Journal of Market Access & Health Policy最新文献

{"title":"Comparative Efficacy of Finerenone versus Canagliflozin in Patients with Chronic Kidney Disease and Type 2 Diabetes: A Matching-Adjusted Indirect Comparison","authors":"D. Cherney, Kerstin Folkerts, P. Mernagh, Mateusz Nikodem, Joerg Pawlitschko, P. Rossing, Neil Hawkins","doi":"10.3390/jmahp12030014","DOIUrl":"https://doi.org/10.3390/jmahp12030014","url":null,"abstract":"This study aimed to close an evidence gap concerning the relative efficacy of finerenone versus SGLT2is in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D). Canagliflozin was selected as a proxy for the SGLT2i class. Patient-level data of two randomized controlled trials (RCTs) of finerenone (FIDELIO-DKD and FIGARO-DKD) were used alongside aggregated data from CREDENCE, an RCT of canagliflozin. To account for meaningful between-study heterogeneity between each finerenone trial and CREDENCE, a matching-adjusted indirect comparison of a range of efficacy outcomes was undertaken for each finerenone study versus CREDENCE. These results were meta-analyzed, enabling the estimation of the relative effects of finerenone against canagliflozin. For the cardiorenal composite endpoint, the hazard ratio (HR) comparing finerenone to canagliflozin was 1.07 (95% CI: 0.83 to 1.36). The corresponding HRs for all-cause mortality, end-stage kidney disease and cardiovascular death were 0.99 (95% CI: 0.73 to 1.34), 1.03 (95% CI: 0.68 to 1.55) and 0.94 (95% CI: 0.64 to 1.37), respectively. The absence of statistically significant differences was consistent throughout the main analysis and a range of sensitivity analyses. Based on this study, using a large sample of data and adjusted for meaningful differences between the baseline characteristics of the included RCTs, there was no statistically significant evidence indicating a difference in the efficacy of finerenone compared to canagliflozin in the treatment of CKD in patients with T2D.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"45 16","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141805338","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Expanding the Availability of Scalp Cooling to All Patients at Risk of Chemotherapy-Induced Alopecia","authors":"Portia Lagmay-Fuentes, Andrea Smith, Shannon Krus, Laurie Lewis, Asma Latif, Tiffany Gagliardo, Manpreet Kohli","doi":"10.3390/jmahp12030013","DOIUrl":"https://doi.org/10.3390/jmahp12030013","url":null,"abstract":"Alopecia is an undesirable side effect of cancer chemotherapy. The mitigation of alopecia is a desirable adjunct treatment for patients with cancer. FDA-cleared scalp cooling (SC) devices have been successfully used to prevent or reduce chemotherapy-induced alopecia (CIA). This paper provides an understanding of the implementation and value of the new Insurance-Based Billing Model used in the USA for SC and its benefits compared with the original self-pay model. This improved compensation change will result in all patients in need, including underserved and disadvantaged populations, receiving equitable healthcare by allowing access to this valuable supportive care technology.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"10 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-07-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141659317","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Opportunities and Challenges in Cross-Country Collaboration: Insights from the Beneluxa Initiative","authors":"Zilke Claessens, Michiel Lammens, L. Barbier, I. Huys","doi":"10.3390/jmahp12030012","DOIUrl":"https://doi.org/10.3390/jmahp12030012","url":null,"abstract":"National pricing and reimbursement agencies face growing challenges with complex health technologies, prompting European policy advancements. Beneluxa is a cross-country collaboration involving Belgium, the Netherlands, Luxemburg, Austria, and Ireland that aims to address sustainable access to medicines. In view of the soon-to-be-implemented EU HTA Regulation, insights and experiences from stakeholders with Beneluxa cross-country collaboration could provide possible transferable learnings. Therefore, this research aims to (i) identify the opportunities and challenges faced by Beneluxa, (ii) gather insights from stakeholders, namely (possible) applicants and policymakers, within and beyond Beneluxa on the initiative and broader cross-country collaboration principles, and (iii) transfer these insights into learnings and recommendations in anticipation of the full implementation of the new HTA Regulation. Fifteen semi-structured interviews were conducted with industry and European HTA/policy stakeholders. The principal challenges discussed by stakeholders encompass hesitancy from the industry toward Beneluxa assessments, which were attributed to procedural and timeline uncertainties, legislative framework ambiguity, and challenges in terms of industry’s internal organization. Another challenge highlighted is the resource-intensive nature of the procedure due to diverse approaches among member states. In addition, industry stakeholders mentioned limited communication and procedural complexity. Despite challenges, both stakeholder groups recognized important opportunities for cross-country collaboration. Transferable insights for future cross-country collaboration include transparent communication, clear legislative embedding, internal industry restructuring to facilitate joint HTAs, and member state support for conducting collaborative assessments. The study underscores diverging views among stakeholders on cross-country collaboration’s potential to support HTA and the market access of complex health technologies. While acknowledging benefits, there still are challenges, including industry hesitancy, emphasizing the need for transparent communication and clear guidance in the evolving EU HTA landscape.