Search Results (298)

Introduction and Objectives: Risk-sharing agreements (RSAs) have emerged as a key strategy for financing high-cost medical technologies while ensuring financial sustainability. These payment mechanisms mitigate clinical and financial uncertainties, optimizing pricing and reimbursement decisions. Despite their widespread adoption globally, Latin America has reported limited implementation, particularly for high-cost medical devices. This study aims to share insights from designing RSAs in the form of Outcome Protection Programs (OPPs) for medical devices in Latin America from the perspective of a medical devices company. Methods: The report follows a structured approach, defining key OPP dimensions: payment base, access criteria, pricing schemes, risk assessment, and performance incentives. Risks were categorized as financial, clinical, and operational. The framework applied principles from prior models, emphasizing negotiation, program design, implementation, and evaluation. A multidisciplinary task force analyzed patient needs, provider motivations, and payer constraints to ensure alignment with health system priorities. Results: Over two semesters, a panel of seven experts from the manufacturer designed n = 105 innovative payment programs implemented in Argentina (n = 7), Brazil (n = 7), Colombia (n = 75), Mexico (n = 9), Panama (n = 4), and Puerto Rico (n = 3). The programs targeted eight high-burden conditions, including Coronary Artery Disease, atrial fibrillation, Heart Failure, and post-implantation arrhythmias, among others. Private providers accounted for 80% of experiences. Challenges include clinical inertia and operational complexities, necessitating structured training and monitoring mechanisms. Conclusions: Outcome Protection Programs offer a viable and practical risk-sharing approach to financing high-cost medical devices in Latin America. Their implementation requires careful stakeholder alignment, clear eligibility criteria and endpoints, and robust monitoring frameworks. These findings contribute to the ongoing dialogue on sustainable healthcare financing, emphasizing the need for tailored approaches in resource-constrained settings. Full article

Patient involvement in health technology assessment (HTA) processes is increasingly recognized as pivotal for informed, equitable, and patient-relevant health care decision-making. With the implementation of Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs) under Regulation (EU) 2021/2282, the European Union has a unique opportunity to design harmonized mechanisms that reflect best practices from established HTA systems. This article, drawing on the Acute Leukemia Advocates Network (ALAN)’s comparative analysis of HTA practices across seven countries (Canada, England, Scotland, France, Germany, Spain, and Italy), examines how current patient involvement processes can inform the JCA framework. It identifies opportunities to replicate effective practices and proposes strategies to embed patient voices meaningfully into the JCA process. By prioritizing robust and inclusive patient involvement, the EU can establish a global benchmark for impactful and consistent HTA processes. By leveraging lessons from international HTA systems and prioritizing clear frameworks, early involvement, and capacity building, the EU can set a global standard for meaningful patient participation in HTA processes. ALAN is an independent global network of patient organizations dedicated to improving outcomes for patients with acute leukemia. Full article

The designs of clinical trials of drugs for rare diseases are challenged by health technology assessment organisations and payers. Phase II pivotal studies, single-arm or open-label designs, the extensive use of non-final endpoints, and the limited use of patient-reported outcomes (PROs) are the main points of contention. The evidence on the actual design of these trials is limited, but corroborates the concerns of the above. Our aim is to scrutinise whether the design of pivotal studies of drugs for rare diseases to be launched into the Italian market by 2026 present similar issues. The drugs and the relevant pivotal studies were retrieved from Biomedtracker and US and European clinical trial databases. We identified 154 new drugs for rare diseases. Single-arm designs account for 36% of trials. Almost 50% of randomised control trials (RCTs) are designed using an active comparator and 61% are double-blinded. Primary endpoints are mostly (82%) surrogate. A total of 59% of studies include PROs. Our findings were partially expected (e.g., extensive use of surrogate endpoints) and partially not (e.g., RCTs and an active comparator), considering previous studies on the same topic. Having more head-to-head studies may reduce uncertainty concerning evidence at market launch, but different issues persist, including the still limited role of PROs. Full article

