Journal of market access & health policy最新文献

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.

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.

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.

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.

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.

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.

This study examines patient access to orphan medicinal products (OMPs) in Central and Eastern Europe (CEE) over the past five years, focusing on seven countries: Bulgaria, Czechia, Hungary, Poland, Romania, Slovakia, and Slovenia. While these jurisdictions have undergone rapid healthcare transformations, significant disparities in OMP access persist compared to Western Europe. This study aimed to address this gap by identifying barriers and enablers to optimize patient access to OMPs in a sustainable and equitable manner. A mixed-methodology approach was utilized, combining systematic literature reviews, in-depth interviews, and advisory board insights. Perspectives were gathered from a wide range of stakeholders, including policymakers, payers, academia, industry associations, and patient advocacy groups. Additionally, the study incorporated data from CEE-specific initiatives to triangulate findings and evaluate barriers, enablers, and best practices in OMP access. The analysis identified sub-optimal OMP access across most CEE countries, marked by prolonged delays and lower reimbursement rates compared to Western Europe, with Slovenia and Czechia as notable exceptions. Key barriers include limited awareness, inadequate health technology assessment (HTA) frameworks, insufficient financing mechanisms, underutilization of novel access schemes, and fragmented patient engagement. Conversely, enablers include the presence of rare disease policies, OMP-specific HTA frameworks, and patient-inclusive decision-making processes.

The HTA R introduces provisions that may benefit member states, in particular the opportunity to share national or regional assessment reports, cooperate outside of clinical domains, or use the methodological guidelines on a local level for technologies that are not subject to joint assessment. Challenges related to the timelines, differences between assessment scopes, and diverging guidance may jeopardize the full potential of the HTA R in Central European EU member states. However, these are more likely to be related to the commitment and vigilance of local competent authorities. We attempt to address these opportunities and mark some challenges imposed by the application of the HTA R by taking the perspective of public health decision-makers in Central European EU member states. We conclude that the foundations for capitalizing on the opportunities offered by the HTA R are already laid in the region, and we foresee policymakers and payers sharing the responsibility of acting as drivers of change in health policy to reduce the duplication (or multiplication) of efforts by HTDs, as well as to increase the efficient use of HTA bodies' resources.

Background: This study explored the readiness and strategic considerations of companies and key stakeholders for the implementation of the Joint Clinical Assessment (JCA) under the European Health Technology Assessment Regulation (HTAR). It examined the implications of the JCA process for jurisdictional submission strategies, and decision-making across Europe. The study aimed at identifying key measures for an efficient and effective JCA process to enable national rollout.

Methods: A survey was conducted with international pharmaceutical companies, followed by a multi-stakeholder workshop that expanded on the findings. The survey and workshop focused on key areas such as time to market, submission strategies, and the role of JCA in national decision-making processes. Descriptive and qualitative analyses were performed to identify recommendations for measuring and improving the JCA process.

Results: 13 companies responded to the survey, respondents were generally prepared for the JCA process (readiness rated 6-7/10), but concerns persist about timeline uncertainties and timely JCA report delivery. In the short term, success for the HTAR from the company perspective is measured by positive recommendations across EU jurisdictions. Long term, the focus shifts to aligning HTA methodologies and evidence requirements across the EU. Establishing metrics to assess the efficiency and effectiveness of the JCA is a key step in the HTAR's ongoing learning journey. To enhance the efficiency of the JCA process, a list of metrics is recommended for continuous improvement, as well as establishing training programs to strengthen member states' capabilities, fostering open dialog for sharing technology-specific insights, and creating open-source tools to support companies. Additionally, research should be conducted to understand agencies' expectations of the JCA and how they will use its reports, grouping agencies by archetype to identify trends. A key recommendation is the development of a product-based scorecard to evaluate JCA submissions and reviews from various perspectives, ensuring the process meets stakeholders' needs and can be effectively utilized in national decision-making.

Conclusions: The JCA process offers a significant opportunity to streamline HTA decision-making across Europe. This study highlights several key measures and consideration for its successful rollout, including the need for clearer communication about the role of JCA in national decisions, measurement of rollout time components, and the development of quality evaluation frameworks. A collaborative, iterative approach, where stakeholders continually refine the system, will be essential for its effectiveness. Addressing these challenges will enable the JCA to enhance efficiency, consistency, and ultimately improve access to treatments for patients.