Journal of market access & health policy最新文献

{"title":"The EU-Joint Clinical Assessment Guidance Documents Fail to Address the Significance of Systematic Literature Reviews and Deviate from the State of the Art.","authors":"Beata Smela, Mondher Toumi, Samuel Aballéa, Steven Simoens, Laurent Boyer, Bruno Falissard, Renato Bernardini, Stefano Capri, Pascal Auquier","doi":"10.3390/jmahp14030037","DOIUrl":"10.3390/jmahp14030037","url":null,"abstract":"<p><p>This paper summarizes an analysis of the Joint Clinical Assessment (JCA) subgroup's recommendations for systematic literature reviews (SLRs). While the JCA offers clear guidance on study classification, exclusion criteria reporting, and PRISMA diagram use, several of its recommendations diverge from established best practices in evidence-based medicine (EBM). A comparison with recognized guidelines, such as those from Cochrane and EUnetHTA, reveals that the JCA guidance may lack reliability, comprehensiveness, and reproducibility. Aligning JCA recommendations with gold standards in SLR methodology would address these shortcomings and enhance methodological rigor.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13397900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148581370","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":"Joint Clinical Assessment in the EU HTA Regulation-Would Drugs Supported by Single-Arm Trials Fit Under Evaluation?","authors":"Krzysztof Kloc, Mondher Toumi, Elżbieta Łukomska, Malwina Kowalska, Inez Tyrała-Chowaniec, Steven Simoens, Jürgen Wasem, Laurent Boyer, Claude Dussart, Pascal Auquier","doi":"10.3390/jmahp14020036","DOIUrl":"10.3390/jmahp14020036","url":null,"abstract":"<p><p>The Joint Clinical Assessment (JCA) evaluates the relative effectiveness (RE) of interventions over comparators. While randomised control trials (RCTs) are considered the gold standard, single-arm trials (SATs) require an external control for accurate RE estimation. This study reviewed Health Technology Assessment (HTA) outcomes for medicinal products supported by SATs in France, Germany, Poland, and Spain, and simulated the JCA for these products based on evidence submitted in France. Among HTA evaluations published in France in 2019-2024, 16% were SAT-driven, and 5.6% of them included external controls. SAT-supported drugs had a high reimbursement approval rate (74%) and showed better HTA outcomes when controls were used. In Germany, 64% of SAT-based HTA outcomes indicated no added benefit and 30% a non-quantifiable benefit. In Poland and Spain, 63% and 72% HTA evaluations recommend reimbursement, respectively. Despite wide acceptance by Member States, experts determined that 94% of SAT-supported products would not qualify for JCA review due to insufficient evidence. Only 6% would qualify for JCA for a likely limited number of PICOs (Population-Intervention-Comparator-Outcome), but the certainty rating would be low. These findings suggest that SATs, as primary evidence, may not be suitable for JCA, potentially undermining HTA in EU Member States.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-06-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13301992/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148321039","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":"Supporting the Implementation and Addressing the Challenges of the EU HTA Regulation.","authors":"Elaine Julian, Mondher Toumi, Fabrizio Gianfrate, Renato Bernardini, Stefano Capri, Mira Pavlovic-Ganascia, Oriol Solà-Morales, Antonella Cardone, Emilia Strycharz-Angrecka, Valentina Strammiello, Jean-François Bergmann, Daniel Widmer, Walter Van Dyck, Frank-Ulrich Fricke, Patrick Tilleul, Jörg Ruof","doi":"10.3390/jmahp14020035","DOIUrl":"10.3390/jmahp14020035","url":null,"abstract":"<p><p>Four years after the European Regulation on Health Technology Assessment (EU HTAR) came into force and a little over a year after the implementation phase of the Regulation started, with the publication of the first Joint Clinical Assessment (JCA) report imminent, it is good timing to take a step back and revisit what has been achieved and look at what the main challenges on the way forward are [...].</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13301416/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148321016","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":"Operative Costs and Anesthesia Exposure Time for Pediatric Glaucoma Surgeries.","authors":"Scott D Sorenson, Raymond G Areaux","doi":"10.3390/jmahp14020034","DOIUrl":"10.3390/jmahp14020034","url":null,"abstract":"<p><p><b>Background</b>: Pediatric glaucomas are sight-threatening conditions that frequently require surgical intervention and may expose vulnerable young patients to repeated episodes of general anesthesia. While the clinical efficacy of pediatric glaucoma surgeries has been described, comparative data on operative cost and anesthesia exposure time are scarce, limiting evidence-based decision-making for surgeons, caregivers, and health systems. <b>Objective</b>: To quantify and compare the operative costs and total operating room (OR) time of common pediatric glaucoma surgeries, with particular attention to unilateral angle surgery versus immediately sequential bilateral angle surgery (ISBAS), and to single-incision ab interno trabeculotomy (SIT) versus other angle techniques. <b>Methods</b>: A retrospective review of patients who received glaucoma surgery by one of three experienced glaucoma surgeons