Health technology assessment (HTA) has become an essential tool for supporting evidence-based decision making in health care systems worldwide. Comprehensive HTA reports, however, are resource-intensive and time-consuming, making them impractical for many decisions that need to be made at the local or institutional level. This gap has led to the development of more pragmatic approaches that aim to balance methodological rigor with feasibility. In Norway, this challenge was addressed through the introduction of mini-HTA, which is a structured, simplified, and context-specific decision-support tool to inform local or institutional decisions about introducing new technologies, procedures, or organizational innovations. Mini-HTA provides a standardized framework for evaluating clinical effectiveness, safety, organizational impact, ethical considerations, and economic consequences while remaining feasible for clinicians and managers to apply in practice. Several key insights emerge from our experience with developing and implementing mini-HTA in Norway. First, adapting HTA approaches to local decision-making contexts increases feasibility and uptake, particularly where time and resources are limited. Second, governance structures, including clear allocation of responsibilities and leadership engagement, are crucial for the consistent use of mini-HTA in practice. Third, the Norwegian experience illustrates how a shared but flexible tool can be applied across different levels of care, including both hospitals and municipalities, while still allowing for contextual adaptation. Finally, the use of mini-HTA as a platform for collaboration across sectors highlights its potential not only as a decision-support tool, but also as a mechanism for coordinating priorities and responsibilities across organizational boundaries.
The approach of Health Technology Assessment, which developed from the Evidence-Based Medicine movement of the 1990s, has proved its value in appraising the value of pharma products but presents methodological challenges when applied to medical devices and digital technologies, whose importance has increased enormously during the last 30 years. The concept of a single assessment, which can summarize relative effectiveness and cost-effectiveness, has been further challenged by the rapidly rising importance of software-driven devices, and latterly Artificial Intelligence. The need to adopt a broader evaluation approach based on product life cycles has been recognized by both regulators and HTA bodies, but practical frameworks for this have not yet been widely adopted. We describe a Life-Cycle-Based Evaluation (LCBE) approach to the assessment of Digital Health Technologies, developed as part of the ASSESS-DHT Horizon Europe 2020 project, EU project 101137347. The LCBE framework is currently being evaluated in pilot studies and aims to support early, proportionate, and continuous assessment and foster collaboration among developers, regulators, and HTA bodies to ensure timely, rigorous, and adaptive evaluations across the technology life cycle.
OBJECTIVES:The use of surrogate endpoints poses challenges to health technology assessment (HTA) agencies and payers when informing the clinical and cost-effectiveness of health technologies. Decision makers often need to make recommendations in the absence of well-established evidence on their validation, particularly when the surrogate endpoint is novel without clear links to the longer-term outcome. This study explored the guidance needs of HTA agencies and identified how they have considered and dealt with surrogate endpoints in cost-effectiveness analysis. METHODS:A qualitative study was used to explore the professional experiences of international HTA staff and their collaborators. Data were collected using three focus groups with 29 participants representing 20 HTA agencies or associated organizations. Framework analysis was used to identify themes from the transcripts. RESULTS:HTA agencies frequently see submissions featuring surrogate endpoints. Issues they face include definitional concerns, absent or weak evidentiary links, and resource-intensive validation methods. Versatile guidance that is mindful of these challenges, such as those faced with rare diseases, is required. This would help to clarify expectations and help ascertain the required information from industry for economic modeling, on how to present the uncertainty arising from surrogate endpoints, and how to appropriately report the findings. CONCLUSIONS:Decision making using surrogate endpoints is a frequent and challenging problem faced by HTA agencies. These results provide the first-hand experiences and reflect the needs of HTA agencies globally. The resulting recommendations and considerations for the use of surrogate endpoints in economic modeling should benefit HTA agencies moving forward.
OBJECTIVES:This systematic review explores which intersectoral costs and benefits (ICBs) are considered in the economic evaluation of pneumococcal conjugate vaccines (PCVs) across different age groups and the related impacts on the results of economic evaluations. METHODS:Seven databases were searched from 2009 to 2024 for full economic evaluations (Ees) of PCVs from a societal perspective. ICBs were presented narratively and in tabular form. Studies were appraised on quality, and PRISMA guidelines were followed. RESULTS:In all, 57 studies were included. ICBs were captured in the following sectors: patient and family, paid labor (productivity), nonpaid productivity, other sectors (education, leisure, consumption), unspecified sectors for ecological effects (herd immunity, serotype replacement, cross-protection), and equity. In cases where the comparator was no vaccination, 10 studies reported that PCVs were dominant when ICBs were included. After excluding dominant analyses, the median QALY-based incremental cost-effectiveness ratio (ICER) reduction ranged from -3.25 percent (for patient/family costs and productivity loss) to -46.02 percent (in children with ecological effects). Among DALY-based studies, the inclusion of ecological effects reduced the median ICER by -22.97 percent. In head-to-head comparisons, 16 studies reported that PCVs were dominant when ICBs were included. CONCLUSION:Inclusion of ICBs, except for serotype replacement, showed favorable cost-effectiveness results. This study provides a framework for identifying the intersectoral costs and benefits for the health economic evaluation of PCVs in children and adults.
