International Journal of Technology Assessment in Health Care最新文献

Objectives: Evaluate how a foundation-supported fellowship employs early health-technology assessment (eHTA) to guide the development and positioning of emerging health innovations.

Methods: We reviewed all eHTA reports conducted under the Fellowship from 2018 to 2021 (n = 10), extracting technology class, development stage, economic modeling, and recommendations. In 2023, we conducted thirty-minute structured video interviews with developers of each technology (eleven invitees, ten responses). The interview comprised Likert questions on perceived usefulness and intention to update the model in later stages, and six open-ended questions on perceived advantages, implementation barriers, and downstream actions. Likert data were summarized descriptively; open-ended responses were summarized and discussed within the research team until consensus on key themes.

Results: The eHTA subject technologies were four diagnostics, three therapeutics, two predictive algorithms, and one curative device, all preclinical. Analyses used six Markov or decision-tree frameworks, four hybrid models or simulations, and six value-based-pricing scenarios. Five technologies were potentially cost-effective, three conditionally cost-effective, one unlikely to be cost-effective without stronger evidence, and one cost-effective yet unlikely to break even. Eight developers rated eHTA "useful" or "very useful"; three had already leveraged results in grant or investor materials and two planned to do so when more data emerged. Reported barriers included evidence gaps, funding constraints, and misalignment with pharmaceutical partners on codevelopment strategies; two projects were discontinued.

Conclusions: eHTA supplies developers with early economic insight, but its guidance is most reliable when interpreted alongside budget impact, feasibility, regulatory, and adoption considerations.

Objectives: Health technology assessment of medical devices (HTA-MDs) presents unique challenges compared to pharmaceuticals. Total MD expenditure continues to grow in Europe, and countries typically conduct their own HTA-MDs evaluations, with varying institutionalization arrangements. European Union's (EU's) HTA Regulation aims to establish collaborative clinical assessments across Member States, potentially expediting the path from EU safety certification of MDs to pricing and reimbursement decisions. This study aims to identify emergent configurations among institutionalizations of HTA-MDs in the EU, European Economic Area (EEA), and European Free Trade Association (EFTA) countries.

Methods: Publicly available data were cross-sectionally collected for EU, EEA, and EFTA countries until August 2024 to allow a cross-country analysis of HTA-MDs institutionalizations. Countries were included if they had at least one publicly mandated body for HTA-MDs. Data sources were scientific databases, institutional websites, and HTA bodies' documentation. A framework of 16 elements, qualitatively describing the institutionalization of HTA-MDs, was developed based on a document review and used as a dataset for agglomerative hierarchical cluster analysis to identify patterns of HTA-MDs institutionalization.

Results: The 21 included countries formed three clusters: Cluster 1 featured regulatory-focused, legally bound HTA-MDs systems with mandatory assessments determining reimbursement decisions; Cluster 2 was characterized by regulatory functions, external expert collaboration, formal prioritization processes, and organized Horizon Scanning; Cluster 3 showed recommendatory functions, nonmandatory assessments, and limited impact on reimbursement decisions.

Conclusions: HTA-MDs institutionalizations could benefit from implementing prioritization processes of evaluations, establishing networks of collaborative assessment centers, and ensuring links between evaluations and reimbursement decisions.

Objective: Researchers propose wider individual and societal benefits (or broad elements of value) be included in economic evaluations (EEs) of medicines. This study investigates opinions of Australian stakeholders regarding the inclusion of broader value elements in reimbursement decisions for medicines for rare diseases in Australia.

Method: Stakeholders were invited via email to complete an online survey about their views on broader elements of value in HTA. Responses were summarised using descriptive statistics and compared using chi-square statistics.

Results: Forty-four respondents (academia (n=11), private sector (n=33)) completed the survey between October 2023 and May 2024. Only 27% of stakeholders agree the current information about the sources of value considered in reimbursement decisions is sufficient. Stakeholders consistently agree labour productivity (>50%), adherence (>80%), reducing uncertainty due to a new diagnostic (>70%), disease severity (>71%), value to caregivers (>70%), and equity (>70%) should be considered in HTA. The majority (>70%) agreed managed entry agreements (MEA), risk share arrangements (RSA), and multi criteria decision analysis (MCDA) be used in reimbursement decision making for medicines for rare diseases. Significantly fewer academic stakeholders (40%) versus private sector (77%), believe an increased willingness-to-pay threshold be applied to medicines for rare disease.

Conclusions: Academic and private sector stakeholders hold similar views when considering medicines for non-rare and rare diseases. Stakeholders favour considering more value elements in HTA than referred to in the Pharmaceutical Benefits Advisory Committee (PBAC) guidelines. This study highlights further advice is needed on the factors considered in reimbursement decisions and how that would influence guidelines.

