International Journal of Technology Assessment in Health Care最新文献

{"title":"Mapping policy instruments and challenges in health innovation development: implications for the role of early HTA - an umbrella review.","authors":"Minji Hong, Langming Mou, Chee Ern Har, Yi Wang","doi":"10.1017/S0266462326104243","DOIUrl":"https://doi.org/10.1017/S0266462326104243","url":null,"abstract":"<p><strong>Objectives: </strong>Health innovation encounters challenges across the innovation lifecycle. Although various policy instruments aim to support innovation, their alignment with stage-specific challenges remains unclear. This study synthesizes evidence to examine how innovation challenges and policy responses are distributed across the health innovation lifecycle, with particular attention to the role of early Health Technology Assessment (early HTA).</p><p><strong>Methods: </strong>We conducted an umbrella review of review-level studies that examined health innovation challenges and related policy instruments. A comprehensive literature search was performed across five databases, including PubMed, Web of Science, Embase, Scopus, and CINAHL. Twenty-four eligible studies were included and synthesized using a well-established analytical framework. Innovation challenges and policy instruments were mapped across stages of the development pathway and linked to the contributions of early HTA.</p><p><strong>Results: </strong>Innovation challenges were most pronounced in product design and clinical trial stages. Institutional barriers, including lengthy approval processes, became more prominent during regulatory approval and market access. Eighty policy responses were identified and clustered into economic, regulatory, and informational instruments. Economic measures were concentrated in product design and clinical trial stages, while regulatory instruments focused on regulatory approval and market access. Early HTA emerged as a complementary decision-support approach that may help address product design and clinical trial stages challenges.</p><p><strong>Conclusions: </strong>There is a clear misalignment between where innovation challenges arise and where policy responses are applied. Integrating early HTA more systematically into innovation policy frameworks could help address challenges in product design and clinical trial stages and improve the likelihood of successful innovation adoption.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e90"},"PeriodicalIF":3.0,"publicationDate":"2026-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149227869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Authors' response to \"Rethinking Slovenia's health technology assessment system\".","authors":"Katarina Beravs Bervar, Wim Goettsch, Iñaki Gutierrez-Ibarluzea, Adam Skali, Alric Rűther, Iga Lipska, Eva Turk","doi":"10.1017/S0266462326104164","DOIUrl":"https://doi.org/10.1017/S0266462326104164","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e94"},"PeriodicalIF":3.0,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Making trade-offs explicit: development and pilot of a Belgian MCDA framework for orphan drug appraisal.","authors":"Alessandra Blonda, Daniel A Ollendorf, Isabelle Huys, Yvonne Denier, Steven Simoens","doi":"10.1017/S0266462326104127","DOIUrl":"https://doi.org/10.1017/S0266462326104127","url":null,"abstract":"<p><strong>Objectives: </strong>Orphan drug reimbursement remains a contested area in health technology assessment (HTA), as decision makers must balance limited evidence, high unmet need, patient-relevant value, and affordability. Multi-criteria decision analysis (MCDA) has been proposed to make such trade-offs more explicit and transparent, yet empirical applications in European national HTA settings remain scarce.</p><p><strong>Methods: </strong>We developed and piloted an MCDA framework for orphan drug reimbursement in Belgium. Candidate criteria were identified through a systematic literature review. A multidisciplinary expert panel (comprising representatives from the national reimbursement agency, sickness funds, patient organizations, pharmaceutical companies, clinicians, and bioethicists) participated in four focus group discussions to select and define criteria. Criterion weights were elicited through a three-round Delphi process. The framework was piloted on four orphan drugs previously appraised in Belgium: ponatinib, pirfenidone, onasemnogene abeparvovec, and metreleptin.</p><p><strong>Results: </strong>Seven quantitative and three qualitative contextual criteria were selected. Unmet medical need received the highest weight, followed by comparative effectiveness and impact on patient-reported outcomes. Onasemnogene abeparvovec achieved the highest overall MCDA score (0.77), followed by metreleptin (0.65), pirfenidone (0.47), and ponatinib (0.39). The pilot also generated policy-relevant insights on criterion design, handling residual uncertainty, separating value assessment from reimbursement feasibility (through cost criteria), and using scores to support-rather than replace-deliberation.</p><p><strong>Conclusion: </strong>This study presents the first MCDA framework for orphan drug reimbursement in Belgium and illustrates how criterion weights and scores can structure committee deliberation, complemented by contextual appraisal, to support more transparent, consistent, and accountable decision making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e96"},"PeriodicalIF":3.0,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dialogue on the use of artificial intelligence in health technology assessment: building a community of practice, transparency, and trust.","authors":"Eon Ting, Nicole Mittmann, Ghayath Janoudi, Joanne Kim","doi":"10.1017/S0266462326104292","DOIUrl":"https://doi.org/10.1017/S0266462326104292","url":null,"abstract":"<p><p>The use of artificial intelligence (AI) in health technology assessment (HTA) is rapidly emerging and evolving worldwide (1;2). While many may tout the potential of AI to enhance efficiency, accuracy, and speed of HTA, these technologies also present risks, concerns, and practical challenges that may limit responsible, ethical, and effective deployment. The 2025 HTAi Global Policy Forum (GPF) brought forward key themes regarding the use of AI in HTA, such as trust, human agency, and risk-based approaches (3). Others have also discussed the policy considerations of AI in HTA (4;5).