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Design and Features of Pricing and Payment Schemes for Health Technologies: A Scoping Review and a Proposal for a Flexible Need-Driven Classification. 卫生技术定价和付款计划的设计与特点:医疗技术定价与支付方案的设计与特点:范围审查与灵活的需求驱动分类建议》。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-01-01 Epub Date: 2024-10-15 DOI: 10.1007/s40273-024-01435-2
Vittoria Ardito, Oriana Ciani, Michael Drummond
{"title":"Design and Features of Pricing and Payment Schemes for Health Technologies: A Scoping Review and a Proposal for a Flexible Need-Driven Classification.","authors":"Vittoria Ardito, Oriana Ciani, Michael Drummond","doi":"10.1007/s40273-024-01435-2","DOIUrl":"10.1007/s40273-024-01435-2","url":null,"abstract":"<p><strong>Background and objective: </strong>In a context of growing clinical and financial uncertainty, pricing and payment schemes can act as possible solutions to the problems of affordability and access to health technologies. However, a comprehensive categorization of the available schemes to help decision makers tackle these challenges is lacking. This work aims at mapping existing types of pricing and payment schemes, and proposes a new approach for their classification, in order to help decision makers and other stakeholders select the best type of scheme to meet their needs.</p><p><strong>Methods: </strong>A Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR)-compliant scoping literature review was performed between 2010 and 2023 in three databases (PubMed, Web of Science, Scopus). The search strategy was developed around two groups of keywords, \"pricing/payment schemes\" and \"scheme innovativeness\". Eligible studies were those illustrating the unique design and features of each scheme type, which were extracted by two independent reviewers, and synthesized using a narrative format, including a detailed tabular description of each type of scheme.</p><p><strong>Results: </strong>A total of 70 unique types of pricing and payment schemes were identified. Around one third (33%) was only specified in principle, while two thirds (67%) had been implemented in practice. About half of the scheme types were proposed for drugs (34/70, 49%), and the vast majority were not designed for a specific therapeutic area (55/70, 79%). Each scheme type was categorized based on distinctive characteristics: the objectives, the outcome component, the timing/modalities of payments, and the evidence collection requirements.</p><p><strong>Conclusions: </strong>Instead of trying to fit the retrieved schemes into a rigid taxonomy, we propose a new approach that suggests a flexible need-driven use of the available scheme types, driven primarily by the specific objective that one might have, and allows leveraging of the other key characteristics of each type of scheme.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"5-29"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724778/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Economic Burden Associated with Pulmonary Arterial Hypertension in the United States. 美国与肺动脉高压有关的经济负担。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-01-01 Epub Date: 2024-10-12 DOI: 10.1007/s40273-024-01427-2
Anna Watzker, Adnan Alsumali, Christine Ferro, Gabriela Dieguez, Clare Park, Dominik Lautsch, Karim El-Kersh
{"title":"Economic Burden Associated with Pulmonary Arterial Hypertension in the United States.","authors":"Anna Watzker, Adnan Alsumali, Christine Ferro, Gabriela Dieguez, Clare Park, Dominik Lautsch, Karim El-Kersh","doi":"10.1007/s40273-024-01427-2","DOIUrl":"10.1007/s40273-024-01427-2","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevated pressure in the pulmonary arteries, commonly resulting in right heart failure. PAH is associated with a high economic burden throughout the duration of the disease.</p><p><strong>Methods: </strong>This retrospective cohort study of the Milliman Contributor Health Source Data, the Medicare 100% Research Identifiable Files, and the Merative Marketscan<sup>®</sup> Commercial dataset between 2018 and 2020 identified adult patients with prevalent PAH based on the earliest qualifying diagnosis date or medication date ('index date') between January 1, 2019 and November 30, 2020. Outcomes were assessed using patient data from index date through the earliest of end of enrollment, end of data, or death (Medicare fee-for-service [FFS] only). All-cause and PAH-related medical and pharmacy costs per-patient per-month (PPPM) and healthcare resource utilization per 1000 patients were summarized.