Journal of market access & health policy最新文献

{"title":"EU-HTA-Why Do Words Matter?","authors":"Mondher Toumi, Bruno Falissard, Steven Simoens, Maarten Postma, Marta Wilk, Laurent Boyer, Renato Bernardini, Stefano Capri, Jaime Espin, Jürgen Wasem, Pascal Auquier","doi":"10.3390/jmahp14020031","DOIUrl":"10.3390/jmahp14020031","url":null,"abstract":"<p><p>Words matter because they have profound power to shape thoughts, emotions, actions, and social realities [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214955/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038349","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Between Rigor and Relevance: Why the EU HTA Guidelines on Indirect Comparisons Miss the Mark.","authors":"Samuel Aballéa, Mondher Toumi, Piotr Wojciechowski, Emilie Clay, Bruno Falissard, Steven Simoens, Pascal Auquier, Stefano Capri, Renato Bernardini, Joerg Ruof, Frank-Ulrich Fricke, Oriol Sola Morales, Laurent Boyer","doi":"10.3390/jmahp14020030","DOIUrl":"10.3390/jmahp14020030","url":null,"abstract":"<p><p>Indirect treatment comparisons (ITCs) are essential in the context of joint clinical assessments (JCAs) under Regulation (European Union [EU]) 2021/2282, bridging evidence gaps where head-to-head data are lacking and enabling assessment across diverse national patient, intervention, comparator, and outcome (PICO) requirements. This paper critically reviews the EU Health Technology Assessment Coordination Group's (HTACG) guidelines on direct and indirect comparisons, with particular focus on ITCs. While the guidelines promote transparency and rigorous evaluation of assumptions, they adopt a restrictive stance on assumption violations, the use of unanchored comparisons, and population-adjusted methods such as matching-adjusted indirect comparisons (MAIC) and simulated treatment comparisons (STC). The guidance shows limited support for Bayesian methods and undervalues meta-regression in favor of subgroup analyses. Operational implications for health technology developers (HTDs) are substantial, including new requirements for dual systematic reviews, multiple network structures, and shifted null hypothesis testing. Moreover, the guidelines effectively dissuade the use of non-randomized comparisons in rare or rapidly evolving indications and may inadvertently hinder access to effective treatments. Emerging practices such as external control arms (ECA) or target trial emulation are underdeveloped. Notably, there is no indication that the guidelines are grounded in systematic methodological validation studies. As JCAs evolve, greater methodological flexibility, empirical grounding, and clear operational guidance will be essential. Refining the guidelines along these principles would enhance their practical utility, mitigate intrinsic assessment variability, support consistent assessments across Member States (MS), and ultimately improve patient access to innovative therapies.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214804/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of a Digital Leakage Notification System on Healthcare Resource Utilisation and Costs for People with Stomas in the United Kingdom: Analysis of Interim Results from a Prospective Longitudinal Study.","authors":"Alexandra Baxter, Esben Bo Boisen, Majken Linnemann Jensen, Aamir Shaikh","doi":"10.3390/jmahp14020029","DOIUrl":"10.3390/jmahp14020029","url":null,"abstract":"<p><p><b>Background/Objectives</b>: Leakage of stomal effluent adversely affects the quality of life for people living with a stoma and increases healthcare resource utilisation and costs. The Heylo™ digital leakage notification system (DLNS) detects early signs of leakage under the baseplate, enabling proactive stoma management. We evaluated the impact of the DLNS on stoma-related costs in a real-world UK setting. <b>Methods</b>: This costing analysis used data from the first 100 DLNS users with 6 months of follow-up in an ongoing longitudinal observational study (NCT06554015). Costs were calculated as 3-month totals before DLNS (baseline): Month 3, and Month 6 for stoma-related healthcare provider consultations, hospitalisations, and ostomy solution components (pouching systems, supporting products, and DLNS sensor layers). Least squares (LS) means were estimated using a mixed model. <b>Results</b>: Total LS mean costs for stoma-related consultations plus ostomy solution use decreased significantly from baseline by 21.9% at Month 3 (-£304 [95% CI: -£456, -£153]; <i>p</i> = 0.001) and 24.5% at Month 6 (-£340 [95% CI: -£504, -£175]; <i>p</i> < 0.001). Stoma-related consultation costs decreased by 50.7% at Month 3 (-£309 [95% CI: -£447, 171]; <i>p</i> < 0.001) and 57.9% at Month 6 (-£353 [95% CI: -£493, -£213]; <i>p</i> < 0.001), driven by fewer physician and stoma care nurse consultations. Mean ostomy solution costs remained similar from baseline (£784/person) to Month 6 (£782/person; <i>p</i> = 0.955) as DLNS sensor layer costs were offset by less use of other ostomy products. <b>Conclusions</b>: Initiation of the Heylo™ DLNS was associated with significant cost savings from reduced stoma-related consultations, pouching systems, and supporting product use.