Colonoscopy vs the Fecal Immunochemical Test: Which is Best?

Although there is no debate around the effectiveness of colorectal cancer screening in reducing disease burden, there remains a question regarding the most effective and cost-effective screening modality. Current United States guidelines present a panel of options that include the 2 most commonly used modalities, colonoscopy and stool testing with the fecal immunochemical test (FIT). Large-scale comparative effectiveness trials comparing colonoscopy and FIT for colorectal cancer outcomes are underway, but results are not yet available. This review will separately state the "best case" for FIT and colonoscopy as the screening tool of first choice. In addition, the review will examine these modalities from a health economics perspective to provide the reader further context about the relative advantages of these commonly used tests.

Methods for Including Adverse Events in Economic Evaluations: Suggestions for Improvement.

OBJECTIVE: Inclusion of relevant effectiveness and safety outcomes in economic evaluation of health technologies is required to aid efficient healthcare decisions. Our objective was to identify the key issues related to the inclusion of adverse events (AEs) in economic evaluation and explore perspectives for good practice recommendations to handle these issues. METHODS: We focused on the frequently encountered methodological issues related to the integration of AEs in economic evaluations of health technologies. We distinguished the following elements: the incorporation of AEs in decision models, the terminology of AEs, the estimation of AEs consequences in terms of quality of life (QoL) and costs, and the exploration of the uncertainty related to the impact of AEs on the economic results. RESULTS: We illustrated and discussed each of the identified issues by giving health technology assessment examples. We focused on the extent to which the integration of AEs in decision models can be improved by dealing with the lack of relevant real-world safety data, estimating the consequences of AEs (eg, for costs and QoL loss), exploring the impacts of AEs that are not adequately captured in current measurement of health-related QoL, and identifying the need for development of a good terminology of relevant types of AEs to be incorporated in economic evaluation. CONCLUSION: Based on a reflection the key methodological issues related to the incorporation of adverse drug events in economic evaluations, we suggested several recommendations to serve a starting point for health technology assessment agencies and researchers to develop good research practices in this field.

Mapping Payment and Pricing Schemes for Health Innovation: Protocol of a Scoping Literature Review.

INTRODUCTION: Innovative pricing and payment/reimbursement schemes have been proposed as one part of the solution to the problem of patient access to new health technologies or to the uncertainty about their long-term effectiveness. As part of a Horizon Europe research project on health innovation next generation pricing and payment models (HI-PRIX), this protocol illustrates the conceptual and methodological steps related to a scoping review aiming at investigating nature and scope of pricing and payment/reimbursement schemes applied to, or proposed for, existing or new health technologies. METHODS: A scoping review of literature will be performed according to the PRISMA guidelines for scoping reviews (PRISMA-ScR) guidelines. The search will be conducted in three scientific databases (i.e., PubMed, Web of Science, and Scopus), over a 2010-2023 timeframe. The search strategy is structured around two blocks of keywords, namely "pricing and payment/reimbursement schemes," and "innovativeness" (of the scheme type or scheme use). A simplified search will be replicated in the gray literature. Studies illustrating pricing and payment/reimbursement schemes with a sufficient level of details to explain their characteristics and functioning will be deemed eligible to be considered for data synthesis. Pricing and payment/reimbursement schemes will be classified according to several criteria, such as their purpose, nature, governance, data collection needs, and foreseen distribution of risk. The results will populate a publicly available online tool, the Pay-for-Innovation Observatory. DISCUSSION: The findings of this review have the potential to offer a comprehensive toolkit with a variety of pricing and payment schemes to policymakers and manufacturers facing reimbursement and access decisions.

Real-world evidence for coverage determination of treatments for rare diseases.

