Search Results (138)

Background: The objective of this work was to support the implementation of the European Health Technology Assessment Regulation (EU HTAR) and optimize performance of the evolving EU HTA system. Therefore, an inclusive multi-stakeholder framework of key performance indicators (KPI) for success measurement was developed. Methods: A modified Delphi-procedure was applied as follows: (1) development of a generic KPI pool at the Fall Convention 2024 of the European Access Academy (EAA); (2) review of initial pool and identification of additional KPIs; (3) development of prioritized KPIs covering patient, clinician, Health Technology Developer (HTD), and System/Member State (MS) perspectives, and (4) consolidation of the stakeholder-centric KPIs after EAA’s Spring Convention 2025. Results: Steps 1 and 2 of the Delphi procedure revealed 14 generic KPI domains. Steps 3 and 4 resulted in four prioritized KPIs for patients (patient input; utilization of patient-centric outcome measures; time to access; equity); six for clinicians (population/intervention/comparator/outcomes (PICO); addressing uncertainty; clinician involvement; transparency; equity and time to access); four for HTDs (PICO; joint scientific consultation (JSC) process; joint clinical assessment (JCA) process; time to national decision making); five from a system/MS perspective (PICO; learning and training the health system; reducing duplication; equity and time to access). The scope of, e.g., the PICO-related KPI, differed between stakeholder groups. Also, several KPIs intentionally reached beyond the remit of EU HTA as they are also dependent on MS-specific factors including national health systems and budgets. Discussion and Conclusions: The KPI framework developed here presents a step towards the generation of systematic multi-stakeholder evidence to support a successful implementation of the EU HTAR. The relevance of the identified stakeholder-centric KPIs is confirmed by their alignment with the Health System Goals suggested in the context of “Performance measurement for health improvement” by the World Health Organisation. Implementation of the framework, i.e., measurement of KPIs, is envisioned to provide evidence to inform the 2028 revision of the EU HTAR. Full article

The PICO framework determines the scope of the Joint Clinical Assessment (JCA) under the EU HTA Regulation (EU HTAR), with PICO consolidation being a critical final step of the scoping process. Due to limited clarity on how consolidation works in practice, Health Technology Developers (HTDs) may simulate PICO scoping as a strategic tool to guide the development of robust JCA submissions. A review of 14 publications, representing 35 individual PICO exercises across 20 indications (74% in oncology), showed an average of 7 countries participating per exercise and 8 consolidated PICOs per analysis. A separate PICO scoping simulation focused on a first-line immuno-oncology treatment for metastatic non-small cell lung cancer (mNSCLC) generated 67 PICOs, reflecting the anticipated perspectives of 25 countries, largely driven by biomarker and histology-based sub-populations. The limited number of published examples and country participation restricts the ability to draw clear conclusions or confidently predict the output of PICO scoping in a real life JCA processes. The simulation also raised questions about whether all sub-populations should be included or consolidated further. Overall, there is a need for greater clarity in the JCA PICO scoping process, in particular the consolidation step, to facilitate high-quality evidence generation and support the EU HTAR to meet its goals of efficiency, transparency, and equity in health technology evaluation across Europe, along with more consistent patient access. Full article