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214903/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038288","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessment and Appraisal of Drug Innovativeness in Italy: Ultimate Evidence on Key Drivers and Consistency.","authors":"Alvise Verde, Federica Turati, Clara Trimarchi, Carlotta Galeone, Claudio Jommi","doi":"10.3390/jmahp14020028","DOIUrl":"10.3390/jmahp14020028","url":null,"abstract":"<p><p>This study aims to update and integrate empirical evidence on the key drivers and consistency of the appraisals of drug innovativeness in Italy by the Italian Medicines Agency (AIFA), and discuss if this evidence is supportive of the reform and requirements implemented in 2025. Appraisals from July 2017 to December 2024 were retrieved from the AIFA website. The association between the innovativeness appraisal, the innovativeness domains (unmet need/added therapeutic value/quality of evidence) and disease/drug/evidence-specific variables was assessed using odds ratios (ORs) from binary/multinomial logistic regression models. Innovativeness status was strongly associated with added therapeutic value (OR > 70). Medicines for rare diseases were more likely to receive conditional innovativeness (OR = 2.95). Full innovativeness was more frequently recognized for indications including paediatric patients (OR = 3.60). References to severe diseases and patient-reported outcomes (PROs) had a higher, not statistically significant, likelihood of innovativeness, whereas reference to indirect treatment comparisons had a lower likelihood (OR = 0.18). The appraisal process showed high internal consistency, but its regulation needs more specific guidance. The innovativeness regulation was reformed in July 2025, including specific recommendations on the criteria to identify the alternative treatments; the role and robustness of indirect comparisons; and the role and requirements for PROs. Our evidence provides an empirical rationale for this reform.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214850/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038317","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Shelf Life of Adrenaline Auto-Injectors at Pharmacies in Denmark, Finland, Sweden, and Norway.","authors":"Jesper Nørregaard, Christoffer Mertz, Anne Danø, Jeppe Hæstrup Kamstrup, Mille Vang Lybech","doi":"10.3390/jmahp14020026","DOIUrl":"10.3390/jmahp14020026","url":null,"abstract":"<p><p>There is a well-documented gap between the prescription of adrenaline auto-injectors (AAIs) and their real-world use during anaphylaxis. Although several aspects of AAI underuse have been investigated, the potential role of shelf life in influencing patient adherence has not been quantified. This study assessed the real-world remaining shelf life of AAIs available at pharmacies in Denmark, Finland, Sweden, and Norway, using pharmacy-level stock data and pharmacy employee-reported perceptions. Across Denmark, Finland, and Sweden, the average remaining shelf life was 9.6 months, and in Norway it was 10.5 months at the point of dispensing. In Denmark, Finland and Sweden, 100%, 91%, and 94% of employees, respectively, considered shelf life an important or very important factor when dispensing AAIs to patients. Our findings suggest that patients and caregivers filling prescriptions for AAIs frequently receive devices with limited remaining shelf life, which may necessitate multiple renewals per year. This has potential implications in terms of adherence to clinical guidelines, dependence of expired devices during emergencies, patient cost, caregiver burden, and overall societal expenditure. These results highlight an unmet need for emergency treatment options with longer shelf life to better support continuous access to life-saving medicine during anaphylaxis.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214919/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038476","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Primary Care or Primary Problem? Aligning Access Pathways with Patient Needs Across the Care Continuum.","authors":"Gregory J Privitera, James J Gillespie, Alexa Walton","doi":"10.3390/jmahp14020027","DOIUrl":"10.3390/jmahp14020027","url":null,"abstract":"<p><p>In the United States, access to healthcare is shaped not only by patient need but also by payer policies that determine which providers are reimbursable, how care is sequenced, and what constitutes a legitimate entry point into the system. These gatekeeping functions, while valuable for supporting clinical prioritization, risk stratification, and continuity of care, can also unintentionally reinforce structural inequities and credential