International Journal of Technology Assessment in Health Care最新文献

Objectives: This assessment aimed to identify the degree and parameters of demand for support from HTAsiaLink, the Asia regional health technology assessment (HTA) hub, for HTA ecosystem development.

Methods: A sequential, exploratory, mixed-method design was implemented, starting with a literature review to define the Asia region's HTA landscape. Then an online survey was sent to 125 Asia-focused HTA practitioners and support organizations to obtain their thoughts on HTA development needs and how a regional hub could serve them. Finally, fifty purposively selected key informants representing government HTA agencies in Asia, funding partner organizations, philanthropic foundations, global HTA support, and regional HTA hub organizations were invited to participate in semi-structured interviews. Nineteen Asian countries and territories were represented in documents reviewed. Twenty-five recipients from ten Asian countries and territories responded to the survey, and twenty-eight individuals from eight Asian countries and territories plus eight international organizations participated in interviews.

Results: Identified needs include support to fill HTA human resources gaps, strengthen the capacity of the existing HTA workforce, produce HTA public goods, improve harmonization within and across country systems, and strengthen political will. Other important considerations include the need to adapt the hub's purpose to an expanding role and adopt sustainable financing approaches accordingly.

Conclusion: Demand for an HTA hub in Asia is high, including to support HTA technical, deliberative processes, and institutional capacity strengthening. Findings underscore the importance of both conducting HTAs and fostering demand for HTA output. HTAsiaLink is recognized as well-positioned to play an expanded support role to address these needs.

Real-world evidence (RWE) is increasingly used and accepted by health technology assessment (HTA) bodies as supportive evidence to inform the approval of new technologies. However, the criteria driving RWE acceptance are often unclear.This study aims to improve understanding of the role and value of RWE in HTA decision-making and outline the best practices in building real-world external control arms (ECAs).A mixed approach of a targeted literature review and HTA expert interviews was applied. The HTA reports of ten selected technologies and the expert interviews from France, Germany, Italy, Spain, and the UK informed the criteria driving the acceptance of RWE. Overall, the UK and Spanish HTA bodies are more receptive to accepting RWE, whereas the French and German are the least accepting. When RWE is used to substantiate efficacy claims, the level of scrutiny from regulators and HTA bodies is considerably higher than when RWE has different intended uses. Representativeness of the data source, overall transparency in the study and robust methodologies are the key criteria driving RWE acceptance across markets.

Objectives: To provide an overview of learning strategies that health technology assessment (HTA) agencies use worldwide to educate laypeople about HTA.

Methods: A scoping review focused on learning strategies to educate laypeople about HTA using the Joanna Briggs Institute frameworks was conducted across databases and gray literature. The study reviewed qualitative, quantitative, and mixed-methods studies from four databases, including practice documents from the HTA and health organization websites.

Results: Fifteen studies were included in this review. The United Kingdom, Spain, and Canada mainly contributed to knowledge about educating laypeople in HTA. The main strategies employed were conference-like events, educational materials, training, and plain language. International HTA and health agencies developed courses, online training, and guidance materials to increase laypeople's participation in the HTA process.

Conclusions: Efforts to improve public involvement in HTA focus on structured consultations, digital platforms, and capacity-building to enhance accessibility. Strategies like workshops and plain language aim to encourage lay participation, but challenges such as technical complexity and limited resources persist. Despite these challenges, incorporating patient perspectives has increased research relevance and public trust. Future studies should examine standardized frameworks for involvement, the impact of lay participation on policy, and solutions to barriers to a more equitable HTA process.

Introduction: Health technology assessment (HTA) reports are written for healthcare decision makers, particularly in relation to reimbursement/pricing, and are intended to assess clinical effectiveness, safety, and cost. Four additional domains are further considered in what is called a "full HTA": ethical, legal, social, and organizational aspects. The ethical aspects have long been the subject of debate regarding how they should be processed. It would be important if the following questions could be answered: Who publishes full HTA reports and how? Which methods are used in the ethics domain? What kind of results do they produce? However, such a "mapping of the field" turns out to be difficult. Despite the existence of international HTA registers, we were not able to compile a comprehensive sample of full HTA reports. Therefore, the aim of our study was rather to explore a) substantially: Which information can be expected to be (easily) found, which can only be obtained with considerable effort, and which remain (for the time being) in the dark? And b) methodologically: Is it possible to do meaningful meta-research in this field?

