Marcus Sellars, Joanna Coast, Emily Lancsar, Cam Donaldson, Stacy M Carter
{"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}
Arie Rahadi, Rizki Tsalatshita Khair Mahardya, Putri Listiani, Eva Herlinawaty, Ryan Rachmad Nugraha, Dani Ramdhani Budiman, Christian Suharlim
{"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}
Rossella Di Bidino, Iga Lipska, Monika Kukla, Marina von Pinoci, Sara Consilia Papavero, Marco Marchetti, Laura Sampietro-Colom, Americo Cicchetti
{"title":"The potential of the hospital-based Health Technology Assessment: Results of a world-wide survey.","authors":"Rossella Di Bidino, Iga Lipska, Monika Kukla, Marina von Pinoci, Sara Consilia Papavero, Marco Marchetti, Laura Sampietro-Colom, Americo Cicchetti","doi":"10.1017/S0266462325000108","DOIUrl":"10.1017/S0266462325000108","url":null,"abstract":"<p><strong>Objectives: </strong>Hospital-Based Health Technology Assessment (HB-HTA) is a heterogeneous phenomenon constantly evolving to respond to the needs of decision-makers at the hospital level. In 2023, The HB-HTA Interest Group of Health Technology Assessment International (HTAi) surveyed HB-HTA activities with the aim to provide an updated description of the actual scenario.</p><p><strong>Methods: </strong>An online survey was conducted to gather data on the main characteristics of hospitals, HB-HTA activities, outputs, role in the decision-making processes, dissemination and training activities, and their interaction and collaboration with other stakeholders and HTA-related regulations. Finally, the survey collected feedback on the perception of and current barriers to HB-HTA. Three categories of responders were identified: Both hospitals performing and not performing HTA and policymakers.</p><p><strong>Results: </strong>Eighty-seven responses were collected from twenty-eight countries. Nearly half of the responders (<i>n</i> = 41) conducted HB-HTA, whereas eighteen consisted of hospitals not performing HTA, and twenty-eight were policy makers. HB-HTA was performed mainly in hospitals with >500 beds. HB-HTA units were organized in 40 percent of cases as an \"independent group.\" The survey showed that HTA units could contribute to all the steps of the decision-making processes, whereas the impact of the assessments on the decisions was mainly perceived as a medium. Furthermore, HB-HTA was not seen as a duplication of effort, even without specific regulations.</p><p><strong>Conclusions: </strong>The survey highlighted the role of HB-HTA in hospital decision-making supporting the vision of HB-HTA as one of the actors in the HTA ecosystem, the success of which depends on collaboration with other stakeholders.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e19"},"PeriodicalIF":2.6,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12018856/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143657150","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":"Navigating change: a comparative analysis of health technology assessment reforms across agencies - processes, drivers, and interdependencies.","authors":"Gayathri Kumar, Priscila Radu, Patricia Cubi-Molla, Martina Garau, Eleanor Bell, Jia Pan, Ramiro Gilardino, Julie Van Bavel, Agnes Brandtmüller, Katherine Nelson, Melinda Goodall","doi":"10.1017/S0266462325000133","DOIUrl":"10.1017/S0266462325000133","url":null,"abstract":"<p><strong>Objectives: </strong>Health technology assessment (HTA) is a critical part of healthcare decision making in many countries. Changes in Methods and Processes (M&P) of HTA agencies can affect the time and degree of patient access to treatments. Published literature focuses on the different M&P adopted by HTA agencies, rather than on how these have come about over time. Our study investigates key HTA reforms and explores their drivers and interdependencies in a set of HTA agencies in Europe, Asia-Pacific, and North America.</p><p><strong>Methods: </strong>We conducted a targeted literature review on M&P guidelines and subsequent changes to those, for 14 HTA agencies. We supplemented and validated initial findings with 29 semi-structured interviews with country-specific experts. We used analytical tools to create process maps, proactivity and influence networks, and clusters of HTA agencies.</p><p><strong>Results: </strong>We found that processes leading to M&P reforms follow similar steps across HTA agencies. The three most important drivers to reforms were HTA practice and guidelines in other countries; the healthcare policy, legal, and political context within the agency's country; and experience of challenges in the assessment by the HTA body itself. International collaborations have the potential to accelerate the evolution of HTA systems and the implementation of reforms.</p><p><strong>Conclusion: </strong>We identified PBAC (Australia), CDA-AMC (Canada), NICE (England), IQWiG (Germany), and ZIN (the Netherlands) as HTA agencies that are catalysts of HTA reforms as well as internationally influential. International collaborations may represent a useful route to accelerate changes as long as they ensure wide stakeholder engagement at an early stage.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e21"},"PeriodicalIF":2.6,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12018853/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143624807","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}
Richmond Owusu, Brian Adu Asare, Genevieve Cecilia Aryeetey, Ivy Amankwah, Emmanuella Abassah-Konadu, Godwin Gulbi, Saviour Yevutsey, Sergio Torres Rueda, Joseph Kazibwe, Francis Ruiz, Joycelyn Zeez, Justice Nonvignon