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"28 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141665039","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Future Possible Changes in Medically Underserved Areas in Japan: A Geographic Information System-Based Simulation Study","authors":"Akihisa Nakamura, Eiji Satoh, Tatsuya Suzuki, Soichi Koike, Kazuhiko Kotani","doi":"10.3390/jmahp12020010","DOIUrl":"https://doi.org/10.3390/jmahp12020010","url":null,"abstract":"Background: A decrease in populations could affect healthcare access and systems, particularly in medically underserved areas (MUAs) where depopulation is becoming more prevalent. This study aimed to simulate the future population and land areas of MUAs in Japan. Methods: This study covered 380,948 1 km meshes, 87,942 clinics, and 8354 hospitals throughout Japan as of 2020. The areas outside a 4 km radius of medical institutions were considered as MUAs, based on the measure of areas in the current Japanese Medical Care Act. Based on the population estimate for a 1 km mesh, the population of mesh numbers of MUAs was predicted for every 10 years from 2020 to 2050 using geographic information system analysis. If the population within a 4 km radius from a medical institution fell below 1000, the institution was operationally assumed to be closed. Results: The number of MUAs was predicted to decrease from 964,310 (0.77% of the total Japanese population) in 2020 to 763,410 (0.75%) by 2050. By 2050, 48,105 meshes (13% of the total meshes in Japan) were predicted to be new MUAs, indicating a 31% increase in MUAs from 2020 to 2050. By 2050, 1601 medical institutions were tentatively estimated to be in close proximity. Conclusions: In Japan, the population of MUAs will decrease, while the land area of MUAs will increase. Such changes may reform rural healthcare policy and systems.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"18 11","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141271832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Evidence to Reinforce Clinical Trial Evidence in Health Technology Assessment: A Critical Review of Real-World Evidence Requirements from Seven Countries and Recommendations to Improve Acceptance","authors":"Katia Thokagevistk, Céline Coppo, Laetitia Rey, Amanda Carelli, Veronica Díez, Sarah Vaselenak, Liana Oliveira, Ajay Patel, Emilia Sicari, Teresa Ramos, Susanne Schach, Erika Schirghuber, Alex Simpson, Remy Choquet, Katell Le Lay","doi":"10.3390/jmahp12020009","DOIUrl":"https://doi.org/10.3390/jmahp12020009","url":null,"abstract":"Background: Real-world evidence (RWE) can reinforce clinical trial evidence in health technology assessment (HTA). Objectives: Review HTA bodies’ (HTAbs) requirements for RWE, real uses, and acceptance across seven countries (Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom) and outline recommendations that may improve acceptance of RWE in efficacy/effectiveness assessments and appraisals processes. Methods: RWE requirements were summarized based on HTAbs’ guidelines. Acceptance by HTAbs was evaluated based on industry experience and case studies. Results: As of June 2022, RWE methodological guidelines were in place in three of the seven countries. HTAbs typically requested analyses based on local data sources, but the preferred study design and data sources differed. HTAbs had individual submission, assessment, and appraisal processes; some allowed early meetings for the protocol and/or results validation, though few involved external experts or medical societies to provide input to assessment and appraisal. The extent of submission, assessment, and appraisal requirements did not necessarily reflect the degree of acceptance. Conclusion: All the countries reviewed face common challenges regarding the use of RWE. Our proposals address the need to facilitate collaboration and communication with industry and regulatory agencies and the need for specific guidelines describing RWE design and criteria of acceptance throughout the assessment and appraisal processes.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"60 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141123296","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EU HTA Regulation and Joint Clinical Assessment—Threat or Opportunity?","authors":"Volker Schuster","doi":"10.3390/jmahp12020008","DOIUrl":"https://doi.org/10.3390/jmahp12020008","url":null,"abstract":"The vision of a unified European HTA is by no means a new endeavor. At its core are the publicly declared ambitions to harmonize assessments of clinical data within the EU and avoid the duplication of efforts. Not surprisingly, these ambitions are publicly announced to be motivating the new 2022 EU HTA regulation. However, industry experts typically see more of a risk for additional bureaucracy resulting in delays, further scrutiny, and one additional EU (clinical) dossier to submit on top of all national HTA dossiers, which could be considered a duplication of effort and therefore counterproductive. Regardless of how the details of the process will be defined and how the entire process will work in practice, we can be sure that EU officials will refer to the EU HTA and Joint Clinical Assessment (JCA) in particular as a learning system. The purpose of this article is to take a closer look at the new EU HTA regulation and analyze threats and opportunities for manufacturers and what the resulting opportunities and threats will be at the affiliate level throughout the EU.