Value in Health最新文献

{"title":"Validating Health Economic Models With the Probabilistic Analysis Check dashBOARD","authors":"","doi":"10.1016/j.jval.2024.04.008","DOIUrl":"10.1016/j.jval.2024.04.008","url":null,"abstract":"<div><h3>Objectives</h3><p>Health economic (HE) models are often considered as “black boxes” because they are not publicly available and lack transparency, which prevents independent scrutiny of HE models. Additionally, validation efforts and validation status of HE models are not systematically reported. Methods to validate HE models in absence of their full underlying code are therefore urgently needed to improve health policy making.</p><p>This study aimed to develop and test a generic dashboard to systematically explore the workings of HE models and validate their model parameters and outcomes.</p></div><div><h3>Methods</h3><p>The Probabilistic Analysis Check dashBOARD (PACBOARD) was developed using insights from literature, health economists, and a data scientist.</p><p>Functionalities of PACBOARD are (1) exploring and validating model parameters and outcomes using standardized validation tests and interactive plots, (2) visualizing and investigating the relationship between model parameters and outcomes using metamodeling, and (3) predicting HE outcomes using the fitted metamodel.</p><p>To test PACBOARD, 2 mock HE models were developed, and errors were introduced in these models, eg, negative costs inputs, utility values exceeding 1. PACBOARD metamodeling predictions of incremental net monetary benefit were validated against the original model’s outcomes.</p></div><div><h3>Results</h3><p>PACBOARD automatically identified all errors introduced in the erroneous HE models. Metamodel predictions were accurate compared with the original model outcomes.</p></div><div><h3>Conclusions</h3><p>PACBOARD is a unique dashboard aiming at improving the feasibility and transparency of validation efforts of HE models. PACBOARD allows users to explore the working of HE models using metamodeling based on HE models’ parameters and outcomes.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023404/pdfft?md5=41158b0742362657a09711b19c7d14aa&pid=1-s2.0-S1098301524023404-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140770494","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"First-Line Treatment with Atezolizumab plus Bevacizumab and Chemotherapy for US Patients with Metastatic, Persistent, or Recurrent Cervical Cancer: A Cost-Effectiveness Analysis.","authors":"Jianying Lei, Jiahao Zhang, Caicong You, Maobai Liu, Na Li","doi":"10.1016/j.jval.2024.07.013","DOIUrl":"https://doi.org/10.1016/j.jval.2024.07.013","url":null,"abstract":"<p><strong>Objective: </strong>The BETAcc clinical trial demonstrated that chemotherapy combined with bevacizumab plus atezolizumab (CBA) significantly prolonged progression-free survival (PFS) and overall survival (OS) in patients with metastatic, persistent, or recurrent cervical cancer. However, to our knowledge, the economic value of using this new therapy for this indication is currently unknown. Therefore, our study aims to evaluate the cost-effectiveness of CBA for the first-line treatment of metastatic, persistent, or recurrent cervical cancer from the United States healthcare payers perspective.</p><p><strong>Methods: </strong>A state-transition Markov model over a 10-year lifetime horizon was developed to compare the cost and effectiveness of CBA versus chemotherapy plus bevacizumab (CB). The primary outcomes of our study included costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the results.</p><p><strong>Results: </strong>CBA was associated with an additional 0.58 QALY at an extra cost of $172,495.90 compared to CB. The ICER was $295,972.43/QALY, significantly higher than the willingness-to-pay (WTP) threshold value of $150,000/QALY. One-way sensitivity analyses revealed that results were most sensitive to the PFD utility, the unit cost of atezolizumab, and PD utility. Probabilistic sensitivity analysis indicated that CBA achieved a 4.3% probability of cost-effectiveness at a $150,000/QALY threshold. To achieve cost-effectiveness, the unit price of atezolizumab must be reduced by approximately 56.6%.</p><p><strong>Conclusions: </strong>CBA treatment is unlikely to be a cost-effective option compared with CB for patients with persistent, recurrent, or metastatic cervical cancer in the United States.