Journal of market access & health policy最新文献

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Optimising Investment in Health Innovations in Europe. 优化欧洲卫生创新投资。
Journal of market access & health policy Pub Date : 2026-02-13 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010011
Tosin Adeyemo, Tim Wilsdon, Christina Vandorou, Fiona Davies, Annabelle Godet
{"title":"Optimising Investment in Health Innovations in Europe.","authors":"Tosin Adeyemo, Tim Wilsdon, Christina Vandorou, Fiona Davies, Annabelle Godet","doi":"10.3390/jmahp14010011","DOIUrl":"10.3390/jmahp14010011","url":null,"abstract":"<p><p>European citizens made it clear in 2024 that healthcare should be the EU's top priority for shaping the future of Europe. This sentiment reflects the escalating health challenges facing the region, driven by ageing populations, rising chronic disease burdens, and persistent disparities in access to healthcare. Despite these growing needs, the most recent data on health spending as a share of gross domestic product (GDP) is just slightly above the pre-COVID-19 pandemic level, and spending on pharmaceuticals specifically has remained a stable proportion of healthcare spending over the last 20 years. Austerity measures have profoundly impacted the health sector and pharmaceutical industry, more so than any other sectors, despite the wide range of health and socioeconomic benefits medicines bring to patients, the health system, and society. Such trends are not keeping pace with evolving population demographics and disease prevalence. To secure a healthier, more equitable future, Europe must urgently increase health investments, optimise health systems, and address unmet needs by supporting fast uptake of pharmaceutical innovations. Policymakers must work with all stakeholders to ensure stronger and sustained investments in health innovations by (i) adopting a long-term vision, moving away from short-term thinking and valuing health appropriately to drive economic growth; (ii) implementing transformative policies that eliminate ineffective and wasteful spending; (iii) promoting value-based approaches to improve patient access and system sustainability, and (iv) creating incentives that attract greater investments to strengthen Europe's competitiveness and safeguard against health threats.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"11"},"PeriodicalIF":0.0,"publicationDate":"2026-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12922146/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273323","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}
引用次数: 0
Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs. 在健康经济评价中采用社会视角:关于如何整合社会成本的9项HTA方法指南分析。
Journal of market access & health policy Pub Date : 2026-02-10 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010010
Chloé Gervès-Pinquié, Hortense Nanoux, Sarah Akarkoub, Rémi Brazeilles, Henri Bonnabau, Katell Le Lay
{"title":"Adopting a Societal Perspective in Health-Economic Evaluation: Analysis of Nine HTA Methodological Guidelines on How to Integrate Societal Costs.","authors":"Chloé Gervès-Pinquié, Hortense Nanoux, Sarah Akarkoub, Rémi Brazeilles, Henri Bonnabau, Katell Le Lay","doi":"10.3390/jmahp14010010","DOIUrl":"10.3390/jmahp14010010","url":null,"abstract":"<p><strong>Objective: </strong>The perspective applied in Health Technology Assessment (HTA) has recently been presented as a central methodological debate in HTA. Beyond expected effects on patients' health and quality of life, health technologies can affect broader societal domains, such as the labor market and informal care. Ensuring comparability between treatments when estimating their impact on society relies strongly on transparency regarding the methodology used to measure and value societal costs. This study aims to describe and summarize current recommendations of HTA bodies on adopting societal perspectives in HTA.</p><p><strong>Methods: </strong>A scoping review of HTA guidelines was conducted, and findings were validated through semi-structured interviews with academic and industry HTA experts. Guidelines from nine countries (Australia, Canada, France, Germany, The Netherlands, Spain, Sweden, United Kingdom, United States of America) were analyzed using an extraction grid covering four domains: out-of-pocket/copayment costs, informal care costs, productivity losses, and unrelated health care costs. Exploratory analyses (multiple factor analysis and clustering) assessed whether recommendations for a societal perspective as a base case were homogeneous across countries.