Methods and results: In the attempt to explore the possibilities of meta-research, we were able to track down and analyze thirty-nine full HTA reports from six countries.

Conclusions: While not representative of the whole field, this analysis shows the possibilities and challenges to meta-research, but nonetheless also provides some substantial insight into the characteristics of such reports, with a particular focus on the methods used to process ethical aspects.

Background: As Ethiopia advances towards efficient resource utilization and UHC through strategic health purchasing, the institutionalization of HTA will play a critical role. This study aims to identify key stakeholders, analyze the political economy surrounding HTA and priority setting in Ethiopia, and assess existing skills and capacities for a robust and sustainable HTA system.

Methods: We employed a mixed-method approach, combining 16 key informant interviews, 24 document reviews, and a cross-sectional survey (n=65) to assess national HTA capacity. We employed the Walt and Gilson policy analysis triangle framework, alongside Campos and Reich's framework, to evaluate the context, process, content, and actors influencing HTA institutionalization, and to explore the complex interplay of institutions, positions, power, and interests among various stakeholders.

Results: While there is a general commitment to implementing HTA across various government agencies and stakeholder groups, the institutionalization process faces several challenges, involving multiple agencies with overlapping mandates, raises bureaucratic challenges and potential conflicts, risking horizontal fragmentation as agencies compete for authority, budget, and influence. The involvement of other key stakeholders, such as professional associations, patients, and the public, is notably lacking. Challenges such as limited HTA expertise, high professional turnover, and gaps in specific HTA knowledge areas persist, with capacity-building efforts often failing to address organizational needs effectively.

Conclusions: The complexity of HTA institutionalization in Ethiopia underscores the necessity of managing intricate inter-agency dynamics, establishing a robust legal framework for an inclusive and transparent HTA process, building local capacity, and securing sustainable, domestically aligned funding.

Objectives: The aim of this policy article is twofold: (i) to provide a summary and update of recent important policy developments, in particular relevant guidance on the use of real-world data/real-world evidence (RWD/RWE) by health technology assessment (HTA) bodies and (ii) to set out our policy recommendations on how the different elements of an "RWE framework" we have previously developed could support, further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies and payers.

Methods: We undertook a targeted review and analysis of recent important policy developments. The aim was to build on our recommendations from previous work on the "RWE Framework," and consider how the relevant tools from our Framework can further enhance and facilitate the use of RWE for HTA purposes and by HTA bodies/payers.

Results: We provide eight conditions that we argue would, in combination, constitute the optimal use and acceptance of RWD/RWE for HTA. We believe that, should the eight conditions hold, RWD/RWE would enable more efficient access to medicines and healthcare technologies for patients.

Conclusions: High-quality, fit-for-purpose RWD/RWE can and should be used more frequently in HTA. Multi-stakeholder and cross-geography collaborative partnerships are needed to align on best practices to optimize the evidence that needs to be generated to satisfy all stakeholders' needs.

Background: Health technology assessment (HTA) is a form of policy analysis that informs decisions about funding and scaling up health technologies to improve health outcomes. An equity-focused HTA recommendation explicitly addresses the impact of health technologies on individuals disadvantaged in society because of specific health needs or social conditions. However, more evidence is needed on the relationships between patient engagement processes and the development of equity-focused HTA recommendations.

Objectives: The objective of this study is to assess relationships between patient engagement processes and the development of equity-focused HTA recommendations.

Methods: We analyzed sixty HTA reports published between 2013 and 2021 from two Canadian organizations: Canada's Drug Agency and Ontario Health.

Results: Quantitative analysis of the HTA reports showed that direct patient engagement (odds ratio (OR): 3.85; 95 percent confidence interval (CI): 2.40-6.20) and consensus in decision-making (OR: 2.27; 95 percent CI: 1.35-3.84) were more likely to be associated with the development of equity-focused HTA recommendations than indirect patient engagement (OR: .26; 95 percent CI: .16-.41) and voting (OR: .44; 95 percent CI: .26-.73).

Conclusion: The results can inform the development of patient engagement strategies in HTA. These findings have implications for practice, research, and policy. They provide valuable insights into HTA.

Objectives: Effective allocation of scarce healthcare resources involves complex ethical and technical evaluations, with decision makers sometimes utilizing a societal perspective in health technology assessment (HTA). This study aimed to explore societal perspectives on healthcare resource allocation within Australia's HTA framework, focusing on the valuation of health gains for children and young people (CYP) compared to adults.