</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e95"},"PeriodicalIF":3.0,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220347","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Strengthening reporting of patient and public involvement in health technology assessment.","authors":"Ferrán Catalá-López, Begonya Nafria Escalera, Laura Tejedor-Romero, Ernesto Ganuza, David Moher","doi":"10.1017/S0266462326104280","DOIUrl":"https://doi.org/10.1017/S0266462326104280","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e93"},"PeriodicalIF":3.0,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149193739","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparing harmonization in medicines regulation and health technology assessment in Europe: lessons for the EU HTA regulation.","authors":"Francine Brinkhuis, Aukje K Mantel-Teeuwisse, Hubert G M Leufkens, Kirti Narsai, Wim G Goettsch, Hendrika A van den Ham","doi":"10.1017/S0266462326104267","DOIUrl":"https://doi.org/10.1017/S0266462326104267","url":null,"abstract":"<p><strong>Objectives: </strong>The European Union (EU) has long pursued harmonization in medicines regulation and, more recently, in health technology assessment (HTA). The European Medicines Agency (EMA) and the EU HTA Regulation (EU HTAR) represent key milestones in these respective trajectories. This study compares the harmonization trajectories of EU medicines regulation and EU HTA to identify lessons relevant for EU HTAR implementation.</p><p><strong>Methods: </strong>We used a multi-method qualitative approach combining a narrative literature review with online focus groups involving eighteen regulatory and policy experts. The review explored the historical development of harmonization in both policy areas. The focus-group analysis examined perspectives on collaboration, harmonization dynamics, and implementation.</p><p><strong>Results: </strong>Both policy areas moved from national arrangements and voluntary cooperation toward more formal EU-level structures. Medicines regulation developed earlier and resulted in more centralized procedures and binding EU-wide authorization decisions. EU HTA retains greater national responsibility for value judgments, pricing, and reimbursement. The two areas also differ in the scope and standardization of assessments. In both trajectories, harmonization was shaped by trust, willingness to collaborate, capacity differences, and legal and procedural contexts. Participants emphasized the importance of stepwise implementation, work sharing, and sustained dialogue, while anticipating variation in national JCA uptake and impact.</p><p><strong>Conclusions: </strong>Experience from EU medicines regulation highlights the importance of building trust and adapting collaboration to differences in national maturity and priorities. Harmonization should develop gradually through learning and refinement across diverse legal and procedural contexts. These lessons may support EU HTAR implementation and further development.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e91"},"PeriodicalIF":3.0,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149181596","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Restricted mean survival time ratio in oncology cost-effectiveness analysis: stability and data-driven support for treatment-effect waning modeling and survival extrapolation.","authors":"Mingye Zhao, Hanqiao Shao, Taihang Shao, Yunong Jiang, Yunlin Jiang, Yuan Li, Hongshu Fang, Wenxi Tang","doi":"10.1017/S026646232610422X","DOIUrl":"https://doi.org/10.1017/S026646232610422X","url":null,"abstract":"<p><strong>Objectives: </strong>We examined long-term treatment-effect patterns relevant to treatment effect waning (TEW) modeling in oncology health technology assessment (HTA), compared the dispersion and update stability of hazard ratios (HRs) and restricted mean survival time ratios (RMSTRs), and developed prediction models to support extrapolation model selection and TEW modeling.</p><p><strong>Methods: </strong>Using the FDA Hematology/Oncology Approvals Database, we identified phase II/III randomized controlled trials (2006-2025) with updates. We included overall survival (OS) and other time-to-event end points (non-OS). We conducted 2/3/4/5-year landmark analyses to estimate post-landmark HRs and RMSTRs, summarized by event-weighted medians and dispersion (coefficient of quartile relative dispersion, CQR). Update-related change was quantified using updated-to-initial ratios. We predicted longer-term RMSTR using elastic-net regression with nested cross-validation and split-conformal 90 percent prediction intervals.</p><p><strong>Results: </strong>We analyzed 168 trials (270 contrasts; 150 OS, 120 non-OS). Across landmark analyses at 2, 3, 4, and 5 years, RMSTR was consistently less dispersed than HR for both OS and non-OS end points, while post-landmark HRs generally remained < 1. Update ratios were more stable for RMSTR than HR. Initial and updated RMSTR were strongly correlated (OS r = .87; non-OS r = .82). Parsimonious regularized models accurately predicted updated RMSTR, with conformal prediction intervals achieving 91 percent and 96 percent coverage for OS and non-OS.