Journal of market access & health policy最新文献

{"title":"Valuation of Medical Innovation in Orphan Diseases with a Focus on Small Investors and Limited Diversifiable Risks.","authors":"Mark Nuijten, Pieter van Gelder","doi":"10.3390/jmahp14030047","DOIUrl":"10.3390/jmahp14030047","url":null,"abstract":"<p><p>This paper assesses the impact of uncertainty for investors on the economic valuation of medical innovation projects for orphan drugs or rare diseases. Conventionally, investor evaluation uses the deterministic discounted cash flow (DCF) method with an appropriate sensitivity analysis that captures some level of uncertainty. In healthcare, and particularly for rare diseases, the levels of uncertainty in financial outcomes (return on investment and net present value (NPV)) are broader than the ones normally captured by the DCF formula. Uncertainties include R&D costs, the approval process (level and timing) for obtaining reimbursement, sales, the production cost, and the failure probabilities of the clinical trial phases, to name a few. Additionally, there is not only one type of investor to consider, but different investors exposed to different levels of risk management of their investment. Our analysis tried to capture those two dilemmas (higher levels of uncertainty and different investor types) in two ways. One way was to identify a better method to enhance the different levels of uncertainty. The real option method of evaluation was proposed instead of DCF. For instance, the real option method better captures the uncertainty of the different phases of product development. The other way is to differentiate the investor types through their level of risk assessment perspectives. Small investors and start-up companies may see more benefit in applying the real option methodology to estimate their NPVs at different time points during product development. In summary, our evaluation identified various types of uncertainty when assessing an investment, along with methods to manage their effect on the economic/financial outcomes of medical innovations. Given the high uncertainty associated with early-stage drug development, such as orphan drugs for rare diseases, the real options approach is preferable to traditional DCF models. The analysis also showed that there is not just a single investor perspective to consider but specific perspectives that enhance the prime use of the real option methodology.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510584/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834932","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":"Guidance on Multiplicity Analysis in Single-Trial Assessments: A No-Solution Equation.","authors":"Mondher Toumi, Bruno Falissard, Asma Jouini, Pascal Auquier, Claude Dussart, Laurent Boyer","doi":"10.3390/jmahp14030046","DOIUrl":"10.3390/jmahp14030046","url":null,"abstract":"<p><p>The European Health Technology Assessment (EU HTA) guidance on Multiplicity of Hypothesis Testing (MHT) [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510491/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834041","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":"The European Union's Health Technology Assessment Regulation (EU-HTA R) Will Prosper Despite Major Setbacks.","authors":"Mondher Toumi, Imen Soussi, Bruno Falissard, Steven Simoens, Asma Jouini, Maarten Postma, Juergen Wasem, Oriol Solà-Morales, Laurent Boyer, Claude Dussart, Borislav Borissov, Renato Bernardini, Stefano Capri, Jaime Espin, Pascal Auquier","doi":"10.3390/jmahp14030045","DOIUrl":"10.3390/jmahp14030045","url":null,"abstract":"<p><p><b>Background:</b> The EU Health Technology Assessment Regulation (EU-HTA R), effective January 2025, mandates Joint Clinical Assessments (JCAs) to harmonize HTA across Member States. However, its implementation raises fundamental questions about methodological coherence, institutional capacity, and epistemological alignment. <b>Objectives:</b> This manuscript (1) systematically assesses whether the stated strategic and operational objectives of the EU-HTA R are achievable under current implementation conditions; (2) examines the implications for EU institutional legitimacy if these objectives are not met; and (3) proposes an epistemological framework as a prerequisite for developing a coherent joint HTA methodology. <b>Methods:</b> We conducted a critical policy analysis of the EU-HTA R, its implementing guidance documents, and published templates, supplemented by a comparative review of Member State HTA methodologies and their underlying philosophical foundations. <b>Results:</b> The analysis reveals that the EU-HTA R is unlikely to achieve its strategic