Journal of market access & health policy最新文献

{"title":"Gene therapy randomised clinical trials in Europe - a review paper of methodology and design.","authors":"Krassimira Ilieva, Borislav Borissov, Mondher Toumi","doi":"10.1080/20016689.2020.1847808","DOIUrl":"10.1080/20016689.2020.1847808","url":null,"abstract":"<p><p><b>Purpose</b>: Gene therapy brings opportunities to discover cures for diseases for which there are no adequate treatments. As most gene therapies target rare diseases, several challenges are associated with their clinical development, such as limited population size, lack of established clinical pathways for development, and sometimes the absence of validated endpoints. The objective of this study was to systematically review and evaluate the methodology and design of European clinical trials (CTs) utilising gene therapy medicinal products (GTMPs). <b>Methods</b>: A systematic search of online CT databases was performed using keywords to identify CTs conducted with GTMPs in Europe, published from 1 January 1995 to 31 July 2019. <b>Results</b>: The search identified 1571 CTs, of which 199 were identified as published articles. A total of 159 CTs remained following the elimination of duplicated CTs, non-gene therapy trials, and those conducted outside Europe. Of these, only nine CTs were randomised, double-blind, with or without parallel groups, and placebo-controlled. <b>Conclusions</b>: The analysed randomised CTs were conducted in accordance with Good clinical practice with low risk of bias across domains. Only one CT was identified with some concerns of bias due to lack of information regarding the randomisation process and changes in protocol.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1847808"},"PeriodicalIF":0.0,"publicationDate":"2020-11-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1847808","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38367394","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":"Transitioning to community-based mental health service delivery: opportunities for Ukraine.","authors":"Eleanor Quirke, Orest Suvalo, Oleksii Sukhovii, York Zöllner","doi":"10.1080/20016689.2020.1843288","DOIUrl":"10.1080/20016689.2020.1843288","url":null,"abstract":"<p><p><b>Background and objectives</b>: Ukraine's mental health system has been found to be inadequate and unresponsive to the needs of the population, in view of its emphasis on inpatient service delivery. This study sought to identify potential changes to the organization and financing of mental health services within the Ukrainian health system that would facilitate the delivery of mental health services in a community-based setting. <b>Methodology</b>: A systematic literature review was undertaken to identify organizational and financing features that have been successfully used to enable and incentivize the delivery of community-based mental health services in Central or Eastern European and/or former Soviet Union countries. <b>Results</b>: There was limited literature on the organizational and financing features that facilitate the delivery of community-based care. Key facilitators for transitioning from institution-based to community-based mental health service delivery include; a clear vision for community-based care, investment in the mental health system, and mechanisms that allow health funding to follow the patient through the health system. <b>Conclusions</b>: Ukraine should adopt strategic purchasing mechanisms to address inefficiency in the financing of its mental health system, and prioritize collaborative planning and delivery of mental health services. Ongoing reform of the Ukrainian health system provides momentum for instituting such changes.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1843288"},"PeriodicalIF":0.0,"publicationDate":"2020-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1843288","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38650015","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":"Economic burden of spinal muscular atrophy: an analysis of claims data.","authors":"Lisa Belter, Rosángel Cruz, Sierra Kulas, Emily McGinnis, Omar Dabbous, Jill Jarecki","doi":"10.1080/20016689.2020.1843277","DOIUrl":"10.1080/20016689.2020.1843277","url":null,"abstract":"<p><p><b>Background</b>: Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disease. <b>Objective</b>: Characterize direct costs associated with SMA management. <b>Data source</b>: Truven Health Analytics MarketScan claims data (2012-2016). <b>Patients</b>: Eligible patients had ≥2 SMA-related medical claims ≥30 days apart. Patients were matched (1:1) to controls by birth year, gender, and geographic region. Patients were categorized as having infantile, child, or juvenile SMA based on diagnosis at age <1, 1-3, or 3-18 years, respectively. <b>Main outcome measures</b>: Annual inpatient and outpatient insurance claims and costs (2019 USD) for cases versus controls. <b>Results</b>: Fifty-eight, 56, and 279 cases and controls comprised the infantile, child, and juvenile cohorts, respectively. Cases had more inpatient claims than controls (infantile: 60.3% vs 1.7%; child: 35.7% vs 3.6%; juvenile: 47.0% vs 4.3%; all <i>P</i> ≤ 0.002). Mean net payments for inpatient admissions were higher for cases versus controls (infantile: $118,609.00 vs $58.79; child: $26,940.01 vs $143.56; juvenile: $39,389.91 vs $701.21; all <i>P</i> ≤ 0.01), as were mean net payments for outpatient services (infantile: $55,537.83 vs $2,047.20; child: $73,093.66 vs $1,307.56; juvenile: $49,067.83 vs $1,134.69; all <i>P</i> ≤ 0.0002). <b>Conclusions</b>: Direct costs of SMA are tremendous, often >50-fold higher compared with matched controls. Efforts are needed to reduce costs through improved standards of care.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1843277"},"PeriodicalIF":0.0,"publicationDate":"2020-11-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0a/d5/ZJMA_8_1843277.PMC7655070.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38641089","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":"Health economic evaluation of gene replacement therapies: methodological issues and recommendations.","authors":"Samuel Aballéa, Katia Thokagevistk, Rimma Velikanova, Steven Simoens, Lieven Annemans, Fernando Antonanzas, Pascal Auquier, Clément François, Frank-Ulrich Fricke, Daniel Malone, Aurélie Millier, Ulf Persson, Stavros Petrou, Omar Dabbous, Maarten Postma, Mondher Toumi","doi":"10.1080/20016689.2020.1822666","DOIUrl":"10.1080/20016689.2020.1822666","url":null,"abstract":"<p><p><b>Objective</b>: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects beyond trial duration; 4) the estimation of costs; 5) the selection of appropriate discount rates; 6) the incorporation of broader elements of value; and 7) affordability. <b>Methods</b>: A literature review on economic evaluations of GRT was performed. Interviews were conducted with 8 European and US health economic experts with experience in evaluations of GRT. Targeted literature reviews were conducted to investigate further potential solutions to specific challenges. <b>Recommendations</b>: Experts agreed on factors to be considered to ensure the acceptability of historical cohorts by HTA bodies. Existing prospective registries or, if not available, retrospective registries, may be used to analyse different disease trajectories and inform extrapolations. The importance of expert opinion due to limited data was acknowledged. Expert opinion should be obtained using structured elicitation techniques. Broader elements of value, beyond health gains directly related to treatment, can be considered through the application of a factor to inflate the quality-adjusted life years (QALYs) or a higher cost-effectiveness threshold. Additionally, the use of cost-benefit analysis and saved young life equivalents (SAVE) were proposed as alternatives to QALYs for the valuations of outcomes of GRT as they can incorporate broader elements of value and avoid problems of eliciting utilities for paediatric diseases. <b>Conclusions</b>: While some of the limitations of economic evaluations of GRT are inherent to limited clinical data and lack of experience with these treatments, others may be addressed by methodological research to be conducted by health economists.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1822666"},"PeriodicalIF":0.0,"publicationDate":"2020-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ec/af/ZJMA_8_1822666.PMC7580851.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38659112","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 complete clearance for assessing treatment efficacy for 5-fluorouracil interventions in actinic keratoses: how baseline lesion count can impact this outcome.","authors":"Khaled Ezzedine,&nbsp;Caroline Painchault,&nbsp;Melanie Brignone","doi":"10.1080/20016689.2020.1829884","DOIUrl":"https://doi.org/10.1080/20016689.2020.1829884","url":null,"abstract":"<p><p><b>Background</b>: Many trials in actinic keratoses (AK) use complete clearance rate (100% reduction in number of lesions) as the primary endpoint. We explore limitations (predominantly baseline factors) associated with this outcome. <b>Objective</b>: This analysis