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Evaluating the Long-Term Cost-Effectiveness of the English NHS Diabetes Prevention Programme using a Markov Model. 使用马尔可夫模型评估英国国家医疗服务体系糖尿病预防计划的长期成本效益。
IF 2
PharmacoEconomics Open Pub Date : 2024-07-01 Epub Date: 2024-04-20 DOI: 10.1007/s41669-024-00487-6
Emma McManus
{"title":"Evaluating the Long-Term Cost-Effectiveness of the English NHS Diabetes Prevention Programme using a Markov Model.","authors":"Emma McManus","doi":"10.1007/s41669-024-00487-6","DOIUrl":"10.1007/s41669-024-00487-6","url":null,"abstract":"<p><strong>Background: </strong>In 2016, England launched the largest nationwide diabetes mellitus prevention programme, the NHS Diabetes Prevention Programme (NHS DPP). This paper seeks to evaluate the long-term cost-effectiveness of this programme.</p><p><strong>Methods: </strong>A Markov cohort state transition model was developed with a 35-year time horizon and yearly cycles to compare referral to the NHS DPP to usual care for individuals with non-diabetic hyperglycaemia. The modelled cohort of individuals mirrored the age profile of referrals received by the programme by April 2020. A health system perspective was taken, with costs in UK £ Sterling (price year 2020) and outcomes in terms of quality-adjusted life-years (QALYs). Probabilistic analysis with 10,000 Monte Carlo simulations was used. Several sensitivity analyses were conducted to explore the uncertainty surrounding the base case results, particularly varying the length of time for which the effectiveness of the programme was expected to last.</p><p><strong>Results: </strong>In the base case, using only the observed effectiveness of the NHS DPP at 3 years, it was found that the programme is likely to dominate usual care, by generating on average 40.8 incremental QALYs whilst saving £135,755 in costs for a cohort of 1000. At a willingness to pay of £20,000 per QALY, 98.1% of simulations were on or under the willingness-to-pay threshold. Scaling this up to the number of referrals actually received by the NHS DPP prior to April 2020, cost savings of £71.4 million were estimated over the 35-year time horizon and an additional 21,472 QALYs generated. These results are robust to several sensitivity analyses.</p><p><strong>Conclusion: </strong>The NHS DPP is likely to be cost-effective. Indeed, in the majority of the simulations, the NHS DPP was cost-saving and generated greater QALYs, dominating usual care. This research should serve as evidence to support the continued investment or recommissioning of diabetes prevention programmes.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11252105/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140870344","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
Introducing Open Science in Teaching Health Economic Modelling. 在健康经济模型教学中引入开放科学。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-01-18 DOI: 10.1007/s41669-023-00469-0
Xavier G L V Pouwels, Hendrik Koffijberg
{"title":"Introducing Open Science in Teaching Health Economic Modelling.","authors":"Xavier G L V Pouwels, Hendrik Koffijberg","doi":"10.1007/s41669-023-00469-0","DOIUrl":"10.1007/s41669-023-00469-0","url":null,"abstract":"<p><p>Open Science is gaining ground in all research fields, including health economics and outcomes research (HEOR). However, teaching Open Science is still in its infancy. This paper describes the design, implementation and evaluation of a teaching activity focusing on introducing Open Science during a Master's course during which participants have to develop a discrete event simulation. The teaching activity was organised as a series of lectures introducing different aspects of the Open Science philosophy and practices, such as good software coding practices, version control systems and reproducible research. The participants' increase in Open Science knowledge was elicited through a survey before and after the teaching innovation. After the teaching innovation, participants' knowledge of Open Science increased and they reported an improvement in Open Science-related skills, such as using a script-based statistical software, identifying and re-using open data, and collaborative script development. During the evaluation at the end of the course, the course participants mentioned that the Open Science-related content was interesting but would fit better within a course in which broader research-related content is taught. Based on this feedback, we will most likely narrow the scope of the Open-Science-related content in this course to Open Source Modelling which may better fit the scope of the course. This paper contains links to the teaching activities we developed and other resources which may be used to design teaching activities on Open Science. Herewith, we hope to inspire other teachers in including Open Science into their teaching.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058179/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139486297","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
