International Journal of Technology Assessment in Health Care最新文献

{"title":"Results of health technology assessments of orphan drugs in Germany-lack of added benefit, evidence gaps, and persisting unmet medical needs.","authors":"Philip Kranz, Natalie McGauran, Can Ünal, Thomas Kaiser","doi":"10.1017/S026646232400062X","DOIUrl":"https://doi.org/10.1017/S026646232400062X","url":null,"abstract":"<p><strong>Background: </strong>The number of orphan drug (OD) approvals has increased sharply in Europe. In Germany, all ODs are initially subject to a limited assessment after market access. Their added benefit over the standard of care is accepted as established upon EU approval; a regular health technology assessment (HTA) is performed only in certain cases.</p><p><strong>Objective: </strong>We assessed whether the increase in OD approvals has led to improvements in patient-relevant outcomes as supported by the evidence submitted by market authorization holders (MAHs) for HTA in Germany. We also examined the extent to which these ODs address unmet medical needs.</p><p><strong>Methods: </strong>The results of limited assessments and regular HTAs of ODs in Germany (January 2011-September 2021, plus January-December 2023) were analyzed to determine their added benefit based on MAH-submitted dossiers. Added benefit was reported separately for each research question generated from the EU-approved therapeutic indications and any sub-indications (e.g., different subpopulations or control interventions) specified for HTA in Germany.</p><p><strong>Results: </strong>Eighty-nine ODs (limited assessments: sixty-nine; regular HTAs: twenty) were evaluated in 175 research questions (limited assessments: ninety-seven; regular HTAs: seventy-eight). The added benefit granted in limited assessments was non-quantifiable in nearly eighty percent of the ninety-seven questions. In regular HTAs, no proof of added benefit was shown in fifty-four percent of the seventy-eight questions, mainly due to insufficient comparative data with the standard of care. Established treatments were available for fifty-eight percent of the seventy-eight questions; more than half of which addressed oncology indications (although these account for only eight percent of rare diseases).</p><p><strong>Conclusions: </strong>Due to evidence gaps in post-approval HTA, many ODs approved in the EU lack proof of added benefit in terms of improving patient-relevant outcomes. Moreover, most approved ODs are indicated for diseases with established treatments and oncology indications, while many unmet medical needs remain. Incentives are required to encourage research in areas of unmet medical need and to generate comparative data with the standard of care.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e68"},"PeriodicalIF":2.6,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768875","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":"Links between accuracy and effectiveness of laboratory medicine equipment: use of the EUnetHTA core model to compare two analyzers by measuring HbA1c.","authors":"Chiara Di Resta, Chiara Sacco, Mladen Trbos, Massimo Locatelli, Giuseppe Banfi, Rossella Tomaiuolo","doi":"10.1017/S0266462324000497","DOIUrl":"https://doi.org/10.1017/S0266462324000497","url":null,"abstract":"<p><strong>Objectives: </strong>In the field of Laboratory Medicine, the evolution of knowledge and the innovation of technologies are the basis of analytical and diagnostic progress, leading to the development of new solutions based on innovative technologies. However, these advances must be accompanied by evidence of appropriateness, diagnostic effectiveness, and organizational efficiency, considering the test's first impact on patient outcomes.</p><p><strong>Methods: </strong>The Health Technology Assessment (HTA) is a valid management tool to support Laboratory Medicine professionals in assessing technologies and which is the most appropriate to adopt. This study is an illustrative case of the application of HTA, exploiting the EUnetHTA Core Model, on two analyzers able to determine the glycated hemoglobin (Hemoglobin A1c, HbA1c), the Capillarys 2 Flex piercing analyzer and the HLC-723G11 analyzer in the Laboratory Medicine Service of the IRCCS San Raffaele Hospital (Milan, IT). The main focus is related to potential differences in methods, organizational aspects, and clinical effectiveness of these approaches for measuring HbA1c.</p><p><strong>Results: </strong>The EUnetHTA Core Model has proven to be the optimal method for HTA in the field of Laboratory Medicine, as it allows to highlight both the peculiarities of the methods on which the analyzers are based and the clinical efficacy of the laboratory test on specific patient populations, considering individual variations in treatment responses, assessing the potential benefits for individual patients or small groups.</p><p><strong>Conclusions: </strong>This granular analysis helps provide insights into the effectiveness and value of healthcare interventions at the patient level, contributing to evidence-based decision-making in clinical practice and healthcare policy.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e67"},"PeriodicalIF":2.6,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768872","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":"How can managed entry agreements contribute to coverage decisions in Latin America?","authors":"Sebastián García Martí, Andrés Pichon-Riviere, Federico Augustovski, Manuel Espinoza","doi":"10.1017/S0266462324000527","DOIUrl":"https://doi.org/10.1017/S0266462324000527","url":null,"abstract":"<p><strong>Context: </strong>Healthcare stakeholders in Latin America, including payers, manufacturers, and patients, seek to expedite access to technologies. However, uncertainty sometimes surrounds their true benefits and budgetary implications. Managed entry agreements (MEAs) are proposed to address this uncertainty by redistributing risks among key actors.</p><p><strong>Objectives: </strong>The objective of Health Technology Assessment International's 2023 Latin American Policy Forum was to examine the potential utility of MEA in technology reimbursement and decision-making processes in the region.