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BACKGROUND: Scientific research and innovation can generate societal impact via different pathways. Productive interactions, such as collaboration between researchers and relevant stakeholders, play an important role and have increasingly gained interest of health funders around the globe. What works, how and why in research partnerships to generate societal impact in terms of knowledge utilisation is still not well-known. To explore these issues, the Netherlands Organization for Health Research and Development (ZonMw) initiated an exploratory research-on-research study with a focus on participatory knowledge infrastructures (PKIs) that they fund in the field of public health and healthcare. PKIs are sustainable infrastructures in which knowledge production, dissemination and utilisation takes place via committed collaboration between researchers and stakeholders from policy, practice and/or education. Examples are learning networks, academic collaborative centres, care networks and living labs. The aim of the study was twofold: to gain insights in what constitutes effective collaboration in PKIs; and to learn and improve the research governance, particularly of ZonMw as part of their dissemination and implementation activities. METHODS: During 2020-2022, we conducted a literature review on long-term research partnerships, analysed available documentation of twenty ZonMw-funded PKIs, surveyed participants of the 2021 European Implementation Event, interviewed steering committee members, organized a Group Decision Room with lecturers, and validated the findings with key experts. RESULTS: We identified eight mechanisms ('how and why') that are conditional for effective collaboration in PKIs: transdisciplinary collaboration; defining a shared ambition; doing justice to everyone's interests; investing in personal relationships; a professional organisation or structure; a meaningful collaborative process; mutual trust, sufficient time for and continuity of collaboration. Several factors ('what') may hinder (e.g., lack of ownership or structural funding) or facilitate (e.g., stakeholder commitment, embeddedness in an organisation or policy) effective collaboration in research partnerships. CONCLUSION: To use the study results in policy, practice, education, and/or (further) research, cultural and behavioural change of all stakeholders is needed. To facilitate this, we provide recommendations for funding organisations, particularly ZonMw and its partners within the relevant knowledge ecosystem. It is meant as a roadmap towards the realisation and demonstration of societal impact of (health) research and innovation in the upcoming years.
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Conducta Cooperativa , Países Bajos , Humanos , Participación de los Interesados , Investigadores , Salud Pública , Investigación sobre Servicios de Salud , Conocimiento , Difusión de la Información , Atención a la SaludRESUMEN
The inherent normativity of HTA can be conceptualized as a result of normative commitments, a concept that we further specify to encompass moral, epistemological and ontological commitments at play in the practice of HTA. Based on examples from literature, and an analysis of the example of assessing Non-Invasive Prenatal Testing (NIPT), we will show that inevitable normative decisions in conducting an assessment commits the HTA practitioner to moral (regarding what makes a health technology desirable), ontological (regarding which effects of health technology are conceivable), and epistemological (regarding how to obtain reliable information about health technology) norms. This highlights and supports the need for integrating normative analysis and stakeholder participation, providing guidance to HTA practitioners when making normative choices. This will foster a shared understanding between those who conduct, use, or are impacted by assessments regarding what are conceivable and desirable outcomes of using health technology, and how to collect reliable information to assess whether these outcomes are (going to be) realized. It also provides more insight into the implications of different normative choices.
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BACKGROUND: Since 2014, several clinical studies focusing on centronuclear myopathies have been conducted, including a prospective natural history study, a gene transfer clinical trial and a clinical trial using an antisense oligonucleotide. Dedicated patient organizations have played an important role in this process. The experience of members of these organizations, either as a study participant, parent or as a patient organization member communicating with the sponsors are potentially very informative for future trial design. METHODS: We investigated the burden of and the lessons learned from the first natural history studies and clinical trials from a patient perspective using a qualitative approach. We arranged 4 focus groups with a total of 37 participants from 3 large international patient organizations: ZNM-ZusammenStark!, the Myotubular Trust, and the MTM-CNM Family Connection. 4 themes, based on a systematic literature search were discussed: Expectations and preparation, Clinical study participation, Communication and Recommendations for future clinical trials. The focus group recordings were transcribed, anonymized, and uploaded to Atlas-ti version 8.1 software. The data were analyzed using a thematic content analysis. RESULTS: Overall, participants were realistic in their expectations, hoping for small improvements of function and quality of life. The realization that trial participation does not equate to a treatment was challenging. Participating in a clinical study had a huge impact on many aspects of daily life, both for patients and their immediate families. First-hand insights into the burden of the design and its possible effect on performance were provided, resulting in numerous compelling recommendations for future clinical studies. Furthermore, participants stressed the importance of clear communication, which was considered to be especially vital in cases of severe adverse events. Finally, while patients were understanding of the importance of adhering to the regulations of good clinical practice, they indicated that they would strongly appreciate a greater understanding and/or acknowledgment of the patient perspective and a reflection of this perspective in future clinical trial design. CONCLUSION: The acknowledgment and inclusion of patients' perspectives and efficient and effective communication is expected to improve patient recruitment and retention in future clinical studies, as well as more accurate assessment of the patient performance related to suitable planning of the study visits.