assessed the effect of baseline lesion count on complete clearance rate using randomized controlled trials (RCTs) that evaluated 5-fluorouracil (5-FU) formulations, alone or with 10% salicylic acid solution, in patients with AK. <b>Methodology</b>: Correlation between baseline lesion count and complete clearance rate at week 8 was assessed using Pearson's coefficient. <b>Results</b>: Five RCTs assessing 5-FU (4%, 5%, or 0.5% in 10% salicylic acid solution) in 1,080 patients with AK were included. Mean lesion count at baseline ranged from 8.1 to 21.2 lesions per patient. Complete clearance rate was negatively associated with number of lesions at baseline. Correlation between mean number of lesions at baseline and complete clearance rate was strong (r² = 0.94) and statistically significant (p < 0.001). <b>Conclusion</b>: This analysis showed that, in a homogenous set of trials, complete clearance rates achieved with 5-FU interventions are inversely related to number of lesions at baseline. These findings highlight the limits of restricting treatment evaluation to complete clearance rate and the relevance of alternative measures.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1829884"},"PeriodicalIF":0.0,"publicationDate":"2020-10-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1829884","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38553863","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":"Chinese guidelines related to novel coronavirus pneumonia.","authors":"Tingting Qiu, Shuyao Liang, Monique Dabbous, Yitong Wang, Ru Han, Mondher Toumi","doi":"10.1080/20016689.2020.1818446","DOIUrl":"10.1080/20016689.2020.1818446","url":null,"abstract":"<p><p><b>Background and Objective:</b> China has managed to control the coronavirus disease (COVID-19) with confinement measurements and treatment strategies, while other countries are struggling to contain the spread. This study discusses the guidelines related to COVID-19 in China in order to provide important references for other countries in the fight against COVID-19. <b>Methods:</b> Chinese guidelines relevant to COVID-19 were systematically searched via the China National Knowledge Infrastructure database, YiMaiTong database, and World Health Organization (WHO) COVID-19 database on March 20^th, 2020. Guideline information was extracted, including date of publication, source, objectives and the target population. Guidelines specific to the pharmacological treatment of COVID-19 were further investigated to identify the types of antivirus drugs recommended and to report on how treatment recommendations for COVID-19 have evolved overtime. <b>Results:</b> A total of 100 guidelines were identified, of which 74 were national guidelines and 26 were regional guidelines. The scope of included guidelines consisted of: the diagnosis and treatment of COVID-19, the management of hospital departments and specific diseases during the outbreak of COVID-19. Fifty-one of the included guidelines targeted overall COVID-19 patients, while the remaining guidelines concentrated on special patient populations (i.e. geriatric population, pediatric population, and pregnant population) or patients with coexisting diseases. Fifteen guidelines focused on the pharmacological treatments for all COVID-19 patients. Interferon, Lopinavir/Ritonavir, Ribavirin, Chloroquine, and Umifenovir represented the most recommended antivirus drugs. Among them, 7 Chinese guidelines have recommended Chloroquine Phosphate or Hydroxychloroquine for the treatment of COVID-19. <b>Conclusions</b>: China has generated a plethora of guidelines covering almost all aspects of COVID-19. Chloroquine, as one widely affordable treatment, was recommended by Chinese national guidelines and provincial guidelines. Considering the continuous debates around Chloroquine, confirmatory studies with robust methodology are awaited to address the unanswered questions on its potential benefits and risks on COVID-19.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1818446"},"PeriodicalIF":0.0,"publicationDate":"2020-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/61/0f/ZJMA_8_1818446.PMC7580738.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38553862","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 non-medical switching of prescription medications on health outcomes: an e-survey of high-volume medicare and medicaid physician providers.","authors":"Craig Coleman,&nbsp;Tabassum Salam,&nbsp;Amy Duhig,&nbsp;Aarti A Patel,&nbsp;Ann Cameron,&nbsp;Jennifer Voelker,&nbsp;Brahim Bookhart","doi":"10.1080/20016689.2020.1829883","DOIUrl":"https://doi.org/10.1080/20016689.2020.1829883","url":null,"abstract":"<p><strong>Background: </strong>Non-medical switching refers to a change in a stable patient's prescribed medication to a clinically distinct, non-generic, alternative for reasons other than poor clinical response, side-effects or non-adherence.