A roadmap towards implementing health technology assessment in Oman.

PURPOSE: Health technologies are advancing rapidly and becoming more expensive, posing a challenge for financing healthcare systems. Health technology assessment (HTA) improves the efficiency of resource allocation by facilitating evidence-informed decisions on the value of health technologies. Our study aims to create a customized HTA roadmap for Oman based on a gap analysis between the current and future status of HTA implementation. DESIGN/METHODOLOGY/APPROACH: We surveyed participants of an advanced HTA training program to assess the current state of HTA implementation in Oman and explore long-term goals. A list of draft recommendations was developed in areas with room for improvement. The list was then validated for its feasibility in a round table discussion with senior health policy experts to conclude on specific actions for HTA implementation. FINDINGS: Survey results aligned well with expert discussions. The round table discussion concluded with a phasic action plan for HTA implementation. In the short term (1-2 years), efforts will focus on building capacity through training programs. For medium-term actions (3-5 years), plans include expanding the HTA unit and introducing multiple cost-effectiveness thresholds while from 6-10 years, publishing of HTA recommendations, critical appraisal reports, and timelines is recommended. ORIGINALITY/VALUE: Although the HTA system in Oman is still in its early stages, strong initiatives are being taken for its advancement. This structured approach ensures a comprehensive integration of HTA into the healthcare system, enhancing decision-making and promoting a sustainable, evidence-based system addressing the population's needs.

Automated Drugs Dispensing Systems in Hospitals: a Health Technology Assessment (HTA) Study Across Six European Countries.

Background: Automated Drug Dispensing (ADD) systems are considered to be strategic hospital assets used to reduce errors and enhance economic and organizational sustainability. With regards to efficacy and safety, the literature evidence demonstrates the incremental benefits of centralised or decentralised systems compared to manual dispensing. Analyses about organisational and economic sustainability are still lacking and the present study aims to perform a Health Technology Assessment (HTA), producing multidimensional evidence on the use of ADD systems within hospitals. Methods: In 2023, a comprehensive HTA draws insights from healthcare professionals across six European nations: Italy, France, Germany, the Netherlands, the United Kingdom, and Belgium. This appraisal juxtaposed four drug dispensing scenarios: manual methods, centralized ADD systems, decentralized ADD systems, and integrated solutions employing cutting-edge technologies in both central pharmacies and wards. The study deployed an Activity-Based Costing approach that was combined with a cost-effectiveness and Budget Impact Analysis to evaluate economic impacts. Qualitative questionnaires were implemented to assess ethical, legal, organizational, safety, and efficacy aspects. Results: From a multidimensional perspective, healthcare professionals acknowledged ADD manifold advantages of ADD systems. From an organizational perspective and within a 12-month timeframe, transitioning to automation may face initial challenges that are attributed to potential resistance from professionals and significant investments. However, 36 months past its adoption, automation's superiority over manual methods was recognized. Economically, savings burgeoned from +17.9% in UK to +26.6% in Belgian hospitals that adopted integrated systems in comparison to traditional manual approaches. Conclusion: Compared to traditional methods, implementing ADD systems could improve the logistic management of drug in the hospital setting, thereby enhancing safety and efficacy, streamlining the healthcare professionals' workflow, and bolstering financial stability.

Application of health technology assessment: a new need for an ethical neonatology.