</p><p><strong>Results: </strong>The study included 11,670 Medicare FFS, 1021 Medicare Advantage, 274 Medicaid, and 1174 commercially insured patients in the US. The annual national burden to payers was estimated to be US$3.1 billion. The PPPM payer costs ranged from US$6500 to US$14,742; out-of-pocket (OOP) costs ranged from US$341 to US$907 PPPM. Inpatient utilization rate ranged from 435 to 770 per 1000 patients for all-cause admissions and from 15 to 58 per 1000 patients for PAH-related admissions.</p><p><strong>Conclusions: </strong>This study demonstrates that PAH continues to be associated with a high economic burden and healthcare resource utilization across all payer types within the US healthcare system.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"83-91"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142471986","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparative Analysis of Traditional and Pharmacometric-Based Pharmacoeconomic Modeling in the Cost-Utility Evaluation of Sunitinib Therapy. 传统药物经济学模型与基于药物计量学的药物经济学模型在舒尼替尼治疗成本效用评估中的对比分析
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-01-01 Epub Date: 2024-09-26 DOI: 10.1007/s40273-024-01438-z
Maddalena Centanni, Janine Nijhuis, Mats O Karlsson, Lena E Friberg
{"title":"Comparative Analysis of Traditional and Pharmacometric-Based Pharmacoeconomic Modeling in the Cost-Utility Evaluation of Sunitinib Therapy.","authors":"Maddalena Centanni, Janine Nijhuis, Mats O Karlsson, Lena E Friberg","doi":"10.1007/s40273-024-01438-z","DOIUrl":"10.1007/s40273-024-01438-z","url":null,"abstract":"<p><strong>Background: </strong>Cost-utility analyses (CUAs) increasingly use models to predict long-term outcomes and translate trial data to real-world settings. Model structure uncertainty affects these predictions. This study compares pharmacometric against traditional pharmacoeconomic model evaluations for CUAs of sunitinib in gastrointestinal stromal tumors (GIST).</p><p><strong>Methods: </strong>A two-arm trial comparing sunitinib 37.5 mg daily with no treatment was simulated using a pharmacometric-based pharmacoeconomic model framework. Overall, four existing models [time-to-event (TTE) and Markov models] were re-estimated to the survival data and linked to logistic regression models describing the toxicity data [neutropenia, thrombocytopenia, hypertension, fatigue, and hand-foot syndrome (HFS)] to create traditional pharmacoeconomic model frameworks. All five frameworks were used to simulate clinical outcomes and sunitinib treatment costs, including a therapeutic drug monitoring (TDM) scenario.</p><p><strong>Results: </strong>The pharmacometric model framework predicted that sunitinib treatment costs an additional 142,756 euros per quality adjusted life year (QALY) compared with no treatment, with deviations - 21.2% (discrete Markov), - 15.1% (continuous Markov), + 7.2% (TTE Weibull), and + 39.6% (TTE exponential) from the traditional model frameworks. The pharmacometric framework captured the change in toxicity over treatment cycles (e.g., increased HFS incidence until cycle 4 with a decrease thereafter), a pattern not observed in the pharmacoeconomic frameworks (e.g., stable HFS incidence over all treatment cycles). Furthermore, the pharmacoeconomic frameworks excessively forecasted the percentage of patients encountering subtherapeutic concentrations of sunitinib over the course of time (pharmacoeconomic: 24.6% at cycle 2 to 98.7% at cycle 16, versus pharmacometric: 13.7% at cycle 2 to 34.1% at cycle 16).</p><p><strong>Conclusions: </strong>Model structure significantly influences CUA predictions. The pharmacometric-based model framework more closely represented real-world toxicity trends and drug exposure changes. The relevance of these findings depends on the specific question a CUA seeks to address.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"31-43"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724784/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142351615","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
