International Journal of Technology Assessment in Health Care最新文献

Objectives: Value frameworks play a crucial role in bridging the gap between evidence and decision making in health care, particularly in settings with limited resources as low- and middle-income countries (LMIC). In this study, we present the development of a value framework (VF) targeted to provide coverage recommendations in rapid health technology assessment reports (rHTA) as well as its first 5 years of implementation.

Methods: We performed an exhaustive literature search with the aim to identify existing VFs and their dimensions followed by the generation of a VF proposal through a mixed methods, qualitative-quantitative approach including a Delphi panel to weigh the criteria and correlate them with the subsequent recommendations. To describe its implementation, we present the results of 264 rHTA reports from 2017 to 2022.

Results: The value framework has three main domains (quality of evidence, net benefit, and economic impact). We adapted widely used methodologies for quality of evidence and net benefit domains. The economic impact domain was the most complex to assess, so an ad hoc method was developed. Analysis of 265 HTAs revealed the distribution of recommendations across different criteria and technology types. Most were for drugs (40.5 percent) or therapeutic procedures (36 percent). With a five-category final recommendation, 0.8 percent were favorable, 19.7 percent were uncertain, and 44 percent were unfavorable.

Conclusion: The VF demonstrated its versatility and practicality in meeting the needs of rHTA audience, and can facilitate evidence-informed decision making. This VF serves as a valuable tool for conducting adaptive rHTAs and supports decision-making processes in Argentina and similar LMIC contexts.

The HTAi Health Technology Assessment (eHTA) Working Group's (WG) development of a consensus definition of early eHTA, as reported in Grutters et al. (1), represents a major step towards the establishment of eHTA as a distinct subdiscipline of HTA. In a global landscape in which growth in pharmaceutical spending is driven by the increasing number of high-cost specialty drugs (2-6), and where the cost of new entrants is not systematically associated with their clinical benefit (7;8), broader uptake of eHTA by pharmaceutical innovators offers a route to improving the value delivered by our collective investments in drug research and development (R&D). As we argue in this commentary, the WG's report provides a coherent framework within which to further define appropriate eHTA methods for specific use cases as well as eHTA's relationship to other decision-making tools currently used by health technology innovators and funders.

Objectives: The increasing relevance of horizon scanning (HS), early awareness (EA), and disinvestment (DIS) highlights the need for a structured approach to capacity building. Although these fields are essential for evidence-based policy decisions, a harmonized education and training framework to develop necessary competencies is lacking. This article presents the development of a curriculum designed to address this gap in training.

Methods: A transdisciplinary working group was established, drawing on international stakeholders from academia, the public sector, and industry. Using an iterative consensus-driven approach, the group developed a modular curriculum. The curriculum design incorporated best practices from existing education programs in related fields and emphasized case-based learning strategies to ensure contextual adaptability.

Results: The resulting curriculum covers theoretical foundations, practical applications, and decision-making processes related to HS, EA, and DIS in eight modules. It supports diverse learner needs, including trainees, training institutions, and public and private organizations, and is designed to be flexible, scalable, and applicable across different regional and organizational contexts.

Conclusions: This curriculum initiative represents a major step toward harmonizing capacity building in HS, EA, and DIS. It fosters sustainability, enhances global health system preparedness, and provides a structured educational platform to support the effective integration of emerging health technologies and evidence-based disinvestment strategies.

Objectives: Prioritization of health technologies for insurance coverage is usually based on explicit and implicit criteria. This study presents the development of the multi-criteria decision analysis (MCDA) model, the Iranian Health Insurance Benefit Optimization Model (IR-HIBOM), to inform the design of basic health insurance benefit packages.

Methods: An initial set of twenty-nine potential allocation criteria was identified through a review of available evidence and other relevant literature. Review of this set by three specialized panels yielded a final set of thirteen criteria. A cross-sectional survey using the best-worst scaling method was then fielded to 163 health system experts to evaluate their preferences regarding the relative importance of the allocation criteria. The mixed logit method was employed to determine the weight of the relative importance of each criterion. Subsequently, a multilevel criteria scoring framework was defined based on a review of similar models and input from a panel of five expert members of the study team. Finally, model's appraisal was conducted.

Results: Thirteen criteria, including relative safety, efficacy, disease severity, access to alternative health technologies, budget impacts, cost-effectiveness, quality of evidence, population size, age, job absenteeism, economic status, daily care needs, and ease-of-use/acceptance were selected. Cost-effectiveness and ease-of-use criteria had the highest and lowest relative importance weights, with 30.5 percent and 1 percent, respectively. Furthermore, scores were determined for the several levels of each criterion, and decision rules were defined for the cost-effectiveness and budget impact criteria. The final model's appraisal, based on weighted scores of thirteen selected technologies, indicated that it was valid and applicable.

Conclusions: The IR-HIBOM demonstrated its potential utility in the health resource allocation.

Objectives: Medicines and devices have significant negative impacts on the environment. Increasingly, Health Technology Assessment (HTA) agencies, which inform healthcare decision making, are expected to integrate environmental issues into their assessments. This study assessed how HTA agencies have responded to these calls, with a focus on two national agencies that have committed to this agenda.

Methods: This descriptive study was conducted using document review. All relevant documents from both agencies were systematically collected and analyzed using descriptive statistics and content analysis.

Results: Thirty documents (2015-2024), from Canada's Drug Agency (CDA) (17) and England's National Institute for Health and Care Excellence (NICE) (13) that included environmental considerations were analyzed. Although no HTAs have assessed environmental data, primarily due to a lack of data and methods, documents demonstrate that CDA and NICE are employing varied strategies to incorporate environmental considerations through technology guidance. The agencies demonstrate both differences and similarities in approach: NICE focused on carbon and the use phase, whereas CDA focused on multiple environmental impacts across the lifecycle; both agencies are beginning to address the environmental impacts of devices, but there is a notable absence of medicines-related work.

Conclusions: This study demonstrates that the agencies are exploring alternative strategies to elevate attention to the environmental impacts of health technologies. Differences in focus (e.g., whether to prioritize carbon emissions) and shared inattention to medicines point to deeper tensions. Thus, although both agencies have taken important steps forward, much work remains to fully address the environmental harms of health technologies.

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.