International Journal of Technology Assessment in Health Care最新文献

{"title":"Actions for stakeholders to develop better real-world evidence for HTA bodies/payers decision making.","authors":"Ashley A Jaksa, Alina N Pavel, Matti Aapro, Niklas Hedberg, Victoria Hodgkinson, Laurie J Lambert, Francois Meyer, Matias Olsen, Piia K Rannanheimo, Karen M Facey","doi":"10.1017/S0266462325100238","DOIUrl":"10.1017/S0266462325100238","url":null,"abstract":"<p><strong>Objective: </strong>In 2020, RWE4Decisions, a multi-stakeholder initiative commissioned by the Belgian payer, published stakeholder actions to support the generation, analysis, and interpretation of real-world evidence (RWE) to inform the decision making of health technology assessment (HTA) bodies/payers for highly innovative medicines in the European Union (EU). Since 2020, changes in the decision-making environment and advancements in RWE have created an impetus to update stakeholder actions for the EU and Canada.</p><p><strong>Methods: </strong>RWE4Decisions' experts led focus groups with individual stakeholder groups (HTA bodies/payers, pharmaceutical industry, clinicians, patients, registry holders, and data analytical experts). Each focus group crafted new actions for their stakeholder, then the actions were discussed and revised in a multi-stakeholder meeting, a public webinar, and a public consultation. Themes across actions and meetings were identified.</p><p><strong>Results: </strong>Detailed new actions for each stakeholder group are presented. Key themes identified are the need to address interorganizational fragmentation regarding secondary data use and methodologies to build robust RWE. HTA bodies/payers need to develop a common vision about the potential use of RWE. The role of the whole clinical team as primary data collectors is critical. Opportunities for scientific advice across the life cycle of a medicine are essential, and the implementation of RWE guidance related to HTA is paramount. Progress requires specific, operational actions and a collective effort by a variety of stakeholders.</p><p><strong>Conclusions: </strong>Carrying out these actions will facilitate the development of methodological best practices for generating RWE to inform HTA of highly innovative medicines and build trust between stakeholders in the use of RWE.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e52"},"PeriodicalIF":3.1,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12350084/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309925","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":"Defining early health technology assessment: building consensus using Delphi technique: a commentary on implementation and diffusion of early HTA.","authors":"Linn Nathalie Støme","doi":"10.1017/S0266462325100214","DOIUrl":"10.1017/S0266462325100214","url":null,"abstract":"<p><p>This article presents the first consensus-based definition of early health technology assessment (HTA): \"an HTA conducted to inform decisions about subsequent development, research, and/or investment by explicitly evaluating the potential value of a conceptual or actual health technology.\" The definition was developed and refined through the involvement of relevant stakeholders in the field, a working group, and a survey panel, aiming to reach a consensus. An important part of this work was distinguishing between early HTA and related concepts, such as early awareness, dialogue, and scientific advice; thus, clarifying its unique role in HTA. Furthermore, the authors discuss how early HTA may guide investment decisions in development and reduce research waste. In addition, the consensus-based definition may enhance clarity for developers in producing early decision support to reach healthcare providers and policymakers. Finally, the article emphasizes the need for standardized terminology to increase the visibility of research, development, and policy in early HTA.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e32"},"PeriodicalIF":2.6,"publicationDate":"2025-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178749/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144215804","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":"Defining early health technology assessment: building consensus using Delphi technique.","authors":"Janneke P C Grutters, Janet Bouttell, Payam Abrishami, Sulafa Y M Ahmed, Amanda Cole, Dalia Dawoud, Carla Fernández-Barceló, Geert W J Frederix, Neil Hawkins, Jonathan Karnon, Sharon Leadbitter, Christopher McCabe, Jani Mueller, Samuel Owusu Achiaw, Andrew Partington, Laura Sampietro-Colom, Abril Seyahian, Rabia Sucu, Michelle Tew, Sasha van Katwyk, Yves Verboven, Yi Wang","doi":"10.1017/S0266462325100123","DOIUrl":"10.1017/S0266462325100123","url":null,"abstract":"<p><p>Although early health technology assessment (HTA) is increasingly being used to guide and inform decisions on product development, a consensus definition is currently lacking. A working group under the HTA International Society was established to develop a consensus-based definition of early HTA. The working group developed a definition using an iterative process that comprised five stages of work and included a two-round Delphi survey with 133 respondents in the first and 99 respondents in the second round of the survey, with various backgrounds and levels of expertise. Following this process, the working group reached the first consensus-based definition of early HTA, which is an HTA conducted to inform decisions about subsequent development, research, and/or investment by explicitly evaluating the potential value of a conceptual or actual health technology. In total, 86 (87 percent) of the 99 panelists who participated in the second round of the Delphi survey either strongly agreed or agreed with this definition. This consensus definition represents an important milestone in early HTA. It will enhance the uniformity of terminology, increasing the visibility of research and policy in this field. We also hope that it will act as a catalyst sparkling further research and developments in this discipline.