Journal of market access & health policy最新文献

Designing an accessible, financially viable healthcare system is a key challenge for society. The value-based healthcare (VBHC) strategic model aims to simultaneously improve the quality of healthcare and the efficiency of health systems. The aim of this research was to describe the perceptions of different stakeholders in the Portuguese health industry about the creation of value and the understanding of VBHC as a competitive advantage. A qualitative study was conducted using the inductive method of Braun and Clarke, designed according to the COREQ criteria. Based on the results of the literature review, a semi-structured script for an interview was created, consisting of eight questions. The initial interview script was based on a thorough narrative literature review and tested with two professionals with practical experience in VBHC. The final version of the semi-structured interview guide consisted of eight open-ended questions. The questions were designed to elicit in-depth, reflective responses, and their neutrality was reviewed to avoid leading language that might introduce bias. As the interviews progressed, minor iterative changes were made to include participant-suggested additions, always maintaining alignment with the research objectives. This iterative process was essential to capture the nuanced perspectives of stakeholders and conformed to COREQ standards for qualitative research. A total of 15 stakeholders in VBHC were interviewed. The interviews were transcribed and coded, and 605 codes were created, divided into subthemes and themes. VBHC implementation faces several challenges, requiring a collaborative effort by the stakeholders involved, to achieve a comprehensive vision of value and appropriate multi-stakeholder alignment. The implementation of VBHC can confer a sustainable competitive advantage, and its adoption as a strategic model will be inevitable in the future.

Background: Expected utility has been deployed in order to predict health behaviour in health economic evaluation. However, only limited variance in health behaviour is explained by this construct. This limited explained variance is often attributed to the dubious foundational postulates underlying the construct (e.g., absolute rationality, complete information, fixed preferences). Due to these limitations it has been hypothesized that substituting or complementing expected utility with experienced utility may enhance predictions of health behaviour. As this hypothesis has not yet been subjected to empirical scrutiny, this study examines if deployment of experienced utility or expected utility and experienced utility combined enhances predictions of health behaviour relative to expected utility separately.

Methods: Online questionnaires were distributed across a panel of Dutch citizens (N = 2550). The questionnaire includes items and scales on sample characteristics, expected utility, experienced utility and health behaviour. Data analysis was conducted by employing descriptive, reliability, validity and model statistics.

Results: Experienced utility has a significant direct effect on health behaviour that is stronger than expected utility. Experienced utility also explains more variance in health behaviour than expected utility. Expected utility and experienced utility combined have a significant direct and indirect effect on health behaviour that is stronger than each type of utility separately. Expected utility and experienced utility combined also explain more variance in health behaviour than each type of utility separately.

Conclusions: Deploying experienced utility separately or in combination with expected utility in health economic evaluation seems pertinent as it has considerable impact on health behaviour and may provide health economists with an even sturdier foundation for conducting health economic evaluation.

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.

Background: With the ongoing development of game-changing technologies, assessing healthcare provider burden is desirable. This requires developing and evaluating subjective outcome measures, but there is no single scale that measures this burden. We developed a measure of quality of life (QOL) to address this, focusing on medical doctors (MDs). Methods: Based on Japan's national statistical distribution of MDs in Japan, we qualitatively interviewed twenty MDs to identify factors that influenced their QOL and another eight MDs to verify the appropriateness and interpretability of the questions. Validity and reliability were evaluated and verified in a quantitative survey of 374 MDs to finalize the questionnaire. Results: Based on our initial research and interviews, we derived nine dimensions and developed the work-related QOL questionnaire for MDs (WQMD-9) accordingly. Correlation coefficients between questionnaire items were 0.3-0.7 and Cronbach's α was 0.897, confirming the validity and reliability of the questionnaire. Conclusions: The WQMD-9 is an original profile-type scale with nine dimensions and five levels. We expect that as new technologies develop, evaluations of the associated medical treatment will involve measuring the QOL of not only patients but also MDs, and the WQMD-9 will facilitate this process.

Our objective is to describe the experience and challenges of using Managed Entry Agreements (MEAs) in Algeria, Morocco, and Tunisia. We conducted online interviews with key decision-makers in Algeria, Morocco, and Tunisia between March 2021 and December 2023. The questionnaire captured experience with MEAs, types of agreements implemented, and challenges to implementing MEAs. Three, five, and seven participants working, respectively, in the Algerian, Moroccan, and Tunisian pharmaceutical sectors, participated in the interviews. Participants were from the public (8/15) and the private sector (7/15). Only Tunisian respondents reported having dealt with MEAs contracts, such as financial-based agreements (FBAs) related to standard discounts and volume-based price discounts. All respondents were aware of the potential need for structuring contracts differently for expensive medicines. Hurdles in implementing MEAs noted by respondents were mainly related to the absence of a legal framework and the lack of budget allocated for new medicines. Most respondents projected an increase in the use of MEAs to improve reimbursement and access to new, highly priced medicines. Recommendations include strengthening pricing, reimbursement processes, and HTA use. The adoption of FBAs is suggested as a practical initial approach.

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.

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.

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.

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.

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.