual revenue threshold was exceeded or the initial appraisal resolution was time limited. Almost all initial appraisals used the limited process, whereas the majority of reappraisals used the regular process. The CBR increased in only 9 and decreased in 21 of 55 reappraisals. There was some evidence that reappraisals with an accepted randomized controlled trial were significantly more likely to achieve a higher CBR. CONCLUSIONS: Findings confirmed that reasons and processes for conducting reappraisals of RDTs in Germany differ. Further, high CBRs in reappraisals were not common and evidence quality in initial appraisals and reappraisals was limited.

Managing diabetes and hypertension in western Kenya: A qualitative study of experiences of patients supported by the primary health integrated care for chronic conditions (PIC4C) model of care.

The Primary Health Integrated Care for Chronic Conditions (PIC4C) pilot project was launched in 2018 to strengthen prevention and control of four non-communicable conditions at primary health care level in western Kenya. We conducted a qualitative study to explore the extent to which PIC4C integrated services supported people with hypertension and/or diabetes towards timely diagnosis and referral, treatment, follow-up and adherence, from the perspective of those receiving care. Semi-structured interviews were conducted with a purposively sampled patient cohort at two time points, with the intention of capturing changes over time (total (n) = 43, completion of both interviews (n) = 37). We extracted existing survey data to describe socio-demographic characteristics and analyzed qualitative data thematically. We identified two cross-cutting contextual factors, individual's financial resources and their social situation, which shaped each stage of their interactions with PIC4C services. The PIC4C model successfully engaged people in accessing screening services to enable timely diagnosis and referred them to enter care. Free community level screening services and decentralization of care to lower level facilities reduced cost barriers for patients. However, retention in care and adherence to treatment were affected by the wider system context in which PIC4C was operating, including inconsistencies in medication availability and patients' limited financial capacity. Individually tailored advice from health care workers to work around some of these challenges supported self-management strategies. Further development of the service should focus on supporting health care workers to adopt flexible, contextually responsive approaches in order to support patients facing economic and other constraints to engage in (self) care.

The impact of patient registration on utilisation and quality of care: a propensity score matching and staggered difference-in-differences analysis of a cohort of 16,775 people with type 2 diabetes.

BACKGROUND: In 2012, Luxembourg introduced a Referring Doctor (RD) policy, whereby patients voluntarily register with a primary care practitioner, who coordinates patients' health care and ensures optimal follow-up. We contribute to the limited evidence base on patient registration by evaluating the effects of the RD policy. METHODS: We used data on 16,775 people with type 2 diabetes on oral medication (PWT2D), enrolled with the Luxembourg National Fund from 2010 to 2018. We examined the utilisation of primary and specialist outpatient care, quality of care process indicators, and reimbursed prescribed medicines over the short- (until 2015) and medium-term (until 2018). We used propensity score matching to identify comparable groups of patients with and without an RD. We applied difference-in-differences methods that accounted for patients' registration with an RD in different years. RESULTS: There was low enrolment of PWT2D in the RD programme. The differences-in-differences parallel trends assumption was not met for: general practitioner (GP) consultations, GP home visits (medium-term), HbA1c test (short-term), complete cholesterol test (short-term), kidney function (urine) test (short-term), and the number of repeat prescribed cardiovascular system medicines (short-term). There was a statistically significant increase in the number of: HbA1c tests (medium-term: 0.09 (95% CI: 0.01 to 0.18)); kidney function (blood) tests in the short- (0.10 (95% CI: 0.01 to 0.19)) and medium-term (0.11 (95% CI: 0.03 to 0.20)); kidney function (urine) tests (medium-term: 0.06 (95% CI: 0.02 to 0.10)); repeat prescribed medicines in the short- (0.19 (95% CI: 0.03 to 0.36)) and medium-term (0.18 (95% CI: 0.02 to 0.34)); and repeat prescribed cardiovascular system medicines (medium-term: 0.08 (95% CI: 0.01 to 0.15)). Sensitivity analyses also revealed increases in kidney function (urine) tests (short-term: 0.07 (95% CI: 0.03 to 0.11)) and dental consultations (short-term: 0.06, 95% CI: 0.00 to 0.11), and decreases in specialist consultations (short-term: -0.28, 95% CI: -0.51 to -0.04; medium-term: -0.26, 95% CI: -0.49 to -0.03). CONCLUSIONS: The RD programme had a limited effect on care quality indicators and reimbursed prescribed medicines for PWT2D. Future research should extend the analysis beyond this cohort and explore data linkage to include clinical outcomes and socio-economic characteristics.