Health technology assessment (HTA) decisions for pharmaceuticals are complex and evolving. New rare disease treatments are often approved more quickly through accelerated approval schemes, creating more uncertainties about clinical evidence and budget impact at the time of market entry. The use of real-world evidence (RWE), including early coverage with evidence development, has been suggested as a means to support HTA decisions for rare disease treatments. However, the collection and use of RWE poses substantial challenges. These challenges are compounded when considered in the context of treatments for rare diseases. In this paper, we describe the methodological challenges to developing and using prospective and retrospective RWE for HTA decisions, for rare diseases in particular. We focus attention on key elements of study design and analyses, including patient selection and recruitment, appropriate adjustment for confounding and other sources of bias, outcome selection, and data quality monitoring. We conclude by offering suggestions to help address some of the most vexing challenges. The role of RWE in coverage and pricing determination will grow. It is, therefore, necessary for researchers, manufacturers, HTA agencies, and payers to ensure that rigorous and appropriate scientific principles are followed when using RWE as part of decision-making.

Como avaliar uma política de saúde? / How to assessment a health policy

Revisa o potencial de uso da avaliaçäo econômica no desenvolvimento de políticas eficientes de saúde. Demonstra que os métodos de avaliaçäo econômica alcançaram padröes científicos aceitáveis e que estudos têm sido realizados em muitos campos clínicos. Discute o uso dos resultados de avaliaçäo na elaboraçäo de políticas, especialmente para tecnologias de saúde

How can European health systems support investment in and the implementation of population health strategies?