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e34"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178750/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199122","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":"Defining early health technology assessment: reclaiming origins and diversity of applications.","authors":"Clifford Goodman","doi":"10.1017/S0266462325100159","DOIUrl":"10.1017/S0266462325100159","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e35"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178747/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199123","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":"Development and validation of a type 2 diabetes model to estimate the cost-effectiveness of diabetes interventions across the care continuum.","authors":"Megan Wiggins, Jeff Round, Erin Kirwin","doi":"10.1017/S0266462325100172","DOIUrl":"10.1017/S0266462325100172","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study is to develop a patient-level model for type 2 diabetes mellitus (T2DM) progression that can estimate the cost-effectiveness of T2DM interventions from prevention to management.</p><p><strong>Methods: </strong>We developed an individual-level microsimulation model, the Institute of Health Economics Diabetes Model (IHE-DM), that simulates: (i) T2DM progression from normal glucose tolerance (NGT) to T2DM, (ii) the occurrence and timing of eight comorbidities and death, and (iii) the correlated progression of risk factors over time. We report model validation and use a case study to investigate the cost-effectiveness of a hypothetical T2DM prevention program.</p><p><strong>Results: </strong>The internal validation indicated excellent performance with mean absolute differences between the predicted and observed values for all endpoints of less than 1 percent. External validation results were mixed. The model under-predicted cumulative T2DM incidence in the first 8 years, predicted well from years eight through eleven, and over-predicted from years twelve through fifteen. Our case study estimated an incremental net monetary benefit of CAD 2,701 (USD 2,289) (95% Uncertainty Interval: CAD 1,316 to 4,000 [USD 1,115 to 3,390]) over the 15-year time horizon.</p><p><strong>Conclusions: </strong>Prominent T2DM models focus on patients with diagnosed T2DM whereas our model simulates progression from NGT to T2DM and incorporates important correlations in the progression of risk factors. These adaptations allow us to evaluate preventative interventions and better capture the long-term impacts, filling an important gap in the evidence base. Our model can be used to inform future funding decisions for T2DM interventions across the care continuum.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e36"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12188100/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199124","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":"Commentary: Reflections on defining Early HTA.","authors":"Iben Fasterholdt, Tue Kjølhede, Lise Kvistgaard Jensen","doi":"10.1017/S0266462325100196","DOIUrl":"10.1017/S0266462325100196","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"1-4"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12178746/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199121","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":"Improving access to innovation from international health technology assessment.","authors":"Carlos Crespo","doi":"10.1017/S0266462325100135","DOIUrl":"10.1017/S0266462325100135","url":null,"abstract":"<p><p>The field of health care has evolved from an emphasis on evidence-based medicine, with a focus on efficacy, safety, and tolerability, to the pursuit of evidence-based efficiency and sustainable innovation in many respects (healthcare budgets, carbon print, etc.). This evolution can be attributed, in part, to the contributions of health technology assessment (HTA) bodies, which have facilitated the incorporation of various factors into the decision-making process (). These factors include comparative effectiveness, quality of life, efficiency, budgetary impact, and organizational impact, among others. Within the domain of health care, irrespective of the perspective of each entity (e.g., Food and Drug Administration (FDA), European Medicines Agency, etc.), there is an imperative for the presence of evidence and its assessment in the most transparent manner possible, with the objective of ensuring the incorporation of healthcare technologies.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e37"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12188096/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199125","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":"Supporting translation with early health technology assessment: from definition to action.","authors":"Sean P Gavan","doi":"10.1017/S0266462325100147","DOIUrl":"10.1017/S0266462325100147","url":null,"abstract":"<p><p>Translating emerging health technologies towards adoption and patient benefit requires timely and effective research and development decisions. Early health technology assessment has a key role to play in supporting these decisions. A new consensus definition of early health technology assessment is a welcome contribution to help bring these activities toward wider use in the field. In parallel, the opportunities to perform early health technology assessment activities are increasing as new types of health technologies begin to enter healthcare systems globally. A greater focus on transparency of reporting, improving awareness around how early health technology assessment can impact decision-making, increased resourcing for these activities, expanding training for analysts, and encouraging collaboration between individuals across healthcare systems will be vital to strengthen the uptake of early health technology assessment from this point forward.