Evaluating the implementation of the Primary Health Integrated Care Project for Chronic Conditions: a cohort study from Kenya.

Introduction: In Kenya, non-communicable diseases (NCDs) are estimated to account for almost one-third of all deaths and this is likely to rise by over 50% in the next 10 years. The Primary Health Integrated Care for Chronic Conditions (PIC4C) project aims to strengthen primary care by integrating comprehensive NCD care into existing HIV primary care platform. This paper evaluates the association of PIC4C implementation on clinical outcomes. Methods: Outcomes included proportion of new patients, systolic blood pressure (SBP), fasting plasma glucose (FPG), diastolic blood pressure, hypertension control, random plasma glucose, diabetes control, viral load and HIV viral suppression. We used interrupted time series and binomial regression with random effects for facility-level data and generalised mixed-effects regression for visit-level data to examine the association between PIC4C and outcomes between January 2017 and December 2021. We conducted sensitivity analysis with restrictions on sites and the number of visits. Results: Data from 66 641 visits of 13 046 patients with hypertension, 24 005 visits of 7267 patients with diabetes and 84 855 visits of 21 186 people with HIV were analysed. We found evidence of association between PIC4C and increase in proportion of new patients per month with hypertension (adjusted OR (aOR) 1.57, 95% CI 1.39 to 1.78) and diabetes (aOR 1.31, 95% CI 1.19 to 1.45), small increase in SBP (adjusted beta (aB) 1.7, 95% CI 0.8 to 2.7) and FPG (aB 0.6, 95% CI 0.0 to 1.1). There was no strong evidence of association between PIC4C and viral suppression (aOR 1.20, 95% CI 0.98 to 1.47). In sensitivity analysis, there was no strong evidence of association between PIC4C and SBP (aB 1.74, 95% CI -0.70 to 4.17) or FPG (aB 0.52, 95% CI -0.64 to 1.67). Conclusions: PIC4C implementation was associated with increase in proportion of new patients attending clinics and a slight increase in SBP and FPG. The immediate post-PIC4C implementation period coincided with the COVID-19 pandemic, which is likely to explain some of our findings.

Governing Integrated Health and Social Care: An Analysis of Experiences in Three European Countries.

Purpose: Achieving greater health and social care integration is a policy priority in many countries, but challenges remain. We focused on governance and accountability for integrated care and explored arrangements that shape more integrated delivery models or systems in Italy, the Netherlands and Scotland. We also examined how the COVID-19 pandemic affected existing governance arrangements. Design/methodology/approach: A case study approach involving document review and semi-structured interviews with 35 stakeholders in 10 study sites between February 2021 and April 2022. We used the Transparency, Accountability, Participation, Integrity and Capability (TAPIC) framework to guide our analytical enquiry. Findings: Study sites ranged from bottom-up voluntary agreements in the Netherlands to top-down mandated integration in Scotland. Interviews identified seven themes that were seen to have helped or hindered integration efforts locally. Participants described a disconnect between what national or regional governments aspire to achieve and their own efforts to implement this vision. This resulted in blurred, and sometimes contradictory, lines of accountability between the centre and local sites. Flexibility and time to allow for national policies to be adapted to local contexts, and engaged local leaders, were seen to be key to delivering the integration agenda. Health care, and in particular acute hospital care, was reported to dominate social care in terms of policies, resource allocation and national monitoring systems, thereby undermining better collaboration locally. The pandemic highlighted and exacerbated existing strengths and weaknesses but was not seen as a major disruptor to the overall vision for the health and social care system. Research limitations: We included a relatively small number of interviews per study site, limiting our ability to explore complexities within sites. Originality: This study highlights that governance is relatively neglected as a focus of attention in this context but addressing governance challenges is key for successful collaboration.

How do we ensure that innovation in health service delivery and organization is implemented, sustained and spread?

Countries in Europe are experimenting with innovative ways of organizing and delivering health care to better meet people’s increasingly diverse health and care needs. In practice, it has been difficult to translate necessary change into large-scale, sustainable and effective strategies. Implementing innovations is complex and there is a need to better understand the key factors that support the successful introduction of service innovation, from adoption to sustaining, spreading and scaling. This policy brief is a contribution to this effort by (i) reviewing the main frameworks and factors that have been identified as supportive for the successful introduction of innovation in service organization and delivery and (ii) illustrating these factors using selected examples of service innovations in European countries. This policy brief was written for the WHO European high-level meeting on "Health systems for prosperity and solidarity: leaving no one behind", held in Tallinn, Estonia on 13-14 June 2018, specifically as a support to the related sessions on harnessing innovations and systems to meet people’s needs.