Abstract Introduction Several Health Technology Assessment (HTA) agencies have shown an interest in the environmental impacts of drugs, technologies, and health services, and some have begun work in this area. However, few agencies have published a strategy for integrating environmental considerations into their core activities. Defining a strategy matters since it makes explicit where an organization aims to go and how it intends to get there. To help bridge the gap, this article aims to describe the strategy developed by the Institut national d’excellence en santé et services sociaux (INESSS) (Québec, Canada), its implementation process, and the lessons learned. Methods The strategy was informed by the scientific and grey literature and consolidated through a consultation process with multiple stakeholders, including experts in environmental sciences, HTA agencies, patients, caregivers, citizens, clinicians, sustainable development officers, and health and social care civil servants. Results The strategy articulates the means to meet three operational objectives: (a) knowing when to integrate environmental considerations into HTA knowledge products; (b) knowing which environmental considerations to integrate; and (c) knowing how to integrate these considerations into a growing number of knowledge products and improve over time. Conclusions Navigating through the novelty and uncertainty associated with environmental sustainability requires scientific humility as well as an organizational ability to learn and continuously improve. The tools and implementation process described in this paper could inspire and benefit other HTA agencies interested in advancing the inclusion of environmental considerations into their core activities.
Title/abstract and full-text screening are among the most time-consuming stages of systematic reviews. Large language models (LLMs) such as ChatGPT can assist in screening, but most prior evaluations focus only on abstract-level decisions and require coding or API access, limiting practical application. This study aimed to develop and evaluate a structured, prompt-based approach that enables LLMs to perform both abstract and full-text screening without programming or API integration. Using datasets from two completed meta-analyses, we implemented a stepwise training framework involving comprehension checks, criterion-specific feedback, and iterative prompt refinement. Study 1 optimized technical parameters, including file format and batch size, using 1,000 abstracts and 50 full texts from a completed meta-analysis. Study 2 validated the approach in an independent dataset on teletherapy for depression (1,321 abstracts, 82 full texts). Human reviewers’ decisions served as the reference standard, and sensitivity, specificity, and accuracy were the primary outcomes. In Study 1, the LLM achieved 98.0% sensitivity and 80.6% specificity at abstract screening, with optimal performance using batches of 50 abstracts in plain-text format. In Study 2, sensitivity reached 100% and specificity 85.6% for abstracts, and 82.1% accuracy for full-text screening, correctly retaining all eligible studies. A structured, prompt-based approach allows LLMs to approximate human-level accuracy in both abstract and full-text screening, with high sensitivity and specificity. This method makes LLM-assisted screening more accessible to review teams. While human oversight remains essential to address false positives and ensure rigor, prompt-based LLM workflows can substantially reduce reviewer burden and accelerate evidence synthesis.
OBJECTIVES:Economic evaluations adopting societal perspectives often include productivity costs, yet these remain inconsistently measured and valued. We aimed to identify existing measurement instruments, valuation methods, and guidance for incorporating productivity costs in economic evaluations. METHODS:We conducted a scoping review of peer-reviewed literature (MEDLINE, Embase, EconLit; 2019-2025) to map methods for measuring and valuing productivity costs, and an environmental scan of grey literature to identify complementary policy and methodological guidance from Health Technology Assessment organizations. Two reviewers independently screened records and extracted data on study characteristics, instruments, valuation methods, and recommendations. We synthesized findings descriptively and thematically. RESULTS:We screened 3,971 records and included 40 studies: 29 on measurement instruments and 11 on valuation methods. Among twenty-five instruments, twenty-three measured productivity loss for patients, two for caregivers; thirteen measured presenteeism only, four captured presenteeism and absenteeism, and eight measured presenteeism, absenteeism, and unpaid work; three were suitable for valuation using the human capital approach (HCA) and friction cost approach (FCA). Studies highlighted limitations, including HCA's tendency to overestimate and FCA's reliance on scarce friction period data. We also identified ten guidance documents: several jurisdictions favored FCA to value paid labor (n = 4) and opportunity cost for unpaid labor (n = 3). CONCLUSIONS:There are many productivity cost instruments, but only few are suitable for valuation. Valuation remains contested, and guidance is fragmented: HCA is simple but risks overestimation, whereas FCA better reflects labor markets but requires data on friction periods. Future work should prioritize valuation-ready tools, improved friction period estimation, and harmonized guidance.