</p><p><strong>Methods: </strong>This article is based on a background document, a survey, and the deliberative work of the country representatives and others who participated in the Policy Forum.</p><p><strong>Results: </strong>Interest in MEA in Latin America is increasing, with financial agreements being more prevalent than those based on clinical outcomes. During the Policy Forum, potential barriers to MEA implementation were identified, such as the lack of legal frameworks, insufficient reliable data, and, in some cases, distrust among stakeholders. Some potential solutions were also identified, including early stakeholder involvement to enhance dialogue and understanding, and piloting shorter-duration MEA to facilitate the revision of agreement terms, especially in situations of epidemiological uncertainty.</p><p><strong>Conclusions: </strong>The Policy Forum served as a valuable platform for discussing the importance of flexible MEA implementation that acknowledges data uncertainty, promotes transparent dialogue to incorporate opinions and values from all stakeholders, and develops legal frameworks to support effective technology access schemes in Latin America.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e65"},"PeriodicalIF":2.6,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768721","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":"Adebrelimab versus serplulimab plus chemotherapy as first-line treatment for extensive-stage small-cell lung cancer: A cost-effectiveness analysis in China.","authors":"Wanglong Hong, Aizong Shen, Miaomiao Zheng, Wentao Zhu, Guoqiang Ma","doi":"10.1017/S0266462324000175","DOIUrl":"https://doi.org/10.1017/S0266462324000175","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"1-23"},"PeriodicalIF":2.6,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142768780","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":"Reporting Standards for Economic Evaluations of Artificial Intelligence (AI) Interventions: A CHEERS Extension.","authors":"Marina Richardson, Gurdeep S Sagoo","doi":"10.1017/S0266462324004756","DOIUrl":"https://doi.org/10.1017/S0266462324004756","url":null,"abstract":"","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"1-4"},"PeriodicalIF":2.6,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750684","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 intersection of prescription drugs and medical devices: the evaluation and funding challenges of two categories of emerging health technologies.","authors":"Caroline Rodrigues, Rui Fu, Turjoy Ghose, Emre Yurga, Eric Nauenberg","doi":"10.1017/S0266462324004768","DOIUrl":"10.1017/S0266462324004768","url":null,"abstract":"<p><p>Health technology assessments (HTAs) are policy analysis frameworks contributing to the approval, reimbursement, and rollout of biotechnology and pharmaceuticals. New innovations in health technologies expose gaps in reimbursement and implementation guidelines. We defined two types of emerging health technologies: (1) therapeutic innovations, such as drug-device combination products or nondrug alternatives to prescription drugs and (2) disruptive health innovations such as novel surgeries and gene replacement therapies. We aimed to determine delineated definitions for these categories through a comprehensive review of HTA guidelines across 20 nations. Utilizing databases such as International Network of Agencies for HTA, International Society for Pharmacoeconomics and Outcomes Research, and European Medical Agency, we identified products falling within these categories. Real-world case studies highlighted the inadequacies stemming from the absence of clear definitions and proposed solutions to enhance current HTA guidelines. These shortcomings apply at the state or provincial level in addition to national jurisdictions as existing funding structures and silos fail to accommodate the unique attributes of these technologies.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":" ","pages":"e1"},"PeriodicalIF":2.6,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750709","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":"Integrating environmental sustainability into health technology assessment: an international survey of HTA stakeholders.","authors":"Michela Bobini, Americo Cicchetti","doi":"10.1017/S0266462324000631","DOIUrl":"https://doi.org/10.1017/S0266462324000631","url":null,"abstract":"<p><strong>Introduction: </strong>Health technologies play a relevant role in environmental sustainability (ES). However, limited evidence exists on approaches and methods to integrate ES into the Health Technology Assessment (HTA).</p><p><strong>Objectives: </strong>The purpose of this study is: (i) to provide an overview of global HTA organizations' progression toward the integration of ES into HTA; (ii) to investigate various paths for this integration, highlighting obstacles, priorities, potential approaches, and methods.</p><p><strong>Methods: </strong>Data were collected via questionnaires from organizations belonging to HTA networks, International Network of Agencies for Health Technology Assessment, and European Network for HTA. To complement the results of the survey, the authors carried out a desk analysis with strategic documents available on institutional websites.</p><p><strong>Results: </strong>The survey included twenty-six respondents from twenty different countries (thirty-three percent response rate). Among the study's participants, there is a notable acknowledgment of the importance of integrating ES into HTA. However, only nine organizations are actively engaged in these integration efforts, each employing unique methodologies and perspectives. There is a substantial consensus on the application of life cycle assessment, with a particular emphasis on the use of environmentally extended input-output analysis, and a stronger preference for cost-utility analysis. Nevertheless, evidence on integrating ES into HTA remains scarce. Major challenges identified include data collection difficulties and the necessity for interdisciplinary teams.