hierarchies that delay or limit timely and equitable care, particularly for historically marginalized populations. While reform efforts often focus on expanding benefits or provider networks, fewer address the underlying design of access itself or the rules that govern how patients enter care. It is argued in this paper that a more equitable and efficient healthcare system requires multi-entry care models, in which nurses, behavioral health clinicians, pharmacists, and community health workers may serve as condition-appropriate, reimbursable first points of contact within coordinated care teams. Drawing on evidence from Medicare, Medicaid, the Veterans Health Administration, and commercial payers, these models may support cost containment, improve care coordination, facilitate appropriate utilization, and promote earlier patient engagement. While findings from these models are not uniform across all settings, evidence suggests that outcomes are highly dependent on implementation context, system design, and supporting infrastructure. When implemented with appropriate safeguards (such as interoperable health records, team-based care requirements, and coordinated referral tracking), multi-entry systems can preserve continuity while expanding access. Payers are uniquely positioned to lead this transformation by aligning reimbursement policy with patient needs, supporting team-based care infrastructure, and embedding accountability into access pathways, thereby creating a system that can be more responsive, inclusive, and sustainable.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214796/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038388","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Introducing an Expanded Value Framework in Health Technology Assessment of Vaccines.","authors":"Farzaneh Eslami, Thi Hao Pham, Angga P Kautsar, Cao Ba Khuong, Cornelis Boersma, Mondher Toumi, Jurjen van der Schans, Maarten J Postma","doi":"10.3390/jmahp14020024","DOIUrl":"10.3390/jmahp14020024","url":null,"abstract":"<p><p>Health Technology Assessment (HTA) frameworks increasingly recognize the broader value elements of vaccines; however, their adoption remains inconsistent across jurisdictions and often incomplete in practice. Many HTA processes continue to prioritize narrow clinical outcomes and direct costs, leading to the underrepresentation of the full preventive and long-term benefits of vaccination. Building on the ISPOR \"Elements of Value\" framework and recent evidence, this study adapts and expands existing models specifically for vaccines to enhance HTA applicability in both high-income and resource-limited settings. We introduce an updated vaccine value framework comprising 21 distinct value elements. Notably, the original model was expanded by introducing four entirely new value drivers: (1) real-world evidence; (2) control of antimicrobial resistance; (3) health system strengthening; and (4) environmental impact. Additionally, existing elements were refined, such as broadening \"fear of contagion\" to \"peace of mind\" and expanding \"productivity\" to capture education and leisure gains. We map these elements to potential data sources and methodological tools to facilitate their inclusion in HTA. This study offers an operational, holistic, and context-sensitive framework that reflects current advancements in assessment. By capturing the full spectrum of vaccine value, this framework aims to support more comprehensive, transparent, and equitable HTA decision-making for global immunization programs, while considering conceptual overlap between value elements to reduce the risk of double counting.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214781/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Market Access Delays on Time to Patient Access: Multi-Country Comparative Analysis Assessing the First Commercial Launch Indications for Five Oncology Medicines Across Europe and Canada.","authors":"Barry Crean, David Parry, Alison Horsfield, James Ryan, Nektarios Oraiopoulos","doi":"10.3390/jmahp14020025","DOIUrl":"10.3390/jmahp14020025","url":null,"abstract":"<p><p><b>Background:</b> The benefit of pharmaceutical innovation manifests when patients access treatment. Following regulatory approval in Europe and Canada, reimbursement decisions depend on health technology assessments (HTAs), which can be prolonged. To quantify the impact of delays on patients, we evaluated market access timelines for olaparib, osimertinib, durvalumab, acalabrutinib, and trastuzumab deruxtecan across six high-income countries with established HTA systems (Canada, England, France, Germany, Italy, Spain). <b>Methods:</b> Time to access was from regulatory approval to reimbursement. Survival benefit was median overall survival (OS) and progression-free survival (PFS) assessed versus the comparator at approval and the latest data cut-off. The number of eligible patients per year multiplied by the years to patient access and survival benefit reflects the lost survival benefit. <b>Results:</b> Efficacy benefits observed at approval continued to the latest data cut-offs. The mean time to patient access was 18 months. Although this varied by country and treatment, with England and Germany typically being the fastest and France and Spain the slowest, timelines often exceeded the 180-day EU target despite identical evidence used in HTA submissions. This resulted in an estimated mean of 2836 patients being unable to access treatment and 3391 OS-derived and 2739 PFS-derived life-years lost. <b>Conclusions:</b> Access processes must evolve to ensure the timely realization of new medicines' benefits.