Key messagesPolicy issue and associated policy challenges• Population health is influenced by a variety of factors, many of whichrequire action outside the health system.• The health and socioeconomic costs of the key contributors to poor healthin Europe are substantial. Many of these health problems are avoidable.• There is a growing body of information not only on the effectiveness butalso on the cost-effectiveness of population health interventions.• Policy measures to help promote investment in effective interventions needto focus on improving both the quality and use of evidence across multiplesectors.Policy options• One option to strengthen the evidence base might be to expand the remitof existing regulatory bodies that assess the cost-effectiveness of healthcare technologies.• A second option in parts of Europe where capacity for evaluation is limitedmight be to adapt existing information to the local context to make thecase for investment.• Various institutional arrangements, including the possibility of a standaloneministry for population health, might help to facilitate coordinationand secure funding for action; other related options include joint budgetsor monetary transfers between sectors.Facilitating implementation• When new assessments are conducted, these might begin by focusing oninterventions likely to be highly effective, cost-effective andnoncontroversial. This can help new institutions to establish their presenceand credibility.• Improving communication between researchers and policy-makers acrosssectors can help facilitate change; knowledge brokers might provide a linkbetween different groups.• Increasing awareness of the health, non-health and economic effects ofinterventions can help to reduce resistance to action outside the healthsector. Health impact assessment may have a role to play in this process.• Mechanisms to monitor the implementation process across sectors mightInvesting in and implementing population health strategiesalso help facilitate change; setting explicit measurable targets onpopulation health objectives might provide further incentives forstakeholders across sectors to take action.Executive summaryPoor health in Europe has substantial health and socioeconomic costs. Much ofthis burden might be avoided by implementing effective population healthstrategies, both within and outside the health system. A broad approach topromoting population health requires a combination of upstream anddownstream measures. Upstream measures may include measures that, amongother goals, can help promote health, such as fiscal redistribution, improvingthe quality of housing and using incentives to encourage students to stay inschool. Downstream measures include health promotion and primary diseaseprevention action, often targeting individual behaviour and lifestyle.To support investment in population health strategies, health systems must beable to identify not only what works and at what cost but also in what context.Mechanisms to allow such information to be fed into the policy deliberationprocess and also to facilitate the implementation of agreed population healthstrategies are then required.Generating and using existing evidence on the effectiveness and costeffectivenessof population health strategiesUsing systematic reviewsSystematic reviewing, which seeks to systematically identify and appraiseeffectiveness (and other evidence) on a given topic, can be particularly useful inassessing whether interventions are effective. Collating and, where feasible,statistically pooling information from studies reduce the probability that oneunrepresentative study would bias the results of any effectiveness analysis.Information from existing high-quality reviews can also provide rapidinformation on the evidence (and evidence gaps) on a particular topic. This mayavoid duplicating time-consuming and costly reviews.Using economic evaluationEconomic evaluation can also strengthen the case for investing in populationhealth interventions. Widely used in the health care, environmental andtransport sectors, economic evaluation compares the costs and effects ofalternative courses of action.Evidence on the cost-effectiveness of population health interventions, whilemodest compared with health care evaluation, has grown rapidly, particularlyfor screening and vaccinations. Examples of complex cost-effectiveinterventions in specific settings and contexts include targeted exerciseprogrammes for older people, measures for controlling tobacco, drugs andalcohol (including taxation), early-year interventions targeting children and theirInvesting in and implementing population health strategiesparents and traffic-calming and transport safety measures. Many interventionsare funded and delivered outside the health system.Policy options moving forward: strengthening the evidence baseHow can countries better use effectiveness and economic evaluation in theirdeliberations on population health strategies? Institutional and regulatoryarrangements might include independent agencies to assess evidence; in-housegovernment agencies; and support for knowledge clearing houses.Establishing and/or expanding the remit of health technologyassessment agenciesOne option might be to expand the remit of existing health technologyassessment agencies. In England, the National Institute for Health and ClinicalExcellence now collates evidence on the effectiveness and cost-effectiveness ofpublic health interventions. Evaluation includes mental and physical well-beinginterventions delivered in the workplace and school-based alcohol educationstrategies. It uses a highly formalized process taking up to one year tocomplete, which includes opportunities for stakeholder input and formal linksto the policy-making process. It adopts a broader economic perspective thanthat used for health care interventions, recognizing effects outside the healthcare system.Adapting results from existing economic evaluationAnother approach, especially when resources are limited, may be to adaptinformation on existing cost-effectiveness studies, such as using the WHOCHOICE(Choosing Interventions that are Cost Effective) programme. Thisallows a range of interventions, including those for prevention, treatment andrehabilitation, to be compared in isolation or in combination to determine anoptimal mix of resources within a health care system. The results are presentedin such a way as to determine the probability of an intervention being costeffectivein low-, medium- and high-resource situations. They can be adapted,using local information, to specific country contexts.Reforming institutional arrangementsInstitutional structures and governance arrangements can also influence thesuccess of the implementation of population health interventions. Onepossibility, given that funding for population health within health care budgetscan be severely limited, would be to establish a dedicated ministry. Very fewattempts have been made to set up such institutions. One rare example is theMinistry of Health Promotion in Ontario, Canada, established in June 2005. It isstill too early to judge how effective this model will be; the challenge ofPolicy briefcoordinating activities with the health ministry might also mean that publichealth becomes more marginalized from mainstream health care policy.Other alternatives include establishing specific governance structures forpopulation health within a health ministry, including a ministerial post,partnership arrangement to encourage intersectoral cooperation at both thenational and local levels and flexible funding structures, including mandatory orvoluntary joint budgets.Identifying alternative ways of bringing about changeWhat mechanisms might be available to ensure that population health is on theagenda of policy-makers in all sectors and at all levels, directing them to beaware of the health consequences of their decisions and to accept theirresponsibilities for health? How can governments provide incentives toadministrative bodies to implement national policy on population health?Giving priority to the low-hanging fruitIn building the evidence base on population health interventions within acountry, initially picking low-hanging fruit ­ interventions likely to be highlyeffective, cost-effective and noncontroversial ­ may be prudent. Several healthtechnology assessment agencies have adopted this policy when they arebecoming established. This can help organizations to establish their presenceand credibility.Improving the communication between researchers and policy-makersResearchers and policy-makers often do not speak in a common language andmay distrust each other. Knowledge brokers, individuals who understandscientific evidence but are comfortable working in a policy-makingenvironment, may help overcome this problem. They can help translateacademic reports into brief messages relevant to policy-makers and help ensurethat policy-makers commission feasible research. With appropriate training,staff from international agencies, such as WHO, could potentially act asknowledge brokers.Improving the awareness of health effects among policy-makers acrosssectorsAwareness of the health effects of policies developed and implemented outsidethe health sector may be limited. One way of improving awareness may bethrough the formalized use of health impact assessment. This has been used inseveral countries, but the extent to which it has facilitated change remainsunclear.Investing in and implementing population health strategiesTargets, monitoring and evaluationInvesting in systems to monitor and evaluate implementation and measuringthe longer-term effects on population health outcomes can also be helpful.Joint targets across government departments might be set and progresstowards achievement monitored. Negative publicity arising from failing toachieve targets may act as a powerful incentive foraction.