</p><p><strong>Results: </strong>A societal perspective was recommended as a base case in 56% of the guidelines. Marked cross-country heterogeneity was observed for measurement methods. Only The Netherlands recommended a specific instrument for both informal care and productivity losses (iPCQ). The most frequently cited valuation approaches were the opportunity cost method (informal care) and the friction cost method (productivity losses). Three clusters of countries were identified: The Netherlands/Canada (1); US/Sweden/UK/Germany (2); and Spain/France/Australia (3). Exploratory analyses indicated limited alignment between endorsing the inclusion of societal costs and recommending the societal perspective as base case.</p><p><strong>Conclusions: </strong>This preliminary work highlights the need for explicit methodological guidance on societal cost estimation within HTA. HTA bodies could draw on the Netherland's guidelines and cost database and develop a national \"societal perspective doctrine\"-including core cost sets, standardized measurement tools (e.g., iPCQ; diary methods), and structured involvement of patients and caregivers-to enhance comparability and decision relevance.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"10"},"PeriodicalIF":0.0,"publicationDate":"2026-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12921813/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273342","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}
引用次数: 0
Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies. 肺动脉高压和癌症:揭示流行病学、临床途径和治疗策略的相似之处。
Journal of market access & health policy Pub Date : 2026-02-06 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010009
Karim El-Kersh, Nadine Zawadzki, Catelyn Coyle, Shurui Zhang, Dhruv Dalal, Anna Watzker, Dominik Lautsch, Jason Shafrin
{"title":"Pulmonary Arterial Hypertension and Cancer: Unveiling Parallels in Epidemiology, Clinical Pathways, and Therapeutic Strategies.","authors":"Karim El-Kersh, Nadine Zawadzki, Catelyn Coyle, Shurui Zhang, Dhruv Dalal, Anna Watzker, Dominik Lautsch, Jason Shafrin","doi":"10.3390/jmahp14010009","DOIUrl":"10.3390/jmahp14010009","url":null,"abstract":"<p><p>Pulmonary arterial hypertension (PAH) and cancer share high mortality and complex prognoses. Due to PAH's rarity, these parallels may be underrecognized by healthcare stakeholders. This study explored similarities between PAH and cancer across epidemiological, clinical, therapeutic, and healthcare resource utilization (HCRU) considerations. A four-step approach was employed: (1) inclusion/exclusion criteria were applied to identify potential PAH cancer analogs; (2) characteristics for comparison were categorized as epidemiologic, clinical, therapeutic landscape, and HCRU; (3) a targeted literature review extracted data on disease characteristics; (4) a similarity ranking was calculated as the absolute difference between each cancer's and PAH's characteristics. Fourteen cancers met the inclusion criteria. Well-differentiated thyroid cancer (WDTC) had the highest number (5) of characteristics closest to PAH. WDTC and medullary thyroid cancer were most similar to PAH in epidemiology; gastrointestinal stromal tumor was most similar in clinical and HCRU characteristics, and anaplastic lymphoma kinase-positive (ALK+) non-small-cell lung cancer and renal cell carcinoma were most similar in therapeutic landscape. Although no single cancer fully mirrors PAH, the identification of multiple analogs underscores PAH's multidimensional complexity and confirms its overlap with oncological conditions. Cancer analogs could serve as a valuable framework for enhancing recognition of PAH's clinical, therapeutic, and HRCU implications among healthcare stakeholders.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"9"},"PeriodicalIF":0.0,"publicationDate":"2026-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12921831/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273280","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}
引用次数: 0
A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting. Sentio骨传导听力植入系统在澳大利亚医疗机构的成本-效果分析。
Journal of market access & health policy Pub Date : 2026-01-27 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010008
Magnus Värendh, Ida Haggren, Helén Lagerkvist, Maria Åberg Håkansson, Jonas Hjelmgren