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214782/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038294","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation.","authors":"Walter Van Dyck, Sissel Michelsen, David Veredas, Isabelle Huys, Jeroen Luyten, Steven Simoens","doi":"10.3390/jmahp14020023","DOIUrl":"10.3390/jmahp14020023","url":null,"abstract":"<p><p>Spreading payments over time by means of annuities has been proposed as a means to address the affordability challenge of funding very expensive advanced therapies, especially within managed entry agreements. This study aims to examine when annuities (in contrast with a single upfront payment) offer a viable solution for both healthcare payers and manufacturers to fund one-time advanced therapies. We put forward four conditions under which annuity-based payments can be considered an acceptable payment strategy: (1) excessive budget impact, (2) cost equivalence with upfront payment, (3) compensation for financial risk and (4) a limited annuity period. We develop an exploratory model that simulates how the budget impact of annuity-based payments for advanced therapies meets these conditions across several economic and epidemiological scenarios. Given our model parameter values, results suggest that annuity-based payments are most suitable when the initial patient volume (prevalence) significantly exceeds annual new cases (incidence), and when the financial risk premium for the annuity-based payment scheme does not exceed the social discount rate. While further refinement of the model is needed, this study demonstrates that annuity-based payments can only help control the annual budget need when the focus is on a high-prevalence disease, and the therapy is financed through health impact bonds issued by a governmental payer. This arrangement ensures a low-risk premium, which is typically only available to public payers.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108133/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791449","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Household Out-of-Pocket Burden Costs for Pediatric Pneumonia in Low- and Middle-Income Countries: Evidence Review and Econometric Framework.","authors":"Ioannis Smaraidos, Maria Kyrmanidou, Asterios Kampouras","doi":"10.3390/jmahp14020022","DOIUrl":"10.3390/jmahp14020022","url":null,"abstract":"<p><p>Pediatric pneumonia remains a major cause of morbidity and mortality in low- and middle-income countries (LMICs), imposing both health and financial burdens. While the clinical aspects of pediatric pneumonia are well-studied, less attention has been paid to its economic implications for households, particularly regarding out-of-pocket (OOP) expenditure. This paper synthesizes current evidence from Kenya, India, Bangladesh, and Vietnam and introduces a proposed econometric framework designed to identify cost determinants and model policy interventions. The framework integrates microeconomic data, identifies cost determinants, and models the effects of clinical and policy factors (e.g., intensive care, vaccination, insurance coverage) on household expenditures. Simulated results illustrate potential findings from such an approach. Existing studies show substantial variability in hospitalization costs, with OOP payments ranging from US$30 to US$250 per episode, often exceeding 20% of monthly household income. Econometric modeling using generalized linear models (GLMs) and difference-in-differences (DiD) can disentangle the impact of hospital practices, disease severity, and policy interventions. Simulated regression results demonstrate that length of stay, intensive care admission, and absence of insurance significantly increase household costs, while pneumococcal conjugate vaccine (PCV) introduction reduces both admissions and financial burden. Hospitalization for pediatric pneumonia imposes significant OOP costs on households in LMICs. An econometric framework provides rigorous tools to estimate cost drivers, evaluate policy impacts, and guide equitable health financing reforms.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13108171/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147791277","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}