How Much Better is Faster? Empirical Tests of QALY Assumptions in Health-Outcome Sequences. 快有多好?健康-收入序列中 QALY 假设的实证检验。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-01-01 Epub Date: 2024-10-04 DOI: 10.1007/s40273-024-01437-0
F Reed Johnson, John J Sheehan, Semra Ozdemir, Matthew Wallace, Jui-Chen Yang
{"title":"How Much Better is Faster? Empirical Tests of QALY Assumptions in Health-Outcome Sequences.","authors":"F Reed Johnson, John J Sheehan, Semra Ozdemir, Matthew Wallace, Jui-Chen Yang","doi":"10.1007/s40273-024-01437-0","DOIUrl":"10.1007/s40273-024-01437-0","url":null,"abstract":"<p><strong>Objectives: </strong>This study was designed to test hypotheses regarding the path dependence of health-outcome values in the form of linear additivity of health-state utilities and diminishing marginal utility of health outcomes.</p><p><strong>Methods: </strong>We employed a discrete-choice experiment to quantify patient treatment preferences for major depressive disorder. In a series of choice questions, participants evaluated seven symptom-improvement sequences and out-of-pocket costs over 6-week durations. Money-equivalent values were derived from a deductive latent-class mixed-logit analysis.</p><p><strong>Results: </strong>The discrete-choice experiment was completed by 751 respondents with self-reported major depressive disorder recruited from an online commercial panel. The class-membership probability was 0.83 for latent-class preferences consistent with supporting relative importance weights for all symptom-improvement sequences in the study design. First, we found strong support for diminishing marginal utility in symptom-improvement sequences. The money-equivalent value of an initial week of normal mood was $147 (95% confidence interval: $128, $166) and a second week of normal mood was $70 ($49, $91). Furthermore, for short treatment durations where conventional discounting was not a factor, equivalent changes in health status were valued more highly for an earlier onset of effect: holding subsequent symptom patterns constant, $338 (211, 454) versus $70 (49, 91) for improvements starting in week 2 versus week 3 and $147 ($128, $166) versus $29 (-$4, $64) for improvements starting in week 3 versus week 4.</p><p><strong>Conclusions: </strong>Our findings imply that conventional quality-adjusted life-year calculations in which health values are assumed to be path independent can understate the value of health improvements that appear earlier in a sequence.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"45-52"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142375715","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The Impact of Tocilizumab Coverage on Health Equity for Inpatients with COVID-19 in the USA: A Distributional Cost-Effectiveness Analysis. 美国 COVID-19 住院患者使用托昔单抗对健康公平的影响:分布式成本效益分析》(The Impact of Tocilizumab Coverage on Health Equity for Inpatient Patients with COVID-19: A Distributional Cost-Effectiveness Analysis)。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2025-01-01 Epub Date: 2024-10-10 DOI: 10.1007/s40273-024-01436-1
Stacey Kowal, Katherine L Rosettie
{"title":"The Impact of Tocilizumab Coverage on Health Equity for Inpatients with COVID-19 in the USA: A Distributional Cost-Effectiveness Analysis.","authors":"Stacey Kowal, Katherine L Rosettie","doi":"10.1007/s40273-024-01436-1","DOIUrl":"10.1007/s40273-024-01436-1","url":null,"abstract":"<p><strong>Objectives: </strong>We conducted a distributional cost-effectiveness analysis to evaluate how coverage of tocilizumab for inpatients with COVID-19 from 2021 to present impacted health equity in the USA.