Can Cyprus Afford Luspatercept? A Budget Impact Analysis of the Reimbursement of Luspatercept for the Management of Thalassaemia in Cyprus. 塞浦路斯能否负担得起 Luspatercept?塞浦路斯用于地中海贫血症治疗的 Luspatercept 的报销预算影响分析》(A Budget Impact Analysis of the Reimbursement of Luspatercept for the Management of Thalassaemia in Cyprus)。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-04-04 DOI: 10.1007/s41669-024-00482-x
Olga Pitsillidou, Panagiotis Petrou, M J Postma
{"title":"Can Cyprus Afford Luspatercept? A Budget Impact Analysis of the Reimbursement of Luspatercept for the Management of Thalassaemia in Cyprus.","authors":"Olga Pitsillidou, Panagiotis Petrou, M J Postma","doi":"10.1007/s41669-024-00482-x","DOIUrl":"10.1007/s41669-024-00482-x","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to estimate the budget impact of luspatercept reimbursement as an adjuvant to the standard management of β-thalassaemia major in Cyprus, from a societal perspective, and assess the financial feasibility of its inclusion in the β-thalassaemia armamentarium.</p><p><strong>Methods: </strong>A 5-year horizon budget impact model was developed to determine the budget impact of reimbursing luspatercept for the management of β-thalassaemia major in Cyprus. Two treatment discontinuation scenarios were elaborated. In the first scenario, luspatercept is reimbursed complementary to best supportive care, and a dropout rate of 40% is assumed based on published real-world data, while for the second scenario a dropout rate of 25%, is assumed as per the clinical trial data. Input parameters were retrieved from the phase III clinical trial of luspatercept, literature, and expert opinion consensus. One-way sensitivity analyses were conducted for both scenarios.</p><p><strong>Results: </strong>The addition of luspatercept to the standard management of β-thalassaemia major in Cyprus imparted an incremental budget impact ranging from €21,300,643 to €25,834,368, depending on the drop-out rate scenario assumed. Results were sensitive to the number of eligible patients and dose per patient.</p><p><strong>Conclusion: </strong>The potential reimbursement of luspatercept will wield a substantial impact on Cyprus total pharmaceutical expenditure and it is therefore imperative to affix a reimbursement framework that will allow the payer to mitigate uncertainty stemming out of the scarce clinical data and the inherently complex therapeutic landscape of β-thalassemia management.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058738/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140865075","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
Behind the Scenes: A Qualitative Investigation of Interviewers' Performance in EQ-5D Valuation Studies. 幕后:对 EQ-5D 估值研究中受访者表现的定性调查。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-04-09 DOI: 10.1007/s41669-024-00486-7
Carlotta Varriale, Giovanni Andrulli, Michela Meregaglia, Fanni Rencz, Aureliano Paolo Finch
{"title":"Behind the Scenes: A Qualitative Investigation of Interviewers' Performance in EQ-5D Valuation Studies.","authors":"Carlotta Varriale, Giovanni Andrulli, Michela Meregaglia, Fanni Rencz, Aureliano Paolo Finch","doi":"10.1007/s41669-024-00486-7","DOIUrl":"10.1007/s41669-024-00486-7","url":null,"abstract":"<p><strong>Background: </strong>The EuroQol Valuation Technology (EQ-VT) protocol is currently employed by the valuation studies of the EQ family of instruments worldwide. To date, all the evidence in support of the quality control (QC) originates from quantitative indicators.</p><p><strong>Objective: </strong>We aimed to explore interviewers' conversational patterns in EQ-VT interviews, beyond quantitative QC indicators, and to provide a preliminary exploration of how the interaction between interviewer and respondent impacts data quality.</p><p><strong>Methods: </strong>Two researchers transcribed and independently coded 24 video-recorded interviews from the Italian EQ-5D-5L valuation study, adopting the conversational analysis framework. The analysis identified positive and negative 'patterns' of conversational practice. These were categorized into themes and sub-themes and were used to score a random sample of 42 video-recorded interviews conducted at different time points by seven interviewers.