Background/Objectives: Venous thromboembolism (VTE) is an important global health concern associated with considerable morbidity and mortality. Despite established guidelines for VTE treatment, there is a gap between clinical recommendations and their implementation in practice because of limited accessibility, particularly in low- and middle-income countries and among patients with cancer. This study aimed to assess the cost-effectiveness of direct oral anticoagulants (DOACs) on VTE in patients with cancer at a Thai university-affiliated hospital. Methods: A cost–utility analysis using a Markov model was developed to estimate costs and quality-adjusted life-years (QALYs) of DOACs and low-molecular weight heparin (LWMH) in Thai patients with cancer aged over 60 years. The model with eight health states, including CAT on treatment, pulmonary embolism (PE), deep vein thrombosis (DVT), clinically relevant nonmajor bleeding (CRNMB), non-intracranial hemorrhage major bleeding (non-ICH MB), intracranial hemorrhage (ICH), off treatment, and any death, was developed with a one-month cycle length and used to estimate costs and health outcomes from a societal perspective with a lifetime horizon. The efficacy and safety of DOACs compared to LMWH were obtained from a network meta-analysis, while the costs were based on a Thai university hospital database. All costs and outcomes were discounted at 3%, and the Thai societal willingness-to-pay (WTP) threshold (THB 160,000 per QALY gained) was applied. The incremental cost-effectiveness ratio (ICER) was calculated to compare costs and QALYs of the interventions. Results: The total lifetime cost of LMWH was THB 70,928 (USD 2,163), while for apixaban, dabigatran, edoxaban, and rivaroxaban, the costs were THB 26,323 (USD 803), THB 33,667 (USD 1,027), THB 29,570 (USD 902), and THB 22,310 (USD 680), respectively. The QALYs for LMWH, apixaban, dabigatran, edoxaban, and rivaroxaban were 0.771, 0.775, 0.746, 0.759, and 0.770 QALYs, respectively. Compared to LMWH, apixaban provided 0.004 additional QALYs, with a decreased cost of THB 44,605 (USD 1,360), resulting in reduced expenses. On the other hand, dabigatran, edoxaban, and rivaroxaban were also associated with lower lifetime costs but reduced life-years and QALYs when compared with LMWH. Conclusions: This study revealed that apixaban is likely to be the preferred option for treating patients with CAT. However, policy decision-making process should take into account the uncertainties related to the implementation of this practice. Full article

In the process of determining whether a health technology should be covered by healthcare systems, patients and their representatives were initially excluded from both evaluations and decision-making. In Europe, direct dialogue between patient organisations and regulatory authorities—particularly in the pharmaceutical sector—began in the early 1990s. It was only decades later, as the high cost of medicines created new challenges, that authorities recognised the necessity of engaging with patients. Patients’ contributions to the assessment of a health technology begin with discussions about the need for the technology in question. Initially, these discussions involve the developer, and later—after research and development—regulators, HTA assessors, and payers. Given that multiple technologies may be under development, patients and their organisations often prioritise those that generate the most interest within the patient community. They can then share their perspectives with evaluators during the horizon-scanning phase. Another key contribution is the role patients play in guiding clinical research by participating in scientific advice. Finally, during the assessment and appraisal stages, various methods are used to gather their views. Full article

Background/Objectives: The coronavirus disease 2019 (COVID-19) pandemic imposed an unprecedented global health and economic burden, prompting rapid implementation of diverse public health interventions. This review aimed to synthesize global evidence on the cost-effectiveness of key COVID-19 control strategies, including vaccination, testing, and social distancing and to identify methodological, contextual, and equity-related determinants of their economic value. Methods: A narrative literature review was conducted using peer-reviewed studies published between January 2020 and September 2025 and indexed in PubMed, Scopus, and Web of Science. Eligible studies included economic evaluations and modeling analyses addressing COVID-19 interventions in healthcare, community, or educational settings. Data on costs, outcomes, and methodological features were extracted and synthesized descriptively. Results: Across 74 included studies, vaccination—particularly with messenger RNA (mRNA) platforms—emerged as the most cost-effective intervention across all settings, often cost-saving among high-risk populations. Combined or layered strategies integrating vaccination, testing, and selective social distancing consistently outperformed single interventions in both health and economic outcomes. Early and targeted implementation yielded the highest cost-effectiveness by preventing exponential transmission and healthcare overload. However, heterogeneity in modeling assumptions, analytic perspectives, and outcome measures limited comparability. Few studies applied extended or distributional cost-effectiveness frameworks to address equity, while indirect and long-term effects such as productivity losses and “long COVID” were frequently omitted. Conclusions: COVID-19 interventions are most efficient when early, targeted, and adaptive to local epidemiologic conditions. Integrating equity, methodological consistency, and broader societal impacts into future evaluations will strengthen evidence-based, economically sustainable pandemic preparedness and response strategies. Full article