</p><p><strong>Methods: </strong>A published, payer-perspective, distributional cost-effectiveness analysis for inpatient COVID-19 treatments was adapted to include information on baseline health disparities across 25 equity-relevant subgroups based on race and ethnicity (5 census-based groups), and county-level social vulnerability (5 geographic quintiles). The underlying cost-effectiveness analysis was updated to reflect patient characteristics at admission, standard of care outcomes, tocilizumab efficacy, and contemporary unit costs. The distributional cost-effectiveness analysis inputs for COVID-19 hospitalization and subgroup risk adjustments based on social vulnerability were derived from published estimates. Opportunity costs were estimated by converting total tocilizumab spend into quality-adjusted life-years (QALYs), distributed equally across subgroups.</p><p><strong>Results: </strong>Tocilizumab treatment was cost effective across all subgroups. Treatment resulted in larger relative QALY gains in more socially vulnerable subgroups than less socially vulnerable subgroups, given higher hospitalization rates and inpatient mortality. Using an opportunity cost threshold of US$150,000/QALY and an Atkinson index of 11, tocilizumab was estimated to have improved social welfare by increasing population health (53,252 QALYs gained) and reducing existing overall US health inequalities by 0.003% since 2021.</p><p><strong>Conclusions: </strong>Use of tocilizumab for COVID-19 since 2021 increased population health while improving health equity, as more patients with lower baseline health were eligible for treatment and received larger relative health gains. Future equitable access to tocilizumab for inpatients with COVID-19 is expected to lead to continued increases in population health and reductions in disparities.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"67-82"},"PeriodicalIF":4.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11724795/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Acknowledgement to Referees. 鸣谢裁判员。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2024-12-14 DOI: 10.1007/s40273-024-01464-x
{"title":"Acknowledgement to Referees.","authors":"","doi":"10.1007/s40273-024-01464-x","DOIUrl":"https://doi.org/10.1007/s40273-024-01464-x","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824286","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evidence Following Conditional NICE Technology Appraisal Recommendations: A Critical Analysis of Methods, Quality and Risk of Bias. NICE 有条件技术评估建议后的证据:对方法、质量和偏差风险的批判性分析。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2024-12-01 Epub Date: 2024-09-09 DOI: 10.1007/s40273-024-01418-3
Yankier Pijeira Perez, Dyfrig A Hughes
{"title":"Evidence Following Conditional NICE Technology Appraisal Recommendations: A Critical Analysis of Methods, Quality and Risk of Bias.","authors":"Yankier Pijeira Perez, Dyfrig A Hughes","doi":"10.1007/s40273-024-01418-3","DOIUrl":"10.1007/s40273-024-01418-3","url":null,"abstract":"<p><strong>Background: </strong>The National Institute for Health and Care Excellence (NICE) may approve health technologies on condition of more evidence generated only in research (OiR) or only with research (OwR). NICE specifies the information needed to comply with its request, although it may not necessarily guarantee good quality and timely evidence for re-appraisal, before reaching a final decision.</p><p><strong>Aim: </strong>This study aimed to critically appraise the methods, quality and risk of bias of evidence generated in response to NICE OiR and OwR technology appraisal (TA) and highly specialised technologies (HSTs) recommendations.</p><p><strong>Methods: </strong>NICE TAs (between March 2000 and September 2020) and HST evaluations (to October 2023) of medicines were reviewed. Conditional recommendations were analysed to identify the evidence requested by NICE for re-appraisal. The new evidence was analysed for compliance with NICE's request and assessed using the Cochrane Collaboration's tools for risk of bias in randomised trials and the ROBINS-I tool for non-randomised evidence.