{"title":"A Cost-Effectiveness Analysis of the Sentio Bone Conduction Hearing Implant System in the Australian Healthcare Setting.","authors":"Magnus Värendh, Ida Haggren, Helén Lagerkvist, Maria Åberg Håkansson, Jonas Hjelmgren","doi":"10.3390/jmahp14010008","DOIUrl":"10.3390/jmahp14010008","url":null,"abstract":"<p><p>Bone conduction hearing implant systems (BCHIs) are established treatments for patients with conductive or mixed hearing loss or single-sided deafness when conventional hearing aids are unsuitable. This study evaluated the cost-effectiveness of the active transcutaneous system Sentio versus a similar system, i.e., Osia in an Australian setting. Scenario analyses also compared Sentio to other systems, i.e., Ponto and Baha Attract. A Markov cohort model was adapted from a previously published source to reflect Australian practice, incorporating device acquisition, surgery, maintenance, battery replacement and adverse event management over a 15-year horizon from a healthcare perspective. Effectiveness inputs were derived from published evidence using a naïve indirect comparison. Extensive sensitivity analyses and external validation tested robustness. In the base case, Sentio was associated with lower costs and a small modelled incremental quality-adjusted life years (QALYs) gain versus Osia. Scenario analyses confirmed cost-effectiveness relative to Ponto and Baha Attract, with outcomes below the Australian willingness-to-pay threshold. Health state utility, device price and reimplantation assumptions were the most influential drivers, yet Sentio remained cost-effective in over 95% of simulations. These findings support Sentio as a clinically and economically efficient BCHI in Australia and highlight the need for direct utility and long-term durability data.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"8"},"PeriodicalIF":0.0,"publicationDate":"2026-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12921733/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273345","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}
引用次数: 0
Evaluating [18F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost-Consequence Comparison with Alternative PET Radiotracers in Spain. 评估[18F]-DCFPyL检测前列腺癌复发:与其他PET示踪剂在西班牙的成本-后果比较。
Journal of market access & health policy Pub Date : 2026-01-23 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010007
Tiago Matos, Mrunmayee Godbole, Rithvik Badinedi, Madhusubramanian Muthukumar, Marina Hodolic, Nicolas Tchouen, Anthony Berthon
{"title":"Evaluating [<sup>18</sup>F]-DCFPyL for Detecting Prostate Cancer Recurrence: A Cost-Consequence Comparison with Alternative PET Radiotracers in Spain.","authors":"Tiago Matos, Mrunmayee Godbole, Rithvik Badinedi, Madhusubramanian Muthukumar, Marina Hodolic, Nicolas Tchouen, Anthony Berthon","doi":"10.3390/jmahp14010007","DOIUrl":"10.3390/jmahp14010007","url":null,"abstract":"<p><p><b>Introduction:</b> [<sup>18</sup>F]-DCFPyL (Piflufolastat [<sup>18</sup>F]) is a prostate-specific membrane antigen (PSMA)-targeted position emission tomography (PET) radiotracer for detecting the biochemical recurrence (BCR) of prostate cancer (PCa). This study evaluates its economic impact compared with [<sup>68</sup>Ga]-PSMA-11, [<sup>18</sup>F]-FCH, and [<sup>18</sup>F]-PSMA-1007 from the Spanish National Healthcare System's perspective. <b>Methods:</b> A cost-consequence model, over a 5-year time horizon, simulated the diagnostic and treatment pathway based on radiotracer-specific accuracy and disease localization. Treatment options included a radical prostatectomy, radiation therapy, androgen deprivation therapy (ADT), and radiation therapy + ADT. Costs were calculated for true/false positives and negatives. Due to limited data availability, key inputs were informed by expert opinions, supported by published meta-analyses, public sources, and literature. Officially published Spanish prices were applied: EUR 2000 for [<sup>18</sup>F]-DCFPyL, [<sup>68</sup>Ga]-PSMA-11, and [<sup>18</sup>F]-PSMA-1007, and EUR 1144 for [<sup>18</sup>F]-FCH. <b>Results:</b> The use of [<sup>18</sup>F]-DCFPyL led to fewer unnecessary therapies; specifically, it led to 11,229 (74%) fewer than [<sup>68</sup>Ga]-PSMA-11, and 5180 (56%) and 7771 (66%) fewer than [<sup>18</sup>F]-FCH and [<sup>18</sup>F]-PSMA-1007, respectively. It achieved significant cost savings for repeated testing: EUR 15M (43%) versus [<sup>68</sup>Ga]-PSMA-11, EUR 37M (65%) versus [<sup>18</sup>F]-FCH, and EUR 27M (58%) versus [<sup>18</sup>F]-PSMA-1007. Cost savings for false positives were EUR 15M (50%) against [<sup>68</sup>Ga]-PSMA-11, EUR 22M (60%) versus [<sup>18</sup>F]-FCH, and EUR 29M (66%) compared with [<sup>18</sup>F]-PSMA-1007. The cost per correct diagnosis was reduced by EUR 198 (8%) compared with [<sup>68</sup>Ga]-PSMA-11 and EUR 377 (15%) relative to [<sup>18</sup>F]-PSMA-1007, while showing a EUR 635 (40%) increase compared with [<sup>18</sup>F]-FCH. <b>Conclusions:</b> [<sup>18</sup>F]-DCFPyL offers a cost-saving option for BCR detection within the Spanish National Healthcare System by reducing the number of unnecessary therapies, the cost of false positives, and repeat testing compared with alternative radiotracers. These improvements support the potential for better diagnostic outcomes and more informed downstream clinical decision-making.