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OBJECTIVES: Our objective was to explore procedures and methods used at health technology assessment (HTA) agencies for assessing medical devices and the underlying views of HTA practitioners about appropriate methodology to identify challenges in adopting new methodologies for assessing devices. We focused on the role of normative commitments of HTA practitioners in the adoption of new methods. METHODS: An online survey, including questions on procedures, scoping, and assessments of medical devices, was sent to members of the International Network of Agencies for Health Technology Assessment. Interviews were conducted with survey respondents and HTA practitioners involved in assessments of transcatheter aortic valve implantation to gain an in-depth understanding of choices made and views about assessing medical devices. Survey and interview questions were inspired by the "values in doing assessments of health technologies" approach towards HTA, which states that HTA addresses value-laden questions and information. RESULTS: The current practice of assessing medical devices at HTA agencies is predominantly based on procedures, methods, and epistemological principles developed for assessments of drugs. Both practical factors (available time, demands of decision-makers, existing legal frameworks, and HTA guidelines), as well as commitments of HTA practitioners to principles of evidence-based medicine, make the adoption of a new methodology difficult. CONCLUSIONS: There is a broad recognition that assessments of medical devices may need changes in HTA methodology. In order to realize this, the HTA community may require both a discussion on the role, responsibility, and goals of HTA, and resulting changes in institutional context to adopt new methodologies.
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Equipos y Suministros , Investigación Cualitativa , Evaluación de la Tecnología Biomédica , Evaluación de la Tecnología Biomédica/organización & administración , Evaluación de la Tecnología Biomédica/normas , Humanos , Equipos y Suministros/normas , Toma de Decisiones , Entrevistas como Asunto , Medicina Basada en la Evidencia , Encuestas y Cuestionarios/normas , Reemplazo de la Válvula Aórtica TranscatéterRESUMEN
PURPOSE: To develop a multidisciplinary outpatient rehabilitation intervention for people with neuromuscular diseases (NMD) based on the capability approach: capability care for persons with NMD. MATERIALS AND METHODS: The development process is described using a framework of actions for intervention development. It has been an iterative process consisting of a design phase based on theoretical insights and project group discussions, and a refine phase involving input from relevant stakeholders. RESULTS: Multidisciplinary efforts have resulted in the development of capability care for rehabilitation of persons with NMD. It can focus both on facilitating and achieving functionings (beings and doings), as well as looking for alternative functionings that fulfil the same underlying value, thereby contributing to the persons' well-being. To facilitate a conversation on broader aspects that impact on well-being, persons with NMD receive a preparation letter and healthcare professionals are provided with guiding questions and practical tools to use. CONCLUSIONS: We have shown that it is possible to develop a healthcare intervention based on the capability approach. We hope that rehabilitation professionals will be encouraged to use capability care and that other medical professionals will be inspired to develop capability care in their respective fields. REGISTRATION: Registered at trialregister.nl NL8946.
The capability approach can be used for development of healthcare interventions.Capability care in rehabilitation focuses on realising what is of real value to the person.The capability approach and the ICF are complementary and can both be used in rehabilitation.