Background: Budget Impact Analysis is an integral part of the Health Technology Assessment in Bulgaria, informing reimbursement decisions of the National Health Insurance Fund. Inaccurate projections risk both fiscal unsustainability and restricted patient access to innovation. Yet projection accuracy methods remains uncertain, particularly given limited epidemiologic data and evolving clinical use. Objectives: This study aimed to assess the empirical validity of Health Technology Assessment budget-impact projections for medicines approved in 2019 by comparing projected patient volumes and expenditures with real-world National Health Insurance Fund reimbursements through 2025, and to identify drivers of divergence across therapeutic areas and reimbursement channels. Methods: We conducted a retrospective cohort analysis linking 2019 Health Technology Assessment submissions with monthly National Health Insurance Fund claims for both hospital and outpatient reimbursement channels. Actual utilization was calculated as the annualized median number of treated patients per month. Projected costs were derived by multiplying HTA-projected patient volumes by the observed unit cost per patient-month. We quantified deviations using observed-to-projected ratios and absolute gaps and assessed the relationship between projected and actual expenditures using a log–log regression model. Results: By September 2025, realized volumes typically exceeded projections (median ratio 1.6; range 0.02–21.3). Large overshoots were observed for Avelumab, Risankizumab, and Guselkumab; Cobimetinib and Abemaciclib remained below forecast. Expenditure deviations were driven predominantly by volume: immunology (+€17.4 million) and oncology (+€5.0 million) accounted for the largest absolute gaps. Elasticity was near proportional overall (β = 1.002; standard error = 0.24; R² = 0.50), lower in hospitals (β = 0.79; p = 0.055) and higher in outpatient care (β = 1.30; p = 0.003). Conclusions: Health Technology Assessment Budget Impact Analyses captured broad cost scaling but systematically missed product-specific uptake, with deviations largely volume-driven. Strengthening national registries and real-world data pipelines, and adopting dynamic, indication-responsive contracting and forecasting, could materially improve budget predictability while preserving access to innovation. Full article

This meta-review evaluated the possibility of more specified recommendations in antibiotic treatment through a narrowed focus on facial trauma. The aim was to analyze the effect of different regimens of antibiotic in treatment of skeletal trauma to the face. The knowledge mapping was based on existing systematic reviews (SRs) on trials specified in a PICO: Participants (P): Adults and children, diagnosed with fractures to the facial skeleton. Interventions (I): Antibiotic intervention. Comparator (C): Placebo, no antibiotics. Outcomes (O): Postoperative infection, pain, re-operation, other complications, healing deficiencies, (Oral) Health related Quality of Life, removal of osteosynthesis, adverse reactions. The literature search in PubMed, The Cochrane Library, and Web of Science according to PRISMA resulted in 1487 records. A COVIDENCE selection process resulted in 29 articles retrieved and read in full text revealing 10 articles eligible for evaluated according to ROBIS. Three SRs were considered to have low risk of bias and constituted the final evidence evaluation. The meta-review of these SRs did not provide sufficient support for prolonged antibiotic treatment after surgical intervention of midfacial fractures in comparison with antibiotics only the first day postoperatively. No support for antibiotic treatment for conservatively managed fractures alone was found. This review is limited by a relatively low number of included SRs. However, tendencies in outcomes suggests a restricted duration of antibiotics in treatment of facial fractures. Full article

Background: Internists formulate diagnostic hypotheses and personalized treatment plans by integrating data from a comprehensive clinical interview, reviewing a patient’s medical history, physical examination and findings from complementary tests. The patient treatment life cycle generates a significant volume of data points that can offer valuable insights to improve patient care by guiding clinical decision-making. Artificial Intelligence (AI) and, in particular, Generative AI (GAI), are promising tools in this regard, particularly after the introduction of Large Language Models. The European Federation of Internal Medicine (EFIM) recognizes the transformative impact of AI in leveraging clinical data and advancing the field of internal medicine. This position paper from the EFIM explores how AI can be applied to achieve the goals of P6 Medicine principles in internal medicine. P6 Medicine is an advanced healthcare model that extends the concept of Personalized Medicine toward a holistic, predictive, patient-centered approach that also integrates psycho-cognitive and socially responsible dimensions. An additional concept introduced is that of Digital Therapies (DTx), software applications designed to prevent and manage diseases and disorders through AI, which are used in the clinical setting if validated by rigorous research studies. Methods: The literature examining the relationship between AI and Internal Medicine was investigated through a bibliometric analysis. The themes identified in the literature review were further examined through the Delphi method. Thirty international AI and Internal Medicine experts constituted the Delphi panel. Results: Delphi results were summarized in a SWOT Analysis. The evidence is that through extensive data analysis, diagnostic capacity, drug development and patient tracking are increased. Conclusions: The panel unanimously considered AI in Internal Medicine as an opportunity, achieving a complete consensus on the matter. AI-driven solutions, including clinical applications of GAI and DTx, hold the potential to strongly change internal medicine by streamlining workflows, enhancing patient care and generating valuable data. Full article