How can the impact of health technology assessments be enhanced?

Policy issues• Health technology assessment (HTA) is an important tool for informing effective regulation of the diffusion and use of health technologies.• The key policy issues surrounding the use of HTA fall into three areas: (a) the bodies, decision-makers and other stakeholders involved, (b) the methods and processes employed; and (c) how the findings of HTAs are implemented.• The impact of HTA can be enhanced if: key stakeholders (e.g. patients, providers and industry) are adequately involved; decision-makers give a prior commitment to use assessment reports (and assessments meet their needs); the necessary resources are available for implementing decisions; there is transparency in the assessment and decision-making processes; and collaboration, knowledge and skills are transferred across jurisdictions.Policy measures• Increased stakeholder involvement throughout the HTA process can help capture and improve the real-world value and applicability of HTAs. Nevertheless, stakeholder involvement needs to be transparent and well-managed in order to ensure that the objectivity of assessments is not influenced.• HTAs must be timely in relation to the decisions they seek to inform. Simpler studies, early-warning systems and conditional approvals are increasingly being used to manage the uncertainty surrounding new and emerging technologies while facilitating the timeliness and relevancy of HTA.• International collaboration among HTA bodies can facilitate the development of methods and more efficient assessment processes, and facilitate knowledge transfer and capacity-building in less established HTA systems and programmes.• To facilitate the use and implementation of HTA reports in decision-making, incentives within a given health care system must be appropriately aligned with the decisions that are based on (or informed by) HTA.Implementation considerations• Problems with applying technical information and national recommendations to local decision-making can be reduced if there are formal links between the producers and users of HTA.• Learning through collaboration and exchange of experience can help to overcome those institutional and capacity barriers that often hinder implementation.

Wie können europäische Gesundheitssysteme Strategien zur Förderung der Gesundheit der Bevölkerung durch Investitionen und Umsetzungsmaßnahmen unterstützen?

Gesundheitliche Probleme führen in der Europäischen Region zu erheblichen Kosten für das Gesundheitssystem und den sozioökonomischen Bereich. Diese Belastung kann größtenteils vermieden werden, wenn sowohl innerhalb als auch außerhalb des Gesundheitssystems wirksame Strategien zur Förderung der Gesundheit der Bevölkerung umgesetzt werden. Zu einer umfassenden Förderung der Bevölkerungsgesundheit gehört eine Kombination von Maßnahmen, die einerseits vorgelagert und andererseits nachgelagert sein können. Vorgelagerte Maßnahmen sind Maßnahmen, die neben weiterenZielen auch der Gesundheitsförderung dienen, z. B. steuerliche Umverteilung, Verbesserung der Wohnsituation und Einführung von Anreizen, die Lernende zur Fortführung ihrer Ausbildung motivieren. Zu den nachgelagerten Maßnahmen zählen Gesundheitsförderung und Maßnahmen zur primären Krankheitsprävention. Diese Maßnahmen sind häufig auf das Verhalten und die allgemeine Lebensführung von Einzelpersonen gerichtet. Um Investitionen in Strategien zur Förderung der Gesundheit der Bevölkerung zu unterstützen, müssen die Gesundheitssysteme nicht nur in der Lage sein,mögliche Lösungen und die mit ihnen verbundenen Kosten zu identifizieren, sondern sie müssen auch den Rahmen für diese Lösungen festlegen. Anschließend müssen Mechanismen bestimmt werden, die die Einbeziehung solcher Informationen in den Entscheidungsfindungsprozess ermöglichen und die Umsetzung der vereinbarten Strategien zur Förderung der Gesundheit der Bevölkerung erleichtern.

