Journal of Market Access & Health Policy

Type 2 diabetes (T2D) constitutes a major health problem, reaching alarming rates over the last decades, especially due to contemporary lifestyle and associated obesogenic environments, as well as the aging population. Diabetes not only causes social consequences but also leads to increasing healthcare costs, posing a significant challenge for the health system. This paper applies a five-step approach for estimating T2D-related costs in Greece. The approach initially estimates the T2D-related ICD10 prevalence and the target population. Next it applies the appropriate therapeutic protocols to identify the most appropriate treatments. Subsequently, it calculates the total cost of medical treatments for each target population, based on the distribution of patients between the different treatments and treatment lines. Finally, based on the diagnostic and treatment protocols, it calculates the annual direct costs associated with the cost categories. Using the estimated future population of the country, the proposed methodology can also project the budget required, under certain conditions, to deal with T2D. The analysis estimated that T2D-related costs in 2021 under rational use of resources were EUR 1,397,871,172.55 billion and EUR 1,512,934,947.63 billion projected in the year 2030 considering the aging effect, per cost category, and in total, presenting an increase of approximately 115 million euros in 2030 compared to 2021. The term “rational use of resources” in this study refers to the use of internationally recognized, evidence-based diagnostic and therapeutic protocols, as adopted by the Greek Ministry of Health. This scenario represents an idealized standard of care rather than actual real-world adherence and is used to estimate the potential resource needs under optimal medical practice conditions. An inflation rate of 4.2% was applied to costs between 2021 and 2030. The analysis showed that the highest percentage (39%) of the total T2D-related healthcare expenditures is associated with complications that occur in T2D patients. Despite a comparatively modest prevalence of T2D in Greece relative to other European and Mediterranean countries, the economic burden associated with its management remains high. The aging of the population will lead to an increase in the total cost of T2D. The applied methodology of estimating budgets by aggregating categories of expenses under a specific disease (ICD10), instead of dividing budgets into categories of expenses, can successfully lead to the optimization and rationalization of expenses according to actual needs. The findings underline the significant economic burden of T2D in Greece, particularly due to complications and population aging. These results emphasize the urgent need for health policy strategies focusing on prevention, early intervention, and the efficient allocation of healthcare resources. The methodology applied can serve as a decision-making tool for forecasting healthcare budgets and optimizing expenditures under different population and treatment scenarios. 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

Access to home- and community-based services (HCBSs) varies substantially between states. Yet, it is unknown how state-level policies and administrative factors impact consumer-reported unmet service needs, an important indicator of HCBS access and quality. Using the National Core Indicators—Aging and Disability Adult Consumer Survey (2016–2019; n = 13,654 community-dwelling older adults, 13 states), we examined associations between unmet HCBS needs with four state-level factors: HCBS spending relative to institutional care spending, HCBS spending per client, percentage of Medicaid beneficiaries in managed care, and Medicaid expansion; and funding program. In the adjusted logistic regression model, the odds of overall unmet HCBS needs were lower with higher percentage Medicaid beneficiaries in managed care (adjusted odds ratio [aOR], 0.92; 95% confidence interval [CI], 0.89–0.96) and Medicaid expansion (aOR, 0.80; 95% CI, 0.73–0.87) but greater with higher HCBS spending relative to institutional care spending (aOR, 1.19; 95% CI, 1.11–1.28). Compared to Medicaid waiver, odds of unmet HCBS needs were significantly lower among consumers in Managed Long-Term Services and Supports (aOR, 0.67; 95% CI, 0.61–0.74) and Program of All-Inclusive Care for the Elderly (PACE; aOR, 0.39; 95% CI, 0.31–0.49). State policies and administrative factors are important place-based determinants of HCBS consumers’ unmet HCBS needs/access; and warrant consideration in HCBS quality assurance and improvement. Full article