</p><p><strong>Results: </strong>NICE made 54 conditional recommendations from TAs (13 OiR and 41 OwR) and five conditional recommendations for HSTs (all OwR). Of these, 16 TAs presented additional evidence for re-appraisal (9 OiR [69%] and 7 OwR [17%]) and three HSTs (3 OwR [60%]). Two of the nine re-appraised TAs with OiR recommendation and four of the seven OwR complied fully with NICE's request for further evidence, while all three from the HSTs complied. The majority of re-appraised TAs and HSTs included evidence that was deemed to be at serious, high, moderate or unclear risk of bias. Among the 26 randomised controlled trials from TAs assessed, eight were categorised as having low risk of bias in all domains and ten had at least one domain as a high risk of bias. Reporting was unclear for the remainder. Twenty-two non-randomised studies, primarily single-arm studies, were susceptible to biases mostly due to the selection of participants and to confounding. Two HSTs provided evidence from randomised controlled trials which were classified as unclear or high risk of bias. All non-randomised evidence from HSTs were categorised as moderate or serious risk of bias.</p><p><strong>Conclusions: </strong>There is widespread non-compliance with agreed data requests and important variation in the quality of evidence submitted in response to NICE conditional approval recommendations. Quality standards ought to be stipulated in respect to evidence contributing to re-appraisals following NICE conditional approval recommendations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1373-1394"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11564307/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154772","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care. 不同的模型,相同的结果:选择不同方法建立嵌合抗原受体 T 细胞疗法与标准疗法的成本效益模型时的考虑因素》。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2024-12-01 Epub Date: 2024-09-07 DOI: 10.1007/s40273-024-01430-7
Amy Gye, Richard De Abreu Lourenco, Stephen Goodall
{"title":"Different Models, Same Results: Considerations When Choosing Between Approaches to Model Cost Effectiveness of Chimeric-Antigen Receptor T-Cell Therapy Versus Standard of Care.","authors":"Amy Gye, Richard De Abreu Lourenco, Stephen Goodall","doi":"10.1007/s40273-024-01430-7","DOIUrl":"10.1007/s40273-024-01430-7","url":null,"abstract":"<p><strong>Objective: </strong>Chimeric antigen-receptor T-cell therapy (CAR-T) is characterised by early phase data at the time of registration, high upfront cost and a complex manufacturing and administration process compared with standard therapies. Our objective was to compare the performance of different models to assess the cost effectiveness of CAR-T using a state-transition model (STM), partitioned survival model (PSM) and discrete event simulation (DES).</p><p><strong>Methods: </strong>Individual data for tisagenlecleucel for the treatment of young patients with acute lymphoblastic leukaemia (ALL) were used to populate the models. Costs and benefits were measured over a lifetime to generate a cost per quality-adjusted life-year (QALY). Model performance was compared quantitatively on the outcomes generated and a checklist developed summarising the components captured by each model type relevant to assessing cost effectiveness of CAR-T.</p><p><strong>Results: </strong>Models generated similar results with base-case analyses ranging from an incremental cost per QALY of $96,074-$99,625. DES was the only model to specifically capture CAR-T wait time, demonstrating a substantial loss of benefit of CAR-T with increased wait time.</p><p><strong>Conclusion: </strong>Although model type did not meaningfully impact base-case results, the ability to incorporate an outcome-based payment arrangement (OBA) and wait time are important elements to consider when selecting a model for CAR-T. DES provided greater flexibility compared with STM and PSM approaches to deal with the complex manufacturing and administration process that can lead to extended wait times and substantially reduce the benefit of CAR-T. This is an important consideration when selecting a model type for CAR-T, so major drivers of uncertainty are considered in funding decisions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1359-1371"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11564325/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142146027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
An Evaluation of an Algorithm for the Selection of Flexible Survival Models for Cancer Immunotherapies: Pass or Fail? 癌症免疫疗法灵活生存模型选择算法评估:通过还是失败?