OBJECTIVES:Pathway models incorporate multiple decision nodes to assess the most cost-effective sequence or the optimal point of introduction of a new technology within a treatment pathway. Pathway models are particularly useful in disease areas such as oncology, where patients may have several lines of therapy. We aimed to review methodologies for modeling and evidence synthesis in pathway models that evaluate the cost-effectiveness of treatment strategies within oncology. METHODS:We designed a search to identify relevant methodological papers and systematic reviews of oncology pathway models that critique their methodological approaches. We also updated a previous review of studies on methods for evidence synthesis to inform pathway models. Best practice recommendations were extracted and summarized. RESULTS:Nine and five studies were included on methods for model structures and evidence synthesis, respectively. Key themes related to data requirements, including a preference for patient-level model structures and data from multi-line sources. There was limited guidance on alternative model structures in the absence of patient-level data. Multi-state network meta-analysis with flexible survival models was the most appropriate method identified for evidence synthesis; however, due to data limitations, it may be necessary to conduct separate syntheses at each line of therapy. CONCLUSIONS:Data limitations may reduce the potential benefits of pathway models. Further method development is needed for pathway models in decision spaces where individual patient data and sources that cover multiple lines of treatment are not available.
Objectives This study aims to investigate the awareness, practices, needs, and challenges of hospital-based health technology assessment (HB-HTA) in China's tertiary public hospitals, drawing on perspectives from both hospital management and clinical practice.Methods Semi-structured interviews were conducted with seventeen participants representing six major tertiary public hospitals in China. Respondents included vice presidents, clinical department directors, and heads of pharmacy. The questionnaire explored four dimensions consistent with China's national performance appraisal framework: medical quality, operational efficiency, sustainable development, and satisfaction. Data were analyzed using NVivo 14 for qualitative responses and Excel 2021 for quantitative metrics.Results The findings revealed that 82.35 percent (14/17) of hospitals lacked dedicated HB-HTA teams, with decisions made primarily by coordination between Medical Affairs Offices (28.89 percent, 13/45) and Pharmacy Departments (22.22 percent, 10/45). Safety (28.26 percent, 13/46), effectiveness (23.91 percent, 11/46), and cost-effectiveness (19.57 percent, 9/46) were prioritized criteria, yet only 29.41 percent (5/17) of institutions used formal HB-HTA tools. Divergence persisted between administrators' organizational priorities and clinicians' patient-centered perspectives. Despite barriers reported by 62.8 percent (27/43) of respondents' institutions, 88.24 percent (15/17) expressed an urgent or recognized need for HB-HTA implementation.Conclusions For effective HB-HTA implementation in China, addressing capacity deficits and reconciling stakeholder perspectives is essential. A project-based strategy, supported by government real-world data platforms and interdisciplinary teams, is recommended for contextually-appropriate evaluation frameworks.
OBJECTIVES:Health technology assessment (HTA) is a multidisciplinary, policy-oriented process designed to inform evidence-based, transparent, and value-based decision making in health systems. Across the Middle East and North Africa (MENA), HTA development remains heterogeneous and unevenly institutionalized. This manuscript reports on a Health Technology Assessment international (HTAi)-led Policy Dialogue designed to identify shared challenges, priorities, and opportunities to strengthen HTA systems across the region. METHODS:Insights were generated through an in-person meeting held in Tunisia in 2025, alongside the HTAi MENA Regional Meeting. The dialogue was complemented by an online survey and a follow-up virtual meeting. Data sources included structured survey responses from experts across twelve countries and qualitative inputs from multi-stakeholder discussions involving policymakers, HTA practitioners, academics, and international organization representatives. Findings were synthesized descriptively to capture perceived challenges, priorities, and expectations for regional and international collaboration. RESULTS:Participants emphasized HTA's role in improving resource allocation, transparency, and access to high-value technologies. Key challenges included limited access to high-quality data, workforce capacity constraints, fragmented governance, and weak legal and institutional frameworks. The highest-ranked priorities were establishing formal legal mandates for all actors involved in HTA processes, building technical and system capacity (including institutional governance, processes, and coordination mechanisms), and strengthening health data infrastructure. Strong support emerged for regional cooperation through joint training, shared assessments, and HTA networking, alongside expectations for international organizations to play a catalytic role. CONCLUSIONS:HTA is gaining prominence across MENA, but most countries remain in early phases of institutional development. Progress will require coordinated action on governance, capacity, data systems, and implementation.
One of the ways in which healthcare's climate impact can be reduced is by taking greenhouse gas emissions into account when deciding which treatments are reimbursed and/or included in clinical guidelines. In a report for the Minister of Health, Welfare, and Sports in the Netherlands, we explored how this idea could be put into practice. We developed a set of two criteria: greenhouse gas emissions per patient (which can be compared to, for example, QALYs gained), and total amount of greenhouse gas emissions for all patients undergoing the intervention (which can be compared to, e.g., healthcare's remaining carbon budget). In the coming three years, the National Health Care Institute will use these criteria in an experimental setting, to gain practical experience and to assess whether there is sufficient support among policy makers, practitioners, and patients for taking into account climate impact regularly.