</p><p><strong>Results: </strong>The conversational analysis identified 20 positive and 14 negative interview patterns, which were grouped into two main themes (i.e., task execution and communication skills). Positive items included appending questions that stimulated respondents' engagement, providing different explanations for an unclear aspect, supporting the participant with useful information for completing the tasks, and increasing the interview's coherence by confirming the respondent answers. Negative patterns included moving forward in the exercise without making sure that the respondent understood, trying to force an answer from the respondent, speaking too fast, and providing incomplete or incorrect explanations of the task. Most interviewers exhibited a moderate increase in positive patterns or a decrease in negative patterns over time. A certain degree of consistency between the quantitative QC results and the qualitative scoring deriving from the interviewer-respondent interaction was observed, with the best and worst performers of the qualitative scoring showing good and bad scores on key QC items, respectively.</p><p><strong>Conclusions: </strong>The identified positive and negative patterns may be useful to inform the training material of EQ-VT studies worldwide and complement the existing QC process.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058132/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140857436","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 Faricimab in the Treatment of Diabetic Macular Oedema (DMO): A UK Analysis. 法利西单抗治疗糖尿病黄斑水肿 (DMO) 的成本效益:英国分析。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-03-04 DOI: 10.1007/s41669-023-00465-4
Christian Bührer, Thomas Paling, Richard Gale, Tatiana Paulo, Marloes Bagijn
{"title":"Cost-Effectiveness of Faricimab in the Treatment of Diabetic Macular Oedema (DMO): A UK Analysis.","authors":"Christian Bührer, Thomas Paling, Richard Gale, Tatiana Paulo, Marloes Bagijn","doi":"10.1007/s41669-023-00465-4","DOIUrl":"10.1007/s41669-023-00465-4","url":null,"abstract":"<p><strong>Aim: </strong>The aim of this work was to evaluate the cost-effectiveness of faricimab against relevant therapeutic alternatives used in clinical practice for the treatment of diabetic macular oedema (DMO) in the UK.</p><p><strong>Methods: </strong>A state-transition (Markov) model, with health states based on visual acuity scores and treatment pathways, was developed to conduct cost-utility analysis of faricimab treat and extend (T&E) regimen versus ranibizumab pro re nata (PRN) and aflibercept PRN over a time horizon of 25 years. Comparison against bevacizumab PRN was considered in scenario analysis. Effectiveness data for faricimab was sourced from the pivotal YOSEMITE and RHINE double-blind randomised controlled trials, and from a network meta-analysis for comparators. Costs and (dis)utilities were taken from nationally published sources or literature. The base case included indirect costs (productivity gains, informal care) given the wider impacts of DMO on society. Sensitivity analyses were conducted.</p><p><strong>Results: </strong>In the base case, faricimab T&E dominated ranibizumab PRN and aflibercept PRN, being more effective and resulting in cost savings (between 0.16 and 0.36 mean QALYs gained, and £5483-9655 mean cost savings). In scenario analysis, faricimab was more effective but costlier compared with bevacizumab, with an incremental cost-effectiveness ratio (ICER) of £8898 per QALY gained. Considering only healthcare payer costs, the ICER of faricimab compared with ranibizumab PRN was £7991 per QALY gained and faricimab dominated aflibercept PRN.</p><p><strong>Conclusions: </strong>Faricimab T&E has the potential to reduce the burden of vision loss on society, giving people living with DMO greater independence and contributing to increased healthcare system capacity. At a threshold of £20,000, faricimab T&E is cost-effective compared with relevant comparators, and potentially cost saving.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058163/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140028662","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
Identification and prioritisation of items for a draft participant-reported questionnaire to measure use of social care, informal care, aids and adaptations. 确定与会者报告的调查问卷草案的项目和优先顺序,以衡量社会护理、非正式护理、辅助工具和改装的使用情况。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-03-07 DOI: 10.1007/s41669-024-00479-6
Kirsty M Garfield, Gail A Thornton, Samantha Husbands, Ailsa Cameron, William Hollingworth, Sian M Noble, Paul Roy, Joanna C Thorn