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141890237","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment Effect Waning Assumptions: A Review of National Institute of Health and Care Excellence Technology Appraisals","authors":"","doi":"10.1016/j.jval.2024.04.016","DOIUrl":"10.1016/j.jval.2024.04.016","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aims to review the National Institute of Health and Care Excellence (NICE) technology assessments to gain insights into the implementation of treatment effect (TE) waning, whereby the hazard or survival in an assessed technology converges to that of the comparator. This analysis aims to contribute to inform future guidance in this area.</p></div><div><h3>Methods</h3><p>Technology appraisals published October 20, 2021 to September 20, 2023 were reviewed and data extracted on TE waning circumstances, methods, and rationale to compile a database based on 3 research questions: When are TE waning assumptions used? What methods are used? Why have the company/Evidence Assessment Group/committee preferred these methods?</p></div><div><h3>Results</h3><p>Both the evidence assessment group/company and the committee included TE waning assumptions in 28 appraisals. There was no pattern of waning assumptions between shorter (&lt;20 years) and longer (&gt;20 years) time horizons. The most prominent time point for applying waning assumptions was at 5 years, with 30 out of 59 (50.8%) of the methods applied used 5 years. Stopping rules were used in 21 out of 30 (70.1%) of the appraisals for which the committee included waning, and waning assumptions were used more in oncology. The most common reason given for including TE waning assumptions was precedent from prior appraisals.</p></div><div><h3>Conclusions</h3><p>Considerable heterogeneity existed in both the methods used and justifications given for TE waning assumptions. This variability poses a risk of inconsistent decision making. Reliance on past appraisals emphasizes the necessity to advocate for evidence-driven approaches and underscores the demand for guidance on suitable methods for incorporating assumptions.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023489/pdfft?md5=a6d63f5c33c911d59fb82bd0ce9943e0&pid=1-s2.0-S1098301524023489-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140870012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Systematic Review and Quality Assessment of Cardiovascular Disease-Specific Health-Related Quality-of-Life Instruments Part I: Instrument Development and Content Validity","authors":"","doi":"10.1016/j.jval.2024.04.001","DOIUrl":"10.1016/j.jval.2024.04.001","url":null,"abstract":"<div><h3>Objectives</h3><p>Health-related quality-of-life (HRQoL) instruments for cardiovascular diseases (CVD) have been commonly used to measure important patient-reported outcomes (PROs) in clinical trials and practices. This study aimed at systematically identifying and assessing the content validity of CVD-specific HRQoL instruments in clinical studies.</p></div><div><h3>Methods</h3><p>The research team searched Cumulative Index to Nursing and Allied Health Literature, Embase, and PubMed from inception to January 20, 2022. The research team included studies that reported the development and content validity for CVD-specific instruments. Two reviewers independently assessed the methodological quality using the Consensus-based Standards for the Selection of Health Measurement Instruments methods on evaluating content validity of PROs. Content analysis was used to categorize the items included in the instruments.</p></div><div><h3>Results</h3><p>The research team found 69 studies reporting the content validity of 40 instruments specifically developed for CVD. Fourteen (35.0%) were rated “sufficient” with very low to moderate quality of evidence. For PRO development, all instruments were rated “doubtful” or “inadequate.” Twenty-eight (70.0%) instruments cover the core concepts of HRQoL.</p></div><div><h3>Conclusions</h3><p>The quality of development and content validity vary among existing CVD-specific instruments. The evidence on the content validity should be considered when choosing a HRQoL instrument in CVD clinical studies and health economic evaluations.