International Journal of Technology Assessment in Health Care最新文献

{"title":"Redefining value assessment and healthcare funding priorities for medicines: the journey to patient-centric decision making in APAC - a systematic literature review.","authors":"Durhane Wong-Rieger, I-Ching Tsai, Jin Yu Tan, David Bin-Chia Wu, DaeYoung Yu, Alison Keetley, Alex Best, Ritu Jain","doi":"10.1017/S0266462325000224","DOIUrl":"https://doi.org/10.1017/S0266462325000224","url":null,"abstract":"<p><strong>Objectives: </strong>This study explored patient involvement in healthcare decision-making in the Asia Pacific region (APAC) by identifying roles and factors influencing differences between healthcare systems. Proposed recommendations to enhance patient engagement were made.</p><p><strong>Methods: </strong>This systematic literature review was conducted using studies from Australia, China, Japan, Malaysia, New Zealand, the Philippines, South Korea, Singapore, Taiwan, and Thailand. Studies were included if they provided data on patient involvement in health technology assessment (HTA) and/or funding decisions for medicines. Extracted data were scored according to eleven parameters adapted from the National Health Council (NHC) rubric, which assessed the level of patient involvement in healthcare system decision-making.</p><p><strong>Results: </strong>We identified 159 records between 2018 and 2022, including methodology guidelines from Government websites. Most mentioned parameters were patient partnership, patient-reported outcome, and mechanism to incorporate patient input. Limited information was available on diversity and patient-centered data sources. Tools for collecting patient experience included quality-of-life questionnaires, focus groups, interviews, and surveys, with feedback options like structured templates, videos, and public sessions.Beyond input in assessment process, involvement of patients in decision-making phase has evolved within HTA bodies over time with considerable variation. Few APAC healthcare systems involve patients in the appraisal process as members of the recommendation or decision-making committee.</p><p><strong>Conclusions: </strong>The findings indicate that while patient involvement in pharmaceutical reimbursement decisions exists, improvements are needed. Effective integration of patient input requires transparency, education, and resource planning. This study establishes a baseline to track progress and assess the long-term impact of patient involvement.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e28"},"PeriodicalIF":2.6,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996122","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":"A SYSTEMATIC ASSESSMENT OF THE DEMAND FOR HTA HUB SERVICES IN ASIA.","authors":"Julie L Hoang, Jeffrey J Sine, Sweta Saxena, Christian Suharlim","doi":"10.1017/S0266462325000236","DOIUrl":"https://doi.org/10.1017/S0266462325000236","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"1-27"},"PeriodicalIF":2.6,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996584","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 role and value of real-world evidence in health technology decision-making in France, Germany, Italy, Spain, and the UK: insights on external control arms.","authors":"Alexa Vidalis, Oscar Dumoulin, Mrunmayee Godbole, Catia C Proenca","doi":"10.1017/S0266462324004720","DOIUrl":"https://doi.org/10.1017/S0266462324004720","url":null,"abstract":"<p><p>Real-world evidence (RWE) is increasingly used and accepted by health technology assessment (HTA) bodies as supportive evidence to inform the approval of new technologies. However, the criteria driving RWE acceptance are often unclear.This study aims to improve understanding of the role and value of RWE in HTA decision-making and outline the best practices in building real-world external control arms (ECAs).A mixed approach of a targeted literature review and HTA expert interviews was applied. The HTA reports of ten selected technologies and the expert interviews from France, Germany, Italy, Spain, and the UK informed the criteria driving the acceptance of RWE. Overall, the UK and Spanish HTA bodies are more receptive to accepting RWE, whereas the French and German are the least accepting. When RWE is used to substantiate efficacy claims, the level of scrutiny from regulators and HTA bodies is considerably higher than when RWE has different intended uses. Representativeness of the data source, overall transparency in the study and robust methodologies are the key criteria driving RWE acceptance across markets.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e25"},"PeriodicalIF":2.6,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12019763/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143990484","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Learning strategies for laypeople to participate in health technology assessment: a scoping review.","authors":"Alex Itaborahy, Quenia Morais, Leny Frossard, Iandy Mateus, Bianca Leite, Marisa Santos","doi":"10.1017/S0266462325000200","DOIUrl":"https://doi.org/10.1017/S0266462325000200","url":null,"abstract":"<p><strong>Objectives: </strong>To provide an overview of learning strategies that health technology assessment (HTA) agencies use worldwide to educate laypeople about HTA.