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"7"},"PeriodicalIF":0.0,"publicationDate":"2026-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12921982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273301","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}
引用次数: 0
Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy. 加强以患者为中心的健康技术评估:脊髓性肌萎缩症PICOS范围的改进德尔菲面板。
Journal of market access & health policy Pub Date : 2026-01-19 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010006
Emanuele Arcà, Adele Barlassina, Adaeze Eze, Valentina Strammiello
{"title":"Enhancing Patient-Centered Health Technology Assessment: A Modified Delphi Panel for PICOS Scoping in Spinal Muscular Atrophy.","authors":"Emanuele Arcà, Adele Barlassina, Adaeze Eze, Valentina Strammiello","doi":"10.3390/jmahp14010006","DOIUrl":"10.3390/jmahp14010006","url":null,"abstract":"<p><strong>Highlights: </strong><b>What is the main finding?</b> This paper illustrates how systematic PICOS scoping can be operationalized within JCAs through Delphi consensus methods for mapping and reconciling multiple national and patient perspectives into a common framework.It highlights concrete examples of harmonization and clarifies the methodological tensions that arise when balancing rigor with flexibility. <b>What are the implications of the main findings?</b> The findings underscore that early, structured stakeholder engagement-particularly involving patients-is essential for JCAs to be inclusive, scientifically robust, and relevant for national reimbursement decisions.Clear PICOS scoping could improve transparency while also enhancing the credibility and uptake of JCA outcomes in healthcare policy.</p><p><strong>Abstract: </strong><b>Objectives:</b> This study explores the feasibility and value of integrating structured patient input into the PICOS (Population, Intervention, Comparator, Outcome, Study design) scoping process for Joint Clinical Assessments under the EU Health Technology Assessment Regulation. <b>Methods:</b> A modified Delphi panel, led by a steering committee composed of two clinicians, one patient expert, and one policy expert, engaged 12 individuals representing patient organizations across 12 European Member States to reach consensus on PICOS elements for CAR-T therapy in pediatric spinal muscular atrophy. <b>Results:</b> The Delphi process effectively facilitated PICOS consolidation and consensus among the 12 patient experts representing diverse EU contexts. Through 3 iterative rounds integrating quantitative rankings and qualitative feedback, the panel achieved strong agreement on key outcomes, intervention delivery, and study design elements, with population eligibility and comparator selection showing heterogeneity. Patient engagement was central: participants emphasized inclusive eligibility criteria, shared decision-making, and the inclusion of caregiver perspectives. The integration of qualitative insights allowed nuanced interpretation of dissent, distinguishing between genuine disagreement and framing effects, thereby enhancing transparency and scientific validity. Importantly, the process revealed patient priorities for outcomes, treatment burden, and evidence trade-offs, informing both PICOS refinement and future health technology assessment (HTA) strategies. This structured, participatory approach demonstrates the feasibility and value of incorporating patient voices systematically into early-stage EU HTA, fostering robust, credible, and context-sensitive consensus on complex rare-disease interventions. <b>Conclusions:</b> The study demonstrates the potential of consensus-building methodologies to enhance transparency, reduce heterogeneity, and support patient-centered evidence generation and decision-making in HTA.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"6"},"PeriodicalIF":0.0,"publicationDate":"2026-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12922062/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147273339","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}
引用次数: 0
Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications. 克服患者访问障碍在复杂的条件:教训精神分裂症更广泛的医疗保健应用。