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023313/pdfft?md5=a377122805b62a03b62264f4d9c7d66b&pid=1-s2.0-S1098301524023313-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140761058","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Utilization, Expenditure, and Treatment Patterns Associated With Calcitonin Gene-Related Peptide Monoclonal Antibodies Reimbursed Subject to a Managed Access Protocol in Ireland","authors":"","doi":"10.1016/j.jval.2024.04.002","DOIUrl":"10.1016/j.jval.2024.04.002","url":null,"abstract":"<div><h3>Objectives</h3><p>Calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) are novel high-cost treatments for the prevention of migraine. This study presents data on utilization, expenditure, and treatment patterns with CGRP mAbs available under a managed access protocol in Ireland, to a cohort of treatment refractory patients (failed 3 or more previous treatments) with chronic migraine.</p></div><div><h3>Methods</h3><p>Data were extracted from the Primary Care Reimbursement Service High Tech claims database and special drug request online system and analyzed using Microsoft Excel and SAS. Treatment persistence was evaluated by refill patterns, and adherence was evaluated using the proportion of days covered method. Expenditure data were extracted directly from the database.</p></div><div><h3>Results</h3><p>Between September 1, 2021 and April 30, 2023, 1517 applications for reimbursement approval for a CGRP mAb were received; 1458 (96.1%) were approved for reimbursement. Total expenditure on CGRP mAbs in year 1 (September 1, 2021 to August 31, 2022) was €3.2 million. The majority of patients initiated treatment with fremanezumab (60.8%) or erenumab (37.1%). Almost 90% of patients were considered adherent, and treatment persistence was high, with more than 75% of patients receiving more than 12 months of treatment in our 18-month study time frame.</p></div><div><h3>Conclusions</h3><p>This study demonstrates the importance of active health technology management, after reimbursement, in enabling cost-effective use of high-cost treatments while providing budget certainty for the healthcare payer. High levels of adherence and persistence suggest that treatment is successfully targeted in situations which unmet clinical need is greatest.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023337/pdfft?md5=a49d055686427376412ab3fa298eb24b&pid=1-s2.0-S1098301524023337-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140755727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Illustrating the Financial Consequences of Outcome-Based Payment Models From a Payers Perspective: The Case of Autologous Gene Therapy Atidarsagene Autotemcel (Libmeldy®)","authors":"","doi":"10.1016/j.jval.2024.05.010","DOIUrl":"10.1016/j.jval.2024.05.010","url":null,"abstract":"<div><h3>Objectives</h3><p>To illustrate the financial consequences of implementing different managed entry agreements (managed entry agreements for the Dutch healthcare system for autologous gene therapy atidarsagene autotemcel [Libmeldy]), while also providing a first systematic guidance on how to construct managed entry agreements to aid future reimbursement decision making and create patient access to high-cost, one-off potentially curative therapies.</p></div><div><h3>Methods</h3><p>Three payment models were compared: (1) an arbitrary 60% price discount, (2) an outcome-based spread payment with discounts, and (3) an outcome-based spread payment linked to a willingness to pay model with discounts. Financial consequences were estimated for full responders (A), patients responding according to the predicted clinical pathway presented in health technology assessment reports (B), and unstable responders (C). The associated costs for an average patient during the time frame of the payment agreement, the total budget impact, and associated benefits expressed in quality-adjusted life-years of the patient population were calculated.</p></div><div><h3>Results</h3><p>When patients responded according to the predicted clinical pathway presented in health technology assessment reports (scenario B), implementing outcome-based reimbursement models (models 2 and 3) had lower associated budget impacts while gaining similar benefits compared with the discount (scenario 1, €8.9 million to €6.6 million vs €9.2 million). In the case of unstable responders (scenario C), costs for payers are lower in the outcome-based scenarios (€4.1 million and €3.0 million, scenario 2C and 3C, respectively) compared with implementing the discount (€9.2 million, scenario 1C).</p></div><div><h3>Conclusions</h3><p>Outcome-based models can mitigate the financial risk of reimbursing atidarsagene autotemcel. This can be considerably beneficial over simple discounts when clinical performance was similar to or worse than predicted.