New health technologies are constantly developing. However, their impacts on health and implications for health systems are not always clear. Faced with this situation, in the last 45 years, Health Technology Assessment (HTA) has taken an important role in the decision-making process related to the implementation of technologies in healthcare systems. According to the Core Model® EUnetHTA, a "full HTA" should cover nine domains: health problems and current use of technology, description and technical characteristics, safety, clinical effectiveness, costs and economic evaluation, ethical analysis, organizational aspects, patients and social aspects, legal aspects. In all domains of a HTA, the approach is evidence-based and uses epidemiological data, systematic reviews to gather the best level of proofs regarding clinical efficacy and safety of interventions and comparators and organizational and economic models. One exception is the ethical analysis, that uses value-based approach. Although in Neonatal Intensive Care Units (NICUs) there are highly advanced technological environments, HTA has not yet been widely used in this field for determining the "value" of the diagnostic and therapeutic procedures. An example of diagnostic tool used in NICUs is the near-infrared spectroscopy (NIRS), a noninvasive device that enables real-time monitoring of the condition of peripheral tissues in critically ill newborns. The availability of this diagnostic tool could improve the choice of the most appropriate treatment to the clinical situation of the newborn. The expected benefit of NIRS motivates the need of a full HTA. Conclusion: HTA is still little used in Neonatal Critical Care, but it may be the appropriate tool to determine the "value" of technologies used in this field. The implementation of clinical trials and HTA may help in an evidence-based evaluation of new technologies for the neonatal critical care. This could facilitate the rapid introduction of the best health technologies into clinical practice. What is Known: • Health Technology Assessment (HTA) has taken an important role in the decision-making process related to the implementation of technologies in healthcare systems • The centrality of ethics in HTA has been known. In fact, ethics is everywhere in HTA, and value judgments permeate all levels of HTA What is New: • HTA is still little used in Neonatal Critical Care, but it may be the appropriate tool to determine the "value" of technologies used in this field • The implementation of clinical trials and HTA may help in an evidence-based evaluation of new technologies for the neonatal critical care and in introduction of the best and ethically acceptable health technologies into clinical practice.

Scenario analysis and multi-criteria decision analysis to explore alternative reimbursement pathways for whole genome sequencing for blood cancer patients.

BACKGROUND: Whole genome sequencing (WGS) has transformative potential for blood cancer management, but reimbursement is hindered by uncertain benefits relative to added costs. This study employed scenario planning and multi-criteria decision analysis (MCDA) to evaluate stakeholders' preferences for alternative reimbursement pathways, informing future health technology assessment (HTA) submission of WGS in blood cancer. METHODS: Key factors influencing WGS reimbursement in blood cancers were identified through a literature search. Hypothetical scenarios describing various evidential characteristics of WGS for HTA were developed using the morphological approach. An online survey, incorporating MCDA weights, was designed to gather stakeholder preferences (consumers/patients, clinicians/health professionals, industry representatives, health economists, and HTA committee members) for these scenarios. The survey assessed participants' approval of WGS reimbursement for each scenario, and scenario preferences were determined using the geometric mean method, applying an algorithm to improve reliability and precision by addressing inconsistent responses. RESULTS: Nineteen participants provided complete survey responses, primarily clinicians or health professionals (n = 6; 32 %), consumers/patients and industry representatives (both at n = 5; 26 %). "Clinical impact of WGS results on patient care" was the most critical criterion (criteria weight of 0.25), followed by "diagnostic accuracy of WGS" (0.21), "cost-effectiveness of WGS" (0.19), "availability of reimbursed treatment after WGS" (0.16), and "eligibility criteria for reimbursed treatment based on actionable WGS results" and "cost comparison of WGS" (both at 0.09). Participants preferred a scenario with substantial clinical evidence, high access to reimbursed targeted treatment, cost-effectiveness below $50,000 per quality-adjusted life year (QALY) gained, and affordability relative to standard molecular tests. Reimbursement was initially opposed until criteria such as equal cost to standard tests and better treatment accessibility were met. CONCLUSION: Payers commonly emphasize acceptable cost-effectiveness, but strong clinical evidence for many variants and comparable costs to standard tests are likely to drive positive reimbursement decisions for WGS.

Equity in national healthcare economic evaluation guidelines: Essential or extraneous?

