PharmacoEconomics最新文献

{"title":"Public Health Impact of Introducing a Pentavalent Vaccine Against Invasive Meningococcal Disease in the United States.","authors":"Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Katherine A Hicks, Justin Carrico, Mei Grace, Diana E Clements, Cindy Burman, Woo-Yun Sohn, Elise Kuylen, Shahina Begum, Zeki Kocaata","doi":"10.1007/s40273-024-01439-y","DOIUrl":"https://doi.org/10.1007/s40273-024-01439-y","url":null,"abstract":"<p><strong>Background: </strong>Invasive meningococcal disease (IMD) is primarily associated with five Neisseria meningitidis serogroups: A, B, C, W, or Y. In the United States (US), available vaccines protect against serogroups B (MenB), A, C, W, and Y (MenACWY), and A, B, C, W, and Y (MenABCWY). The Advisory Committee on Immunization Practices is re-evaluating the adolescent meningococcal vaccination schedule with varying recommendation formats. This analysis aimed to predict which schedule could avert the most IMD cases and have the most positive public health impact (PHI).</p><p><strong>Methods: </strong>An epidemiological model compared the 15-year PHI of vaccination schedules using MenB, MenACWY, and/or MenABCWY vaccines versus current US standard of care (SoC). Varying coverage rates reflected routine, shared clinical decision making, and risk-based recommendations. Sensitivity analyses assessed robustness of the results to different inputs/assumptions.</p><p><strong>Results: </strong>The most positive PHI compared with SoC was observed with one dose of MenACWY at 11 years of age and two doses of MenABCWY (6 months apart) at 16 years of age, assuming routine recommendation and coverage reflecting real-world uptake of MenACWY. This strategy resulted in 123 IMD cases averted (MenB: 59, MenACWY: 64), 17 deaths prevented, 574 life-years saved, and 757 quality-adjusted life-years gained versus SoC. Eliminating MenACWY vaccination at 11 years was found to result in an additional IMD burden.</p><p><strong>Conclusion: </strong>A routinely recommended two-dose pentavalent vaccine, with doses administered 6 months apart at 16 years of age, alongside the routinely recommended MenACWY vaccine at 11 years of age, would improve the PHI and benefits of IMD vaccination to society.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710883","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}

{"title":"A Multistate Model Incorporating Relative Survival Extrapolation and Mixed Time Scales for Health Technology Assessment.","authors":"Enoch Yi-Tung Chen, Paul W Dickman, Mark S Clements","doi":"10.1007/s40273-024-01457-w","DOIUrl":"https://doi.org/10.1007/s40273-024-01457-w","url":null,"abstract":"<p><strong>Background: </strong>Multistate models have been widely applied in health technology assessment. However, extrapolating survival in a multistate model setting presents challenges in terms of precision and bias. In this article, we develop an individual-level continuous-time multistate model that integrates relative survival extrapolation and mixed time scales.</p><p><strong>Methods: </strong>We illustrate our proposed model using an illness-death model. We model the transition rates using flexible parametric models. We update the hesim package and the microsimulation package in R to simulate event times from models with mixed time scales. This feature allows us to incorporate relative survival extrapolation in a multistate setting. We compare several multistate settings with different parametric models (standard vs. flexible parametric models), and survival frameworks (all-cause vs. relative survival framework) using a previous clinical trial as an illustrative example.</p><p><strong>Results: </strong>Our proposed approach allows relative survival extrapolation to be carried out in a multistate model. In the example case study, the results agreed better with the observed data than did the commonly applied approach using standard parametric models within an all-cause survival framework.</p><p><strong>Conclusions: </strong>We introduce a multistate model that uses flexible parametric models and integrates relative survival extrapolation with mixed time scales. It provides an alternative to combine short-term trial data with long-term external data within a multistate model context in health technology assessment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142716834","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}