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023702/pdfft?md5=9c5483bcff35ffc881043b9014cebd32&pid=1-s2.0-S1098301524023702-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141136573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EQ-5D-5L Population Norms for China Derived From a National Health Survey","authors":"","doi":"10.1016/j.jval.2024.04.014","DOIUrl":"10.1016/j.jval.2024.04.014","url":null,"abstract":"<div><h3>Objectives</h3><p>To develop the EQ-5D-5L (5L) population norms for China and to assess the relationship between various factors and 5L data.</p></div><div><h3>Methods</h3><p>This study used data derived from the Psychology and Behavior Investigation of Chinese Residents, a national sample survey of 21 909 representative participants aged 12 years and above. Participants’ health-related quality of life<span> (HRQoL) was measured by the 5L. Their socioeconomic characteristics, behavioral factors, and health conditions were also obtained from the survey. Norm scores were generated and compared for different socioeconomic variables. Multiple linear and logistic regressions were used to assess the relationships of the 3 kinds of variables with the 5L utility, visual analog scale (VAS) scores and 5L health problems.</span></p></div><div><h3>Results</h3><p>The mean (SD) age of participants was 39.4 (18.9) years, and 50.0% of them were female. The mean (SD) utility and VAS scores were 0.940 (0.138) and 73.4 (21.6), respectively. Participants reported considerably more problems in anxiety/depression (26.2%) and pain/discomfort (22.2%) dimensions. The gender difference in HRQoL is attenuated. The participants older than 75 years suffered from a sharp decline in HRQoL; the participants in Shanghai and Tibet provinces reported lower utility and VAS scores and more health problems. Those who were younger, with better socioeconomic status and healthier lifestyles, and without diseases tended to report higher utility and VAS scores and fewer health problems.</p></div><div><h3>Conclusions</h3><p>This study derived the 5L population norms for China based on a representative population sample.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140858178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Navigating Public Policy Responses to a Pandemic: The Balancing Act Between Physical Health, Mental Health, and Household Income","authors":"","doi":"10.1016/j.jval.2024.04.019","DOIUrl":"10.1016/j.jval.2024.04.019","url":null,"abstract":"<div><h3>Objective</h3><p>During COVID-19, governments imposed restrictions that reduced pandemic-related health risks but likely increased personal and societal mental health risk, partly through reductions in household income. This study aimed to quantify the public’s willingness to accept trade-offs between pandemic health risks, household income reduction, and increased risk of mental illness that may result from future pandemic-related policies.</p></div><div><h3>Methods</h3><p>A total of 547 adults from an online panel participated in a discrete choice experiment where they were asked to choose between hypothetical future pandemic scenarios. Each scenario was characterized by personal and societal risks of dying from the pandemic, experiencing long-term complications, developing anxiety/depression, and reductions in household income. A latent class regression was used to estimate trade-offs.</p></div><div><h3>Results</h3><p>Respondents state a willingness to make trade-offs across these attributes if the benefits are large enough. They are willing to accept 0.8% lower household income (0.7-1.0), 2.7% higher personal risk of anxiety/depression (1.8-3.6), or 3.2% higher societal rate of anxiety/depression (1.7-4.7) in exchange for 300 fewer deaths from the pandemic.</p></div><div><h3>Conclusion</h3><p>Results reveal that individuals are willing to accept lower household income and higher rates of mental illness, both personal and societal, if the physical health benefits are large enough. Respondents placed greater emphasis on maintaining personal, as opposed to societal, mental health risk and were most interested in preventing pandemic-related deaths. Governments should consider less restrictive policies when pandemics have high morbidity but low mortality to avoid the prospect of improving physical health while simultaneously reducing net social welfare.