Wie kann die Gesundheitstechnologiebewertung wirkungsvoller gestaltet werden?

Die zunehmende Verbreitung neuer Gesundheitstechnologien hat zu bemerkenswertenVerbesserungen in Bezug auf Gesundheit und Lebensqualität geführt. Allerdings werfen diese Vorteile auch die problematische Frage auf, welcher Gegenwert für die Investitionen entsteht und ob die Steuer- bzw. Beitragszahler und die Patienten bereit sind, die hohen Kosten für Behandlungen, Geräte und Arzneimittel zu übernehmen. Da die Politik bestrebt ist, mit begrenzten Mitteln einen maximalen Nutzen zu erzielen und dies auf legitime und transparente Weise im Einklang mit den Grundwerten der Gesundheitssysteme zu tun,wird die Gesundheitstechnologiebewertung (HTA) in zunehmendem Maße als Instrument zur Unterstützung dieses Ziels und zur Förderung einer effizienten Nutzung von Gesundheitstechnologien verwendet. In dem vorliegenden Grundsatzpapier werden ausgewählte Sachfragen in Bezug auf die Anwendung und Akzeptanz von HTA in der Europäischen Region untersucht.

Ensuring value for money in health care: the role of health technology assessment in the European Union

This book provides a detailed review of the role of health technology assessment (HTA) in the European Union. It examines both method and process in the prioritization and financing of modern health care, and presents extensive case studies from Sweden, the Netherlands, Finland, France, Germany and the United Kingdom. The book examines a number of issues, with particular emphasis on the responsibility and membership of HTA bodies, assessment procedures and methods, the application of HTA evidence to decision-making, and the dissemination and implementation of findings. It aims to highlight ways to improve the HTA process in Europe by examining key challenges and identifying opportunities to support value and innovation in health care. It will be vital reading for a wide range of stakeholders including policy-makers, HTA bodies, manufacturers, health-care professionals and patient organizations.

Как могут европейские системы здравоохранения помочь в инвестировании в стратегии охраны и укрепления здоровья населения и в их реализации?

Нездоровье людей в Европе сопряжено со значительными медицинскими и социально-экономическими издержками. Немалую часть этого бремени можно было бы избежать, если бы были реализованы действенные стратегии охраны и укрепления здоровья населения, причем как в системе здравоохранения, так и в других сферах жизни общества. Широкий подход к делу укрепления здоровья требует сочетания мер, принимаемых на верхнем и на нижнем уровнях воздействия на детерминанты здоровья. Меры, принимаемые на верхнем уровне, могут включать в себя такие действия, которые в дополнение к другим целям могут способствовать укреплению здоровья – например, перераспределение налоговыхпоступлений, повышение качества жилья и применение стимулов, поощряющих учащихся к тому, чтобы они не прекращали учебы. Меры,принимаемые на нижнем уровне, включают непосредственные действия по укреплению здоровья и первичной профилактике заболеваний, часто нацеленные на поведение и образ жизни отдельного человека. Для того, чтобы способствовать вложению средств в стратегии охраны и укрепления здоровья населения, системы здравоохранения должны быть в состоянии не только показать то, что дает положительный эффект и какой ценой, но и определить, в каких условиях этот эффект может быть достигнут. А для этого нужны механизмы, позволяющие вводить такуюинформацию в процесс обдумывания политики и облегчающие реализацию согласованных стратегий.