{"title":"Identification and prioritisation of items for a draft participant-reported questionnaire to measure use of social care, informal care, aids and adaptations.","authors":"Kirsty M Garfield, Gail A Thornton, Samantha Husbands, Ailsa Cameron, William Hollingworth, Sian M Noble, Paul Roy, Joanna C Thorn","doi":"10.1007/s41669-024-00479-6","DOIUrl":"10.1007/s41669-024-00479-6","url":null,"abstract":"<p><strong>Background: </strong>Resource-use measurement is integral for assessing cost-effectiveness within trial-based economic evaluations. Methods for gathering resource-use data from participants are not well developed, with questionnaires typically produced for each trial and rarely validated. The healthcare module of a generic, modular resource-use measure, designed for collecting self-report resource-utilisation data, has recently been developed in the UK. The objective of this research is to identify and prioritise items for new, bolt-on modules, covering informal care, social care and personal expenses incurred due to health and care needs.</p><p><strong>Methods: </strong>Identification and prioritisation, conducted between April and December 2021, involved a rapid review of questionnaires included in the Database of Instruments for Resource Use Measurement and economic evaluations published from 2011 to 2021 to identify candidate items, an online survey of UK-based social care professionals to identify omitted social care items and focus groups with UK-based health economists and UK-based people who access social care services either for themselves or as carers to prioritise items.</p><p><strong>Results: </strong>The review identified 203 items. Over half of the 24 survey respondents reported no missing items. Five academic health economists and four people who access social care services participated in focus groups. Feedback shaped the social and informal care modules and indicated that no specific personal expenses were essential to collect in all trials. Aids/adaptations were highlighted as costly personal expenses when relevant; therefore, the personal expenses module was narrowed to aids/adaptations only.</p><p><strong>Conclusion: </strong>Draft informal care, social care and aids/adaptations modules were developed, ready for further testing.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058127/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140060143","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
Updated Public Health Impact and Cost Effectiveness of Recombinant Zoster Vaccine in Canadian Adults Aged 50 Years and Older. 重组带状疱疹疫苗对加拿大 50 岁及以上成人的最新公共卫生影响和成本效益。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-04-11 DOI: 10.1007/s41669-024-00483-w
Sydney George, Justin Carrico, Katherine A Hicks, Dessi Loukov, Cheryl Ng, Jessica Regan, Nikolaos Giannelos
{"title":"Updated Public Health Impact and Cost Effectiveness of Recombinant Zoster Vaccine in Canadian Adults Aged 50 Years and Older.","authors":"Sydney George, Justin Carrico, Katherine A Hicks, Dessi Loukov, Cheryl Ng, Jessica Regan, Nikolaos Giannelos","doi":"10.1007/s41669-024-00483-w","DOIUrl":"10.1007/s41669-024-00483-w","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study was to update previously estimated public health impact and cost effectiveness of recombinant zoster vaccine (RZV) for the prevention of herpes zoster (HZ) in Canadians aged ≥50 years using longer-term RZV efficacy and waning data and real-world coverage and completion.</p><p><strong>Methods: </strong>A multicohort Markov model was used to conduct a cost-utility analysis comparing RZV with no HZ vaccination among Canadians aged ≥50 years. Real-world data were used for first-dose coverage (17.5%) and second-dose completion (65%). Vaccine efficacy and waning data were applied from up to 8-year follow-up from the ZOE-50 and ZOE-70 clinical trials. Incremental costs and benefits were calculated using a lifetime horizon from the healthcare payer (base case) and societal perspectives. A discount rate of 1.5% was applied to costs and quality-adjusted life-years (QALYs).</p><p><strong>Results: </strong>The model estimated that RZV would prevent 303,835 HZ cases, 83,256 post-herpetic neuralgia (PHN) cases, 39,653 other complications, and 99 HZ-related deaths compared with no HZ vaccination. Incremental cost-effectiveness ratios (ICERs) were estimated to be $27,486 and $22,097 per QALY (2022 Canadian dollars [CAN$]) from the healthcare payer and societal perspectives, respectively. The base-case ICER was most sensitive to a lower percentage of initial HZ cases with PHN. Almost all probabilistic sensitivity analysis simulations (98.1%) resulted in ICERs <CAN$50,000 per QALY.</p><p><strong>Conclusions: </strong>RZV is expected to remain a cost-effective option for Canadian adults aged ≥50 years when using longer-term RZV efficacy and waning estimates, although the estimated public health impact was smaller than in the previous analysis (due to lower coverage/completion estimates).</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058134/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140865531","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 Routine Monitoring of Long-Term Conditions in Primary Care: Informing Decision Modelling with a Systematic Review in Hypertension, Type 2 Diabetes and Chronic Kidney Disease. 基层医疗机构对长期病症进行常规监测的成本效益:通过对高血压、2 型糖尿病和慢性肾脏病的系统回顾为决策建模提供依据。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-02-23 DOI: 10.1007/s41669-024-00473-y