{"title":"Standards for the conduct and reporting of health technology assessments: Ghana reference case of HTA and economic evaluations.","authors":"Richmond Owusu, Brian Adu Asare, Genevieve Cecilia Aryeetey, Ivy Amankwah, Emmanuella Abassah-Konadu, Godwin Gulbi, Saviour Yevutsey, Sergio Torres Rueda, Joseph Kazibwe, Francis Ruiz, Joycelyn Zeez, Justice Nonvignon","doi":"10.1017/S026646232500011X","DOIUrl":"10.1017/S026646232500011X","url":null,"abstract":"<p><strong>Background: </strong>The methods of economic evaluation and HTA should be based on best practices and standards, tailored to unique country contexts that can be systematically applied to inform decisions. This paper outlines standards for the conduct of economic evaluations for HTA in Ghana.</p><p><strong>Methods: </strong>A five-step process was followed to develop the HTA reference case as a methodological and reporting benchmark. These include (a) a review of literature and evidence synthesis, (b) a review of country policies, (c) a review and adaption of international frameworks, (d) expert/stakeholder consultations, and (e) the development of a methodological framework. A series of stakeholder consultations were done to refine, finalize, and validate the outcomes of the processes to generate a finalized reference case.</p><p><strong>Results: </strong>The Ghana reference case is made up of 14 components comprising: evidence synthesis, evaluation type, perspectives on cost, perspectives of outcomes, choice of comparator, data sources, outcome measures, discount rate, uncertainty, equity considerations, time horizon, heterogeneity, transparency, and budget impact. These provide methodological considerations and reporting requirements for economic evaluations for HTA. It provides a framework to ensure the best research methods are adopted to harmonize the evidence-generation process with the expectations of policy and decision-makers and ensure that policy decisions are based on uniform evidence.</p><p><strong>Conclusion: </strong>Recommendations set out in this reference case when followed can provide context-specific evidence to support a rigorous and transparent system for evaluating healthcare interventions and technologies. It will support decision-making, ultimately improving the quality and efficiency of healthcare delivery in the country.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e17"},"PeriodicalIF":2.6,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536921","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}
P Lehoux, I Ganache, O Demers-Payette, H P Silva, G Plamondon, M de Guise
{"title":"HTA responsiveness to today's challenges to health systems: a responsible innovation in health perspective.","authors":"P Lehoux, I Ganache, O Demers-Payette, H P Silva, G Plamondon, M de Guise","doi":"10.1017/S0266462325000121","DOIUrl":"10.1017/S0266462325000121","url":null,"abstract":"<p><strong>Introduction: </strong>Though Health Technology Assessment (HTA) has steadily grown over the past decades, less attention has been paid to the way HTA may prove more responsive to the broader economic, social, and environmental challenges that health systems are facing today. In view of climate change, chronic diseases, an aging population, inequalities, and workforce issues, the HTA community's unique set of skills nonetheless holds great potential to help decision-makers strengthen many publicly funded health systems around the world.</p><p><strong>Methods: </strong>This article adopts an integrated system-wide perspective guided by the Responsible Innovation in Health (RIH) framework to explore how the HTA community may not only adapt to the <i>speed</i> of innovation but also consider its direction.</p><p><strong>Results: </strong>Because RIH aims to steer innovation toward a more sustainable pathway, it can help HTA agencies anticipate decision-makers' informational needs regarding four systemic challenges: (1) equitable access; (2) workforce issues; (3) accountable policy trade-offs; and (4) environmental sustainability. We clarify how key elements of the RIH framework may be used by HTA agencies to: (1) supplement their evaluation process; (2) align their priority-setting or strategic planning activities with their health system challenges; or (3) inform the production of early HTAs, horizon scans, or reports that are broader in scope than a single technology review.</p><p><strong>Conclusions: </strong>The article concludes with three practical implications that were identified by the <i>Institut National d'Excellence en Santé et Services Sociaux</i> (INESSS) (Québec, Canada) and may inspire other HTA agencies.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e16"},"PeriodicalIF":2.6,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11936730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536746","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":"Value and Implementation of Patient and Public Involvement and Engagement in Health Technology Assessment for Japan: implications from systematic searches.","authors":"Takako Kaneyasu","doi":"10.1017/S026646232500008X","DOIUrl":"10.1017/S026646232500008X","url":null,"abstract":"<p><strong>Objectives: </strong>This study comprehensively reviewed reports on patient and public involvement and engagement (PPIE) in health technology assessment (HTA) overseas and identified the status and possible future measures, of PPIE in Japanese HTA.</p><p><strong>Methods: </strong>The series of reviews targeted systematic reviews (SR#1), references in SR#1 (SR#2), and new articles after SR#1 (SR#3). The MEDLINE and Embase databases were searched through August 2024 using the terms \"patient involvement/engagement,\" \"patient participation,\" \"community participation,\" \"public involvement/engagement,\" and \"health technology assessment.