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BACKGROUND: There is growing evidence to support the benefits of transcatheter aortic valve implantation (TAVI) over surgical aortic valve replacement (SAVR) in patients with symptomatic severe aortic stenosis (sSAS) who are at high- or intermediate-risk of surgical mortality. The PARTNER 3 trial showed clinical benefits with SAPIEN 3 TAVI compared with SAVR in patients at low risk of surgical mortality. Whether TAVI is also cost-effective compared with SAVR for low-risk patients in the Dutch healthcare system remains uncertain. This article presents an analysis using PARTNER 3 outcomes and costs data from the Netherlands to inform a cost-utility model and examine cost implications of TAVI over SAVR in a Dutch low-risk population. METHODS: A two-stage cost-utility analysis was performed using a published and validated health economic model based on adverse events with both TAVI and SAVR interventions from a published randomized low risk trial dataset, and a Markov model that captured lifetime healthcare costs and patient outcomes post-intervention. The model was adapted using Netherlands-specific cost data to assess the cost-effectiveness of TAVI and SAVR. Uncertainty was addressed using deterministic and probabilistic sensitivity analyses. RESULTS: TAVI generated 0.89 additional quality-adjusted life years (QALYs) at a 4742 increase in costs per patient compared with SAVR over a lifetime time horizon, representing an incremental cost-effectiveness ratio (ICER) of 5346 per QALY gained. Sensitivity analyses confirm robust results, with TAVI remaining cost-effective across several sensitivity analyses. CONCLUSIONS: Based on the model results, compared with SAVR, TAVI with SAPIEN 3 appears cost-effective for the treatment of Dutch patients with sSAS who are at low risk of surgical mortality. Qualitative data suggest broader societal benefits are likely and these findings could be used to optimize appropriate intervention selection for this patient population.
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The design of a clinical trial for a rare disease can be challenging. An optimal study design is required to effectively study the clinical outcomes for possible therapies for these types of disorders. Understanding the study participants' experiences as well as barriers and facilitators of participation are important to optimize future research and to inform clinical trial management. Centronuclear myopathies (CNMs) including X-linked myotubular myopathy (XLMTM) are a group of rare congenital myopathies for which there is no cure currently. Since 2014, a number of natural history studies and clinical trials have been conducted in CNMs. Two trials have been prematurely terminated because of severe adverse events. Since no research has been conducted regarding trial experience in CNM, we performed a scoping literature research on clinical trial experience of patients with neuromuscular disorders in general. The most common barriers to trial participation of patients comprise concerns about potential harmful effects, opportunity loss and the expected burden on daily life. The most common facilitators were an expected benefit on the disease course, altruism and collateral benefit. While several results are in line with trial experiences of other types of patients, for example oncological patients, distinctions can be made for patients with CNM and other neuromuscular disorders. However, the limited availability of relevant literature suggests that future (qualitative) research should focus on trial experiences in CNM patients.
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Ensayos Clínicos como Asunto , Miopatías Estructurales Congénitas , Enfermedades Neuromusculares , Enfermedades Raras , Humanos , Miopatías Estructurales Congénitas/terapia , Enfermedades Neuromusculares/terapia , Participación del PacienteRESUMEN
BACKGROUND: Countries around the world are increasingly rethinking the design of their health benefit package to achieve universal health coverage. Countries can periodically revise their packages on the basis of sectoral cost-effectiveness analyses, i.e. by evaluating a broad set of services against a 'doing nothing' scenario using a budget constraint. Alternatively, they can use incremental cost-effectiveness analyses, i.e. to evaluate specific services against current practice using a threshold. In addition, countries may employ hybrid approaches which combines elements of sectoral and incremental cost-effectiveness analysis - a country may e.g. not evaluate the comprehensive set of all services but rather relatively small sets of services targeting a certain condition. However, there is little practical guidance for countries as to which kind of approach they should follow. METHODS: The present study was based on expert consultation. We refined the typology of approaches of cost-effectiveness analysis for benefit package design, identified factors that should be considered in the choice of approach, and developed recommendations. We reached consensus among experts over the course of several review rounds. RESULTS: Sectoral cost-effectiveness analysis is especially suited in contexts with large allocative inefficiencies in current service provision and can, in theory, realize large efficiency gains. However, it may be challenging to implement a comprehensive redesign of the package in practice. Incremental cost-effectiveness analysis is especially relevant in contexts where specific new services may impact the sustainability of the health system. It may potentially support efficiency improvement, but its focus has typically been on new services while existing inefficiencies remain unchallenged. The use of hybrid approach may be a way forward to address the strengths and weaknesses of sectoral and incremental analysis areas. Such analysis may be especially useful to target disease areas with suspected high inefficiencies in service provision, and would then make good use of the available research capacity and be politically rewarding. However, disease-specific analyses bear the risk of not addressing resource allocation inefficiencies across disease areas. CONCLUSIONS: Countries should carefully select their approach of cost-effectiveness analyses for benefit package design, based on their decision-making context.