between January 2012 and August 2019 at the University of Minnesota Medical Center. Each surgery was classified by type, cost, and operative time. <b>Results</b>: A total of 160 surgical encounters were analyzed. Average total cost was $6564 for all unilateral procedures, $6782 (±1952.03) for unilateral angle surgeries, and $11,391 (±2396) for ISBAS. Mean OR time was 121 min for all unilateral procedures, 120 min (±46.3) for unilateral angle surgeries, and 208 min (±53.8) for ISBAS-approximately the cost and time of two unilateral angle surgeries combined into a single anesthetic encounter. Compared to separate encounters for bilateral angle surgeries, ISBAS saved $2174 (<i>p</i> = 0.008) and trended toward saving 33 min of OR time (<i>p</i> = 0.068). Single-incision ab interno trabeculotomy (SIT) was $1647 more expensive than conventional incisional goniotomy or trabeculotome trabeculotomy (<i>p</i> < 0.0001) and reduced OR time by 23.5 min (<i>p</i> = 0.011). SIT was as expensive as 360-degree trabeculotomy ab externo with the iScience^TM catheter (<i>p</i> = 0.098) and reduced OR time by 74.4 min (<i>p</i> = 0.0004). There was no difference in complications between unilateral surgery and ISBAS. <b>Conclusions</b>: Reduced cost and a trend toward reduced anesthesia time support the use of ISBAS. SIT substantially reduced general anesthesia exposure for a neutral or slightly increased cost.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13302323/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148321063","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 Decision Tree Cost Analysis of Intracranial Bleed Detection Using a Near-Infrared Device Across Various Healthcare Levels.","authors":"Mamta Patel, Amit Kumar Mittal, Mohit Agrawal, Oshima Sachin, Kavitha Rajsekar, Bharat Choudhary, Suryanarayanan Bhaskar, Kuldeep Singh","doi":"10.3390/jmahp14020033","DOIUrl":"10.3390/jmahp14020033","url":null,"abstract":"<p><p>Traumatic brain injury (TBI), mainly caused by road traffic accidents, is a serious global public health concern. Computed tomography (CT) is the best way to detect intracranial haemorrhage (ICH), but it is not always feasible because it is hard to access, exposes people to radiation, and is expensive, especially in low- and middle-income countries. Portable near-infrared spectroscopy (NIRS) devices offer a non-invasive, point-of-care option for early detection of ICH. The objectives of this study were to estimate the cost per case detected for patients with mild-to-moderate TBI, to estimate incremental cost and to perform a budget impact analysis to assess the financial feasibility of implementing this technology. This study employed a decision tree model from a health system perspective to calculate the cost per detected case and the incremental cost of NIRS across three tiers of care: ambulances, community health centres (CHCs), and tertiary hospitals. The cost per mild-to-moderate TBI case found was Rs. 2177.90 in ambulances, Rs. 748.09 in CHCs, and Rs. 628.14 in tertiary hospitals. The extra cost per patient was Rs. 984.15, Rs. 360.90, and Rs. 289.78, respectively. At the system level, NIRS raised the total costs for 264 ambulance patients from Rs. 37.71 lakh to Rs. 40.31 lakh and for 858 CHC patients from Rs. 115.64 lakh to Rs. 118.73 lakh. National extrapolation indicates a first-year budgetary impact of approximately Rs. 442 crores for ambulances and Rs. 187 crores for CHCs. These results support the strategic, phased implementation of NIRS to use resources better and improve early diagnosis of TBI.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13302714/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148320994","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":"Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies.","authors":"Oliver Blandy, Pierluigi Lembo, Rebecca Folorunso, Karl-Johan Myren, Helene Chevrou-Severac, Simu K Thomas","doi":"10.3390/jmahp14020032","DOIUrl":"10.3390/jmahp14020032","url":null,"abstract":"<p><p><b>Background:</b> Real-world evidence (RWE) can complement clinical trials to inform health technology assessments (HTAs). This study examined the extent to which RWE is considered in HTAs of non-oncology orphan medicinal products across six agencies globally. <b>Methods:</b> Published European Medicines Agency decisions were reviewed to identify approved non-oncology orphan medicinal products (2018-2023) that included RWE within their submission package, which was anticipated to align with the inclusion of RWE in HTA submissions. Data were extracted from the corresponding HTA reports published by six national agencies (Australia, Canada, France, Germany, Sweden, and the UK). <b>Results:</b> RWE was included in 105 regulatory submissions and 52.6% of the corresponding HTA reports (range: 29.9% [Germany] to 78.8% [Canada]), nearly 90% of which received a positive decision (range: 44.4% [Australia] to 100.0% [Germany]). RWE was derived from a variety of study designs and commonly supported clinical efficacy across many therapeutic areas. <b>Conclusions:</b> RWE commonly supports HTAs of recently approved non-oncology orphan medicinal products, strengthening the evidence base and contributing to demonstration of product value.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2026-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148038543","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":"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}