goals under current conditions. Key findings include: guidance documents of substandard methodological quality; a restricted assessment scope that excludes scientific judgement and contextualization; insufficient resources and additional workload for national HTA bodies without reducing existing obligations; unresolved epistemological divergences among Member States spanning Bayesian vs. frequentist approaches, Fisher vs. Neyman-Pearson frameworks, and utilitarian vs. deontological ethical foundations; and procedural shortcomings in stakeholder consultation and expert involvement. These shortcomings risk undermining the epistemic authority and legitimacy of EU institutions. <b>Conclusions:</b> Prior epistemological and normative alignment across Member States is a prerequisite for any robust shared HTA methodology. Revisions to the EU-HTA R and comprehensive updates of guidance documents are necessary, with concrete safeguards-including independent peer review, identified authorship, and adequate resourcing-to ensure substantive rather than merely nominal implementation. A phased roadmap is proposed: establishing clear objectives, aligning epistemological foundations, developing institutional structures, and creating operationally consistent guidance.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510476/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834068","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":"Direct Public-Payer Costs of Dialysis Therapies in Greece: A Patient-Level Micro-Costing Study Across Modality and Provider Sector.","authors":"Vasileios Zavvos, John Fanourgiakis, Michael A Talias, Aimilia Zachariou, Marios Papasotiriou, Christos Iatrou, Nikolaos Kontodimopoulos, Christos Ntais","doi":"10.3390/jmahp14030044","DOIUrl":"10.3390/jmahp14030044","url":null,"abstract":"<p><strong>Background: </strong>Dialysis is a life-sustaining but resource-intensive treatment for patients with kidney failure. Contemporary Greek evidence on the public-payer cost of dialysis is limited, particularly for comparisons across modality, provider sector and treatment subtype.</p><p><strong>Methods: </strong>We performed a retrospective observational micro-costing study using 2022 patient-level data from five hemodialysis units and one peritoneal dialysis unit in Greece. The perspective was that of the public payer and public health budget. Annual costs included dialysis treatment and consumables, patient allowances and transport reimbursement, dialysis-related drugs, laboratory and imaging tests, hospitalizations and public dialysis-unit operating costs where applicable.</p><p><strong>Results: </strong>The analytic sample included 359 patients: 337 receiving hemodialysis and 22 receiving peritoneal dialysis. Median annual total cost was EUR 35,902.53 for hemodialysis and EUR 57,136.44 for peritoneal dialysis per patient. Peritoneal dialysis generated higher annual cost than hemodialysis, mainly because of treatment and consumable costs, and remained more costly after excluding dialysis-unit operating costs in sensitivity analysis. Public-sector hemodialysis had higher recorded median annual public-payer/public-budget cost than private-sector hemodialysis (EUR 38,794.55 versus EUR 34,836.98), but this difference was no longer statistically significant after excluding dialysis-unit operating costs. Hemodiafiltration was more costly than conventional hemodialysis, and automated peritoneal dialysis was more costly than continuous ambulatory peritoneal dialysis. National extrapolation estimated annual public expenditure of EUR 466.6 million using mean costs and EUR 446.8 million using median-based sensitivity estimates.</p><p><strong>Conclusions: </strong>Dialysis imposes substantial annual public expenditure in Greece. In this dataset and reimbursement context, peritoneal dialysis was more costly than hemodialysis, while private-sector hemodialysis generated lower recorded public-payer cost than public-sector provision. The apparent public-private hemodialysis cost difference was sensitive to the treatment of operating costs. These results may help inform reimbursement review, procurement policy and future budget-impact analyses of dialysis service redesign.