Journal of market access & health policy最新文献

{"title":"Quantitative revenue estimates and qualitative assessments of innovative fundraising sources for treating rare diseases in Colombia.","authors":"Surrey M Walton,&nbsp;Wilson Mayorga,&nbsp;Angela Rodríguez Narváez,&nbsp;Maria Alejandra Chavez,&nbsp;Natalia Cortés Guesguán,&nbsp;Luis Durango,&nbsp;Ludy Alexandra Parada","doi":"10.1080/20016689.2023.2211868","DOIUrl":"https://doi.org/10.1080/20016689.2023.2211868","url":null,"abstract":"<p><p><b>Background:</b> Like many developing countries, Colombia faces difficulties in financing health-care services as well as programs for health promotion and health education and there is evidence that its health-care system is underperforming. <b>Objective:</b> To provide evidence-based estimates of potential funding levels and assess the strengths, weaknesses, and viability of innovative funding mechanisms with a focus on treating rare diseases in Colombia. <b>Methods:</b> The strategy involved evidence-based projections of potential funding levels and a qualitative viability assessment using an expert panel. <b>Results:</b> Crowdfunding, corporate donation, and social impact bonds (SIBs) were deemed to be the most viable of numerous potential strategies. Expected funding levels over 10 years for rare diseases in Colombia from crowdfunding, corporate donations, and SIBs were roughly $7,200, $23,000, and $12,400, respectively. <b>Conclusions:</b> Based on the combination of projected funding potential along with expert consensus regarding viability and operability, crowdfunding, corporate donations, and SIBs, especially in combination, have the potential to substantially improve funding for vulnerable patient populations in Colombia.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/8c/e8/ZJMA_11_2211868.PMC10177688.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10248677","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-related quality of life in health states corresponding to different stages of perianal fistula associated with Crohn's disease: a quantitative evaluation of patients and non-patients in Japan.","authors":"Masafumi Kato,&nbsp;Mariko Yoneyama-Hirozane,&nbsp;Katsuhiko Iwasaki,&nbsp;Mao Matsubayashi,&nbsp;Ataru Igarashi","doi":"10.1080/20016689.2023.2166374","DOIUrl":"https://doi.org/10.1080/20016689.2023.2166374","url":null,"abstract":"<p><strong>Background: </strong>Perianal fistula (PF), a complication of Crohn's disease (CD), affects health-related quality of life (QOL).</p><p><strong>Objective: </strong>To elucidate QOL of health states corresponding to different stages of PF associated with CD in Japan.</p><p><strong>Method: </strong>This cross-sectional, observational, web-based questionnaire survey assessed eight different health states in patients with CD and PF and individuals without CD (non-patients) from the Medilead Healthcare Panel (MHP) and determined the utility values (QOL scores) in each health state by the time trade-off method. In patients, we determined also the utility value of the current health state associated with CD and the PF. The analysis excluded respondents with logical inconsistencies.</p><p><strong>Results: </strong>The analysis included 82 patients and 576 non-patients with the same sex and age distribution as the Japanese population. In both groups, mean utility values were higher in remission (patients, 0.78; non-patients, 0.51) than in non-remission states, with lowest values for poor prognosis after proctectomy (patients, 0.13; non-patients, -0.10) and highest values for the state with mild symptoms (patients, 0.60; non-patients, 0.30). In patients, the mean utility value of the current health state was 0.71.</p><p><strong>Conclusion: </strong>QOL decreases with increasing severity of PF and is lower for good prognosis after proctostomy than for remission.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/41/db/ZJMA_11_2166374.PMC9848226.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9146604","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":"Systematic literature reviews over the years.","authors":"Beata Smela,&nbsp;Mondher Toumi,&nbsp;Karolina Świerk,&nbsp;Konrad Gawlik,&nbsp;Emilie Clay,&nbsp;Laurent Boyer","doi":"10.1080/20016689.2023.2244305","DOIUrl":"https://doi.org/10.1080/20016689.2023.2244305","url":null,"abstract":"<p><p><b>Purpose:</b> Nowadays, systematic literature reviews (SLRs) and meta-analyses are often placed at the top of the study hierarchy of evidence. The main objective of this paper is to evaluate the trends in SLRs of randomized controlled trials (RCTs) throughout the years. <b>Methods:</b> Medline database was searched, using a highly focused search strategy. Each paper was coded according to a specific ICD-10 code; the number of RCTs included in each evaluated SLR was also retrieved. All SLRs analyzing RCTs were included. Protocols, commentaries, or errata were excluded. No restrictions were applied. <b>Results:</b> A total of 7,465 titles and abstracts were analyzed, from which 6,892 were included for further analyses. There was a gradual increase in the number of annual published SLRs, with a significant increase in published articles during the last several years. Overall, the most frequently analyzed areas were diseases of the circulatory system (<i>n</i> = 750) and endocrine, nutritional, and metabolic diseases (<i>n</i> = 734). The majority of SLRs included between 11 and 50 RCTs each. <b>Conclusions:</b> The recognition of SLRs' usefulness is growing at an increasing speed, which is reflected by the growing number of published studies. The most frequently evaluated diseases are in alignment with leading causes of death and disability worldwide.