The continued migration of physicians from independent practice to affiliation with larger entities has garnered significant scrutiny. These affiliation models include hospitals and health systems, payers and corporate entities, and management services organizations, which may or may not be private equity (PE)-backed. Data on the impact of different physician affiliation models on cost of care is limited. We examined the relationship between provider affiliation model, site of care (SOC), and cost of care for certain high-volume procedures in procedure-intensive specialties for both Medicare and commercial insurance. We found that hospital-affiliated physicians are least likely—and PE-affiliated physicians are most likely—to provide care in lower-cost settings. For both Medicare and commercial insurance, SOC contributes meaningfully to procedure unit price, which is consistently greater in hospital-based settings. These findings suggest that the physician affiliation model and associated SOC cost differentials contribute materially to healthcare expenditures. As the Medicare cost differentials are set by statute and regulations, strategies such as site-neutral payments are needed to mitigate the monetary impact of historical and future physician practice migration. Full article

The European Health Technology Assessment (HTA) regulation (HTAR) came into effect in January 2025 and impacts the HTA process in all European Member States. Member States must give due consideration to the joint clinical assessment (JCA) report. This may require adaptations at the national level. This paper describes the anticipated changes to the Dutch national HTA process and how the Dutch National Health Care Institute (Zorginstituut Nederland, ZIN) prepared for this, because sharing experience between Member States can be of general interest for future expansion of the EU HTAR. ZIN’s implementation activities were facilitated by a project-governance structure and by a continuous gap analysis of the current national assessment and appraisal process of medicinal products, resulting in a concrete action plan. The implementation of the HTAR has two major implications for ZIN’s HTA process, namely that the scoping phase starts much earlier and that the JCA report is the starting point for the national assessment. Gaps, challenges and issues were identified in the categories: information and knowledge, IT and template, communication and stakeholder engagement, capacity and resources, and financial aspects. Based on a thorough and well-defined implementation plan, ZIN is ready to implement the HTAR in national HTA processes and to take on (co-)assessor roles for JCA of medicinal products in 2025. Full article

In respiratory patients, limited adherence to and misuse of devices hinder the effectiveness of inhalation therapy. Switching inhalers for non-clinical reasons poses a risk of deterioration of respiratory disease and/or promotes poor adherence to therapy. The objective of this work was to explore the impact of device changes for non-clinical reasons on clinical outcomes (primary) and costs (secondary), including carbon emissions in Spain. After a comprehensive literature search, the increased use of resources following worsening outcomes was apportioned using Spanish cost data and following the recommended pathways for care. We calculated the cost of re-training these patients and attributed carbon emissions in metric tons of CO₂ equivalent (tCO₂eq) to the excess resource use. In Spain, the impact of uncontrolled switching for non-clinical reasons in COPD has an annual estimated cost of EUR 923/patient, leading to an excess annual expenditure of more than EUR 216 million. For asthma patients, the annual impact is almost EUR 263/patient, representing an additional EUR 118 million excess annual expenditure. The environmental consequence of both conditions can be equated to almost 45 thousand tCO₂eq. Training all these patients on the new device would cost around EUR 35 million and would generate an extra impact reduction of about 2.6 thousand tCO₂eq in carbon emissions levy. Full article

With the European Union (EU) Health Technology Assessment (HTA) regulation, Joint Clinical Assessments (JCA) are now required for oncological and advanced therapy medicinal products. The JCA assessment scope is determined through the PICO framework (Population, Intervention, Comparator, Outcome). Given the tight JCA timelines, Health Technology Developers (HTD) must anticipate PICO elements early to prepare dossiers effectively. This study investigates whether PICO can be predicted across EU member states using publicly available information. A systematic literature review was conducted to identify relevant peer-reviewed articles. Additionally, an extensive search of publicly available HTA documents, including reports, methodological guidelines, submission templates, and market access information was performed across 29 European countries. Relevant information for PICO anticipation was extracted. For many member states, a wealth of relevant information is publicly accessible: 66% have HTA reports publicly available, 79% have HTA methodological guidelines, 69% have dossier templates, and 100% have market access status lists. Between countries, the requirements for population and outcomes are largely aligned, making comparator the central element in PICO anticipation. PICO can be anticipated reliably based on public information. HTDs must be prepared to adjust their strategies as national procedures adapt, ensuring alignment with both current and emerging EU and national requirements. Full article