Objectives: Predictive biomarkers can identify patients who are more likely to respond to immunotherapy, which can guide treatment decisions. The objective of this study was to assess the potential value of predictive biomarkers in advanced NSCLC patients to guide the development of cost-effective biomarkers in this field.

Methods: A decision analytical model was constructed to compare theoretical new strategies with biomarkers to the current standard of care. The analysis was performed for three different patient groups based on PD-L1 status. Differences in health outcomes (QALYs) and costs were assessed between the current practice and these biomarker strategies.

Results: Omitting immunotherapy in NSCLC patients with a PD-L1 score < 1 percent or between 1 and 49 percent, and a negative biomarker test, could potentially reduce healthcare costs significantly a small loss in QALYs. In these groups, a biomarker test is potentially cost-effective as the incremental cost-effectiveness ratio largely exceeds a willingness-to-accept threshold of €80,000 saved per QALY lost. For patients with a PD-L1 score > 50 percent, a considerable QALY gain can potentially be realized by adding chemotherapy to patients with a negative biomarker test. However, this comes at a significant increase in costs and appears not to be cost-effective.

Conclusions: In general, predictive biomarkers seem to have the potential to increase the cost-effectiveness of treatment with immunotherapy in patients with advanced NSCLC. Optimal positioning of a biomarker depends on the weighing between health impact and costs.

Health Technology Assessment international (HTAi) supports global collaboration and innovation in HTA through its dynamic network of Interest Groups (IGs). These thematic communities provide a dedicated platform for members to engage in focused, collaborative efforts that drive professional exchange, advance methodologies, and develop best practices in HTA. This commentary offers a panoramic overview of all IGs, their evolution, aim, and initiatives. By drawing on diverse stakeholder perspectives, spanning academia, clinical practice, industry, and patient communities, the IGs foster inclusiveness and extend HTAi's influence to significantly contribute to the broader HTA community. Through activities such as workshops, conference sessions, webinars, publications, and research projects, they offer opportunities for professional development and thought leadership. The IGs' cross-cutting contributions position them as engines of innovation to ensure HTAi remains at the forefront of shaping a globally relevant, responsive, and ethically grounded HTA ecosystem.

Objectives: Globally, several health technology assessment (HTA) agencies have started to incorporate environmental considerations into their assessments, given healthcare systems' substantial environmental footprint. In Canada, two HTA agencies, the Canadian Drug Agency and the Institut national d'excellence en santé et en services sociaux, have announced measures to help mitigate healthcare's contribution to climate change. Our aim was to review reports from both agencies to identify those incorporating environmental considerations.

Methods: We retrieved reports published between 1 May 2023 and 1 December 2024 by the two agencies.

Results: We identifed 202 reports, of which eleven were included. These reports covered diverse technologies, with greenhouse gas emissions and waste production being the most frequently considered environmental dimensions. Parallel evaluation was the predominant method for integrating environmental considerations. We believe that the limited number of reports included may reflect the challenges of incorporating such considerations into HTAs.

Conclusion: By addressing these challenges, HTA agencies could play a pivotal role in guiding decisions that align with environmental goals.

Objectives: Life-cycle health technology assessment (HTA) requires an index economic model to establish how estimated cost-effectiveness evolves with emerging evidence. We developed an open-source index economic evaluation of entrectinib, a tumor-agnostic therapy with conditional market authorization. Our objective was to replicate the initial HTA report from publicly available information, aiming to identify key operational and methodological aspects for operationalizing life-cycle decision-making.

Methods: We used partitioned survival analysis to determine tumor-agnostic and tumor-specific cost-effectiveness, using publicly available HTA reviews for parameterization. We estimated incremental costs in 2021 Canadian and US dollars (CAD and USD) from a public-payer healthcare perspective, quality-adjusted life years (QALYs), and incremental net monetary benefit (INMB). We assessed the impact of treatment effectiveness, extrapolation assumptions, and next-generation sequencing (NGS) costs.

Results: One-third of the parameters (n = 30) were unavailable in the Canadian reimbursement review and were sourced from international reviews. Tumor-agnostic incremental costs were CAD 68,451 (95 percent confidence interval: 35,466, 92,155) and USD 54,608 (28,294, 73,518), and QALYs were 0.13 (-0.42, 0.42), yielding INMB CAD -55,803 at 100,000/QALY (USD -44,518). Full extrapolation of treatment effectiveness also yielded negative INMB (CAD -66,664). Inclusion of NGS costs diminished the expected value. Heterogeneity was considerable across tumor indications.

Conclusions: We developed an open-source index economic evaluation to operationalize life-cycle HTA for a conditionally authorized tumor-agnostic therapy. Our findings outline key operational and methodological considerations necessary for the development of index economic models that support life-cycle HTA, offering insights into their potential integration into regular HTA and policy decision-making processes.

Objective: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

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.

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.