Journal of market access & health policy Pub Date : 2025-12-23 eCollection Date: 2026-03-01 DOI: 10.3390/jmahp14010002
Saartje Burgmans, Anne Rieper Hald, Sayagi Tina Markandaier, Nicolas Hall, Rafael Loiseau, Xandra Lie, Bregt Kappelhoff
{"title":"Overcoming Patient Access Barriers in Complex Conditions: Lessons from Schizophrenia for Broader Healthcare Applications.","authors":"Saartje Burgmans, Anne Rieper Hald, Sayagi Tina Markandaier, Nicolas Hall, Rafael Loiseau, Xandra Lie, Bregt Kappelhoff","doi":"10.3390/jmahp14010002","DOIUrl":"10.3390/jmahp14010002","url":null,"abstract":"<p><p>Patient access to innovative care for complex conditions like schizophrenia remains limited by systemic, clinical and policy-level barriers. Cognitive impairment associated with schizophrenia (CIAS) illustrates how critical symptom domains are often overlooked despite their impact on long-term outcomes. This study examines how systemic, infrastructural and economic factors shape access to CIAS care across eight mid-sized European countries to identify shared constraints and opportunities for improvement. Semi-structured interviews were conducted with 32 healthcare professionals and 9 health policy experts. Thematic analysis identified consistent barriers across countries, including fragmented care pathways, insufficient capacity for cognitive assessment, underdeveloped community-based rehabilitation services and reimbursement structures that favour pharmacological over psychosocial interventions. Variability across countries was shaped by differences in community infrastructure, professional training and the breadth of health technology assessment perspectives applied to non-pharmacological care. Countries with stronger community infrastructure and broader reimbursement frameworks were better positioned to deliver comprehensive care. These findings highlight that structural constraints, rather than clinical uncertainty, are the primary impediments to care in complex therapeutic areas. Addressing them will require coordinated reforms that strengthen early identification, expand multidisciplinary rehabilitation capacity and align reimbursement with functional and long-term outcomes.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"14 1","pages":"2"},"PeriodicalIF":0.0,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12821714/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146031900","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}
引用次数: 0
How Patients Can Contribute to the Assessments of Health Technologies. 病人如何对卫生技术的评估作出贡献。
Journal of market access & health policy Pub Date : 2025-12-15 eCollection Date: 2025-12-01 DOI: 10.3390/jmahp13040061
François Houÿez, Julien Delaye
{"title":"How Patients Can Contribute to the Assessments of Health Technologies.","authors":"François Houÿez, Julien Delaye","doi":"10.3390/jmahp13040061","DOIUrl":"10.3390/jmahp13040061","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"13 4","pages":"61"},"PeriodicalIF":0.0,"publicationDate":"2025-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12733746/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145835485","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}
引用次数: 0
The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines. 医药创新指数:支持创新药品的价值经济调控。
Journal of market access & health policy Pub Date : 2025-12-08 eCollection Date: 2025-12-01 DOI: 10.3390/jmahp13040060
Ludmila Peres Gargano, Marcus Carvalho Borin, Wallace Mateus Prata, Juliana Alvares-Teodoro, Francisco de Assis Acurcio, Roberto F Iunes, Augusto Afonso Guerra
{"title":"The Pharmaceutical Innovativeness Index: Supporting Value-Based Economic Regulation of Innovative Medicines.","authors":"Ludmila Peres Gargano, Marcus Carvalho Borin, Wallace Mateus Prata, Juliana Alvares-Teodoro, Francisco de Assis Acurcio, Roberto F Iunes, Augusto Afonso Guerra","doi":"10.3390/jmahp13040060","DOIUrl":"10.3390/jmahp13040060","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"13 4","pages":"60"},"PeriodicalIF":0.0,"publicationDate":"2025-12-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12733516/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145835446","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}
引用次数: 0
Cost-Effectiveness of Electrical Stimulation Therapy in the Treatment of Chronic Wounds: A Systematic Review, Meta-Analysis and Economic Analysis. 电刺激治疗慢性伤口的成本-效果:系统回顾、meta分析和经济分析。
Journal of market access & health policy Pub Date : 2025-11-24 eCollection Date: 2025-12-01 DOI: 10.3390/jmahp13040059
Jennifer M Smith, John Posnett, Emma J Woodmansey
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