The dynamic nature of infectious diseases introduces inherent challenges to the design of vaccine clinical trials, which consequently makes vaccine indirect treatment comparisons (ITCs) and meta-analyses (MAs) more challenging compared with regular pharmaceuticals. However, comparisons of efficacy and safety between vaccines are being frequently required in vaccine decision making due to a low number of head-to-head clinical trials in the vaccine landscape. The introduction of the European Union Health Technology Assessment (HTA) Regulation (EU HTAR) aims to harmonize HTA efforts across Europe. However, the EU HTAR could also escalate existing challenges for conducting vaccine MAs and ITCs. Such challenges include generating efficacy evidence in time for Joint Clinical Assessment (JCA), incorporating high levels of heterogeneity due to infectious disease-specific characteristics, and tackling a high number of PICOs per submission—likely driven by heterogeneity in the available data and differences in national vaccine calendars. Opportunities to tackle these challenges include introducing a stepwise approach to vaccine assessment in JCA, best-practice recommendations for conducting/interpreting vaccine MAs and ITCs, and condensing the number of PICOs to create larger ‘catch-all’ ITC networks. This perspective article explores these challenges and opportunities further. Full article

Background and Objective: Innovation is a key enabler of patient-centered care in cardiology, with new medical devices and digital health technologies offering the potential to improve outcomes and efficiency. However, the evaluation of these innovations poses challenges for clinicians, regulators, and procurement stakeholders, particularly within the complex European healthcare landscape. This review aims to explore the current state of health technology assessment (HTA) for cardiology-related medical devices in Europe, offering a clinical perspective. Material and Methods: Three independent scoping reviews were conducted following the PRISMA-ScR guidelines. Keywords included “innovation”, “health technology assessment”, and “cardiology”. The search was supplemented by the relevant literature on European HTA policies, regulatory directives, and emerging technologies. Results: The review identified three central themes: (1) the evolving role of clinicians in HTA processes, (2) the integration of innovative technologies such as digital tools and artificial intelligence within HTA frameworks, and (3) the considerable variation in HTA practices and policies across EU member states. Conclusions: HTA in Europe is undergoing a transformation, with increasing emphasis on interdisciplinary collaboration and frameworks that support innovation. While the goal of harmonization across the EU remains a work in progress, new regulatory efforts, such as the HTA Regulation (HTAR), offer promising avenues for aligning clinical practice with evidence-based assessment and reimbursement decisions. Full article

Managed competition is a key driver in healthcare systems in countries like Germany, Switzerland, and The Netherlands. Trust in health insurers is vital but currently low in The Netherlands. This may be due to perceptions regarding profit motives, negative experiences, media coverage, and a lack of understanding of insurers’ roles. This study explores how enrollees perceive health insurers and how the aforementioned factors contribute to these perceptions. Semi-structured interviews were conducted with 17 participants from the Nivel Dutch Health Care Consumer Panel in March and April 2023. Data were analysed using Braun and Clarke’s six-step method for inductive thematic analysis. Participants generally view health insurers positively in terms of managing finances and ensuring care accessibility. However, some perceive insurers as profit-driven and prioritising cost reduction over individual needs, leading to dissatisfaction. Negative experiences and media coverage also shape these perceptions. Participants believe that insurers should ensure care accessibility and quality, distribute costs fairly, provide guidance, and prioritise preventive measures. To foster trust, insurers should communicate their non-profit status and use of benefits, increase transparency in purchasing decisions, and maintain clear communication about payment obligations. Enhancing communication about their contributions to healthcare and raising awareness of their broader roles may also help build trust. Full article