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OBJECTIVES: Uncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface. METHODS: Six online discussions among WG members (Dec 2021-Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions. RESULTS: The WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: "unavailable," "inaccurate," "conflicting," "not understandable," "random variation," "information," "prediction," "impact," "risk," "relevance," "context," and "judgment." These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed. CONCLUSIONS: The systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.
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Formulación de Políticas , Evaluación de la Tecnología Biomédica , Incertidumbre , Políticas , Costos y Análisis de CostoRESUMEN
Kazakhstan strives to obtain Universal Health Coverage (UHC) by using health technology assessment (HTA) for determining their health benefit package. This paper reports on employing evidence-informed deliberative processes (EDPs), a practical and stepwise approach to enhance legitimate health benefit package design in Kazakhstan. METHODS: The Ministry of Health of Kazakhstan approved the operationalization and application of EDPs during March 2019 and December 2020. We used a combination of desk research, conducting HTA, online surveys as well as a face-to-face workshop in Nur-Sultan, Kazakhstan, and two online workshops to prioritize 25 selected health technologies. During the latter, we tested two alternative approaches to prioritization: quantitative multicriteria decision analysis (MCDA) and the use of decision rules. RESULTS: For each of the HTA reports, evidence summaries were developed according to the decision criteria (safety, social priority disease, severity of disease, effectiveness, cost-effectiveness, level of evidence, financial risk protection and budget impact). When appraising the evidence, the advisory committee preferred using quantitative MCDA, and only when this would result in any controversy could decision rules be applied. CONCLUSIONS: Despite several challenges, including a partial disruption because of the COVID-19 pandemic, implementation of the process will likely play a key role in determining an evidence-informed and transparent health benefit package.
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COVID-19 , Pandemias , COVID-19/epidemiología , Humanos , Kazajstán , Evaluación de la Tecnología Biomédica , Cobertura Universal del Seguro de SaludRESUMEN
The unique task and contribution of health technology assessment (HTA) is to help identify those health technologies and their uses that are most likely to preserve and restore a population's health in a way that is consonant with its values, including, for example, equity and access to high-quality care (1). Such a task is challenging for at least two reasons. First, because of the vast and constantly evolving number and diversity of health technologies and their applications. Second, because of the usual wide variety of competing views within communities and their stakeholders regarding what strategies are likely to be conducive to the goal of preserving and restoring population health. Although perhaps tempting, it would be a grave mistake to hold that the controversies that result from such competing views can be resolved by taking recourse to the facts only. For such controversies are usually fueled by different notions of health and disease and different specifications of values such as equity and individual and collective responsibility for health. For this reason, they cannot be resolved in a satisfactory way without also addressing those normative issues and their interplay with empirical analysis.
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Tecnología Biomédica , Evaluación de la Tecnología BiomédicaRESUMEN
Health technology assessment (HTA) aims, through empirical analysis, to shed light on the value of health technologies (O'Rourke et al. [2020, International Journal of Technology Assessment in Health Care 36, 187-90]). HTA is, then, where facts and values meet. But how, where, and when do facts and values meet in HTA? Currently, HTA is usually portrayed as a sequential process, starting with empirical analysis (assessment), followed by a deliberation on the implications of the findings for a judgment of a health technology's value (appraisal). In this paper, we will argue that in HTA, empirical analysis and normative inquiry are much more closely entwined. In fact, as we hope to show, normative commitments act as an indispensable guide for the collection and interpretation of empirical evidence. Drawing on policy sciences, we will suggest a concrete methodology that can help HTA practitioners to integrate empirical analysis and normative inquiry in a transparent way. The proposed methodology can be conceived as a concrete means for conducting a scoping exercise in HTA. Moreover, it offers a distinct way of giving stakeholders a structural and constructive role in HTA. This paper outlines the approach developed by the values in doing assessments of health technologies project, a project funded by the Erasmus+ program (contract number 2018-1-NL01-KA203-038960), which is the European Union's program to support education, training, youth, and sport in Europe. The project has resulted in an E-learning course, an accompanying handbook, and a consensus statement, all freely available from the project's website www.validatehta.eu.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Europa (Continente) , Evaluación de la Tecnología Biomédica/métodosRESUMEN
OBJECTIVES: Deliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR-The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap. METHODS: The joint Task Force consisted of fifteen members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to two rounds of peer review. RESULTS: A deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation. CONCLUSIONS: The guidance and the checklist contain a series of questions, grouped by six phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Comités ConsultivosRESUMEN