Methods: In-depth, semistructured interviews were conducted with ten young people (aged 15-17) and twenty adults between October 2021 and April 2022. Participants were purposively sampled for diverse characteristics and completed an online information survey prior to the interviews, introducing relevant concepts. Interviews were analyzed using inductive coding, categorization, and constant comparison.

Results: Participants expressed nuanced perspectives on HTA processes, generally opposing numeric weighting and preferring a deliberative approach based on committee judgment. Although most participants acknowledged some moral relevance of CYP status in HTA, opinions varied on its operationalization. A sizable minority, including those with extensive health system experience, did not view CYP status as morally relevant, though some noted specific service gaps for CYP (e.g., mental health care, pain management). Participants identified a spectrum of factors, both person-centered and intervention related, that often surpassed the relevance of CYP status, including addressing severity, unmet needs, prevention, and early intervention, with an emphasis on Aboriginal and Torres Strait Islander communities.

Conclusion: Our findings highlight the inherent challenges in navigating the complexities of HTA and the critical need for HTA frameworks to be adaptable and inclusive, effectively integrating societal preferences to enhance healthcare policy's equity and responsiveness.

Objectives: Cost-effectiveness models fully informed by real-world epidemiological parameters yield the best results, but they are costly to obtain. Model calibration using real-world data/evidence (RWD/E) on routine health indicators can provide an alternative to improve the validity and acceptability of the results. We calibrated the transition probabilities of the reference chemotherapy treatment using RWE on patient overall survival (OS) to model the survival benefit of adjuvant trastuzumab in Indonesia.

Methods: A Markov model comprising four health states was initially parameterized using the reference-treatment transition probabilities, obtained from published international evidence. We then calibrated these probabilities, targeting a 2-year OS of 86.11 percent from the RWE sourced from hospital registries. We compared projected OS duration and life-years gained (LYG) before and after calibration for the Nelder-Mead, Bound Optimization BY Quadratic Approximation, and generalized reduced gradient (GRG) nonlinear optimization methods.

Results: The pre-calibrated transition probabilities overestimated the 2-year OS (92.25 percent). GRG nonlinear performed best and had the smallest difference with the RWD/E OS. After calibration, the projected OS duration was significantly lower than their pre-calibrated estimates across all optimization methods for both standard chemotherapy (~7.50 vs. 11.00 years) and adjuvant trastuzumab (~9.50 vs. 12.94 years). LYG measures were, however, similar (~2 years) for the pre-calibrated and calibrated models.

Conclusions: RWD/E calibration resulted in realistically lower survival estimates. Despite the little difference in LYG, calibration is useful to adapt external evidence commonly used to derive transition probabilities to the policy context, thereby enhancing the validity and acceptability of the modeling results.

Objectives: Hospital-Based Health Technology Assessment (HB-HTA) is a heterogeneous phenomenon constantly evolving to respond to the needs of decision-makers at the hospital level. In 2023, The HB-HTA Interest Group of Health Technology Assessment International (HTAi) surveyed HB-HTA activities with the aim to provide an updated description of the actual scenario.

Methods: An online survey was conducted to gather data on the main characteristics of hospitals, HB-HTA activities, outputs, role in the decision-making processes, dissemination and training activities, and their interaction and collaboration with other stakeholders and HTA-related regulations. Finally, the survey collected feedback on the perception of and current barriers to HB-HTA. Three categories of responders were identified: Both hospitals performing and not performing HTA and policymakers.

Results: Eighty-seven responses were collected from twenty-eight countries. Nearly half of the responders (n = 41) conducted HB-HTA, whereas eighteen consisted of hospitals not performing HTA, and twenty-eight were policy makers. HB-HTA was performed mainly in hospitals with >500 beds. HB-HTA units were organized in 40 percent of cases as an "independent group." The survey showed that HTA units could contribute to all the steps of the decision-making processes, whereas the impact of the assessments on the decisions was mainly perceived as a medium. Furthermore, HB-HTA was not seen as a duplication of effort, even without specific regulations.

Conclusions: The survey highlighted the role of HB-HTA in hospital decision-making supporting the vision of HB-HTA as one of the actors in the HTA ecosystem, the success of which depends on collaboration with other stakeholders.