OP523 Towards A Health Technology Assessment Framework For Omics-Technologies: Preliminary Results Of The ExACT project.

IntroductionThe EDiHTA project aims to deliver an innovative health technology assessment (HTA) framework for digital health technologies (DHT) that integrates existing methods with new ones to inform decision-making across Europe at different levels. In the development phase of the EDiHTA framework, it has been essential to identify all relevant stakeholders and their roles in the innovation process, while also capturing their needs and requirements.MethodsThe Innovation of Health Technology Assessment Methods framework was applied to identify relevant stakeholders and their needs and requirements regarding the design of the EDiHTA framework. Once stakeholders and their roles (practitioners or beneficiaries) were identified, their needs were assessed by reviewing existing and new HTA methodologies for DHT as used in 17 countries across Europe. Future scenarios were examined and gaps to be addressed were determined. Various methods were used to elicit the views of different stakeholders (policymakers, HTA agencies and bodies, industry, healthcare professionals, patients). including literature reviews, two online surveys, semi-structured online interviews, and focus groups.ResultsWe engaged policymakers from 15 European countries; 15 HTA agencies from nine European countries; 29 developers (startups and big companies) from 10 different European markets; 14 healthcare professionals from nine European countries plus eight from Brazil, Canada, and the USA; and 15 patient representatives from 10 European countries. The analysis highlighted the heterogeneity among European health systems in assessing, implementing, and reimbursing DHT. All stakeholder groups emphasized the need for a harmonized HTA framework to assess DHT efficiently and accurately, including elements specific to DHT, such as cybersecurity, economic and organizational impact, and patient-centered outcomes.ConclusionsThere is a clear need among European stakeholders for an agile, flexible, and multidimensional HTA framework that facilitates stepwise assessments across the technology life cycle and involves all relevant stakeholders in the assessment to inform decision-making. The stakeholder interaction also identified new HTA topics and domains essential for effectively addressing the unique challenges of DHT within the EDiHTA framework.

The assessment of health technology offers a framework to analyze whether the costs of health interventions match benefits to patients. Achieving universal health coverage is an important goal for Rwanda. When choosing which new medical technology (medical devices) to adopt, health technology assessment processes should be applied. The objective of this study was to create health technology assessment framework for Rwanda. Quantitative study was conducted among 45 participants from unknown population by using systematic sampling technique. A self-made survey instrument called questionnaire was created for data collection. Data analysis was performed using Statistical Package for Social Sciences software version 21. Significance and correlation of health technology assessment application in healthcare decision-making were tested using binary logistic regression analysis and chi-square test, respectively. Based on the outcomes attained, 66.7% suggested that health facilities should use health technology assessment in decision-making based on health technology assessment framework. Chi-square (x2 ) test and Binary logistic analysis test showed the significant correlation between health technology assessment framework and willingness to use health technology assessment in decision-making Health technology assessment has a well-recognized potential to enhance healthcare decision-making in Rwanda. And for future research studies, proposed health technology assessment framework needs to be validated through consultation with experts and policymakers.

ObjectivesTo develop a context-specific health technology assessment (HTA) framework tailored to the healthcare needs and system of Iran, to improve evidence-based decision-making, optimise resource allocation and support progress towards universal health coverage.DesignA mixed-methods Delphi consensus study conducted using a three-phase, sequential approach: document review, qualitative focus group discussions and Delphi consensus rounds. The study reporting follows the Accurate Consensus Reporting Document guideline to ensure transparent reporting of consensus methods.SettingA national-level study conducted in Iran’s healthcare system between January 2023 and March 2024, including perspectives from public and academic institutions, policy bodies and patient organisations.ParticipantsThe study involved 18 purposively selected stakeholders in three focus group discussions, including policymakers, healthcare professionals, researchers and patient representatives. Subsequently, 20 HTA experts participated in three iterative Delphi rounds to refine and reach consensus on the framework components.Outcome measuresIdentification of core components and operational steps required to develop and implement a comprehensive HTA framework in Iran.ResultsThe final HTA framework includes nine core components: (1) establishing a national HTA body; (2) engaging stakeholders; (3) building capacity through training and research; (4) developing standard HTA methodologies; (5) implementing prioritisation and evaluation processes; (6) ensuring sustainable funding; (7) enhancing transparency and accountability; (8) promoting continuous improvement and (9) fostering innovation. Detailed operational steps and micro-activities were developed for each component. The framework achieved an 84% consensus among Delphi panellists, indicating strong agreement on its content and applicability.ConclusionsThis tailored HTA framework provides a structured roadmap to institutionalise evidence-based decision-making in Iran’s healthcare system. Its implementation can strengthen the efficiency, equity and sustainability of healthcare planning and policy. Pilot testing is recommended to assess feasibility and scalability, with potential to serve as a model for other low-income and middle-income countries.

