J. Mark. Access Health Policy, Volume 13, Issue 4 (December 2025) – 15 articles

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

The regulation of new medicine prices must balance financial sustainability with equitable access to innovation. Value-Based Pricing (VBP) strategies seek to align drug prices with their clinical and societal impact. The Pharmaceutical Innovativeness Index (PII) is a transparent and reproducible tool proposed to assess the degree of innovativeness of new medicines, with potential to support pricing decisions within economic regulation frameworks. An exploratory qualitative study was conducted through a focus group study was conducted with experts in health economics and pharmaceutical regulation to evaluate the applicability of the PII and to discuss key domains relevant to the assessment of pharmaceutical innovation. Responses were collected anonymously using an interactive digital platform and analyzed through inductive thematic content analysis. Based on these findings, the research team developed a conceptual pricing model integrating the PII with additional value-based criteria. Two hypothetical case studies were created to explore its practical feasibility. Participants identified Added Therapeutic Value (ATV) and Unmet Therapeutic Need (UTN) as the most relevant domains, while Methodological Quality (MQ) and Study Design (SD) were also recognized as essential to ensure rigor and reduce bias. The PII scores showed strong alignment with expert judgment in the illustrative case studies. The proposed model incorporates international best practices—such as the efficiency frontier approach—and additional dimensions including safety and incremental innovation. Overall, the PII demonstrated potential to enhance transparency, consistency, and regulatory efficiency in drug pricing decisions in Brazil. However, it should currently be regarded as an exploratory framework requiring further empirical validation and regulatory adaptation before implementation. Full article

Hard-to-heal wounds are a major burden to healthcare systems. Electrical stimulation therapy (EST) is known to improve clinical outcomes, but cost-effectiveness analysis is lacking. The aim was to explore the cost-effectiveness of EST with standard of care (SoC) versus SoC alone. A systematic review and meta-analysis of randomised controlled studies (RCTs) were conducted. Fourteen RCTs were identified, representing 783 patients. EST + SoC, versus SoC alone, significantly increased the proportion of wounds healed (odds ratio [OR] 2.46 [95% CI, 1.75–3.46], p < 0.0001) and significantly decreased the mean time to healing (−2.67 weeks (95% CI, 1.49–3.84, p < 0.00001). A cost-effectiveness model was developed based on these findings and on the usage and cost of the EST device used in the largest included RCT. Weekly costs of community wound care were taken from published estimates and inflated to 2024 levels, reflecting costs in the UK. In a hypothetical cohort of 100 patients treated over 12 weeks, EST + SoC was estimated to save over GBP 38,000 overall, reduce nursing visits by 385 and lead to 154 more ulcer-free weeks. In conclusion, EST used in the community is a cost-effective addition to SoC with the ability to improve outcomes and reduce human and financial burden of hard-to-heal wounds. Full article

This study aimed to determine the relative efficacy of bevacizumab gamma (an ophthalmic formulation of bevacizumab) versus alternative interventions relevant to the treatment of neovascular age-related macular degeneration (nAMD) in the United Kingdom (UK) via a systematic literature review (SLR) and network meta-analysis (NMA). An SLR was conducted to identify randomized controlled trials (RCTs) of anti-vascular endothelial growth factor (anti-VEGF) therapies for the treatment of nAMD in adult patients relevant to the UK context. The included anti-VEGF treatments were ranibizumab, aflibercept, faricimab, and bevacizumab gamma. Bayesian NMA models were used to estimate relative efficacy in terms of change from baseline (CFB) in best-corrected visual acuity (BCVA) at 12 months, the proportion of patients gaining 15 or more letters at 12 months, and the proportion of patients losing less than 15 letters at 12 months. Twenty-two relevant RCTs were included in the NMA. At 12 months, all anti-VEGF treatments were similarly efficacious to ranibizumab 0.5 mg every four weeks (Q4W) in terms of CFB in BCVA, the proportion of patients gaining 15 or more letters, and the proportion of patients losing less than 15 letters (except for ranibizumab 0.5 mg every 12 weeks [Q12W] and ranibizumab 0.5 mg pro re nata [PRN]). Bevacizumab gamma provided similar improvements in visual acuity to other anti-VEGF treatments. Full article