BACKGROUND: The focus on health maximisation in a healthcare economic evaluation (HEE) - that is health gains are of equal value regardless of the recipient- has significant implications as health systems attempt to address persistent and growing health inequities. This study aimed to systematically compare and contrast the equity principles of different health technology assessment (HTA) agencies and how equity is addressed in HEE guidelines. METHODS: HTA agencies were identified through the ISPOR, GEAR, iDSI, HTAi, INAHTA, HTAsiaLink, and RedETSA websites in June 2021 and updated in August 2023. Agencies websites were then searched to retrieve HEE guidelines. The guidelines were grouped into two categories: well-established and newly-developed agency guidelines, based on whether or not they published their first guidelines before 2009. Data extracted summarised the methodological details in the reference cases, including specifics on how equity featured and in what role. In those agencies where equity did not feature explicitly in the HEE guidelines, an additional search of the agency website was undertaken to understand if equity featured in those agencies' decision-making frameworks. RESULTS: The study included 46 guidelines from 51 countries. Only 30% of the guidelines were explicit about the equity assumptions. Health equity (using a broad definition) was mentioned in 29 guidelines and 14 included a specific definition while only seven recommended specific methods to incorporate inequalities. Addressing equity concerns was usually suggested as an additional analyses rather than a key part of the assessment. It was unclear how equity was incorporated into decision-making processes. In addition, equity was mentioned in other guidance - such as decision-making frameworks - provided by five agencies that did not mention it in the HEE guidelines, and 7 of 14 topic selection criteria that were identified. CONCLUSION: Equity is given less attention than efficiency in HEE guidelines. This indicates that HTA agencies while subscribing to an extra-welfarist approach have a narrow evaluative space - focusing on maximising health and not considering the opportunity cost of the equity constraint. The omission of equity and the lack of systematic approaches in guidelines poses a threat to the international endeavours to reduce inequities. It is timely for HTA agencies to reconsider their positions on equity explicitly.

The maze of real-world evidence frameworks: from a desert to a jungle! An environmental scan and comparison across regulatory and health technology assessment agencies.

Aim: Regulatory and health technology assessment (HTA) agencies have increasingly published frameworks, guidelines, and recommendations for the use of real-world evidence (RWE) in healthcare decision-making. Variations in the scope and content of these documents, with updates running in parallel, may create challenges for their implementation especially during the market authorization and reimbursement phases of a medicine's life cycle. This environmental scan aimed to comprehensively identify and summarize the guidance documents for RWE developed by most well-established regulatory and reimbursement agencies, as well as other organizations focused on healthcare decision-making, and present their similarities and differences. Methods: RWE guidance documents, including white papers from regulatory and HTA agencies, were reviewed in March 2024. Data on scope and recommendations from each body were extracted by two reviewers and similarities and differences were summarized across four topics: study planning, choosing fit-for-purpose data, study conduct, and reporting. Post-authorization or non-pharmacological guidance was excluded. Results: Forty-six documents were identified across multiple agencies; US FDA produced the most RWE-related guidance. All agencies addressed specific and often similar methodological issues related to study design, data fitness-for-purpose, reliability, and reproducibility, although inconsistency in terminologies on these topics was noted. Two HTA bodies (National Institute for Health and Care Excellence [NICE] and Canada's Drug Agency) each centralized all related RWE guidance under a unified framework. RWE quality tools and checklists were not consistently named and some differences in preferences were noted. European Medicines Agency, NICE, Haute Autorité de Santé, and the Institute for Quality and Efficiency in Health Care included specific recommendations on the use of analytical approaches to address RWE complexities and increase trust in its findings. Conclusion: Similarities in agencies' expectations on RWE studies design, quality elements, and reporting will facilitate evidence generation strategy and activities for manufacturers facing multiple, including global, regulatory and reimbursement submissions and re-submissions. A strong preference by decision-making bodies for local real-world data generation may hinder opportunities for data sharing and outputs from international federated data networks. Closer collaboration between decision-making agencies towards a harmonized RWE roadmap, which can be centrally preserved in a living mode, will provide manufacturers and researchers clarity on minimum acceptance requirements and expectations, especially as novel methodologies for RWE generation are rapidly emerging.