IntroductionThe widespread adoption of digital health technologies (DHTs) brings new methodological challenges to health technology assessment (HTA). Different tools, guidelines, and methods have been defined by different stakeholders at national/international levels. In this scenario, the real thread for HTA is the lack of coordination and harmonization in the efforts to finalize the definition of an HTA framework for DHTs.MethodsThe European Digital Health Technology Assessment (EDiHTA) project aims to provide the first framework co-created by all stakeholders along the value chain for DHTs. The consortium is comprised of 15 partners from nine countries (Italy, the Netherlands, Denmark, Spain, Germany, Norway, UK, Belgium, and Poland). Stakeholders included are HTA agencies, regulators, hospitals, patient/citizen representatives, academics/universities, notified bodies, and developers. The project covers all types of DHTs and Technology Readiness Levels (TRLs). A multistakeholder, multidomain, and modular approach is adopted with the final goal to provide a validated, ready-to-use digital HTA framework for DHTs for the European ecosystem.ResultsThe EDiHTA is articulated in eight working packages and covers four years. Starting from the mapping of the current scenario, after the definition of a common taxonomy, EDiHTA will develop a DHT-dedicated HTA framework. A common digital platform to support the assessment will be developed. Both the HTA framework and platform will be tested, refined, and validated through a piloting process involving disparate DHTs (mApp, telemedicine, artificial intelligence, and others) with different TRLs, available in different countries, with the participation of all stakeholders. As a consequence, evidence requirements will be defined and made more transparent; this will support health technology.ConclusionsThe EDiHTA project will deliver the first European digital HTA framework able to support and inform decision-making (at the macro [policy], meso [management providers], and micro [clinicians] level) towards faster and safer access to DHTs of added value, taking European regulations and legal requirements into account and assessing all HTA domains.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil. A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients). Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were "relevance of outcomes for a rare disease," "impact on patient's quality of life," "price negotiation," and "adjusted cost-effectiveness threshold." On the other hand, "budget impact threshold," "innovative nature of treatment," and "willingness to accept greater uncertainty in clinical evidence" received negative evaluations and were excluded from the final framework. A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.

BackgroundArtificial intelligence (AI)–based medical devices have garnered attention due to their ability to revolutionize medicine. Their health technology assessment framework is lacking.ObjectiveThis study aims to analyze the suitability of each health technology assessment (HTA) domain for the assessment of AI-based medical devices.MethodsWe conducted a scoping literature review following the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) methodology. We searched databases (PubMed, Embase, and Cochrane Library), gray literature, and HTA agency websites.ResultsA total of 10.1% (78/775) of the references were included. Data quality and integration are vital aspects to consider when describing and assessing the technical characteristics of AI-based medical devices during an HTA process. When it comes to implementing specialized HTA for AI-based medical devices, several practical challenges and potential barriers could be highlighted and should be taken into account (AI technological evolution timeline, data requirements, complexity and transparency, clinical validation and safety requirements, regulatory and ethical considerations, and economic evaluation).ConclusionsThe adaptation of the HTA process through a methodological framework for AI-based medical devices enhances the comparability of results across different evaluations and jurisdictions. By defining the necessary expertise, the framework supports the development of a skilled workforce capable of conducting robust and reliable HTAs of AI-based medical devices. A comprehensive adapted HTA framework for AI-based medical devices can provide valuable insights into the effectiveness, cost-effectiveness, and societal impact of AI-based medical devices, guiding their responsible implementation and maximizing their benefits for patients and health care systems.