</p><p><strong>Methods: </strong>A scoping review focused on learning strategies to educate laypeople about HTA using the Joanna Briggs Institute frameworks was conducted across databases and gray literature. The study reviewed qualitative, quantitative, and mixed-methods studies from four databases, including practice documents from the HTA and health organization websites.</p><p><strong>Results: </strong>Fifteen studies were included in this review. The United Kingdom, Spain, and Canada mainly contributed to knowledge about educating laypeople in HTA. The main strategies employed were conference-like events, educational materials, training, and plain language. International HTA and health agencies developed courses, online training, and guidance materials to increase laypeople's participation in the HTA process.</p><p><strong>Conclusions: </strong>Efforts to improve public involvement in HTA focus on structured consultations, digital platforms, and capacity-building to enhance accessibility. Strategies like workshops and plain language aim to encourage lay participation, but challenges such as technical complexity and limited resources persist. Despite these challenges, incorporating patient perspectives has increased research relevance and public trust. Future studies should examine standardized frameworks for involvement, the impact of lay participation on policy, and solutions to barriers to a more equitable HTA process.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e26"},"PeriodicalIF":2.6,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144009739","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":"Ethics domains in full health technology assessment reports: an attempt to begin mapping the field.","authors":"Hannes Kahrass, Antje Schnarr, Clovis Mariano Faggion, Marcel Mertz","doi":"10.1017/S026646232400480X","DOIUrl":"10.1017/S026646232400480X","url":null,"abstract":"<p><strong>Introduction: </strong>Health technology assessment (HTA) reports are written for healthcare decision makers, particularly in relation to reimbursement/pricing, and are intended to assess clinical effectiveness, safety, and cost. Four additional domains are further considered in what is called a \"full HTA\": ethical, legal, social, and organizational aspects. The ethical aspects have long been the subject of debate regarding how they should be processed. It would be important if the following questions could be answered: Who publishes full HTA reports and how? Which methods are used in the ethics domain? What kind of results do they produce? However, such a \"mapping of the field\" turns out to be difficult. Despite the existence of international HTA registers, we were not able to compile a comprehensive sample of full HTA reports. Therefore, the aim of our study was rather to explore a) substantially: Which information can be expected to be (easily) found, which can only be obtained with considerable effort, and which remain (for the time being) in the dark? And b) methodologically: Is it possible to do meaningful meta-research in this field?</p><p><strong>Methods and results: </strong>In the attempt to explore the possibilities of meta-research, we were able to track down and analyze thirty-nine full HTA reports from six countries.</p><p><strong>Conclusions: </strong>While not representative of the whole field, this analysis shows the possibilities and challenges to meta-research, but nonetheless also provides some substantial insight into the characteristics of such reports, with a particular focus on the methods used to process ethical aspects.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e22"},"PeriodicalIF":2.6,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12019511/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143803284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Towards institutionalizing HTA in Ethiopia: using a political economy analysis to explore stakeholder perspectives and assessing capacity needs.","authors":"Daniel Asfaw Erku, Ararso Desalegn, Tesfaye Mesele Mekonnen, Ermias Dessie, Firmaye Bogale Wolde, Sabit Ababor Ababulgu, Paul A Scuffham, Damian Walker, Rabia Sucu, Samuel Abera","doi":"10.1017/S0266462325000170","DOIUrl":"10.1017/S0266462325000170","url":null,"abstract":"<p><strong>Background: </strong>As Ethiopia advances towards efficient resource utilization and UHC through strategic health purchasing, the institutionalization of HTA will play a critical role. This study aims to identify key stakeholders, analyze the political economy surrounding HTA and priority setting in Ethiopia, and assess existing skills and capacities for a robust and sustainable HTA system.</p><p><strong>Methods: </strong>We employed a mixed-method approach, combining 16 key informant interviews, 24 document reviews, and a cross-sectional survey (n=65) to assess national HTA capacity. We employed the Walt and Gilson policy analysis triangle framework, alongside Campos and Reich's framework, to evaluate the context, process, content, and actors influencing HTA institutionalization, and to explore the complex interplay of institutions, positions, power, and interests among various stakeholders.