Usefulness of dalbavancin in early discharge and nonhospitalization. It's time to throw your heart over the obstacle?

Introduction: Dalbavancin is a semisynthetic lipoglycopeptide long-acting antibiotic approved for the treatment of acute bacterial skin and skin structure infections (ABSSSIs). Its features can be useful in the current healthcare scenario characterized by the shortage of available hospital beds. Materials methods and results: We implemented several actions in order to optimize the use of dalbavancin allowing an improvement strategy both from the healthcare system and the patient's perspective in two hospital settings. In the Emergency Department we hospitalized only patients who met the clinical criteria and not the logistic criteria (i.e., the need for antibiotic therapy infusion). During the years 2017-2023, this strategy was applied in 40 cases, thus avoiding 40 hospitalizations for a total saving of 280 days of hospitalization.In the Internal Medicine ward and surgery department when there was no longer any need for hospitalization, we discharged the patient as early as possible. During the years 2017-2023, this strategy was applied in 189 cases, saving at least 1,134 days of hospitalization. The outcome of the treated patients was favorable in 228 out of 229 patients (99.5%). Conclusions: Our experience using dalbavancin in ABSSSI has been very satisfactory overall. The efficacy was close to 100%. Minor adverse events of slight severity occurred rarely. At the same time, this strategy allowed a more efficient allocation of hospital beds. Dalbavancin presents an ideal pharmacodynamic/pharmacokinetic profile for the management of ABSSSI especially in settings where shortage of hospital beds is critical.

Putting meat on the bone: how to fast-track innovative medicines to those who need them and generate data to justify continued use.

Regulatory agencies worldwide have taken significant steps to expedite approval and market authorization of medicines based on their potential to address areas of significant unmet medical need and severe disease burden. However, initial approval of such medicines is often accompanied by limited evidence of benefit, posing a conundrum for payers and health systems who may desire greater certainty of their value. This paper describes a system of "accelerated access" to manage these tensions and coordinate activities across stakeholders, based on discussions held at a multi-stakeholder convening in June 2023. We focus on 6 core, near-term actions that can be taken to improve the current system: clarifying criteria for expedited regulatory approval, enhancing stakeholder coordination, creating expedited pathways in payer and health technology assessment settings, developing joint regulatory/payer/health technology assessment guidance on study design and data needs, linking pricing policy to data uncertainty, and improving patient and public understanding of the processes involved as well as the risks and benefits of the relevant medicines. Many of these actions will require additional resources and personnel, and some will necessitate unprecedented levels of coordination. Nevertheless, each action is designed to work with minimal adjustments to the current system rather than demanding an entirely new approach.

Designing a Roadmap for Health Technology Assessment Implementation in Algeria.

Background The scarcity of resources restricts healthcare financing decisions, affecting the population's health. Health technology assessment (HTA) balances restricted budgets with the best possible health outcomes. We aim to characterize the current status of HTA in Algeria and describe the future directions for HTA implementation according to the priorities set by local stakeholders. Methods Stakeholders from the public and private sectors responded to a policy survey about the current and preferred future status of HTA implementation in Algeria. The survey was administered during an online workshop and used a widely accepted international scorecard covering eight domains: capacity building, HTA financing, process and organizational structure, scope of HTA implementation, decision criteria, standardization of methodology, use of local data, and international collaboration. After that, one-on-one interviews with another local expert were conducted to validate and modify the draft recommendations. The interviewees were representatives from government agencies, hospitals, and pharmaceutical companies. Results Thirty-one experts filled out the HTA scorecard survey; most of them were from the public sector (74%). They highlighted that project-based HTA workshops or short courses were the most common form of HTA education in Algeria and recommended the establishment of postgraduate HTA training programs in the future to build sustainable capacities. They reported a lack of funding for HTA research and critical appraisal and recommended an increased public budget for HTA and the introduction of submission fees by manufacturers. There was consensus about the need for local HTA evidence generation in the future. Most of the experts advocated an explicit soft decision threshold. The interviewees further recommended using multi-criteria decision analysis in the short term. The application of quality indicators was believed to improve the reliability of the HTA process. Conclusion The results of our policy research delineate the gap between the current and preferred future status of HTA in Algeria based on insights from multiple stakeholders. The need to improve the educational HTA programs in Algeria, use local data in policy decisions, and increase funding for HTA were the most advocated recommendations.