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"115 14","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140985694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Conceptualisation and Role of Market Access in Pharmaceutical Industry: A Scoping Review","authors":"Clara Fatoye, Gillian Yeowell, Eula Miller, Isaac Odeyemi, C. Mbada","doi":"10.3390/jmahp12020007","DOIUrl":"https://doi.org/10.3390/jmahp12020007","url":null,"abstract":"Background: Understanding the concept and dynamic process of the evolution of professional identity and roles of market access (MA) in the pharmaceutical industry (pharma) is critical to personal, interpersonal, and professional levels of development and impact. Objective: The aim was to carry out a scoping review of the conceptualisation of MA within pharma. Data Sources: BioMed Central, WorldCat.org, and Directory of Open Access Journals were searched from 2003 to 2023. Study Selection: All articles on concepts or definitions and other surrogate terms on MA in pharma were selected. Data Extraction: Keywords generated from an initial cursory literature search on MA in pharma were used in conjunction with AND/OR as search terms. Using the data charting method, key findings were mapped and summarised descriptively. inductive analysis was performed, allowing codes/themes that are relevant to the concept to emerge. Data Synthesis: Arskey and O’Malley’s six-stage framework and the PRISMA extension for scoping reviews extension checklist were used as the review and reporting templates. The databases search yielded 222 results. Following title and abstract screening, a total of 146 papers were screened, and 127 of them were excluded. Full-text review was conducted for 19 papers that were deemed by two reviewers to meet the eligibility criteria. One of the authors arbitrated on disputed papers for inclusion. Only 14 of the included papers were found to meet the criteria for the final analysis. Five conceptual dimensions of MA in pharma were identified as “right products”, “right patient”, “right price”, “right point” (time), and “right place” (setting). Conclusions: Market access in pharma is a process that commences with the development and availability of the right products that are proven to be efficacious and disease/condition-specific (including medications, medical devices, and vaccines); specifically produced for the right patients or end users who will maximise best clinical outcomes and economic value; delivered at the right point in a timely, sustained, and efficient manner, given at the right price (commercially viable or reimbursed price that represents good value); and conducted within the economic, policy, societal, and technological contexts, with the overarching goal of achieving the best patient outcomes and ensuring product profitability.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"9 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141046311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators","authors":"Thomas Desmet, Elaine Julian, Walter Van Dyck, I. Huys, Steven Simoens, Rosa Giuliani, M. Toumi, Christian Dierks, Juliana Dierks, Anontella Cardone, F. Houÿez, Mira Pavlovic, Michael Berntgen, Peter Mol, Anja Schiel, Wim Goettsch, F. Gianfrate, S. Capri, James Ryan, P. Ducournau, O. Solà-Morales, Jörg Ruof","doi":"10.3390/jmahp12010004","DOIUrl":"https://doi.org/10.3390/jmahp12010004","url":null,"abstract":"Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"49 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140244716","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Clinical Effectiveness and Cost-Effectiveness of the Cochlear Osia System and Baha Attract System in Patients with Conductive or Mixed Hearing Loss or Single-Sided Deafness","authors":"Matthias Brunner, Manjula Schou, Robert J. Briggs, Dell Kingsford Smith","doi":"10.3390/jmahp12010003","DOIUrl":"https://doi.org/10.3390/jmahp12010003","url":null,"abstract":"The aim of this study was to evaluate the comparative clinical effectiveness and cost-utility of the active transcutaneous Osia® System versus the passive transcutaneous Baha® Attract System for patients with conductive or mixed hearing loss or single-sided deafness in an Australian healthcare setting. In the absence of direct comparative evidence, an indirect treatment comparison (ITC) of the clinical effectiveness and utility gains was needed. The ITC was informed by three studies identified through a systematic literature review. A Markov model was developed to evaluate the cost-utility of the Osia System. The literature review identified three studies suitable to inform an ITC: Mylanus et al. 2020 and Briggs et al. 2022 (Osia System) and den Besten et al. 2019 (Baha Attract System). The Osia System was found to be clinically superior to the Baha Attract System, across objective audiological outcomes resulting in a clinically meaningful utility benefit of 0.03 measured by the Health Utility Index with at least equivalent safety. In conclusion, the Osia System is more effective than the Baha Attract System, providing better hearing and health-related quality of life outcomes. In an Australian healthcare setting, the Osia System is cost-effective as demonstrated in a cost-utility analysis versus the Baha Attract System.","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"52 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140260775","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Welcome to the Journal of Market Access and Health Policy under MDPI Wings","authors":"M. Toumi","doi":"10.3390/jmahp12010002","DOIUrl":"https://doi.org/10.3390/jmahp12010002","url":null,"abstract":"The Journal of Market Access and Health Policy (JMAHP) is the first peer-reviewed open access journal that focuses on the concept of ‘market access’ from economic, technical, scientific, sociological, anthropological, psychological, and policy perspectives [...]","PeriodicalId":434317,"journal":{"name":"Journal of Market Access & Health Policy","volume":"29 48","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139394167","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}