This article provides an overview of Ecuador’s Methodological Manual for Economic Evaluations of Health Technologies, emphasizing its importance, key methodological aspects, and relevance within the country’s health technology assessment (HTA) framework. The manual establishes standardized guidelines for conducting economic evaluations, incorporating international best practices while adapting them to the national context. It outlines essential methodological components, including decision problem definition, economic evaluation types, cost estimation, use of decision models, and sensitivity analysis. Additionally, it highlights the role of quality-adjusted life years (QALYs) and cost-effectiveness thresholds in supporting evidence-based decision-making. Beyond its methodological contributions, the manual plays a crucial role in institutionalizing economic evaluations in Ecuador’s healthcare system. By promoting transparency, consistency, and technical rigor, it strengthens the integration of economic evidence into policy decisions, ensuring efficient resource allocation. However, challenges persist, including limitations in technical capacity, data availability, and the need for further refinement of local cost-effectiveness thresholds and indirect cost valuation. This article also explores the broader policy implications of adopting the manual within Ecuador’s HTA framework. Strengthening institutional capacity, improving data infrastructure, and fostering international collaboration will be essential to its successful implementation. By embedding economic evaluations into healthcare decision-making, Ecuador can enhance the sustainability of its health system, optimize expenditures, and improve access to cost-effective health technologies.

Evaluation of Health Technology Assessment Frameworks for In Vivo Diagnostics: Assessing Methodological Gaps and Implications for Market Access.

PNS66 NON-INFERIORITY TRIALS IN HEALTH TECHNOLOGY ASSESSMENT - DO THEY NECESSITATE A COST-MINIMISATION APPROACH?

Radiotheranostic therapy (RTT) is an emerging form of personalized cancer care that combines radiopharmaceutical imaging and radiopharmaceutical therapy (RPT). Although promising clinically, the value for money of RTT remains uncertain. The objective of this study is to review the cost-effectiveness evidence of RTT in cancer and critically appraise the methodologies utilized in conducting the included economic evaluations. Methods: A systematic literature review was conducted in accordance with the PRISMA guidelines. A comprehensive search strategy encompassing key search terms related to economic evaluations of RTT in oncology was performed across 7 electronic databases (CINAHL, Econlit, Embase, PubMed, Scopus, Web of Science, and International HTA database), as well as databases of health technology assessment (HTA) agencies (Canada's Drug Agency, Medical Services Advisory Committee, and the National Institute for Health and Care Excellence). The final search was conducted in September 2025 and covered published articles, reports, and HTA appraisals from 2015 to 2025. Methodologic quality was assessed using the CHEQUE framework, whereas reporting quality was evaluated using the CHEERS checklist. Results: Sixteen articles or reports and 9 HTA appraisals were included. Among the radiopharmaceutical imaging studies, 80% evaluated 68Ga whereas all RPT studies assessed 177Lu. Half of the model-based economic evaluations focused on prostate cancer and the rest on neuroendocrine tumors. The modeling approach in the evaluations varied, and the reported incremental cost-effectiveness ratio ranged from $21,350 to $146,526/quality-adjusted life-year for radiopharmaceutical imaging and from $66,761 to $397,342/quality-adjusted life-year for RPTs. HTA agencies identified uncertainties in evidence, comparator choice, and model assumptions and highlighted the absence of real-world considerations such as supply, workforce, and infrastructure constraints. Conclusion: This review identifies substantial heterogeneity and methodologic gaps in the economic evaluation of RTTs, contributing to uncertainty around their value for money. These findings underscore the need for fit-for-purpose HTA frameworks that integrate the full theranostic pathway and address system-level constraints and equity considerations.