Market Access Challenges and Solutions in Cell and Gene Therapy in The Netherlands.

With the increasing pipeline of cell and gene therapies (CGTs) and the expected surge in the number of approvals, understanding the market access landscape becomes crucial for timely patient access. This study evaluates the challenges Dutch stakeholders encounter in CGT market access, offering insights for improving time-to-patient access. A traditional literature review was conducted to identify market access challenges and solutions for CGTs. Based on the findings, participants for semi-structured interviews, designed using an interview guide adapted to the Dutch context, were selected to capture diverse perspectives on market access. This review included 124 relevant articles out of 2449, covering several aspects of market access of CGTs. Subsequently, interviews with 16 stakeholders from academia, patient advocacy groups, manufacturers, health insurers, payers, hospital pharmacists, healthcare practitioners, and the Association of Innovative Medicines were conducted. Stakeholders identified challenges and proposed solutions for reimbursement package management, clinical trials, health economics, payment models, and procedural and organisational aspects. Thematic analysis revealed unique country-specific challenges and solutions in the Netherlands. This research provides insights into these challenges and potential solutions, emphasising the need for collaborative efforts among stakeholders to develop practical and multidisciplinary measures to improve the market access landscape for CGTs in the country.

Trends in NICE technology appraisals of non-small cell lung cancer drugs over the last decade.

OBJECTIVES: The aim of this study is to analyse the trends in technology appraisals for non-small cell lung cancer (NSCLC) treatments performed by the National Institute for Health and Care Excellence (NICE) over the last ten years. METHODS: A systematic search was conducted for single technology appraisals of NSCLC drugs in the online NICE database from 2012 to 2022. Search terms used were 'non small cell lung cancer', and 'NSCLC'. Appraisals that were under development or terminated as well as multiple technology appraisals were considered out of scope. RESULTS: In the 30 included appraisals for targeted therapies and immunotherapies within NSCLC, a total of 53 different comparators were included by NICE for 41 assorted indications or subgroups. Partitioned survival models were most frequently used, often including three health states and time horizons of up to 30 years. Throughout the decade the use of indirect comparisons was high and became more established and complex over time. Of all appraisals, 90% positively recommended the treatment for use in the UK. CONCLUSION: Technology appraisals became more complex over time due to the emergence of targeted therapies and immunotherapies, leading to multiple different indications, subpopulations and comparators that needed to be included in appraisals. Partitioned Survival Analysis (PartSA) models became the cornerstone within NSCLC, with time horizons up to 30 years and over time methods for indirect treatment comparisons became more established. The majority of the appraisals resulted in a positive recommendation for reimbursement.

Bridging Real-World Data Gaps: Connecting Dots Across 10 Asian Countries.