Vaccination has resulted in substantial public health benefits for human populations worldwide since it was first introduced more than a century ago. This article presents an overview of the history of vaccine development, its implementation, and price setting, the latter mainly from a developed world perspective. It considers potential issues and challenges. Over time, vaccine development and production has evolved to a market-driven approach, conducted largely by private commercial entities. The complex processes of identifying potential vaccine targets and developing and producing vaccines at scale have now become more efficient. However, vaccine pricing is an emerging concern. The elements that maximize the overall health benefit of vaccination include high volume, high coverage, and rapid initial implementation to achieve the high coverage with the vaccine as quickly as possible. It therefore requires substantial initial investment. Consequently, the price set for the vaccine should be reasonable to avoid limiting the coverage given the available budget. Suboptimal coverage leads to suboptimal benefit if herd protection is not fully achieved. This may disappoint health authorities and may result in program discontinuation. Conventional cost-effectiveness analysis is therefore not ideally suited to vaccine price setting, as it is based on the concept of ‘more for more’, i.e., higher health gain achieved at a higher reimbursement cost that does not account for limited budgets. Constrained optimization (CO) combines value assessment with constrained budget allocation into one analysis method and may therefore be the better option for vaccine pricing. Full article

Long-term intravenous therapies often necessitate the use of peripherally inserted central catheters (PICCs). Antimicrobial-coated PICCs have been introduced to minimize central line-associated bloodstream infections (CLABSIs). A decision-analytic cost-effectiveness model was developed from a societal perspective, utilizing real-world data concerning PICC-related complications and costs from Class 3A hospitals and community hospital settings in China. The analysis compared the quality-adjusted life years (QALYs) for patients receiving antimicrobial-coated PICCs versus standard PICCs, with catheter-associated costs included. Incremental cost-effectiveness ratios (ICERs) were calculated in Chinese Yuan (CNY) per QALY gained. Patients with antimicrobial-coated PICCs experienced slightly fewer complication-related events, leading to significantly lower costs for managing complications. In the Class 3A hospital setting, the average total cost per patient was lower with antimicrobial-coated PICCs (CNY 62,800) compared to standard PICCs (CNY 102,900), primarily due to the reduced expenses for treating CLABSIs and related unknown fever. The ICER demonstrated that the coated PICC was the strongest option, showing a negative ICER (cost-saving of approximately CNY 4 million per QALY gained in the base-case Class 3A hospital scenario). In community hospital care scenarios, the cost advantage of antimicrobial-coated PICCs remained. Thus, the antimicrobial-coated PICC strategy was cost-saving, providing equal or improved health outcomes at lower costs in China’s medical center and community hospitals, making it a more efficient choice for long-term vascular access. Full article

Wound infection significantly hinders the healing process. Clinical signs and symptoms (CSS) of infection are used to assess the presence of infection and guide whether to intervene. However, CSS may not be dependable, lacking sensitivity and specificity, and may not accurately reflect bacterial load. The interpretation of CSS can be subjective and can vary between clinicians since they depend on patient characteristics, type of wound, and stage of infection. In addition, conditions such as peripheral vascular disease or diabetes can mask the signs and symptoms of infection. Inaccurate or late diagnosis of infected wounds can be costly to the patient and to healthcare systems. Fluorescence imaging (FLI) provides a safe, objective, highly sensitive approach to detect clinically significant bacterial levels in wounds. This information allows individualized treatment plans and a way to monitor bacterial burden and wound healing longitudinally. This publication reviews the evidence for point-of-care FLI as a means of improving wound identification with a high bacterial burden and the clinical and healthcare economic benefits of earlier and more accurate detection of bacteria. 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

Pharmacy technicians (PT) are vital to the efficient and safe operation of hospital pharmacy services, fulfilling a range of technical and clinical responsibilities that directly impact patient care. However, increasing healthcare demands have underscored the importance of adequate staffing levels to sustain service quality and safeguard patient outcomes. This perspective paper explores how appropriate staffing levels for PT in hospital settings are essential and important to support safe, efficient care and a sustainable workforce. It compares evidence-informed staffing models, highlights real-world benchmarks, and proposes governance recommendations to guide policies that strengthen pharmacy services. Recommendations are made to inform clinical governance, suggesting that staffing policies, continuous training, and professional development programs are essential to supporting PT effectiveness and retention. The findings advocate for regulated staffing ratios and governance measures to foster an environment where PTs can deliver high-quality care and uphold safety standards within hospital pharmacies. Full article

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. Full article

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. 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

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. Full article

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. Full article

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