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/93/41/ZJMA_11_2244305.PMC10443963.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10251437","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":"Transformative potential of artificial intelligence in global health policy.","authors":"Ridwan Islam Sifat,&nbsp;Upali Bhattacharya","doi":"10.1080/20016689.2023.2230660","DOIUrl":"https://doi.org/10.1080/20016689.2023.2230660","url":null,"abstract":"ChatGPT is an extraordinary example of how far artificial intelligence has come regarding conversational interfaces. Its exceptional ability to mimic human interaction by utilizing advanced natural language processing techniques has completely transformed how people interact with technology today [1,2]. It offers a highly immersive experience for users; their conversations feel natural and realistic as if they are interacting with another human being [3]. What distinguishes ChatGPT from other chatbots or virtual assistants is its incredible aptitude: when answering questions, it acknowledges user assumptions and challenges them using sophisticated algorithms. This feature significantly contributes to creating valuable insights and enhancing overall learning experiences for the users who engage with ChatGPT [4,5]. Overall, through its remarkable features, powerful machine learning models, and capacity to improve throughout interactions, ChatGPT represents one of the most innovative technologies available currently and continues to evolve every day, thus transforming communication [6]. Its ability to generate accurate responses without additional user input has made it a valuable resource beyond simple inquiries, with users relying on it for academic papers and essays due to its capability to provide supporting references upon request. However, it is important to acknowledge that these references may contain errors, as noted by researchers [7,8]. The remarkable transformation of artificial intelligence technology has revolutionized multiple fields, particularly public policy. Artificial intelligence systems are indispensable tools in policymaking due to their proficiencies in recognizing patterns in vast amounts of information [9]. With these capabilities, policymakers can make more informed decisions precisely based on insights rather than relying entirely on intuition or assumptions. The potential for AI’s continued evolution is immense and wildly anticipated to facilitate unparalleled advancements that could change how we shape our policies. ChatGPT is a promising AI technology that can revolutionize global health policy. It can facilitate communication between humans and machines, enabling vital contributions to complex decision-making processes at all levels of government agencies. However, whether ChatGPT will significantly impact governance regarding efficiency improvements or transparency development within society is unclear. 2. Advantages of integrating ChatGPT in global health policy","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/eb/fa/ZJMA_11_2230660.PMC10316731.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10195134","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":"Access to innovative drugs and the National Reimbursement Drug List in China: Changing dynamics and future trends in pricing and reimbursement.","authors":"Bérengère Macabeo,&nbsp;Liam Wilson,&nbsp;Jianwei Xuan,&nbsp;Ruichen Guo,&nbsp;Petar Atanasov,&nbsp;Linda Zheng,&nbsp;Clement François,&nbsp;Philippe Laramée","doi":"10.1080/20016689.2023.2218633","DOIUrl":"https://doi.org/10.1080/20016689.2023.2218633","url":null,"abstract":"<p><strong>Background and objectives: </strong>Multiple reforms aimed at improving the Chinese population's health have been introduced in recent years, including several designed to improve access to innovative drugs. We sought to review current factors affecting access to innovative drugs in China and to anticipate future trends.</p><p><strong>Methods: </strong>Targeted reviews of published literature and statistics on the Chinese healthcare system, medical insurance and reimbursement processes were conducted, as well as interviews with five Chinese experts involved in the reimbursement of innovative drugs.</p><p><strong>Results: </strong>Drug reimbursement in China is becoming increasingly centralized due to the removal of provincial pathways, the establishment of the National Healthcare Security Administration and the implementation of the National Reimbursement Drug List (NRDL), which is now the main route for drug reimbursement in China. There is also an increasing number of other channels via which patients may access innovative treatments, including various types of commercial insurance and special access. Health technology assessment (HTA) and health economic evidence are becoming pivotal elements of the NRDL decision-making process. Alongside the optimization of HTA decision making, innovative risk-sharing agreements are anticipated to be increasingly leveraged in the future to optimize access to highly specialized technologies and encourage innovation while safeguarding limited healthcare funds.</p><p><strong>Conclusions: </strong>Drug public reimbursement in China continues to align more closely with approaches widely used in Europe in terms of HTA, health economics and pricing. Centralization of decision-making processes for public reimbursement of innovative drugs allows consistency in assessment and access, which optimizes the improvement of the Chinese population's health.