The economic trend and the health care landscape are rapidly evolving across Asia. Effective real-world data (RWD) for regulatory and clinical decision-making is a crucial milestone associated with this evolution. This necessitates a critical evaluation of RWD generation within distinct nations for the use of various RWD warehouses in the generation of real-world evidence (RWE). In this article, we outline the RWD generation trends for 2 contrasting nation archetypes: "Solo Scholars"-nations with relatively self-sufficient RWD research systems-and "Global Collaborators"-countries largely reliant on international infrastructures for RWD generation. The key trends and patterns in RWD generation, country-specific insights into the predominant databases used in each country to produce RWE, and insights into the broader landscape of RWD database use across these countries are discussed. Conclusively, the data point out the heterogeneous nature of RWD generation practices across 10 different Asian nations and advocate for strategic enhancements in data harmonization. The evidence highlights the imperative for improved database integration and the establishment of standardized protocols and infrastructure for leveraging electronic medical records (EMR) in streamlining RWD acquisition. The clinical data analysis and reporting system of Hong Kong is an excellent example of a successful EMR system that showcases the capacity of integrated robust EMR platforms to consolidate and produce diverse RWE. This, in turn, can potentially reduce the necessity for reliance on numerous condition-specific local and global registries or limited and largely unavailable medical insurance or claims databases in most Asian nations. Linking health technology assessment processes with open data initiatives such as the Observational Medical Outcomes Partnership Common Data Model and the Observational Health Data Sciences and Informatics could enable the leveraging of global data resources to inform local decision-making. Advancing such initiatives is crucial for reinforcing health care frameworks in resource-limited settings and advancing toward cohesive, evidence-driven health care policy and improved patient outcomes in the region.

Next-generation sequencing impact on cancer care: applications, challenges, and future directions.

Fundamentally precision oncology illustrates the path in which molecular profiling of tumors can illuminate their biological behavior, diversity, and likely outcomes by identifying distinct genetic mutations, protein levels, and other biomarkers that underpin cancer progression. Next-generation sequencing became an indispensable diagnostic tool for diagnosis and treatment guidance in current clinical practice. Nowadays, tissue analysis benefits from further support through methods like comprehensive genomic profiling and liquid biopsies. However, precision medicine in the field of oncology presents specific hurdles, such as the cost-benefit balance and widespread accessibility, particularly in countries with low- and middle-income. A key issue is how to effectively extend next-generation sequencing to all cancer patients, thus empowering treatment decision-making. Concerns also extend to the quality and preservation of tissue samples, as well as the evaluation of health technologies. Moreover, as technology advances, novel next-generation sequencing assessments are being developed, including the study of Fragmentomics. Therefore, our objective was to delineate the primary uses of next-generation sequencing, discussing its' applications, limitations, and prospective paths forward in Oncology.

Experiences of patient organizations' involvement in medicine appraisal and reimbursement processes in Finland - a qualitative study.

BACKGROUND: This study investigated how patient representatives have experienced their involvement in medicines appraisal and reimbursement processes with the Council for Choices in Health Care in Finland (COHERE) and the Pharmaceuticals Pricing Board (PPB) and how authorities perceive the role of patient organizations' input. METHODS: Semi-structured thematic individual and pair interviews were conducted in 2021 with representatives (n = 14) of patient organizations and government officials (n = 7) of the Ministry of Social Affairs and Health. The interview data were analyzed using qualitative content analysis. RESULTS: Patient representatives expressed their appreciation for the PPB and the COHERE in creating consultation processes and systematic models that support involvement. However, there were many challenges: patient representatives were uncertain about how their submissions were utilized in official processes and whether their opinions had any significance in decision-making. Patients or patient organizations lack representation in appraisal and decision-making bodies, and patient representatives felt that decision-making lacked transparency. The importance of patient involvement was highlighted by the authorities, but they also emphasized that the patient organizations' contributions were complementary to the other materials. Submissions regarding the medications used to treat rare diseases and those with limited research evidence were considered particularly valuable. However, the submissions may not necessarily have a direct impact on decisions. CONCLUSIONS: The interviews provided relevant input for the development of involvement processes at the PPB and COHERE. The interviews confirmed the need for increased transparency in the medicines assessment, appraisal, and decision-making procedures in Finland.

Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative.