</p><p><strong>Conclusions: </strong>Our study represents a preliminary effort to systematize initiatives aimed at integrating ES into HTA. Further research is required to customize methods and tools for appropriately evaluating the environmental impacts of technologies. The findings suggest that achieving ES-HTA integration demands a multi-tiered, interdisciplinary approach.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e64"},"PeriodicalIF":2.6,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750714","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 transition of assessing health technologies to social interventions in Sweden.","authors":"Sophie Söderholm Werkö, Titti Mattsson, Sofia Tranæus, Pernilla Östlund, Knut Sundell","doi":"10.1017/S0266462324000606","DOIUrl":"10.1017/S0266462324000606","url":null,"abstract":"<p><p>Since the 1970s the Swedish government has been promoting social work based on research into methods which work in practice for practitioners and patients. In 2015, the Swedish Agency for Health Technology Assessment (SBU), a government agency instigated in 1987, was commissioned to expand its remit, to review empirical research on social work interventions and to disseminate the results to stakeholders. SBU was then renamed The Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU). This article describes the fusion of health technology assessment (HTA) and Social Intervention Assessment (SIA), including advantages and challenges.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e66"},"PeriodicalIF":2.6,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750717","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":"Impact of COVID-19 pandemic on HTAsiaLink network members.","authors":"Ryan Jonathan Sitanggang, Kinanti Khansa Chavarina, Sarin K C, Raina Wadhwa, Budi Wiweko, Fredrick Dermawan Purba, Izzuna Mudla Mohamed Ghazali, Jonathan Henry Webster Jacobsen, Piyameth Dilokthornsakul, Mayfong Mayxay, Wanrudee Isaranuwatchai, Yaoling Wang, Ying-Li Chen, Benjamin Shao Kiat Ong, Sitanshu Kar, Andrey Avdeyev, Yot Teerawattananon","doi":"10.1017/S0266462324000357","DOIUrl":"https://doi.org/10.1017/S0266462324000357","url":null,"abstract":"<p><strong>Objectives: </strong>This study investigates the impact of coronavirus disease 2019 (COVID-19) pandemic on HTAsiaLink members at the organizational level and provides recommendations for mitigating similar challenges in the future.</p><p><strong>Methods: </strong>A survey was disseminated among HTAsiaLink members to assess the COVID-19 impact in three areas: (i) inputs, (ii) process, and (iii) outputs of the Health Technology Assessment organizations' (HTAOs) research operations and HTA process in general.</p><p><strong>Results: </strong>Survey results showed that most HTAOs hired more staff and secured similar or higher funding levels during COVID-19. Nevertheless, some organizations reported high staff turnover. COVID-19-relevant research was prioritized, and most of the organizations had to adapt their research design to meet the needs of policymakers. Time constraints in conducting research and inability to collect primary data were reported as impacts on the research process. Overall, the number of research projects and accessibility of respondents' publications increased during COVID-19.</p><p><strong>Conclusions: </strong>Research demand for HTAOs increased during COVID-19 and impacted their research process; however, they demonstrated resilience and adaptability to provide timely evidence for policymakers. With the growing reliance on HTA, HTAOs require adequate financial support, continuous capacity building, collaboration, and partnership, innovative HTA methods, and a pragmatic yet robust, evidence-to-policy process in preparation for future pandemics.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e63"},"PeriodicalIF":2.6,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142716216","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":"Impact of indication changes on scoping for European Union Joint Clinical Assessment: scale of the problem and how to address it.","authors":"Inka Heikkinen, Melinda Goodall, Natalie Steck, Maria Poulakou, Katherine Piso","doi":"10.1017/S0266462324004641","DOIUrl":"https://doi.org/10.1017/S0266462324004641","url":null,"abstract":"<p><strong>Objectives: </strong>The European Union Joint Clinical Assessment (JCA) process aligns with the regulatory process to promote faster patient access. The PICO (population, intervention, comparator, and outcome) scoping for the JCA must occur before the regulatory process concludes. The risk of indication change during this period is one of the concerns for the success of the JCA process. We investigated the frequency and type of changes that are made to proposed indications and examined how such changes could impact the PICO scoping for JCA.</p><p><strong>Methods: </strong>Twenty-seven recently approved oncology and 15 Advanced Therapy Medicinal Products (ATMP) products were included. Observed indication changes were categorized into editorial or population changes population changes were graded based on the anticipated impact on JCA scope depending on their nature.</p><p><strong>Results: </strong>The majority of products had only editorial changes between proposed and approved indications (67 percent). Once amended, it was common for the indicated population to be narrowed, and rare for it to be broadened. The most common change observed was the shift to a later treatment line. The greatest risk for PICO rescoping would be when new populations would have been added, or new subpopulations or subgroups would have been omitted from the initial scope.</p><p><strong>Conclusion: </strong>The impact on JCA scope depends on the proposed indication wording and how the PICO scoping would have been conducted. Rescoping warrants a considered decision, and to mitigate the risk of delays, dialogue between the assessors and the developer is recommended for informed decision-making.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":"40 1","pages":"e62"},"PeriodicalIF":2.6,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710124","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}