Syed G Mohiuddin, Mary E Ward, William Hollingworth, Jessica C Watson, Penny F Whiting, Howard H Z Thom
{"title":"Cost-Effectiveness of Routine Monitoring of Long-Term Conditions in Primary Care: Informing Decision Modelling with a Systematic Review in Hypertension, Type 2 Diabetes and Chronic Kidney Disease.","authors":"Syed G Mohiuddin, Mary E Ward, William Hollingworth, Jessica C Watson, Penny F Whiting, Howard H Z Thom","doi":"10.1007/s41669-024-00473-y","DOIUrl":"10.1007/s41669-024-00473-y","url":null,"abstract":"<p><strong>Background: </strong>Long-term conditions (LTCs) are major public health problems with a considerable health-related and economic burden. Modelling is key in assessing costs and benefits of different disease management strategies, including routine monitoring, in the conditions of hypertension, type 2 diabetes mellitus (T2DM) and chronic kidney disease (CKD) in primary care.</p><p><strong>Objective: </strong>This review aimed to identify published model-based cost-effectiveness studies of routine laboratory testing strategies in these LTCs to inform a model evaluating the cost effectiveness of testing strategies in the UK.</p><p><strong>Methods: </strong>We searched the Medline and Embase databases from inception to July 2023; the National Institute for Health and Care Institute (NICE) website was also searched. Studies were included if they were model-based economic evaluations, evaluated testing strategies, assessed regular testing, and considered adults aged >16 years. Studies identified were summarised by testing strategies, model type, structure, inputs, assessment of uncertainty, and conclusions drawn.</p><p><strong>Results: </strong>Five studies were included in the review, i.e. Markov (n = 3) and microsimulation (n = 2) models. Models were applied within T2DM (n = 2), hypertension (n = 1), T2DM/hypertension (n = 1) and CKD (n = 1). Comorbidity between all three LTCs was modelled to varying extents. All studies used a lifetime horizon, except for a 10-year horizon T2DM model, and all used quality-adjusted life-years as the effectiveness outcome, except a TD2M model that used glycaemic control. No studies explicitly provided a rationale for their selected modelling approach. UK models were available for diabetes and CKD, but these compared only a limited set of routine monitoring tests and frequencies.</p><p><strong>Conclusions: </strong>There were few studies comparing routine testing strategies in the UK, indicating a need to develop a novel model in all three LTCs. Justification for the modelling technique of the identified studies was lacking. Markov and microsimulation models, with and without comorbidities, were used; however, the findings of this review can provide data sources and inform modelling approaches for evaluating the cost effectiveness of testing strategies in all three LTCs.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058158/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139932316","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 Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany. 德国治疗中重度血友病 B 的 Etranacogene Dezaparvovec 与延长半衰期预防疗法的成本效益分析。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-03-23 DOI: 10.1007/s41669-024-00480-z
Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks
{"title":"Cost-Effectiveness Analysis of Etranacogene Dezaparvovec Versus Extended Half-Life Prophylaxis for Moderate-to-Severe Haemophilia B in Germany.","authors":"Niklaus Meier, Hendrik Fuchs, Katya Galactionova, Cedric Hermans, Mark Pletscher, Matthias Schwenkglenks","doi":"10.1007/s41669-024-00480-z","DOIUrl":"10.1007/s41669-024-00480-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Haemophilia B is a rare genetic disease that is caused by a deficiency of coagulation factor IX (FIX) in the blood and leads to internal and external bleeding. Under the current standard of care, haemophilia is treated either prophylactically or on-demand via intravenous infusions of FIX. These treatment strategies impose a high burden on patients and health care systems as haemophilia B requires lifelong treatment, and FIX is costly. Etranacogene dezaparvovec (ED) is a gene therapy for haemophilia B that has been recently approved by the United States Food and Drug Administration and has received a recommendation for conditional marketing authorization by the European Medicines Agency. We aimed to examine the cost-effectiveness of ED versus extended half-life FIX (EHL-FIX) prophylaxis for moderate-to-severe haemophilia B from a German health care payer perspective.