</p><p><strong>Results: </strong>While there is a general commitment to implementing HTA across various government agencies and stakeholder groups, the institutionalization process faces several challenges, involving multiple agencies with overlapping mandates, raises bureaucratic challenges and potential conflicts, risking horizontal fragmentation as agencies compete for authority, budget, and influence. The involvement of other key stakeholders, such as professional associations, patients, and the public, is notably lacking. Challenges such as limited HTA expertise, high professional turnover, and gaps in specific HTA knowledge areas persist, with capacity-building efforts often failing to address organizational needs effectively.</p><p><strong>Conclusions: </strong>The complexity of HTA institutionalization in Ethiopia underscores the necessity of managing intricate inter-agency dynamics, establishing a robust legal framework for an inclusive and transparent HTA process, building local capacity, and securing sustainable, domestically aligned funding.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e24"},"PeriodicalIF":2.6,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12019762/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143772291","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world evidence to support health technology assessment and payer decision making: is it now or never?","authors":"Linda A Murphy, Ron Akehurst, David Cunningham, Gérard de Pouvourville, Oriol Solà-Morales","doi":"10.1017/S0266462325000145","DOIUrl":"10.1017/S0266462325000145","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this policy article is twofold: (i) to provide a summary and update of recent important policy developments, in particular relevant guidance on the use of real-world data/real-world evidence (RWD/RWE) by health technology assessment (HTA) bodies and (ii) to set out our policy recommendations on how the different elements of an \"RWE framework\" we have previously developed could support, further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies and payers.</p><p><strong>Methods: </strong>We undertook a targeted review and analysis of recent important policy developments. The aim was to build on our recommendations from previous work on the \"RWE Framework,\" and consider how the relevant tools from our Framework can further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies/payers.</p><p><strong>Results: </strong>We provide eight conditions that we argue would, in combination, constitute the optimal use and acceptance of RWD/RWE for HTA. We believe that, should the eight conditions hold, RWD/RWE would enable more efficient access to medicines and healthcare technologies for patients.</p><p><strong>Conclusions: </strong>High-quality, fit-for-purpose RWD/RWE can and should be used more frequently in HTA. Multi-stakeholder and cross-geography collaborative partnerships are needed to align on best practices to optimize the evidence that needs to be generated to satisfy all stakeholders' needs.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e20"},"PeriodicalIF":2.6,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12018852/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143752444","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient engagement for the development of equity-focused health technology assessment (HTA) recommendations: a case study of two Canadian HTA organizations.","authors":"Rosiane Simeon, Abdulah Al Ameer, Shehzad Ali, Kumanan Wilson, Janet H Roberts, Ian D Graham, Peter Tugwell, Vivian A Welch","doi":"10.1017/S0266462325000182","DOIUrl":"10.1017/S0266462325000182","url":null,"abstract":"<p><strong>Background: </strong>Health technology assessment (HTA) is a form of policy analysis that informs decisions about funding and scaling up health technologies to improve health outcomes. An equity-focused HTA recommendation explicitly addresses the impact of health technologies on individuals disadvantaged in society because of specific health needs or social conditions. However, more evidence is needed on the relationships between patient engagement processes and the development of equity-focused HTA recommendations.</p><p><strong>Objectives: </strong>The objective of this study is to assess relationships between patient engagement processes and the development of equity-focused HTA recommendations.</p><p><strong>Methods: </strong>We analyzed sixty HTA reports published between 2013 and 2021 from two Canadian organizations: Canada's Drug Agency and Ontario Health.</p><p><strong>Results: </strong>Quantitative analysis of the HTA reports showed that direct patient engagement (odds ratio (OR): 3.85; 95 percent confidence interval (CI): 2.40-6.20) and consensus in decision-making (OR: 2.27; 95 percent CI: 1.35-3.84) were more likely to be associated with the development of equity-focused HTA recommendations than indirect patient engagement (OR: .26; 95 percent CI: .16-.41) and voting (OR: .44; 95 percent CI: .26-.73).</p><p><strong>Conclusion: </strong>The results can inform the development of patient engagement strategies in HTA. These findings have implications for practice, research, and policy. They provide valuable insights into HTA.