</p><p><strong>Objective: </strong>To assess the perceptions of high-volume Medicare and/or Medicaid physician providers regarding the impact non-medical switching has on their patients' medication-related outcomes and health-care utilization.</p><p><strong>Methods: </strong>We performed an e-survey of high-volume Medicare and/or Medicaid physicians (spending >50% of their time caring for Medicare and/or Medicaid patients), practicing for >2 years but <30 years post-residency and/or fellowship; working in a general, internal, family medicine or specialist setting; spending ≥40% of their time providing direct care and having received ≥1 request for a non-medical switch in the past 12 months. Physicians were queried on 15-items to assess perceptions regarding the impact non-medical switching on medication-related outcomes and health-care utilization.</p><p><strong>Results: </strong>Three-hundred and fifty physicians were included. Respondents reported they felt non-medical switching, to some degree, increased side-effects (54.0%), medication errors (56.0%) and medication abandonment (60.3%), and ~50% believed it increased patients' out-of-pocket costs. Few physicians (≤13.4% for each) felt non-medical switching had a positive impact on effectiveness, adherence or patients' or physicians' confidence in the quality-of-care provided. Non-office visit and prescriber-pharmacy contact were most frequently thought to increase due to non-medical switching. One-third of physicians felt office visits were very frequently/frequently increased, and ~ 1-in-5 respondents believed laboratory testing and additional medication use very frequently/frequently increased following a non-medical switch. About 1-in-10 physicians felt non-medical switching very frequently/frequently increased the utilization of emergency department or in-hospital care.</p><p><strong>Conclusion: </strong>This study suggests high-volume Medicare and/or Medicaid physician providers perceive multiple negative influences of non-medical switching on medication-related outcomes and health-care utilization.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1829883"},"PeriodicalIF":0.0,"publicationDate":"2020-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1829883","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38659111","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 critical analysis and review of Lancet COVID-19 hydroxychloroquine study.","authors":"Mondher Toumi, Malgorzata Biernikiewicz, Shuyao Liang, Yitong Wang, Tingting Qiu, Ru Han","doi":"10.1080/20016689.2020.1809236","DOIUrl":"10.1080/20016689.2020.1809236","url":null,"abstract":"<p><p><b>Purpose</b> A international registry analysis led by Mehra et al. to investigate the use of hydroxychloroquine (HCQ) and chloroquine (CQ) with or without a macrolide in 96,032 hospitalised COVID-19 patients were published on Lancet, which has raised considerable discussions in the public health community. This study aimed to critically review the quality and limitations of the Mehra et al. publication and discuss the potential influences on the use of HCQ/CQ worldwide. <b>Method</b> A critical review of this publication was conducted to examine the potential study bias in the study objectives, methodology, confounding factors and outcomes and summarise the external reviews. <b>Results</b> The very high homogeneity of the patients' characteristics at baseline was inconsistent with region specific epidemiology and several critical confounding factors. The results indicated that angiotensin converting enzyme inhibitors were associated with a hazard ratio of 0.5, which suggested a technical problem in the estimation of the propensity scores. Several major risk factors for mortality identified in the analysis were treated as a minor risk or neutral or even protective factors. Antiviral treatments were recognised as an effective method to reduce mortality and were neither further studied nor integrated in the multivariate Cox model. <b>Conclusion</b> This research appeared to carry multiple biases. An extensive audit of the study, conditions of review and acceptance for publication in the Lancet of that study are requested to avoid damage to the publics' trust on the scientific community at this critical time of COVID-19 pandemic.