Оценка ведения хронических заболеваний в европейских системах здравоохранения: Принципы и подходы

Растущее бремя хронических заболеваний, в частности быстрое увеличение числа людей с множественной патологией, – сложная проблема для систем здравоохранения во всех странах мира. Связанные с этим реждевременная смертность и сниженное физическое функционирование, а также повышенный спрос на услуги здравоохранения и сопряженные с этим расходы – лишь некоторые из основных причин для беспокойства у лиц, принимающих решения, и специалистов практического звена. Назрела явная необходимость переформатировать системы медико-санитарной помощи для более полного удовлетворения потребностей при хронических заболеваниях, что предполагает переход от традиционной модели оказания помощи при острых и эпизодических проблемах со здоровьем к модели, позволяющей лучше координировать работу специалистов и учреждений при активном участии самих получателей услуг и лиц, ухаживающих за ними. Многие страны уже начали работать в этом направлении, однако испытывают трудности с выбором наилучшего из возможных подходов: модели оказания помощи сильно зависят от местныхусловий, а научных оценок эффективности таких подходов пока нет. Оценка ведения хронических заболеваний в европейских системах здравоохранения рассматривает ряд ключевых вопросов – от интерпретации имеющейся базы фактических данных до оценки политического контекста и подходов к ведению хронических больных в Европе. По данным из 12 подробных отчетов о ситуации встранах (см. второй том в интернете), авторы исследования представляют глубокий анализ целого ряда моделей оказания помощи и функций вовлеченного в этот процесс персонала, механизмов оплаты и доступа пользователей к услугам, а также трудностей, которые страны вынуждены преодолевать в процессе внедрения и оценки этих новых подходов. В основу этой книги легли выводы проекта DISMEVAL (Разработка и подтверждение эффективности методов оценки ведения больных в европейских системах здравоохранения), осуществляемого под руководством исследовательского института RAND Europe и финансируемого за счет средств Седьмой рамочной программы (FP7) Европейского союза (Соглашение № 223277).

Оценка экономических потерь от нездорового питания и низкой физической активности: Обзор фактических данных и предлагаемая общая схема оценки

Нездоровый рацион питания и недостаточная физическая активность способствуют развитию многих хронических болезней и инвалидности. В глобальном масштабе на них приходится около 40% смертей и около 30% бремени болезней. Вместе с тем, мы на удивление мало знаем об экономических потерях, обусловленных этими факторами риска, с точки зрения как медико-санитарной помощи, так и общества в целом. В настоящем исследовании анализируются фактические данные об экономическом бремени, которое можно отнести на счет нездорового питания и недостаточной физической активности. Его авторы попытались дать ответы на следующие вопросы: как варьируются определения и к чему это приводит; насколько сложно оценить экономическое бремя; как найти лучшие способы оценки издержек, обусловленных нездоровым рационом питания и недостаточной физической активностью, на примере диабета. В обзоре делается вывод о том, что нездоровый рацион питания и недостаточная физическая активность влекут за собой значительные расходы на медико-санитарную помощь, но при этом их оценки широко варьируются. Существующие исследования недооценивают истинное экономическое бремя, рассматривая лишь расходы систем здравоохранения. В то же время, косвенные издержки, обусловленные снижением производительности труда, могут быть примерно вдвое выше прямых расходов на медико-санитарную помощь. В сумме такие затраты составляют примерно 0,5% национального дохода. Также авторы исследования изучили возможность для оценки издержек от нездорового питания и недостаточной физической активности в Регионе, на основании одной болезни: они утверждают, что в 2020 г. общее экономическое бремя от новых случаев диабета 2 типа, вызванных этими двумя факторами риска, составит 883 млн евро только лишь в Германии, Испании, Италии, Соединенном Королевстве и Франции. "Истинные" издержки могут быть еще выше, поскольку нездоровое питание и недостаточная физическая активность ассоциируют и с целым рядом других болезней. Результаты исследования позволяют лучше понять экономическое бремя, которое может быть обусловлено двумя основными факторами риска потери здоровья. На их основании лица, формирующие политику, смогут устанавливать свои приоритеты и более эффективно поддерживать здоровое питание и физическую активность.