Health Technology Assessment (HTA) is a multidimensional and multidisciplinary approach for analyzing the medical–clinical, social, organizational, economic, ethical, and legal implications of a technology, through the evaluation of multiple dimensions such as efficacy, safety, costs, and social–organizational impact. In the healthcare context, “technology” refers to any tool—including pharmaceuticals (or, in this case, vaccines)—that is applied to healthcare practice. HTA focuses on assessing both the real and potential effects of a given technology, either prospectively or throughout its life cycle, as well as the consequences that the introduction or exclusion of an intervention may have on the healthcare system, the economy, and society at large. Full article

The introduction of the European Union (EU) Joint Clinical Assessment (JCA) under Regulation (EU) 2021/2282 marks a transformative step in harmonizing health technology assessments (HTAs) across EU member states. This article explores the implications of JCA, particularly in oncology, for member states who utilize cost-effectiveness (CE) analysis and health technology developers (HTDs) who produce this evidence. The JCA framework attempts to standardise the assessment of relative clinical effectiveness and safety across the EU to input into national appraisals. Importantly, it excludes economic evaluations that may be required nationally, necessitating HTDs to align their CE models with the JCA PICO (Population/Intervention/Comparator/Outcome) parameters outlined by member states. This article discusses the challenges and opportunities for aligning JCA and CE modelling outcomes, contrasting evidence requirements between JCA and CE frameworks. It highlights the potential increase in complexity due to the diverse comparators in PICO surveys, necessitating the use of indirect comparison methodologies. It further underscores the importance of early communication between HTDs and HTA bodies to ensure timely, relevant, and pragmatic decision-making. By sharing national PICOs upfront to support national evidence generation, the JCA framework’s potential to aid high-quality decision-making and improve patient access to innovative medicines can be maximised. Full article

The implementation of the European (EU) Health Technology Assessment (HTA) Regulation 2021/2282 (EU HTAR) offers many opportunities, aimed at harmonizing HTA procedures and improving access to innovations; it also represents a significant challenge for the European healthcare system. Within the 2024 Health Policy Forum Italy meeting, different actors, stakeholders, and institutions had the opportunity to discuss major criticism and opportunities coming from the EU-HTA Regulation addressing future developments in the healthcare sector. Two groups, EU & Italy Pharmaceuticals and EU-Italy Medical Devices, worked distinctively on the EU HTAR by highlighting key issues that may pose challenges at both European and national levels, proposing potential solutions. The allocation of participants into two groups, according to their affiliation with either the pharmaceutical or the medical device sector, enhances the diversity of professional backgrounds and institutional perspectives, thereby fostering a more comprehensive and informed discussion. The recommendations highlighted by the two groups emphasize the need to promote cooperation among Member States, strengthen training for decision-makers, and develop a monitoring system to evaluate EU HTA’s impact. Full article