Objective: This confirmatory survey aimed to verify the criterion-related validity and reliability of the final version of the Medical Doctors’ Work-Related Quality of Life Questionnaire (WQMD-9), following partial revision of its content. This study also explored the questionnaire’s structure and scoring methods. Method: From June to July 2022, the WQMD-9 was administered to 98 MDs selected to match the statistical distribution of MDs in Japan. Criterion-related validity was evaluated using a visual analogue scale (VAS) as the reference standard, and reliability was examined using inter-dimension correlations and Cronbach’s α. Results: The correlation coefficient between the VAS score and the simple sum of WQMD-9 dimensions scores was 0.7891, supporting criterion-related validity. Cronbach’s α was 0.87, indicating acceptable reliability. Conclusions: The profile-type WQMD-9 consists of nine dimensions—“Workload,” “Working time,” “Collaboration,” “Clinical practice,” “Working conditions,” “Working environment,” “Feelings of fatigue,” “Work-life balance,” and “Career”—with five levels. In the confirmatory survey population, the WQMD-9 demonstrated criterion-related validity and reliability, suggesting that it can be utilized with simple total scoring approach. Full article

There remains a persistent lack of patient-centered evidence on insurance reform and real-world experiences of patients with chronic disease. This study gathered insights around insurance design from chronic disease beneficiaries. This mixed-methods analysis comprised an online survey and virtual focus group sessions (August to December 2023) involving US residents with chronic disease and health insurance. Patients’ perspectives on insurance design were explored. Survey data were analyzed descriptively. Key themes were identified from focus group transcripts and direct observations. In total, 146 patients across 15 chronic diseases completed the survey; 29 then participated in focus groups. Although most beneficiaries were satisfied with their health plan, concerns centered on prescription medication affordability due to high deductibles and cost exposure, the disproportionate effects of cost exposure based on income, and the unpredictability of out-of-pocket costs. For some, the financial burden led to financial debt, therapy abandonment, mental health issues, and/or worsening of their condition. Overall, there was broad support for policy solutions to redesign insurance and adjust cost exposure for patients with chronic disease. This research offers valuable patient insights into health insurance design in the US to ensure patients’ needs are addressed. Full article

Copay accumulator (CA) and copay maximizer (CM) programs in the United States, which prevent manufacturer copay assistance from counting toward deductibles or out-of-pocket (OOP) maximums, are increasingly used, raising concerns about costs and outcomes for patients with major depressive disorder (MDD) or bipolar disorder (BPD) treated with branded atypical antipsychotics (AAPs) and/or antidepressants (ADs). This retrospective claims study used Kythera commercial data (2020–2024) in the United States to identify adults with MDD or BPD who had at least 1 diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- (2020–2021) and post-index (2023–2024) and at least three months of post-index branded medication use. This retrospective claims study used Kythera commercial data (2020–2024) to identify adults with MDD or BPD who had at least one diagnosis and one branded AAP or AD prescription between 2021 and 2023, requiring 12 months’ continuous enrollment pre- and post-index and at least 3 months of post-index branded medication use. Patients were stratified into CA, CM, or standard copay plan (SCP) cohorts, and propensity score matching was used to compare treatment patterns and costs. Both CA and CM groups had significantly higher median OOP costs than SCPs (e.g., $75/$60 vs. $16 for MDD+AAP; p < 0.0001), and higher pharmacy costs among adherent patients. CA patients had poorer adherence and persistence, shorter treatment duration, and higher discontinuation and abandonment rates than SCPs. These findings highlight higher OOP burden and adherence challenges with CA and CM programs, underscoring the need for careful benefit design for US mental health patients. Full article

Background: Stakeholders’ perception plays a crucial role in shaping pharmaceutical strategies. Stakeholders are groups interested in pharmaceutical companies’ success and outcomes. Stakeholders’ perceptions are multifaceted and impact pharmaceutical strategies, from shaping research to enhancing market access, pricing, and corporate reputation. Understanding and actively managing stakeholders’ perceptions is vital for pharmaceutical companies to succeed in an increasingly complex and competitive industry. Methods: In this case study, knowledge contributions from stakeholders offered insights and strategies for application in the pharmaceutical sector. Results: Qualitative, exploratory research was conducted, which included the participation of sixteen stakeholders from different countries in Latin America, who responded to a semi-structured interview script, whose data were understood through lexical analysis in the Interface de R pour les Analyses Multimensionnelles de Texts et de Questionnaires (IRaMuTeQ). Conclusions: The results of this study underscore the importance of regulatory knowledge for professionals’ support and implementation of international strategies. Regulatory knowledge provides professionals with tools and insights to navigate complex regulatory environments, make informed decisions, and enhance organizational performance in global markets. Full article

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