Introduction: Health technology assessment (HTA) aims to provide decision makers with relevant data to make informed choices. Recent changes in the Portuguese HTA framework have altered substantially the assessment methodology with regard to economic evaluation, with potential impacts on access and process efficiency. The HTA Reshaping Project had as its main objective informing the debate on HTA in Portugal, thereby identifying improvement opportunities and solutions to the HTA framework that address future challenges. Methods: The project comprised several phases, i.e., (1) mapping and evaluation of different HTA frameworks across Europe, identifying best practices and key variables to consider in future frameworks; (2) conduction of in-depth interviews with relevant stakeholders (n = 11); and (3) development of 2 workshops – one with young professionals (n = 12) and another with relevant HTA stakeholders (n = 19) – to consolidate and further explore vital elements of HTA, aimed at brainstorming ideas and developing solutions to improve some of the most critical points, with a view to addressing future challenges. Results: The comparison of HTA frameworks showed that their purpose and sophistication vary across European countries. For example, the need for economic evidence is not unanimous, and reimbursement agreements vary considerably. Among the stakeholders interviewed there was a high level of agreement on priorities that should be addressed, e.g., expanding and creating national level registries and assuring patient participation throughout the HTA process. The possibility of using managed entry agreements to enhance patients’ access, applying multi-indication pricing for medicines with different therapeutic values per indication, and improvement of registry/system interoperability gathered a moderate level of agreement. Conclusions: The Portuguese HTA framework might be further adapted to upcoming challenges and should evolve to improve access to innovative therapies. There is still a long path towards the convergence of HTA frameworks in EU member states.

The objectives of the study were to establish a benchmarking tool to collect metrics to enable increased clarity regarding the differences and similarities across health technology assessment (HTA) agencies, to assess performance within and across HTA agencies, identify areas in the HTA processes in which time is spent and to enable ongoing performance improvement. Common steps and milestones in the HTA process were identified for meaningful benchmarking among agencies. A benchmarking tool consisting of eighty-six questions providing information on HTA agency organizational aspects and information on individual new medicine review timelines and outcomes was developed with the input of HTA agencies and validated in a pilot study. Data on 109 HTA reviews from five HTA agencies were analyzed to demonstrate the utility of this tool. This study developed an HTA benchmarking methodology, comparative metrics showed considerable differences among the median timelines from assessment and appraisal to final HTA recommendation for the five agencies included in this analysis; these results were interpreted in conjunction with agency characteristics. It is feasible to find consensus among HTA agencies regarding the common milestones of the review process to map jurisdiction-specific processes against agreed metrics. Data on characteristics of agencies such as their scope and remit enabled results to be interpreted in the appropriate local context. This benchmarking tool has promising potential utility to improve the transparency of the review process and to facilitate both quality assurance and performance improvement in HTA agencies.

INTRODUCTION:Priority setting in health care has been long recognized as an intrinsically complex and value-laden process. Yet, Health Technology Assessment (HTA) agencies presently employ value assessment frameworks that are ill-fitted to capture the range and diversity of stakeholder values, and thereby risk to compromise the legitimacy of their recommendations. We propose ‘evidence-informed deliberative processes’ as an alternative framework with the aim to enhance this legitimacy.METHODS:The framework is based on an integration of two increasingly popular and complementary frameworks for priority setting: multi-criteria decision analysis (MCDA) and accountability for reasonableness (A4R), Evidence-informed deliberative processes are, on the one hand, based on early, continued stakeholder deliberation to learn about the importance of relevant social values. On the other hand, they are based on rational decision-making – through evidence-informed evaluation of the identified values.RESULTS:The framework has important implications for how HTA agencies should ideally organize their processes. Firstly, HTA agencies should take the responsibility to organize stakeholder involvement. Second, agencies are advised to integrate their assessment and appraisal phase, allowing for the timely collection of evidence on values that are considered relevant. Third, HTA agencies should subject their specification of decision-making criteria to public scrutiny. Fourth, agencies are advised to use a checklist of potentially relevant criteria, and to provide argumentation how each criterion affected the recommendation. Fifth, HTA agencies must publish their argumentation and install options for appeal.CONCLUSIONS:Adopting ‘evidence-informed deliberative processes’ as a value assessment framework could be an important step forward for HTA agencies to optimize the legitimacy of their priority setting decisions. Agencies can incorporate elements according to their needs and affordances.