OBJECTIVES: Deliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR-The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap. METHODS: The joint Task Force consisted of 15 members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to 2 rounds of peer review. RESULTS: A deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation. CONCLUSIONS: The guidance and the checklist contain a series of questions, grouped by 6 phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Comités Consultivos , Lista de Verificación , Economía Médica , Humanos , Evaluación de la Tecnología Biomédica/métodosRESUMEN
OBJECTIVES: To conduct a formative evaluation of applying the VALIDATE approach in practice by (i) assessing how students appreciated the e-learning course, (ii) exploring how, for what purposes and with what outcomes the acquired VALIDATE competences subsequently were used in internships in different institutional contexts, and how this was shaped by these contexts, and (iii) what this shows on real-world use of VALIDATE. METHODS: Comparative discussion of experiences of applying the VALIDATE approach via a semistructured survey and oral feedback from e-course students; final reports on internships in health technology assessment (HTA) practice, followed by semistructured interviews with interns and supervisors to complement and interpret results. RESULTS: All students considered the VALIDATE approach an enlightening and important addition to current HTA knowledge, especially regarding understanding the relation between empirical analysis and normative inquiry, identifying policy relevant questions and using the method of reconstructing interpretive frames for scoping. The latter appeared intellectually challenging and requiring some prior HTA knowledge. The use the VALIDATE approach in practice shows that interns productively redefined the HTA problem, based on appreciation of different stakeholders' definition of the issue; they experienced constraints from retrieving all relevant perspectives from existing literature as well as from institutional rules and routines. CONCLUSIONS: Some challenges in applying the VALIDATE approach deserve attention for its future use: currently used research approaches often assume a problem as "given"; and the data needed on different perspectives is often not reported in scientific literature. Finally, data gathering on and evaluation of value dimensions was experienced as challenging.
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Tecnología Biomédica , Evaluación de la Tecnología Biomédica , Humanos , Conocimiento , Proyectos de Investigación , Evaluación de la Tecnología Biomédica/métodosRESUMEN
Prioritization of COVID-19 vaccines is one of the most relevant topics in the current pandemic emergency. Prioritization decisions are political decisions that are value-laden, and as such of ethical nature. Despite the clear political and ethical nature of this topic, prioritization decisions are often interpreted and presented as scientific decisions. The aim of this article is twofold. First, we aim to show critical points that characterize certain pandemic vaccination plans from the ethical viewpoint using four dimensions (problem definitions, incorporation of different perspectives, context, and specification). The four dimensions were drawn from findings of the European project "VALIDATE" (VALues In Doing Assessments of healthcare TEchnologies", https://validatehta.eu). Second, we aim to reframe the issue about prioritization itself in the light of the four dimensions mentioned. Our conclusion is that policy-problem definitions, incorporation of different perspectives, contextual considerations and specification of moral principles seem to be common critical points of some vaccination plan documents. The European project "VALIDATE" seems to be able to provide a useful and profitable approach to address many of these critical points.
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Vacunas contra la COVID-19 , COVID-19 , COVID-19/prevención & control , Humanos , Pandemias/prevención & control , VacunaciónRESUMEN
BACKGROUND: Countries around the world are increasingly rethinking the design of their health benefit packages to achieve universal health coverage (UHC). Health technology assessment (HTA) bodies support governments in these decisions, but employ value frameworks that do not sufficiently account for the intrinsically complex and value-laden political reality of benefit package design. METHODS: Several years ago, evidence-informed deliberative processes (EDPs) were developed to address this issue. An EDP is a practical and stepwise approach for HTA bodies to enhance legitimate health benefit package design based on deliberation between stakeholders to identify, reflect and learn about the meaning and importance of values, and to interpret available evidence on these values. We further developed the conceptual framework and initial 2019 guidance based on academic knowledge exchange, analysing practices of HTA bodies, surveying HTA bodies and experts around the globe, and implementation of EDPs in several countries around the world. RESULTS: EDPs stem from the general concept of legitimacy, which is translated into four elements - stakeholder involvement ideally operationalised through stakeholder participation with deliberation; evidence-informed evaluation; transparency; and appeal. The 2021 practical guidance distinguishes six practical steps of a HTA process and provides recommendations on how these elements can be implemented in each of these steps. CONCLUSION: There is an increased attention for legitimacy, deliberative processes for HTA and health benefit package design, but the development of theories and methods for such processes remain behind. The added value of EDPs lies in the operationalisation of the general concept of legitimacy into practical guidance for HTA bodies.