The integration of health and social care is increasingly recognized as essential to address population ageing, the rise in chronic diseases, and persistent health inequities. Across Europe, diverse models have been developed to improve service coordination, resource efficiency, and person-centered care. This paper aims to explore international experiences in integrating health and social care, identify common strategies and challenges, and provide insights to inform policy development in countries where integration remains incipient, with a focus on Portugal. A qualitative comparative approach was employed. A systematic literature review was conducted across PUBMED, MEDLINE, and Google Scholar, including peer-reviewed articles, policy reports, and government documents. Thematic analysis was used to identify integration models, enablers, and barriers across different countries. Different models reveal that joint governance, pooled funding, strong community involvement, and digital innovation are key enablers of integration. However, common challenges persist, including fragmented governance, inconsistent implementation, and financial sustainability. In Portugal, structural separation between the health and social sectors continues to limit strategic alignment. Successful integration depends on political commitment, shared vision, and active stakeholder collaboration. European models offer adaptable lessons for Portugal and similar systems, especially regarding intersectoral coordination and preventive care. Integrating health and social care is vital for building resilient, equitable systems. Portugal must adopt a cohesive national strategy; strengthen local implementation; and embrace person-centered, sustainable solutions to ensure long-term impact. Integrating the health and social sectors is indispensable in navigating the ever-evolving healthcare landscape and promoting holistic well-being. Full article

The evolving landscape of Health Technology Assessment (HTA) in Europe, shaped by the implementation of the new EU HTA Regulation (HTAR), places an emphasis on engaging all stakeholders, including patients, in collaborative evidence generation. Yet integrating patients’ perspectives into critical processes like PICO scoping remains a challenge, with concerns around subjectivity, representativeness, and methodological robustness. This opinion paper examines the complexities of patient engagement in HTA, highlighting both the opportunities for patients to make meaningful contributions and the barriers that stand in the way. We propose a framework that employes the Delphi panel methodology to (1) foster scientific validity and increase transparency in patient contributions, (2) establish a structured and consistent patient engagement framework, and (3) and understand European patients’ perspectives while promoting collaboration among EU countries. By facilitating iterative feedback and fostering agreement among diverse groups of patients and caregivers contributing with their expertise, consensus methods like Delphi panels can help refine PICO criteria, align diverse stakeholders’ expectations, and increase the relevance of HTA outcomes. A study is now underway to evaluate the feasibility and value of using the modified Delphi panel methodology for patient engagement in PICO scoping. The authors propose that embracing patient engagement through carefully designed consensus frameworks could enhance the legitimacy and completeness of HTA processes, driving more patient-centered decision making across Europe. Full article

Systemic therapy (ST) and transarterial radioembolization (TARE) are widely used treatments for advanced-stage hepatocellular carcinoma (HCC). This study quantified the significant variability in treatment costs for unresectable HCC from payer and provider perspectives. An Excel-based price analysis model was developed to estimate the prices of ST and TARE over a 21-month time horizon using 2015–2021 data. Median prices were calculated from Medicare Average Sales Price (ASP), provider Wholesale Acquisition Cost (WAC), and Average Wholesale Price (AWP). Sensitivity analyses evaluated price fluctuations associated with a ±10% variation in treatment duration. ST prices demonstrated marked variability across perspectives, with the median ASP at $175,625, WAC at $198,719, and AWP at $262,892. However, TARE prices were stable, ranging from $21,594 to $24,052. Sensitivity analyses revealed that treatment duration variation resulted in price changes of $35,000–$50,000 for ST, compared with ~$5000 for TARE. The variability in ST pricing was driven by treatment duration and drug-specific pricing mechanisms, particularly immunotherapy-based regimens, which accounted for the higher cost range. Conversely, TARE’s consistent pricing is attributed to standardized procedural costs. Substantial variability exists in ST prices compared with the consistent costs of TARE, underscoring the economic advantage of TARE in appropriate clinical contexts. Full article