Organization and financing of public health services in Europe

How are public health services in Europe organized and financed? With European health systems facing a plethora of challenges that can be addressed through public health interventions, there is renewed interest in strengthening public health services. Yet, there are enormous gaps in our knowledge. How many people work in public health? How much money is spent on public health? What does it actually achieve? None of these questions can be answered easily. This volume brings together current knowledge on the organization and financing of public health services in Europe. It is based on country reports on the organization and financing of public health services in nine European countries and an in-depth analysis of the involvement of public health services in addressing three contemporary public health challenges (alcohol, obesity and antimicrobial resistance). The focus is on four core dimensions of public health services: organization, financing, the public health workforce, and quality assurance. The questions the volume seeks to answer are: How are public health services in Europe organized? Are there good practices that can be emulated? What policy options are available?; How much is spent on public health services? Where do resources come from? And what was the impact of the economic crisis?; What do we know about the public health workforce? How can it be strengthened?; How is the quality of public health services being assured? What should quality assurance systems for public health services look like? This study is the result of close collaboration between the European Observatory on Health Systems and Policies and the WHO Regional Office for Europe, Division of Health Systems and Public Health. It accompanies two other Observatory publications: Organization and financing of public health services in Europe: country reports and The role of public health organizations in addressing public health problems in Europe: the case of obesity, alcohol and antimicrobial resistance.

The role of public health organizations in addressing public health problems in Europe: the case of obesity, alcohol and antimicrobial resistance

Growing levels of overweight and obesity, continued harmful consumption of alcohol, and the growing threat of AMR are some of the greatest challenges to the health of European populations. While the magnitude of these problems varies from country to country, they affect all countries in Europe. For each problem, it is clear that public health agencies and organizations must play a part in any response, with intersectoral action beyond the health system needed. What is less clear is what role public health organizations currently play in addressing these problems.This is the gap that this volume aims to fill. It is based on country reports from eight European countries (England, France, Germany, Italy, the Republic of Moldova, the Netherlands, Poland, and Sweden) on the involvement of public health organizations in addressing alcohol consumption and obesity and on reports from nine European countries (England, France, Germany, Italy, the Republic of Moldova, the Netherlands, Poland, Slovenia and Sweden) on their involvement in addressing antimicrobial resistance.This web edition includes Country reports – appendix to The role of public health organizations in addressing public health problems in Europe: the case of obesity, alcohol and antimicrobial resistance (2018; ISBN 9789289051712).

The role of public health organizations in addressing public health problems in Europe: the case of obesity, alcohol and antimicrobial resistance

Growing levels of obesity (including among children), continued harmful consumption of alcohol and the growing threat of antimicrobial resistance (AMR) are some of the greatest contemporary challenges to the health of European populations. While their magnitude varies from country to country, all are looking for policy options to contain these threats to population health. It is clear that public health organizations must play a part in any response, and that intersectoral action beyond the health system is needed. What is less clear, however, is what role public health organizations currently play in addressing these problems. This is the gap that this volume aims to fill. It is based on detailed country reports from nine European countries (England, France, Germany, Italy, the Republic of Moldova, the Netherlands, Poland, Slovenia and Sweden) on the involvement of public health organizations in addressing obesity, alcohol and antimicrobial resistance. These reports explore the power and influence of public health organizations vis-a-vis other key actors in each of the stages of the policy cycle (problem identification and issue recognition, policy formulation, decision-making, implementation, and monitoring and evaluation). A cross-country comparison assesses the involvement of public health organizations in the nine countries covered. It outlines the scale of the problem, describes the policy responses, and explores the role of public health organizations in addressing these three public health challenges. This study is the result of close collaboration between the European Observatory on Health Systems and Policies and the WHO Regional Office for Europe, Division of Health Systems and Public Health. It accompanies two other Observatory publications: Organization and financing of public health services in Europe and Organization and financing of public health services in Europe: country reports.

Organization and financing of public health services in Europe: country reports

What are “public health services”? Countries across Europe understand what they are, or what they should include, differently. This study describes the experiences of nine countries, detailing the ways they have opted to organize and finance public health services, and train and employ their public health workforce. It covers England, France, Germany, Italy, the Netherlands, Slovenia, Sweden, Poland and the Republic of Moldova, and aims to give insights into current practice that will support decision-makers in their efforts to strengthen public health capacities and services. Each country chapter captures the historical background of public health services and the context in which they operate; sets out the main organizational structures; assesses the sources of public health financing and how it is allocated; explains the training and employment of the public health workforce; and analyses existing frameworks for quality and performance assessment. The study reveals a wide range of experience and variation across Europe and clearly illustrates two fundamentally different approaches to public health services: integration with curative health services (as in Slovenia or Sweden) or organization and provision through a separate parallel structure (Republic of Moldova). The case studies explore the context that explain this divergence and its implications. This study is the result of close collaboration between the European Observatory on Health Systems and Policies and the WHO Regional Office for Europe, Division of Health Systems and Public Health. It accompanies two other Observatory publications: Organization and financing of public health services in Europe and The role of public health organizations in addressing public health problems in Europe: the case of obesity, alcohol and antimicrobial resistance.