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/de/68/ZJMA_11_2218633.PMC10266112.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10545126","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 systematic review of the characteristics of data assessment tools to measure medical doctors' work-related quality of life.","authors":"Miyuki Ezura,&nbsp;Katsuhiko Sawada,&nbsp;Yusuke Takushima,&nbsp;Ataru Igarashi,&nbsp;Lida Teng","doi":"10.1080/20016689.2023.2234139","DOIUrl":"https://doi.org/10.1080/20016689.2023.2234139","url":null,"abstract":"<p><strong>Purpose: </strong>Remarkable progress in healthcare technology has recently been made alongside changes in concepts related to drugs and medical devices. It is speculated that this progress benefits not only patients but also healthcare professionals, such as medical doctors. We performed a systematic review of the characteristics of current data assessment tools to measure medical doctors' work-related quality of life (QOL).</p><p><strong>Methods: </strong>A literature search was conducted through PubMed and Ichushi-Web in 2020. The related search terms used were 'medical doctor,' 'quality of work life,' and 'questionnaire/interview.' Two reviewers independently screened the studies, and the characteristics of the QOL assessment tools used in the identified studies were qualitatively reviewed and summarized.</p><p><strong>Results: </strong>In total, 5,443 and 760 articles were retrieved from PubMed and Ichushi-Web, respectively, of which 82 studies were included in this review. Sixty-five (79%) studies used structured questionnaires, and 17 (21%) studies used semistructured questionnaires. In terms of the study purpose, the identified studies mainly included four: mental health, the work or labor situation, satisfaction, and QOL. Components used to measure work-related QOL included satisfaction, burnout, QOL, the work environment, stress, mental health, work-life balance, and others. None of the studies used an originally developed QOL questionnaire to assess the work-related benefits of medical doctors.</p><p><strong>Conclusion: </strong>This systematic review found that there is a lack of studies directly assessing the work-related QOL of medical doctors and a lack of effective data collection tools to assess all work-related QOL components.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b4/71/ZJMA_11_2234139.PMC10367570.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10194366","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":"Budget impact analysis for avatrombopag in the treatment of chronic primary immune thrombocytopenia in adult patients refractory to other treatments.","authors":"Andrea Aiello,&nbsp;Elisa Elena Mariano,&nbsp;Mariangela Prada,&nbsp;Cristina Teruzzi,&nbsp;Nicoletta Martone,&nbsp;Stefano Capri,&nbsp;Giuseppe Carli,&nbsp;Sergio Siragusa","doi":"10.1080/20016689.2023.2230663","DOIUrl":"https://doi.org/10.1080/20016689.2023.2230663","url":null,"abstract":"<p><p><b>Introduction:</b> Primary immune thrombocytopenia is a rare autoimmune disease characterised by a decreased platelet count resulting in an increased risk of bleeding events and even life-threatening haemorrhages. Thrombopoietin receptor agonists (TPO-RAs) are the standard of care second-line therapy for adult patients with chronic immune thrombocytopenia. The first TPO-RAs approved and reimbursed in Italy, eltrombopag and romiplostim, while effective, pose some issues in terms of safety (e.g., hepatotoxicity) or general management (e.g., dietary restrictions). Avatrombopag, an effective and well-tolerated TPO-RA, was recently granted reimbursement. <b>Methods:</b> A 3-year (2023-2025) budget impact analysis (BIA) was conducted to estimate its impact on the Italian National Health Service (NHS). Two scenarios were compared, of which one represents the current situation, without avatrombopag, and the other provides for an increasing market share of avatrombopag (up to 26.6%). <b>Results:</b> BIA shows that the increase in the use of avatrombopag correlates with savings for NHS: in the first year, saving would be €1,300,564, increasing to €2,774,210 in the third year, for a total of €6,083,231 over the 3-year period. The sensitivity analysis confirmed these savings in the scenario with avatrombopag. <b>Conclusions:</b> Based on this BIA, the introduction and reimbursement of avatrombopag is an efficient and advantageous choice for the Italian NHS.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/a1/4c/ZJMA_11_2230663.PMC10316730.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10195136","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":"Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.","authors":"Alexa C Klimchak,&nbsp;Lauren E Sedita,&nbsp;Louise R Rodino-Klapac,&nbsp;Jerry R Mendell,&nbsp;Craig M McDonald,&nbsp;Katherine L Gooch,&nbsp;Daniel C Malone","doi":"10.1080/20016689.2023.2216518","DOIUrl":"https://doi.org/10.1080/20016689.2023.2216518","url":null,"abstract":"<p><p><b>Background</b>: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene therapy that may delay progression of Duchenne muscular dystrophy (DMD), a severe, rare neuromuscular disease caused by <i>DMD</i> gene mutations. Early cost-effectiveness analyses are important to help contextualize the value of gene therapies for reimbursement decision making. <b>Objective</b>: To determine the potential value of delandistrogene moxeparvovec using a cost-effectiveness analysis. <b>Study design</b>: A simulation