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140890587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Can Generalized Cost-Effectiveness Analysis Leverage Meaningful Use of Novel Value Elements in Pharmacoeconomics to Inform Medicare Drug Price Negotiation?","authors":"","doi":"10.1016/j.jval.2024.04.013","DOIUrl":"10.1016/j.jval.2024.04.013","url":null,"abstract":"<div><h3>Objectives</h3><p>Decision makers considering using cost-effectiveness analysis (CEA) to inform health-technology assessment must contend with documented and controversial shortfalls of CEA, including its assumption of disease severity independence and static pricing. ISPOR has recently introduced novel value elements besides direct healthcare cost and effectiveness for the patient, and these should be captured in CEA. Although novel value elements advance our understanding of “what” should be measured (value of hope, severity of disease, health equity, etc), there is limited direction on “how” to measure them in conventional CEA. Furthermore, with Medicare empowered to set drug prices under the Inflation Reduction Act, it is not clear what role CEA might have on where prices are set, given objections to the quality-adjusted life year in conventional approaches.</p></div><div><h3>Methods</h3><p>We critically reviewed the evidence for expanding conventional CEA methods to a more generalized approach of generalized CEA (GCEA).</p></div><div><h3>Results</h3><p>GCEA accounts for methods that address objections to the quality-adjusted life year and incorporate novel value elements. Although GCEA offers advantages, it also requires further research to develop “off-the-shelf” resources to help inform, for example, maximum fair price in the context of Medicare drug price negotiation.</p></div><div><h3>Conclusions</h3><p>Should a shift toward GCEA reveal that the societal value of novel medicines exceeds their market-based costs, which will raise the key question of what market failure Medicare negotiation is meant to solve, if any, and therefore what the appropriate role of such negotiation might be to maximize the value society might garner from the development of novel medicines.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023453/pdfft?md5=9797369e21b7bfd81f6b1126047d8b58&pid=1-s2.0-S1098301524023453-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140858096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Implementing Multilevel Network Meta-Regression for Time-To-Event Outcomes: A Case Study in Relapsed Refractory Multiple Myeloma","authors":"","doi":"10.1016/j.jval.2024.04.017","DOIUrl":"10.1016/j.jval.2024.04.017","url":null,"abstract":"<div><h3>Objectives</h3><p>Multilevel network meta-regression (ML-NMR) leverages individual patient data (IPD) and aggregate data from a network of randomized controlled trials<span> (RCTs) to assess the comparative efficacy of multiple treatments, while adjusting for between-study differences. We provide an overview of ML-NMR for time-to-event outcomes and apply it to an illustrative case study, including example R code.</span></p></div><div><h3>Methods</h3><p><span>The case study evaluated the comparative efficacy of idecabtagene vicleucel (ide-cel), selinexor+dexamethasone (Sd), belantamab mafodotin (BM), and conventional care (CC) for patients with triple-class exposed relapsed/refractory </span>multiple myeloma<span> in terms of overall survival<span>. Single-arm clinical trials and real-world data were naively combined to create an aggregate data artificial RCT (aRCT) (MAMMOTH-CC versus DREAMM-2-BM versus STORM-2-Sd) and an IPD aRCT (KarMMa-ide-cel versus KarMMa-RW-CC). With some assumptions, we incorporated continuous covariates with skewed distributions, reported as median and range. The ML-NMR models adjusted for number of prior lines, triple-class refractory status, and age and were compared using the leave-one-out information criterion. We summarized predicted hazard ratios and survival (95% credible intervals) in the IPD aRCT population.</span></span></p></div><div><h3>Results</h3><p>The Weibull ML-NMR model had the lowest leave-one-out information criterion. Ide-cel was more efficacious than Sd, BM, and CC in terms of overall survival. Effect modifiers had minimal impact on the model, and only triple-class refractory was a prognostic factor.</p></div><div><h3>Conclusions</h3><p>We demonstrate an application of ML-NMR for time-to-event outcomes and introduce code that can be used to aid implementation. Given its benefits, we encourage practitioners to utilize ML-NMR when population adjustment is necessary for comparisons of multiple treatments.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140861983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}