National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings. Therefore, this research aims to (i) identify the opportunities and challenges faced by Beneluxa, (ii) gather insights from stakeholders, namely (possible) applicants and policymakers, within and beyond Beneluxa on the initiative and broader cross-country collaboration principles, and (iii) transfer these insights into learnings and recommendations in anticipation of the full implementation of the new HTA Regulation. Fifteen semi-structured interviews were conducted with industry and European HTA/policy stakeholders. The principal challenges discussed by stakeholders encompass hesitancy from the industry toward Beneluxa assessments, which were attributed to procedural and timeline uncertainties, legislative framework ambiguity, and challenges in terms of industry's internal organization. Another challenge highlighted is the resource-intensive nature of the procedure due to diverse approaches among member states. In addition, industry stakeholders mentioned limited communication and procedural complexity. Despite challenges, both stakeholder groups recognized important opportunities for cross-country collaboration. Transferable insights for future cross-country collaboration include transparent communication, clear legislative embedding, internal industry restructuring to facilitate joint HTAs, and member state support for conducting collaborative assessments. The study underscores diverging views among stakeholders on cross-country collaboration's potential to support HTA and the market access of complex health technologies. While acknowledging benefits, there still are challenges, including industry hesitancy, emphasizing the need for transparent communication and clear guidance in the evolving EU HTA landscape.

The Role of Medical Societies and the Relevance of Clinical Perspective in the Evolving EU HTA Process: Insights Generated at the 2023 Fall Convention and Survey of the European Access Academy.

BACKGROUND: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies' role; (ii) role of clinical guidelines; (iii) interface with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS); and (iv) approaching 'best-available evidence' (BAE). A descriptive analysis of questionnaire outcomes was conducted to inform the European Access Academy (EAA) Fall Convention 2023. Within the working groups (WGs), action points were identified and prioritised. RESULTS: A total of 57 experts from 15 countries responded to the survey. The WGs were attended by (i) 11, (ii) 10, (iii) 12, and (iv) 12 experts, respectively, representing a variety of national backgrounds and stakeholder profiles. The most relevant action points identified were as follows: (i) incorporation of clinical context into population, intervention, comparator, outcomes (PICO) schemes, (ii) timely provision of up-to-date therapeutic guidelines, (iii) ensuring the inclusion of MCBS insights into the EU HTA process, and (iv) considering randomized controlled trials (RCTs) as the gold standard and leveraging regulatory insights if development programs only include single-arm trials. CONCLUSIONS: The involvement of medical societies is a critical success factor for the EU HTA. The identified key action points foster the involvement of patient associations and medical societies.

HTA model for laboratory medicine technologies: overview of approaches adopted in some international agencies.

The Health Technology Assessment (HTA) Working Group of the Emerging Technology Division of International Federation of Clinical Chemistry and Laboratory Medicine (IFCC) aims to develop a methodological approach for producing structured HTA information for laboratory medicine technologies. This approach seeks to support decision-making processes at the country, regional, and/or hospital levels regarding the introduction of specific technologies. The focus of this model will primarily be on defining assessment elements within the domains of 'organizational aspects' and 'costs and economic evaluations', potentially differentiated by the type of diagnostic technology (e.g., genetic tests, molecular tests). To achieve this project's goal, a literature review and examination of websites of international HTA agencies have been conducted. The research aims to identify multidisciplinary methodological approaches used to assess laboratory diagnostic technologies and to pinpoint the domains and assessment elements utilized. We found 7 methodological articles describing methodological approaches adopted to assess laboratory diagnostic technologies. Among the HTA organizations considered, 23 reports were found, of which 7 were produced by the European Network of HTA (EUnetHTA), 4 by the National Institute for Health and Care Excellence Diagnostic Assessment Program (NICE DAP), and 12 by other HTA agencies. The EUnetHTA reports were rapid collaborative assessments covering various domains, while the NICE DAP reports focused on diagnostic guidances, including descriptions of technologies, clinical need and practice, diagnostic tests, accuracy, effectiveness, and cost-effectiveness. Finally, a survey targeting laboratory professionals will be conducted to introduce assessment elements, differentiated by the type of diagnostic technology, primarily for organizational and economic domains.