PHP308 - Institute for Clinical and Economic Review (ICER) VS. Health Technology Assessment (HTA) Agencies: The Case of Multiple Myeloma and PCSK9S

Recommendations by countries' health technology assessment (HTA) agencies are used to decide which new therapies warrant the allocation of limited health-care resources to make them available through publicly funded health systems. This process is of public health importance for balancing the dual aims of optimising patient outcomes while ensuring financial sustainability. We evaluated which factors affect HTA outcomes and the time to positive HTA outcome, focussing on the role of clinical benefit evaluated with the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS). In this retrospective analysis, data were extracted from publicly available HTA reports and related sources from six country settings and their respective HTA agencies (Australia, Canada, England, France, the Canadian province of Quebec, and Scotland). We evaluated new cancer medicines for treating solid tumours in a non-curative setting with published ESMO-MCBS scores and that had been assessed by at least three HTA agencies between Jan 1, 2011, and Dec 31, 2020. Using ESMO-MCBS score as an independent variable, we did descriptive and multivariable regression analyses to evaluate: (1) factors associated with the time between marketing authorisation and positive (unrestricted [List] and restricted [List with Constraints]) HTA outcome; and (2) factors associated with HTA outcomes. 67 medicine-indication pairs used in non-curative settings were identified, totalling 360 HTA submissions (medicine-indication-country triplets) reviewed by the six HTA agencies. Factors significantly associated with a reduced interval between marketing authorisation and a positive (unrestricted or restricted) HTA outcome included a high ESMO-MCBS score (ie, 4 or 5, vs a low or average score of 1-3; hazard ratio [HR] per 1 month increment 1·42 [95% CI 1·11-1·81], p=0·0055), parallel review (vs standard marketing authorisation process; HR 1·69 [1·13-2·54], p=0·011), having a risk-sharing agreement or special funding arrangements (vs no funding agreement, HR 4·62 [95% CI 2·51-8·51], p<0·0001, and HR 4·16 [2·03-8·50], p=0·0001, respectively), and assessment by particular HTA agencies (pan-Canadian Oncology Drug Review vs National Institute for Health and Care Excellence [NICE], HR 2·82 [1·68-4·75], p=0·0001; and Haute Autorité de Santé vs NICE, HR 5·70 [2·87-11·33], p<0·0001). Accelerated marketing authorisation was significantly associated with a longer time to positive HTA outcome (vs standard authorisation process; HR 0·70 [95% CI 0·51-0·95], p=0·024). Positive HTA outcomes (both unrestricted and restricted) were significantly associated with a high ESMO-MCBS score (vs low or average ESMO-MCBS score; relative risk ratio [RRR] 14·10 [95% CI 3·54-56·20], p=0·0002, and RRR 4·52 [1·90-10·75], p=0·0006, respectively) and acknowledgment of unmet medical need (vs unmet need not recorded, RRR 22·73 [5·51-93·73], p<0·0001, and RRR 1·87 [1·18-2·97], p=0·0075, respectively). By contrast, positive HTA outcomes (unrestricted and restricted) were inversely associated with uncertainties regarding inputs to economic models informing HTA submissions (vs uncertainties not recorded, RRR 0·28 [0·10-0·78], p=0·014, and RRR 0·45 [0·25-0·82], p=0·010, respectively). Regarding country-relevant effects, inverse associations with positive HTA outcomes (both unrestricted and restricted) were observed for assessment in Quebec (vs England; RRR 1·15×10-6 [1·44×10-7-9·09×10-6], p<0·0001, and RRR 0·33 (0·24-0·46), p<0·0001, respectively) and for assessment in Australia (vs England; RRR 1·78×10-6 [1·04×10-8-3·00×10-4], p<0·0001, and RRR 0·30 [0·15-0·61], p=0·0008, respectively). Several factors informed HTA outcomes for new cancer medicines. A high ESMO-MCBS score, defined as indicating substantial clinical benefit, increased the likelihood of a positive HTA outcome and shortened the interval between marketing authorisation and HTA outcome, and this association was not affected by other variables. Additional factors informing HTA outcomes include evidence uncertainties and unmet medical need. Country-relevant differences exist in the time-to-HTA outcome and the propensity of some countries to achieve positive (restricted or unrestricted) outcomes compared with others. None.