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2024-12-01 Epub Date: 2024-09-20 DOI: 10.1007/s40273-024-01429-0
Nicholas R Latimer, Kurt Taylor, Anthony J Hatswell, Sophia Ho, Gabriel Okorogheye, Clara Chen, Inkyu Kim, John Borrill, David Bertwistle
{"title":"An Evaluation of an Algorithm for the Selection of Flexible Survival Models for Cancer Immunotherapies: Pass or Fail?","authors":"Nicholas R Latimer, Kurt Taylor, Anthony J Hatswell, Sophia Ho, Gabriel Okorogheye, Clara Chen, Inkyu Kim, John Borrill, David Bertwistle","doi":"10.1007/s40273-024-01429-0","DOIUrl":"10.1007/s40273-024-01429-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Accurately extrapolating survival beyond trial follow-up is essential in a health technology assessment where model choice often substantially impacts estimates of clinical and cost effectiveness. Evidence suggests standard parametric models often provide poor fits to long-term data from immuno-oncology trials. Palmer et al. developed an algorithm to aid the selection of more flexible survival models for these interventions. We assess the usability of the algorithm, identify areas for improvement and evaluate whether it effectively identifies models capable of accurate extrapolation.</p><p><strong>Methods: </strong>We applied the Palmer algorithm to the CheckMate-649 trial, which investigated nivolumab plus chemotherapy versus chemotherapy alone in patients with gastroesophageal adenocarcinoma. We evaluated the algorithm's performance by comparing survival estimates from identified models using the 12-month data cut to survival observed in the 48-month data cut.</p><p><strong>Results: </strong>The Palmer algorithm offers a systematic procedure for model selection, encouraging detailed analyses and ensuring that crucial stages in the selection process are not overlooked. In our study, a range of models were identified as potentially appropriate for extrapolating survival, but only flexible parametric non-mixture cure models provided extrapolations that were plausible and accurately predicted subsequently observed survival. The algorithm could be improved with minor additions around the specification of hazard plots and setting out plausibility criteria.</p><p><strong>Conclusions: </strong>The Palmer algorithm provides a systematic framework for identifying suitable survival models, and for defining plausibility criteria for extrapolation validity. Using the algorithm ensures that model selection is based on explicit justification and evidence, which could reduce discordance in health technology appraisals.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1395-1412"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11564353/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142292932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Incorporating Complexity and System Dynamics into Economic Modelling for Mental Health Policy and Planning. 将复杂性和系统动力学纳入心理健康政策和规划的经济模型。
IF 4.4 3区 医学
PharmacoEconomics Pub Date : 2024-12-01 Epub Date: 2024-10-01 DOI: 10.1007/s40273-024-01434-3
Paul Crosland, Deborah A Marshall, Seyed Hossein Hosseini, Nicholas Ho, Catherine Vacher, Adam Skinner, Kim-Huong Nguyen, Frank Iorfino, Sebastian Rosenberg, Yun Ju Christine Song, Apostolos Tsiachristas, Kristen Tran, Jo-An Occhipinti, Ian B Hickie
{"title":"Incorporating Complexity and System Dynamics into Economic Modelling for Mental Health Policy and Planning.","authors":"Paul Crosland, Deborah A Marshall, Seyed Hossein Hosseini, Nicholas Ho, Catherine Vacher, Adam Skinner, Kim-Huong Nguyen, Frank Iorfino, Sebastian Rosenberg, Yun Ju Christine Song, Apostolos Tsiachristas, Kristen Tran, Jo-An Occhipinti, Ian B Hickie","doi":"10.1007/s40273-024-01434-3","DOIUrl":"10.1007/s40273-024-01434-3","url":null,"abstract":"<p><p>Care as usual has failed to stem the tide of mental health challenges in children and young people. Transformed models of care and prevention are required, including targeting the social determinants of mental health. Robust economic evidence is crucial to guide investment towards prioritised interventions that are effective and cost-effective to optimise health outcomes and ensure value for money. Mental healthcare and prevention exhibit the characteristics of complex dynamic systems, yet dynamic simulation modelling has to date only rarely been used to conduct economic evaluation in this area. This article proposes an integrated decision-making and planning framework for mental health that includes system dynamics modelling, cost-effectiveness analysis, and participatory model-building methods, in a circular process that is constantly reviewed and updated in a 'living model' ecosystem. We describe a case study of this approach for mental health system policy and planning that synergises the unique attributes of a system dynamics approach within the context of economic evaluation. This kind of approach can help decision makers make the most of precious, limited resources in healthcare. The application of modelling to organise and enable better responses to the youth mental health crisis offers positive benefits for individuals and their families, as well as for taxpayers.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1301-1315"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11564312/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142361896","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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