Assessing the economic costs of unhealthy diets and low physical acitivity: an evidence review and proposed framework

Unhealthy diets and low physical activity contribute to many chronic diseases and disability; they are responsible for some 2 in 5 deaths worldwide and for about 30% of the global disease burden. Yet surprisingly little is known about the economic costs that these risk factors cause, both for health care and society more widely. This study pulls together the evidence about the economic burden that can be linked to unhealthy diets and low physical activity and explores: how definitions vary and why this matters; the complexity of estimating the economic burden and; how we can arrive at a better way to estimate the costs of an unhealthy diet and low physical activity, using diabetes as an example. The review finds that unhealthy diets and low physical activity predict higher health care expenditure, but estimates vary greatly. Existing studies underestimate the true economic burden because most only look at the costs to the health system. Indirect costs caused by lost productivity may be about twice as high as direct health care costs, together accounting for about 0.5% of national income. The study also tests the feasibility of using a disease-based approach to estimate the costs of unhealthy diets and low physical activity in Europe, projecting the total economic burden associated with these two risk factors as manifested in new type 2 diabetes cases at €883 million in 2020 for France, Germany, Italy, Spain and the United Kingdom alone. The ‘true’ costs will be higher, as unhealthy diets and low physical activity are linked to many more diseases. The study’s findings are a step towards a better understanding of the economic burden that can be associated with two key risk factors for ill health and they will help policy-makers in setting priorities and to more effectively promoting healthy diets and physical activity.

Armenia: health system review

The Health Systems in Transition (HiT) profiles are country-based reports that provide a detailed description of a health system and of policy initiatives in progress or under development. HiTs examine different approaches to the organization, financing and delivery of health services and the role of the main actors in health systems; describe the institutional framework, process, content and implementation of health and health care policies; and highlight challenges and areas that require more in-depth analysis. Since independence, the health system in Armenia has undergone numerous changes that have effectively transformed a centrally run state system into a fragmented one that is largely financed from out-of-pocket payments. The population, especially those in need, meet with limited access to health services, and those services which are available are often of questionable quality, as health care standards and quality assessment systems are absent. Drugs on the essential drugs list are generally not affordable to those in need. Many health facilities, especially in rural areas, lack modern medical technology and what is available is not distributed efficiently. The commitment to free health care thus remains more declarative than factual, as informal payments are still expected in many cases. Despite significant investments in primary care, a disproportionate share of resources has been invested in secondary and tertiary care. Nevertheless, Armenia is increasingly engaged in reforming the system from one that emphasizes the treatment of diseases and response to epidemics towards a system that emphasizes prevention, family care and community participation. The shift towards a primary care orientation is noticeable, with gradually increased roles for health workers to influence the determinants of health.

Assessing chronic disease management in European health systems: country reports

Many countries are exploring innovative approaches to redesign delivery systems to provide appropriate support to people with long-standing health problems. Central to these efforts to enhance chronic care are approaches that seek to better bridge the boundaries between professions, providers and institutions, but, as this study clearly demonstrates, countries have adopted differing strategies to design and implement such approaches. This book systematically examines experiences of 12 countries in Europe, using an explicit comparative approach and a unified framework for assessment to better understand the diverse range of contexts in which new approaches to chronic care are being implemented, and to evaluate the outcomes of these initiatives. The study focuses on the content of these new models, which are frequently applied from different disciplinary and professional perspectives and associated with different goals and does so through analysing approaches to self-management support, service delivery design and decision-support strategies, financing, availability and access. Significantly, it also illustrates the challenges faced by individual patients as they pass through the system. This book complements the earlier published study Assessing chronic disease management in European health systems; it builds on the findings of the DISMEVAL project (Developing and validating DISease Management EVALuation methods for European health care systems), led by RAND Europe and funded under the European Union's (EU) Seventh Framework Programme (FP7) (Agreement no. 223277).

What is the evidence on the economic impacts of integrated care?

This policy summary reviews the existing evidence on the economic impact of integrated care approaches. Whereas it is generally accepted that integrated care models have a positive effect on the quality of care, health outcomes and patient satisfaction, it is less clear how cost effective they are. As the evidence-base in this field is rather weak, the authors suggest that we may have to revisit our understanding of the concept and our expectations in terms of its assessment. Integrated care should rather be seen as a complex strategy to innovate and implement long-lasting change in the way services in the health and social-care sectors are delivered. This policy summary is based on a report for the European Commission to inform the discussions of the EU’s Reflection process on modern, responsive and sustainable health systems on the objective of integrated care models and better hospital management.