</p><p><strong>Conclusions: </strong>Reliance on current HR-based TEW modeling practices alone may be insufficient to fully characterize long-term treatment-effect uncertainty. RMSTR was consistently less dispersed than HR and predictable from early trial data, providing an anchor for extrapolation model selection and TEW uncertainty analysis in oncology economic evaluations.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e89"},"PeriodicalIF":3.0,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149181637","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reconsidering the classification of health technology assessment status in Iran: a comment on \"Policy dialogue on health technology assessment in Middle East and North Africa\".","authors":"Masoud Behzadifar","doi":"10.1017/S0266462326104279","DOIUrl":"https://doi.org/10.1017/S0266462326104279","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e92"},"PeriodicalIF":3.0,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149181566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The societal perspective in HTA: why it should matter.","authors":"Linda Mundy, Guy Maddern","doi":"10.1017/S0266462326104255","DOIUrl":"https://doi.org/10.1017/S0266462326104255","url":null,"abstract":"<p><p>Traditional health technology assessments (HTAs) primarily focus on safety, clinical effectiveness, and economic impact, typically through cost-effectiveness analyses conducted from the healthcare payer perspective using metrics such as cost per quality-adjusted life year. This approach overlooks the broader societal impacts of new health interventions and may lead to under- or overestimation of their overall value. Broadening the value framework to include the societal perspective would allow for the consideration of direct and indirect costs beyond health system costs, including lost productivity, caregiver impact, as well as the effect on other sectors such as education or welfare. Despite many HTA guidelines recommending its inclusion, few countries provide guidance on valuing non-health sector impacts or mandating the use of the societal perspective in the reference case. Although there are many challenges associated with the integration of the societal perspective into HTA evaluations, the lack of an internationally standardized context-specific framework remains the major barrier. Regional collaboration between HTA agencies to develop a societal perspective framework, build technical capacity, as well as share methodologies and data may address some challenges. Pilot studies should be encouraged to test the inclusion of societal elements, alongside the routine conduct of sensitivity analyses for evaluations undertaken from both healthcare sector and societal perspectives. This would enable policy makers to address uncertainty without compromising the core economic analysis. A broader, more flexible value framework may better align HTA with societal needs and values, and reward industry innovation while informing equitable funding decisions, capturing the full value of health technologies.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e87"},"PeriodicalIF":3.0,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149166528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Barriers and facilitators for conducting and implementing health technology assessment in Jordan: researchers and decision-makers' perspectives.","authors":"Rimal Hamdan Mousa, Samar Melhem, Shiekha S AlAujan, Saja Almazrou, Emad Almomani, Abeer Al Rabayah, Eman A Hammad, Rasha M Arabyat, Ibrahim Alabbadi, Abdallah Y Naser","doi":"10.1017/S0266462326104231","DOIUrl":"https://doi.org/10.1017/S0266462326104231","url":null,"abstract":"<p><strong>Objectives: </strong>Health technology assessment (HTA) is a multidisciplinary approach that evaluates the clinical data and cost-effectiveness of health technologies within broader policy and societal contexts. This study aimed to identify barriers and facilitators to conducting HTA and implementing its findings in Jordan and to determine key technical and context-specific priorities to enhance HTA use.</p><p><strong>Methods: </strong>A cross-sectional study was conducted using an online, self-administered questionnaire distributed between 1 June and 29 July 2024. Participants were purposively sampled and invited via email. The questionnaire's face and content validity were assessed by two local experts. Responses from researchers and decision-makers were analyzed descriptively.</p><p><strong>Results: </strong>A total of fifty-one participants were included. Both groups identified \"lack of routinely collected local effectiveness data and national health statistics\" and \"absence of health state preference values for estimating quality-adjusted life-years (QALYs)\" as major barriers to conducting HTA. The top-rated facilitator was \"the development of a good-quality national real-world database.\" Researchers additionally highlighted \"financial incentives for particular interventions\" as an important barrier, whereas decision-makers emphasized \"the lack of formal willingness-to-pay thresholds\" as the primary barrier to HTA implementation. Both groups identified \"prioritization of economic evaluations targeting high-burden conditions\" as the most important facilitator for implementation.</p><p><strong>Conclusions: </strong>Significant systemic and methodological barriers hinder HTA conduct and implementation in Jordan - including limited local data, a lack of health state preference values, and the absence of explicit willingness-to-pay thresholds. Strengthening national data infrastructure, establishing clear policy frameworks, and prioritizing economic evaluations are critical for enhancing HTA integration and supporting evidence-informed decision-making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"42 1","pages":"e86"},"PeriodicalIF":3.0,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149080608","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}