Introduction: Market access, pricing, and reimbursement of digital health technologies (DHTs) in Europe are significantly challenged by regulatory fragmentation and various assessment methodologies. Understanding the challenges and priorities of technology developers is essential for developing effective and relevant policy responses. This study explores perceived barriers and developer-driven priorities to inform the development of a harmonized health technology assessment (HTA) framework under the EDiHTA project. Methods: A mixed-methods approach was adopted, including a scoping review to identify key challenges, a survey of 20 DHT developers, and interviews and focus groups with 29 industry representatives from startups to multinational companies across 10 European countries during 2024. Results: Key challenges included a lack of transparency in reimbursement processes, fragmented HTA requirements, and misalignment between traditional evidence models and the agile development of DHTs. Developers highlighted the need to integrate real-world evidence, consider usability and implementation factors, and provide structured, lifecycle-based guidance. Financial barriers and procedural burdens were particularly significant for small and medium-sized enterprises. Conclusions: These findings highlight the need for an HTA framework that reflects the iterative nature of digital development, integrates real-world evidence, and reduces uncertainty for developers. The EDiHTA project aims to respond to these challenges by building a harmonized and flexible approach that aligns with the goals of the European HTA Regulation. Full article

Gene therapies that induce the body to produce therapeutic anti-vascular endothelial growth factor (anti-VEGF) proteins are an emerging topic related to neovascular age-related macular degeneration (nAMD). Continuous delivery of anti-VEGF protein directly to the target tissue offers the possibility of lifelong efficacy without the need for repeated and frequent eye injections. This novel approach could revolutionize patient management through optimizing clinical outcomes while simplifying service delivery. However, such gene therapies are anticipated to face unique challenges related to patients’ access and health technology assessment (HTA), and their integration into real-world eyecare practices. This article presents key elements raised at the European Access Academy (EAA) Fall convention (held in Rome in October 2024) regarding anticipated HTA challenges for gene therapies in nAMD. The important role of HTA and policymakers in ensuring that emerging gene therapies are accessible to all eligible patients is also highlighted. This article mainly focuses on the need for a fit-for-purpose EU HTA framework to address the widely varying utilization of standard of care in nAMD clinical practice, and to incorporate considerations about the long-term durability of gene therapies in nAMD. The importance of integrating real-world evidence (RWE) into the EU HTA framework is also discussed. Full article

Objectives: Type1 diabetes (T1D) is one of the most common chronic diseases in pediatric age. Continuous glucose monitoring (CGM) has been shown to improve glycaemic control in adults compared to self-monitoring of blood glucose (SMBG); however, evidence about its use in the pediatric field is limited and fragmented and needs to be improved. This paper aims to address all the critical aspects linked to the use of CGM in a pediatric population while also describing a methodology for conducting health technology assessment (HTA) to support the decision-making process. Methods: The use of CGM and SMBG in a pediatric population was compared by using a decision-making support tool (DoHTA method). Twenty-seven Key Performance Indicators (KPIs) were identified, defining safety, clinical effectiveness, organizational, patient perspective, and economic aspects. Performance scores for both monitoring systems were assessed based on these KPIs, leading to a final comparative ranking. Results: CGM demonstrated a 29.3% performance advantage over SMBG, highlighting its benefits in terms of clinical effectiveness, patient perspectives, safety, and economic evaluation. No substantial differences were identified in terms of organizational aspects. Conclusions: This study critically evaluates the benefits and drawbacks of the use of CGM in a pediatric population. It integrates the assessment of the clinical effectiveness with the organizational aspects, the cost, the patient perspective, and the safety, providing a valuable proof of evidence as well as a reliable and transferable method for conducting decision-making processes in a hospital setting. Full article

The health technology assessment (HTA) is a multidisciplinary process utilized to determine the clinical, economic, social, and ethical value of new health technologies before they are incorporated into healthcare systems. In the case of rare diseases, such as Cystic Fibrosis (CF), challenges arise due to limited evidence and high treatment costs. Indeed, although CF transmembrane conductance regulator (CFTR) modulators are breakthrough therapies for CF, their incorporation into public health systems has been complex with considerable challenges, especially in low- and middle-income countries. This article presents a descriptive and exploratory case study of the regulatory and policy journey for CFTR modulators to be approved in Brazil. Based on a narrative review and document analysis, we highlight the importance of building lay society knowledge to shape policy decisions and promote equitable access to innovative therapies. In parallel, we critically reflect on the HTA process and highlight efforts in the training, education, and coordination required to enable meaningful public engagement and landmark achievements. Full article