\" The implementation details were extracted from information published on the websites of the HTA agencies.</p><p><strong>Results: </strong>Three review articles in SR#1, 12 references in SR#2, and 10 articles in SR#3 were selected. The opportunities for countries, including Japan, to participate in discussions on the HTA process did not differ significantly; however, information on PPIE in Japan was scarce and did not indicate their purpose and value.</p><p><strong>Conclusions: </strong>Collected articles indicated that the value of PPIE in HTA includes relevance, equity, fairness, legitimacy, and capacity building. The participation of patient and public representatives in Japanese discussions since 2005 appeared to be very limited to consider PPIE in HTA. In countries that implement PPIE in HTA, the value of PPIE is explicit: the process guidelines are specific and provide an appropriate environment for input that includes education, training, and feedback. Future reforms of the Japanese system will require discussions on PPIE purpose and value, implementation, and creating an environment in which a diverse range of patients and the public can easily express their views.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e14"},"PeriodicalIF":2.6,"publicationDate":"2025-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894397/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523454","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":"Health technology assessment capacity to support Zambia's health benefits package reform policy.","authors":"Warren Mukelabai Simangolwa, Kaymarlin Govender, Josue Mbonigaba","doi":"10.1017/S0266462325000030","DOIUrl":"10.1017/S0266462325000030","url":null,"abstract":"<p><strong>Background: </strong>The need for more local technical capacity in Health Technology Assessment (HTA) is a leading challenge to its use in low- and middle-income countries. Zambia has been considering using HTA to support its universal health coverage initiative, which includes health benefits package design and implementation. This study assesses the local HTA capacity for the steering committee tasked with supporting the design and implementation of the national health benefits package in Zambia.</p><p><strong>Methods: </strong>The study applied a cross-sectional web-based survey design and the consensus-based Checklist for Reporting of Survey Studies. Data were collected from the steering committee of the benefits package working group, tasked with leading the design process of the health benefits package using the Instrument for the Assessment of Skills to Conduct a Health Technology Assessment tool.</p><p><strong>Results: </strong>The majority of respondents had not served on a selection and reimbursement committee. Clinical effectiveness skills in structuring a search strategy, handling missing data, conducting qualitative evidence synthesis, and grading the certainty of evidence were low. Skills for leadership, networking, conflict management, and project coordination, public and patient involvement were mid-level to low. Most of the respondents were aware of ethical issues with health technologies. Health economics skills in economic evaluations and decision analytic modeling, equity and health system efficiency measurement, budget impact analysis, and quality of life were identified for capacity strengthening.</p><p><strong>Conclusion: </strong>Available technical capacities to revise and implement the national benefits package were lower in health economics, synthesis for clinical effectiveness evidence, ethics, patient and public involvement, and soft skills, in that order.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e15"},"PeriodicalIF":2.6,"publicationDate":"2025-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894396/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523442","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}
Hung Manh Nguyen, Jason Robert Guertin, Daniel Reinharz
{"title":"PERCEPTION OF NON-LAYPERSON ADVISORY COMMITTEE MEMBERS ON THE APPLICATION OF A DISCRETE CHOICE EXPERIMENT INSTRUMENT TO PATIENTS AND ADVISORY COMMITTEE MEMBERS: A QUALITATIVE STUDY.","authors":"Hung Manh Nguyen, Jason Robert Guertin, Daniel Reinharz","doi":"10.1017/S0266462325000029","DOIUrl":"https://doi.org/10.1017/S0266462325000029","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"1-30"},"PeriodicalIF":2.6,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143492102","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":"Use of surrogate endpoints in health technology assessment: a review of selected NICE technology appraisals in oncology.","authors":"Lorna Wheaton, Sylwia Bujkiewicz","doi":"10.1017/S0266462325000017","DOIUrl":"10.1017/S0266462325000017","url":null,"abstract":"<p><strong>Objectives: </strong>Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in the use of surrogate endpoints has been accompanied by literature describing the frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales.</p><p><strong>Methods: </strong>This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.</p><p><strong>Results: </strong>Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.</p><p><strong>Conclusions: </strong>This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals, which can lead to uncertainty in decision making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e11"},"PeriodicalIF":2.6,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894387/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143448363","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}