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13511122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148835565","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":"A Real-World Clinical Trial Evaluating Satisfaction and Experiences with a Dexamethasone Mouth Film Compared to Corticosteroid Tablets as Rescue Medication for Moderate to Severe Acute Allergic Reactions.","authors":"Leif Bjermer, Göran Tornling, James Kereki, Karin Wahlberg, Bahram Javizian, Jonas Hjelmgren","doi":"10.3390/jmahp14030043","DOIUrl":"10.3390/jmahp14030043","url":null,"abstract":"<p><p>Despite the need for immediate treatment during acute allergic reactions (AARs), many patients do not consistently carry medication. A new mouth-dissolving dexamethasone film offers a portable, easily administered alternative to corticosteroid tablets. This non-randomized, open-label, low-interventional real-world trial assessed satisfaction (accessibility and safety/security) with the mouth film versus betamethasone tablets in adults prescribed tablets for moderate to severe AARs at a Swedish primary care center. Over six months, participants had access to both treatments and reported monthly via an electronic diary; qualitative responses were converted to Likert scales where applicable. Of 50 enrolled, 44 provided diary data (mean age 50 years, 74% female, 58% had an epinephrine autoinjector). In total, 189 responses were collected: 98% stated that the participant was satisfied or very satisfied with mouth film accessibility versus 58% for tablets (mean Likert scores 3.5-3.7 vs. 2.5-2.8; <i>p</i> < 0.001). A higher feeling of safety with the mouth film versus tablets was reported in 78% of responses and improved medication carriage compliance in 77%. At six months, 69% preferred the film. Of 18 AARs in total reported by 13 participants, the mouth film was chosen for treatment of 16 (11 participants); in 94%, the mouth film was found immediately accessible, and in 94%, the efficacy was rated as good or very good. In conclusion, the mouth film improved perceived accessibility, safety/security, and medication-carriage compliance compared with tablets for the treatment of AARs.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13511584/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148835522","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":"Multi-Criteria Decision-Making Framework for Sustainable Innovation Management in the Mexican Medical Device Manufacturing Industry: An Exploratory and Interdisciplinary Analysis.","authors":"José Cozain-Hernández, Josué Aarón López-Leyva, Miguel Angel Ponce-Camacho, Víctor Manuel Ramos-García","doi":"10.3390/jmahp14030041","DOIUrl":"10.3390/jmahp14030041","url":null,"abstract":"<p><p>The medical device manufacturing industry in Mexico faces a critical risk of losing competitiveness and sustainability due to its concentration on low-value-added manufacturing activities and limited integration into advanced stages of the value chain, such as R&D. This research addresses the lack of validated quantitative methodologies to identify the critical factors that promote sectoral sustainability in the national context. Through a literature review and the analysis of MCDM, a taxonomy of the problem was developed that integrates dimensions of governance, technological innovation, and human capital. The findings emphasize the need to transition toward circular economy and additive manufacturing models, supported by hybrid algorithms such as AHP, TOPSIS, and DEMATEL to mitigate uncertainty in strategic decision making. As a main result, an innovation management flow aligned with international standards and several maturity levels (TRLs, MRLs, CRLs, and PRLs) is proposed, providing a structured roadmap to scale the Mexican industry toward more-sophisticated global segments.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510750/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148834114","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":"Analysis of How the Term \"Shall\" Is Understood and Applied to the EU-HTA Regulation.","authors":"Mondher Toumi, Samuel Aballéa, Bruno Fallissard, Claude Dussart, Pascal Auquier, Laurent Boyer","doi":"10.3390/jmahp14030042","DOIUrl":"10.3390/jmahp14030042","url":null,"abstract":"<p><p>The European Health Technology Assessment (EU-HTA) Regulation extensively uses the word \"shall\", and its use has caused confusion among audiences unfamiliar with legal terminology [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510832/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148835559","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":"Epistemological Tensions in the EU HTA Joint Clinical Assessment: The Illusion of Judgment-Free Evaluation.","authors":"Mondher Toumi, Bruno Falissard, Asma Jouini, Imen Soussi, Steven Simoens, Maarten Postma, Laurent Boyer, Borislav Borissov, Renato Bernardini, Stefano Capri, David Danko, Claude Dussart, Frank-Ulrich Fricke, Pascal Auquier","doi":"10.3390/jmahp14030040","DOIUrl":"10.3390/jmahp14030040","url":null,"abstract":"<p><p>The practice of Health Technology Assessment (HTA) inherently involves moral, epistemological, and ontological commitments, which shape how assessments are conducted, interpreted, and used for decision-making [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13510361/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148833978","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":"Market Care and Custody: A Health Policy Analysis of Incarceration and Long-Term Care Systems in the U.S.","authors":"Travis W Milburn, Iffath Unissa Syed","doi":"10.3390/jmahp14030039","DOIUrl":"10.3390/jmahp14030039","url":null,"abstract":"<p><p>The United States has one of the world's largest criminal justice systems, with nearly 5.5 million people under correctional supervision and almost 2 million incarcerated. This scale of confinement, coupled with the rise of privatization across correctional and related services, reflects a broader neoliberal trend in public governance. This paper explores the consequences of privatization and marketization of the U.S. criminal justice system-particularly the proliferation of private prisons and immigrant detention centers-and draws parallels to the consequences of privatization of health and social care, especially long-term care (LTC). Both systems reveal shared logics of marketization that prioritize profit maximization, efficiency, and cost-cutting at the expense of care, justice, and equity. Relying on interdisciplinary perspectives from public health and criminology, this paper situates private corrections within the health policy framework of the Commercial Determinants of Health (CDoH), arguing that privatized carceral institutions not only harm incarcerated individuals but also endanger workers, families, and surrounding communities through systemic under-resourcing, precarious labor conditions, and structural violence. By comparing the private, for-profit prison industry with private for-profit LTC systems, we illustrate how these structures have commodified both care and correctional systems. These findings suggest that privatization within carceral and care sectors perpetuates health inequities and reinforces cycles of racial, gendered, and economic disadvantage. Accordingly, this paper calls for strengthening publicly held models and a reassertion of public accountability and interdisciplinary collaboration to restore social justice, health, and human dignity as central organizing principles of both systems for residents, workers, their families, and communities.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13397998/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148581395","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":"The Analytical Framework of Clinical Trials Evaluating Clinical Outcomes of Artificial Intelligence-Based Digital Health Interventions: A Systematic Literature Review.","authors":"Vladimir Zah, Dimitrije Grbic, Carl Asche, Filip Stanicic","doi":"10.3390/jmahp14030038","DOIUrl":"10.3390/jmahp14030038","url":null,"abstract":"<p><p><b>Introduction:</b> This systematic literature review (SLR) provides an analytical framework for clinical trials evaluating clinical outcomes of artificial intelligence-based digital health interventions (AI-DHI). <b>Methods:</b> The SLR was conducted in accordance with the PRISMA guidelines. Search was conducted (September 2025) in PubMed and Embase. Population included patients using AI-DHI. Only clinical trials exploring clinical outcomes, written in English, were considered. NICE checklist was used to assess studies' quality. Results were analyzed descriptively. <b>Results:</b> Final sample had 84 studies, with metabolic (28.6%), musculoskeletal (20.2%), and mental health disorders (19.0%) as the most common indications. Most studies (75.0%) were controlled, parallel-group trials with 2+ arms, mostly comparing AI-DHI with standard-of-care or waitlist. Although type of intervention often precludes blinding (64.3% were open-label), a double-blinding is strongly recommended (only 6.0%). Only 9.5% of studies were conducted at multiple sites across different countries. Dropout rates in the total sample and each study arm should be <20% at all endpoints (64.3%). Statistical tests were used based on the outcome measures. The small sample sizes and limited generalizability of findings were reported as the main limitations. <b>Conclusions:</b> This SLR emphasized current methodological gaps and an urgent need for unified global guidelines. Standard SLR limitations apply to this research.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13398107/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148581424","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}