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e27"},"PeriodicalIF":2.6,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143729977","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":"\"Blanket rules just don't work\": Qualitative exploration of the relative value of child and adult quality-adjusted life year (QALY) gains for health technology assessment.","authors":"Marcus Sellars, Joanna Coast, Emily Lancsar, Cam Donaldson, Stacy M Carter","doi":"10.1017/S0266462325000194","DOIUrl":"10.1017/S0266462325000194","url":null,"abstract":"<p><strong>Objectives: </strong>Effective allocation of scarce healthcare resources involves complex ethical and technical evaluations, with decision makers sometimes utilizing a societal perspective in health technology assessment (HTA). This study aimed to explore societal perspectives on healthcare resource allocation within Australia's HTA framework, focusing on the valuation of health gains for children and young people (CYP) compared to adults.</p><p><strong>Methods: </strong>In-depth, semistructured interviews were conducted with ten young people (aged 15-17) and twenty adults between October 2021 and April 2022. Participants were purposively sampled for diverse characteristics and completed an online information survey prior to the interviews, introducing relevant concepts. Interviews were analyzed using inductive coding, categorization, and constant comparison.</p><p><strong>Results: </strong>Participants expressed nuanced perspectives on HTA processes, generally opposing numeric weighting and preferring a deliberative approach based on committee judgment. Although most participants acknowledged some moral relevance of CYP status in HTA, opinions varied on its operationalization. A sizable minority, including those with extensive health system experience, did not view CYP status as morally relevant, though some noted specific service gaps for CYP (e.g., mental health care, pain management). Participants identified a spectrum of factors, both person-centered and intervention related, that often surpassed the relevance of CYP status, including addressing severity, unmet needs, prevention, and early intervention, with an emphasis on Aboriginal and Torres Strait Islander communities.</p><p><strong>Conclusion: </strong>Our findings highlight the inherent challenges in navigating the complexities of HTA and the critical need for HTA frameworks to be adaptable and inclusive, effectively integrating societal preferences to enhance healthcare policy's equity and responsiveness.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e23"},"PeriodicalIF":2.6,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12019764/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143729973","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Calibration of transition probabilities to model survival of adjuvant trastuzumab for early breast cancer in Indonesia.","authors":"Arie Rahadi, Rizki Tsalatshita Khair Mahardya, Putri Listiani, Eva Herlinawaty, Ryan Rachmad Nugraha, Dani Ramdhani Budiman, Christian Suharlim","doi":"10.1017/S0266462325000157","DOIUrl":"10.1017/S0266462325000157","url":null,"abstract":"<p><strong>Objectives: </strong>Cost-effectiveness models fully informed by real-world epidemiological parameters yield the best results, but they are costly to obtain. Model calibration using real-world data/evidence (RWD/E) on routine health indicators can provide an alternative to improve the validity and acceptability of the results. We calibrated the transition probabilities of the reference chemotherapy treatment using RWE on patient overall survival (OS) to model the survival benefit of adjuvant trastuzumab in Indonesia.</p><p><strong>Methods: </strong>A Markov model comprising four health states was initially parameterized using the reference-treatment transition probabilities, obtained from published international evidence. We then calibrated these probabilities, targeting a 2-year OS of 86.11 percent from the RWE sourced from hospital registries. We compared projected OS duration and life-years gained (LYG) before and after calibration for the Nelder-Mead, Bound Optimization BY Quadratic Approximation, and generalized reduced gradient (GRG) nonlinear optimization methods.</p><p><strong>Results: </strong>The pre-calibrated transition probabilities overestimated the 2-year OS (92.25 percent). GRG nonlinear performed best and had the smallest difference with the RWD/E OS. After calibration, the projected OS duration was significantly lower than their pre-calibrated estimates across all optimization methods for both standard chemotherapy (~7.50 vs. 11.00 years) and adjuvant trastuzumab (~9.50 vs. 12.94 years). LYG measures were, however, similar (~2 years) for the pre-calibrated and calibrated models.</p><p><strong>Conclusions: </strong>RWD/E calibration resulted in realistically lower survival estimates. Despite the little difference in LYG, calibration is useful to adapt external evidence commonly used to derive transition probabilities to the policy context, thereby enhancing the validity and acceptability of the modeling results.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e18"},"PeriodicalIF":2.6,"publicationDate":"2025-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11955306/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143709708","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}