International Journal of Technology Assessment in Health Care最新文献

Objectives: The objective of this paper is to present the Environmental Sustainability in Health Technology Assessment (ESHTA) Working Group's (WG's) opinion on the definition and scope of early Health Technology Assessment (HTA) developed by a WG under HTA International. The aim is to provide suggestions on how early HTA can support the goals of enhancing environmental sustainability in healthcare.

Methods: The HTAi ESHTA WG presents our opinion on the proposed definition and scope of early HTA. This includes a broad range of perspectives from stakeholder groups including patient experts, a policy maker, a statistician, HTA researchers and a healthcare professional, located across lower to higher resource settings and several jurisdictions. We suggest how early HTA can support the goals of enhancing environmental sustainability in healthcare.

Results: HTA agencies play a crucial role in embedding sustainability into their evaluations and practices. Integrating environmental sustainability into HTA at three critical stages - product conceptualization, reimbursement decisions, and point of care - can optimize resource use and reduce environmental impacts. Developing sustainability metrics, defining environmental impact categories, and identifying suitable methods for assessing health technologies are essential steps. Early engagement is also vital for optimizing trade-offs and increasing acceptance by diverse stakeholders.

Conclusions: Incorporating environmental sustainability into early HTA can enhance the likelihood of regulatory approval and reimbursement, ultimately benefiting patients and healthcare systems. By integrating sustainability considerations at the design stage, the potential for environmental impact reduction is maximized. Future efforts should focus on developing comprehensive guidelines and methods, ensuring collaboration between early HTA and ESHTA WGs.

Background: Short-course regimens are currently explored to improve multidrug-resistant tuberculosis effects, reduce costs, as well as enhance patient adherence. Currently, we are determining the most cost-effective shorter regimen out of seven short-course regimens (6-9 months) to treat drug-resistant tuberculosis (DR-TB) compared to the current standard of care (SoC) 9- to 11-month regimen.

Methods: Cost-effectiveness of various short-course DR-TB treatment regimens, namely BEAT, BPaL, BPaLM, BPaLC, mBPaL1, mBPaL2, and mBPaL3, was compared to the current SoC in India. Decision tree model was used from a health system perspective. The information on various costs - such as preinvestigations, regimens, adverse drug reactions (ADRs) management, inpatient treatment - and on effect - such as clinical outcomes and ADRs - was collected from different published sources. It estimated costs, quality-adjusted life years, and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were performed to validate outcomes against the willingness-to-pay threshold.

Results: When all the short-course regimens were compared with the current SoC regimen, the ICERs were ₹5,385, ₹2,014, ₹2,008, ₹2,435, ₹1,462, ₹1,159, and ₹1,895 for BEAT, BPaL, BPaLM, BPaLC, mBPaL1, mBPaL2, and mBPaL3, respectively. Among the short-course regimens, mBPaL2 is the dominant strategy, and mBPaL1 has extended dominance. For all Bedaquiline-containing regimens, the cost of the drug is a crucial factor in determining cost effectiveness. The cost-effectiveness acceptability curve showed that all shorter regimens were 100 percent cost-effective.

Conclusion: The implementation of Bedaquiline-based regimen to treat DR-TB has become more effective, shorter in duration, and less burdensome to the health system.

This commentary examines how early health technology assessment can mitigate risks during the development of innovative technologies.

Objectives: Health economic evaluations are important for healthcare resource allocation. Reviews of health economic evaluations for medical devices have highlighted concerns about the quality of these studies. The complexity of medical devices, including learning curve effects, organizational impact, dynamic pricing, low evidence, and incremental innovation presents unique challenges compared with pharmaceuticals. To support developing a methodological quality assessment instrument for medical device economic evaluations, we conducted a systematic review to identify and evaluate existing economic evaluation quality assessment instruments for suitability in medical device evaluations.

Methods: A comprehensive search of databases (MEDLINE, EMBASE, EconLit, CINAHL, and Web of Science) and grey literature was conducted. Two reviewers screened titles and abstracts. Full-text, peer-reviewed primary studies introducing original instruments were included. Only methodological quality assessment instruments were considered for data extraction. Each item was assessed for its suitability in evaluating medical device economic evaluations and inclusion of medical device-specific features.

Results: The search identified 4203 citations and 77 grey literature sources. Fifteen results underwent full-text assessment, with five relevant instruments identified. A previous systematic review identified 10 additional instruments, which we also considered. Of these 25 articles, 13 were included in the review. These instruments lack specificity for medical devices, particularly in addressing features like learning curve effects, organizational impact, and incremental innovation. Instruments should include items specific to these unique characteristics.

Conclusions: Existing instruments contain general items related to health economic evaluation studies, highlighting the need for an instrument specifically tailored to evaluate the methodological quality of medical device economic evaluation studies.

Objective: To systematize the information and perspectives shared during the 2024 LATAM policy forum, which explored advancements in horizon scanning and early dialogue processes in the region, by analyzing the main discussion and identifying the main lessons.

Methods: This article is based on the discussions and background materials provided during the 1.5 days in-person 2024 Latin American Policy Forum (59 representatives from 11 countries). We gathered and systematized the information shared during the forum, including the results of a pre-forum survey. The Forum agenda included keynote presentations, breakout group activities, and plenary discussions to identify the main lessons and key messages from all different stakeholders' points of view.

Results: The forum highlighted the growing recognition of the need for structured horizon scanning and early dialogue processes in Latin America. Key barriers were identified, including the absence of clear legal frameworks, limited data availability, and the need for capacity-building. Potential solutions included fostering regional cooperation, improving transparency, and creating pilot programs for early engagement. Engaging patients and the pharmaceutical industry was deemed essential for trust and foster alignment between HTA agencies and regulators.

Conclusions: Horizon scanning and early dialogue represent critical tools for improving health system preparedness and aligning innovation with local needs. Their implementation, however, requires coordinated efforts across multiple stakeholders, enhanced dialogue, and the development of supportive legal and regulatory frameworks.

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.