calculated lifetime costs and equal value of life years gained (evLYG). Inputs included extrapolated clinical trial results and published utilities/costs. As a market price for delandistrogene moxeparvovec has not been established, threshold analyses established maximum treatment costs as they align with value, including varying willingness-to-pay up to $500,000, accounting for severity/rarity. <b>Setting</b>: USA, healthcare system perspective <b>Patients</b>: Boys with DMD <b>Intervention</b>: Delandistrogene moxeparvovec plus standard of care (SoC; corticosteroids) versus SoC alone <b>Main outcome measure</b>: Maximum treatment costs at a given willingness-to-pay threshold <b>Results</b>: Delandistrogene moxeparvovec added 10.30 discounted (26.40 undiscounted) evLYs. The maximum treatment cost was approximately $5 M, assuming $500,000/evLYG. Varying the benefit discount rate to account for the single administration increased the estimated value to #$5M, assuming $500,000/evLYG. <b>Conclusion</b>: In this early economic model, delandistrogene moxeparvovec increases evLYs versus SoC and begins to inform its potential value from a healthcare perspective.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/29/7e/ZJMA_11_2216518.PMC10228300.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10195653","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":"Implementation of equity and access in Indian healthcare: current scenario and way forward.","authors":"Canna Ghia,&nbsp;Gautam Rambhad","doi":"10.1080/20016689.2023.2194507","DOIUrl":"https://doi.org/10.1080/20016689.2023.2194507","url":null,"abstract":"<p><strong>Introduction: </strong>The Indian healthcare system is evolving towards better healthcare implementation and coverage. However, even today, the health-care system faces several challenges, a few of which are yet to be addressed. The present review is aimed to delineate the past and present healthcare scenarios in India, health-care policies, and other initiatives for achieving universal health coverage (UHC).</p><p><strong>Methods: </strong>A literature search was done on various government databases, websites, and PubMed for obtaining data and statistics on healthcare funding, health insurance schemes, healthcare budget allocations, categories of medical expenses, government policies, and health technology assessment (HTA) in India.</p><p><strong>Results: </strong>The available data indicates 37.2% of the total population is covered by any health insurance of which 78% are covered by public insurance companies. Around 30% of the total health expenditure is borne by the public sector, and there is high out-of-pocket (OOP) expenditure on healthcare.</p><p><strong>Discussion: </strong>Several new health policies and schemes, an increase in 2021 budget for healthcare by 137%, vaccination drives, augmenting manufacturing of medical devices, special training packages, Artificial Intelligence/Machine Learning (AI/ML)-based standard treatment workflow systems to ensure proper treatment and clinical decision-making have been initiated by the government for improving healthcare funding, equity, and access.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/43/34/ZJMA_11_2194507.PMC10044314.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9224173","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":"Cost-utility and value of information analysis of tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma in the Irish healthcare setting.","authors":"Niamh Carey,&nbsp;Joy Leahy,&nbsp;Lea Trela-Larsen,&nbsp;Laura Mc Cullagh,&nbsp;Michael Barry","doi":"10.1080/20016689.2023.2166375","DOIUrl":"https://doi.org/10.1080/20016689.2023.2166375","url":null,"abstract":"<p><strong>Background: </strong>The evidence base of tisagenlecleucel is uncertain.</p><p><strong>Objective: </strong>To evaluate the cost-effectiveness of tisagenlecleucel. To conduct expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.</p><p><strong>Study design: </strong>A three-state partitioned survival model. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 5 years; general population mortality with a standardised mortality ratio was then applied. EVPI and EVPPI were scaled up to population according to the incidence of the decision.</p><p><strong>Setting: </strong>Irish healthcare payer.</p><p><strong>Participants: </strong>Patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL).</p><p><strong>Interventions: </strong>Tisagenlecleucel versus Salvage Chemotherapy (with or without haematopoietic stem cell transplant).</p><p><strong>Main outcome measure: </strong>Incremental cost-effectiveness ratio (ICER). Population EVPI and EVPPI.</p><p><strong>Results: </strong>At list prices, the ICER was €119,509 per quality-adjusted life year (QALY) (incremental costs €218,092; incremental QALYs 1.82). Probability of cost-effectiveness, at a €45,000 per QALY threshold, was 0%. Population EVPI was €0.00. Population EVPI, at the price of tisagenlecleucel that reduced the ICER to €45,000 per QALY, was €3,989,438. Here, survival analysis had the highest population EVPPI (€1,128,053).</p><p><strong>Conclusion: </strong>Tisagenlecleucel is not cost-effective, versus salvage chemotherapy (with or without haematopoietic stem cell transplant), for R/R DLBCL in Ireland. At list prices, further research to decrease decision uncertainty may not be of value.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/12/0c/ZJMA_11_2166375.PMC9858398.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10582070","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}