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1809236"},"PeriodicalIF":0.0,"publicationDate":"2020-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/bc/5f/ZJMA_8_1809236.PMC7733902.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38393601","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":"Organizational aspect in healthcare decision-making: a literature review.","authors":"Amélie Dubromel, Marie-Audrey Duvinage-Vonesch, Loïc Geffroy, Claude Dussart","doi":"10.1080/20016689.2020.1810905","DOIUrl":"10.1080/20016689.2020.1810905","url":null,"abstract":"<p><strong>Background: </strong>Organizational aspect is rarely considered in healthcare. However, it is gradually seen as one of the key aspects of the decision-making process as well as clinical and economic dimensions. Our primary objective was to identify criteria already used to assess the organizational impact of medical innovations. Our secondary objective was to structure them into an inventory to support decision-makers to select the relevant criteria for their complex decision-making issues.</p><p><strong>Materials and methods: </strong>A search using the Medline database was conducted in June 2019. The records published between January, 1990 and December, 2018 were identified. The publications cited by the authors of the included articles and the websites of health technology assessment agencies, units or learned societies identified during the search were also consulted. The identified criteria were structured in an inventory.</p><p><strong>Results: </strong>We selected 107 records of a wide range of evidence mostly published after the 2000s. We identified 636 criteria that we classified into five categories: people, task, structure, technology, and surroundings.</p><p><strong>Conclusion: </strong>Criteria selection is a crucial step in any multi-criteria decision analysis (MCDA). This work is the first step in the development of a validated MCDA method to assess the organizational impact of medical innovations.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1810905"},"PeriodicalIF":0.0,"publicationDate":"2020-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/aa/c7/ZJMA_8_1810905.PMC7482895.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38394400","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world cost-effectiveness of rivaroxaban and apixaban vs VKA in stroke prevention in non-valvular atrial fibrillation in the UK.","authors":"Kevin Bowrin,&nbsp;Jean-Baptiste Briere,&nbsp;Pierre Levy,&nbsp;Aurélie Millier,&nbsp;Jean Tardu,&nbsp;Mondher Toumi","doi":"10.1080/20016689.2020.1782164","DOIUrl":"https://doi.org/10.1080/20016689.2020.1782164","url":null,"abstract":"<p><strong>Background: </strong>Morbidity and mortality associated with non-valvular atrial fibrillation (NVAF) imposes a substantial economic burden on the UK healthcare system.</p><p><strong>Objectives: </strong>An existing Markov model was adapted to assess the real-world cost-effectiveness of rivaroxaban and apixaban, each compared with a vitamin K antagonist (VKA), for stroke prevention in patients with NVAF from the National Health Service (NHS) and personal and social services (PSS) perspective.</p><p><strong>Methods: </strong>The model considered a cycle length of 3 months over a lifetime horizon. All inputs were drawn from real-world evidence (RWE): baseline patient characteristics, clinical event and persistence rates, treatment effect (meta-analysis of RWE studies), utility values and resource use. Deterministic and probabilistic sensitivity analyses were performed.</p><p><strong>Results: </strong>The incremental cost per quality-adjusted life year was £14,437 for rivaroxaban, and £20,101 for apixaban, compared with VKA. The probabilities to be cost-effective compared with VKA were 90% and 81%, respectively for rivaroxaban and apixaban, considering a £20,000 threshold. In both comparisons, the results were most sensitive to clinical event rates.</p><p><strong>Conclusions: </strong>These results suggest that rivaroxaban and apixaban are cost-effective vs VKA, based on RWE, considering a £20,000 threshold, from the NHS and PSS perspective in the UK for stroke prevention in patients with NVAF. This economic evaluation may provide valuable information for decision-makers, in a context where RWE is more accessible and its value more acknowledged.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1782164"},"PeriodicalIF":0.0,"publicationDate":"2020-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1782164","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38394399","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}