</p><p><strong>Methods: </strong>A microsimulation model was implemented in R. The model used data from the ED phase 3 clinical trial publication and further secondary data sources to simulate and compare patients receiving ED or EHL-FIX prophylaxis over a lifetime horizon, with the potential for ED patients to switch treatment to EHL-FIX prophylaxis when the effectiveness of ED waned. Primary outcomes of this analysis included discounted total costs, discounted quality-adjusted life years (QALYs), incremental cost-effectiveness, and the incremental net monetary benefit. The annual discount rate for costs and effects was 3%. Uncertainty was examined via probabilistic analysis and additional univariate sensitivity analyses.</p><p><strong>Results: </strong>Probabilistic analysis indicated that patients treated with ED instead of EHL-FIX prophylaxis gained 0.50 QALYs and experienced cost savings of EUR 1,179,829 at a price of EUR 1,500,000 per ED treatment. ED was the dominant treatment strategy. At a willingness to pay of EUR 50,000/QALY, the incremental net monetary benefit amounted to EUR 1,204,840.</p><p><strong>Discussion: </strong>Depending on the price, ED can save costs and improve health outcomes of haemophilia patients compared with EHL-FIX prophylaxis, making it a potentially cost-effective alternative. These results are uncertain due to a lack of evidence regarding the long-term effectiveness of ED.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058170/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140194336","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 Analysis of Digital Breast Tomosynthesis Added to Synthetic Mammography in Breast Cancer Screening in Brazil. 巴西乳腺癌筛查中数字乳腺断层成像与合成乳腺 X 线照相术的成本效益分析。
IF 2
PharmacoEconomics Open Pub Date : 2024-05-01 Epub Date: 2024-01-18 DOI: 10.1007/s41669-023-00470-7
Henrique Lima Couto, Ludmila Peres Gargano, Vilmar Marques de Oliveira, Bertha Andrade Coelho, Eduardo Carvalho Pessoa, Augusto Tufi Hassan, Agnaldo Lopes Silva, Linei Augusta Brolini Delle Urban, Luciano Chala Fernandes, Nisha Sharma, Ritse Mann, Stuart A McIntosh, Fernando Zanghelini
{"title":"Cost-Effectiveness Analysis of Digital Breast Tomosynthesis Added to Synthetic Mammography in Breast Cancer Screening in Brazil.","authors":"Henrique Lima Couto, Ludmila Peres Gargano, Vilmar Marques de Oliveira, Bertha Andrade Coelho, Eduardo Carvalho Pessoa, Augusto Tufi Hassan, Agnaldo Lopes Silva, Linei Augusta Brolini Delle Urban, Luciano Chala Fernandes, Nisha Sharma, Ritse Mann, Stuart A McIntosh, Fernando Zanghelini","doi":"10.1007/s41669-023-00470-7","DOIUrl":"10.1007/s41669-023-00470-7","url":null,"abstract":"<p><strong>Background: </strong>Literature meta-analysis results show that digital breast tomosynthesis (DBT) combined with synthesized two-dimensional (s2D) mammograms can reduce recalls and improve breast cancer detection. Uncertainty regarding the screening of patients with breast cancer presents a health economic challenge, both in terms of healthcare resource use and quality of life impact on patients.</p><p><strong>Objective: </strong>This study aims to estimate the cost effectiveness of DBT + s2D versus digital mammography (DM) used in a biennial breast cancer screening setting of women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts in the Brazilian supplementary health system.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was performed on the basis of clinical data obtained from a systematic review with meta-analysis performed to evaluate the analytical validity and clinical utility of DBT + s2D compared with DM. The search was conducted in the PubMed, Cochrane Library and Embase databases, with the main descriptors of the technology, a comparator, and the clinical condition in question, on 9 June 2022. The hybrid economic model (decision tree plus Markov model) simulated costs and outcomes over a lifetime for women aged 40-69 years with scattered areas of fibroglandular breast density and heterogeneous dense breasts. We analyzed incremental cost-effectiveness ratio (ICER) to measure the incremental cost difference per quality-adjusted life year (QALY) of adding DBT + s2D to breast cancer screening.</p><p><strong>Results: </strong>DBT + s2D incurred a cost saving of € 954.02 per patient, in the time horizon of 30 years, compared with DM, and gained 5.1989 QALYs, which would be considered a dominant intervention. These results were confirmed in sensitivity analyses.</p><p><strong>Conclusion: </strong>Switching from DM to biennial DBT + s2D was cost effective. Furthermore, reductions in false-positive recall rates should also be considered in decision making.</p>","PeriodicalId":19770,"journal":{"name":"PharmacoEconomics Open","volume":null,"pages":null},"PeriodicalIF":2.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11058155/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139486292","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
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