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e39"},"PeriodicalIF":2.6,"publicationDate":"2025-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12226196/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144199126","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":"Identifying characteristics for a cost-effective psoriatic arthritis biomarker test: a development-focused health technology assessment.","authors":"Alexander C T Tam, Vinod Chandran, Dafna Gladman, Vathany Kulasingam, Eldon Spackman, Nick Bansback","doi":"10.1017/S0266462325000091","DOIUrl":"10.1017/S0266462325000091","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to evaluate the required test characteristics that a psoriatic arthritis (PsA) biomarker test would need to achieve to be considered cost-effective.</p><p><strong>Methods: </strong>We adapted an existing Markov model to compare a hypothetical biomarker with current practice. The model followed a patient cohort aged 45 years with moderate psoriasis (PsO) in which PsA was prevalent but unrecognized over a 40-year time horizon. Patients were assumed to be routinely seen at a dermatology clinic. In the current practice arm, patients with PsA were clinically detected. In the biomarker arm, a hypothetical test was assumed to be administered at baseline. Patients who screened positive would accept a combination of conventional disease-modifying antirheumatic drugs and targeted treatment to slow disease progression. Progression was modeled as linear changes in Health Assessment Questionnaire (HAQ) scores. We varied the sensitivity, specificity, and biomarker price based on current development progress. Scenario analyses considered alternative patient cohorts with mild and severe PsO separately.</p><p><strong>Results: </strong>The base case showed that a biomarker test with 70 percent sensitivity, 80 percent specificity, and a price of US$500 would be cost-effective (incremental cost-effectiveness ratio US$47,566 per quality-adjusted life-year [QALY]). Three-way analyses showed that a test with 80 percent specificity could be cost-effective at a US$50,000 per QALY threshold with a sensitivity as low as 66 percent at US$500. Only a near-perfect test would be cost-effective at a US$1,000 price point. Results were sensitive to HAQ progression under treatment, therapy costs, and the patient population.</p><p><strong>Conclusion: </strong>This study supports the continued product development of candidate PsA biomarkers.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"41 1","pages":"e29"},"PeriodicalIF":2.6,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12123161/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144127559","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":"A value framework for lymphoma therapies based on MACBETH method.","authors":"Yumei He, Wei Li, Xiaochen Zhu, Zhifeng Nie, He Zhu, Yingyao Chen, Sheng Han","doi":"10.1017/S0266462325000169","DOIUrl":"10.1017/S0266462325000169","url":null,"abstract":"<p><strong>Objectives: </strong>The rising cost of oncology care has motivated efforts to quantify the overall value of cancer innovation. This study aimed to apply the MACBETH approach to the development of a value assessment framework (VAF) for lymphoma therapies.</p><p><strong>Methods: </strong>A multi-attribute value theory methodological process was adopted. Analogous MCDA steps developed by the International Society for Health Economics and Outcomes Research (ISPOR) were carried out and a diverse multi-stakeholder group was recruited to construct the framework. The criteria were identified through a systematic literature review and selected according to the importance score of each criterion given by stakeholders, related research and expert opinions. The MACBETH method was used to score the performance of alternatives by establishing value functions for each criterion and to assign weight to criteria.</p><p><strong>Results: </strong>Nine criteria were included in the final framework and a reusable model was built: quality adjusted life years (QALYs), median progression-free survival, objective response rate, the incidence of serious adverse events (grade 3-4), rates of treatment discontinuation due to adverse events, annual direct medical costs, dosage and administration, the number of alternative medicines with the same indication and mechanism, mortality of the disease. The weights of each criterion in the order presented above are 17.43 percent, 16.11 percent, 14.39 percent,13.54 percent,11.83 percent,11.30 percent,7.08 percent,4.59 percent, and 3.73 percent.</p><p><strong>Conclusions: </strong>A criterion-based valuation framework was constructed using multiple perspectives to provide a quantitative assessment tool in facilitating the delivery of affordable and valuable lymphoma treatment. Further research is needed to optimize its use as part of policy-making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e49"},"PeriodicalIF":3.1,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12322853/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093839","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}