Early health technology assessment (HTA) has recently been defined as a process for evaluating the "potential value" of technologies at early development stages. This analysis examines how value is characterized in early stages compared to standard HTA. Using the MAPS framework (Methods, Attributes, Procedures, and Social Preferences), the findings indicate that a broader value framework may be applicable to early HTA relative to standard HTA. This framework includes systematic consideration of attributes beyond health-related benefits, such as real option value and innovation, as well as additional methods like fiscal impact analysis and social return on investment. Additionally, there is potential for less variation in early HTA processes globally, given that these assessments are less tied to local funding decisions. Finally, the role of social preferences in early HTA may also involve considering trade-offs and impact modifiers that are particularly relevant in initial stages, and which deserve further research.
Objectives The role of health technology assessment (HTA) is evolving. Once seen primarily as a gatekeeper, HTA is increasingly viewed as a potential shaper of health system transformation. The first plenary session of the HTA International 2025 Annual Meeting explored the potential for HTA to adopt this broader role.Methods Drawing on perspectives from across the globe, it was discussed how HTA can help shape more equitable, responsive, and sustainable health systems by informing benefit package design, supporting universal health coverage, and addressing systemic inequities from a societal perspective.Results Audience polling and live commentary underscored a shared sense of urgency, as well as recognition of the methodological, political, and practical constraints to this evolution. Rather than a binary choice, a continuum of roles was suggested depending on the context in which HTA is applied.Conclusions Ultimately, HTA can shape health systems - not through ambition alone, but through evidence-informed, inclusive, and context-aware decision-making.
Objectives The European Union health technology assessment (HTA) regulation increases pressure on European HTA organizations' capacity and new domains of expertise continue to emerge quickly in the HTA field. Therefore, this study aimed to identify emerging competencies described in the literature and to update and verify an existing HTA competency framework, encompassing both process-related and domain-specific expertise for conducting HTA.Methods Using a scoping review approach, PubMed, Embase, PsycINFO, ERIC, CINAHL, and Web of Science were searched from 1 January 2018 until 13 May 2024. The websites of HTA-related societies were searched on 22 April 2024. Documents describing competencies for HTA professionals were included. Deductive content analysis of the identified competencies was performed, utilizing the competencies from the original framework.Results A total of nineteen documents were included, consisting of six competency frameworks, one handbook, and twelve scientific articles. The identified competencies from these documents mapped appropriately to the original competency framework. New competencies within existing domains that emerged were related to organizational sustainability, patient and public involvement, real-world data, interpreting and developing HTA reports, and stakeholder identification. In addition, three new competency domains were identified, which were artificial intelligence use for HTA, environmental aspects, and performance and achievements.Conclusions The newly identified competencies and competency domains highlight emerging areas of expertise for HTA professionals and need to be added to the original competency framework. An updated competency framework can support capacity building efforts and the development of training programs to ensure that HTA professionals are well-equipped to address current healthcare challenges.
Health technology assessment (HTA) has long been the cornerstone of evidence-informed policy for healthcare decision-making. While randomized controlled trials (RCTs) remain fundamental, they do not always reflect the realities of everyday clinical practice. Real-world evidence (RWE) is increasingly recognized as an important complement, capable of capturing variations in population, setting, and practice that influence health outcomes. In Malaysia and across ASEAN, the growing focus on RWE reflects both global momentum and regional aspirations to strengthen data-driven policymaking. This perspective reflects on Malaysia's experience in integrating RWE into HTA and considers how this evolution can inform regional efforts. It argues that the adoption of RWE signifies not only a methodological advance but a transformation in how evidence is conceptualized, governed, and applied. Developing robust data systems, analytical expertise, and cross-country collaboration will be essential to ensure that real-world data are translated into meaningful evidence for equitable and sustainable healthcare decisions.
The effect of Early Technology Review (ETR) through early engagement with multiple stakeholders on strategic development for technologies at prototype development and proof of concept was examined through two generic case studies of relevant outcomes. In both examples, advice to companies could have significantly changed strategic direction to become more relevant to payers and clinical experts. In one instance, the advice was followed and resulted in an expedited first-in-human study and was considered for a second ETR to inform the proof-of-concept study. In the second example, it was reported that changes in strategic direction were being considered.These reports provide descriptive accounts of very early applications of the ETR process that now spans the entire preclinical trajectory. Had the second case study at proof of concept been able to benefit from this approach at the point of prototype development, it could have avoided the costs and research through earlier advice. This begs the question whether a sequential iterative approach to evidentiary multiple stakeholder advice across the technology life cycle may reduce risk and cost while benefitting from efficiencies of applying adaptive design.