{"title":"Economic Evaluations of Non-Pharmacological Interventions for Treating Disorders of Gut-Brain Interaction: A Scoping Review.","authors":"Anton Pak, Madeline O'Grady, Gerald Holtmann, Ayesha Shah, Haitham Tuffaha","doi":"10.1007/s40273-024-01455-y","DOIUrl":"https://doi.org/10.1007/s40273-024-01455-y","url":null,"abstract":"<p><strong>Background and objectives: </strong>Disorders of gut-brain interaction are highly prevalent and burdensome conditions for both patients and healthcare systems. Given the limited effectiveness of pharmacotherapy in treating disorders of gut-brain interaction, non-pharmacological interventions are increasingly used; however, the value for money of non-pharmacological treatments is uncertain. This is the first review to assess the economic evaluation evidence of non-pharmacological interventions for disorders of gut-brain interaction.</p><p><strong>Methods: </strong>A scoping review was conducted in line with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews (PRISMA-ScR) guidelines. Reporting adhered to ISPOR's good practices for systematic reviews with cost and cost-effectiveness outcomes. Comprehensive searches were performed on 24 October, 2023, and an updated search was run on 18 May, 2024 in PubMed/MEDLINE, Embase, Web of Science, Scopus and the International HTA database, with two reviewers screening studies in parallel. The novel Criteria for Health Economic Quality Evaluation (CHEQUE) framework was used to assess methodological and reporting quality. Reporting quality was further assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022.</p><p><strong>Results: </strong>Fifteen studies were included. Most studies examined treatments for irritable bowel syndrome. Cognitive behavioural therapy, dietary interventions and sacral neuromodulation were cost effective. Acupuncture and physiotherapy were not. CHEQUE assessment showed 12 studies met at least 70% of the methodological criteria, and 14 studies achieved 70% or more for reporting quality.</p><p><strong>Conclusions: </strong>This review highlights gaps in the current evidence base, particularly in the robustness and generalisability of results due to methodological inconsistencies. Future research should incorporate longer follow-ups, comprehensive cost assessments, subgroup analyses, equity considerations and clearer justifications for modelling assumptions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142681671","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}

{"title":"Unravelling the Association Between Uncertainties in Model-based Economic Analysis and Funding Recommendations of Medicines in Australia.","authors":"Qunfei Chen, Martin Hoyle, Varinder Jeet, Yuanyuan Gu, Kompal Sinha, Bonny Parkinson","doi":"10.1007/s40273-024-01446-z","DOIUrl":"https://doi.org/10.1007/s40273-024-01446-z","url":null,"abstract":"<p><strong>Objective: </strong>Health technology assessment is used extensively by the Pharmaceutical Benefits Advisory Committee (PBAC) to inform medicine funding recommendations in Australia. The PBAC often does not recommend medicines due to uncertainties in economic modelling that result in delaying access to medicines for patients. The systematic identification of which uncertainties can be reduced with alternative evidence or the collection of additional data can help inform recommendations. This study aims to characterise different types of uncertainty in economic models and empirically assess their association with the PBAC recommendations.</p><p><strong>Methods: </strong>A framework was developed to characterise four types of uncertainties: methodological, structural, generalisability and parameter uncertainty. The first two types were further subcategorised into parameterisable and unparameterisable uncertainty. Data on uncertainty and other factors were extracted from PBAC's Public Summary Documents of first submissions for 193 medicine (vaccine)-indication pairs including economic modelling between 2014 and 2021. Logistic regression was used to estimate the average marginal effect of each type of uncertainty on the probability of a positive recommendation.</p><p><strong>Results: </strong>The PBAC more often raised issues regarding parameter uncertainty (95%) and parameterisable structural uncertainty (83%) than generalisability uncertainty (48%) and unparameterisable methodological uncertainty (56%). The logistic regression results suggested that the PBAC was more likely to recommend a medicine without unparameterisable methodological, generalisability, and parameterisable structural uncertainty by 15.0%, 10.2 %, and 17.6%, respectively. Parameterisable methodological, unparameterisable structural and parameter uncertainty were not significantly associated with the PBAC recommendations.</p><p><strong>Conclusions: </strong>This study identified the uncertainties that had significant associations with PBAC recommendations based on the first submission. This may help improve model quality and reduce resubmissions in the future, thus improving patients' access to medicines.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638770","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}

{"title":"Cost Effectiveness of Tremelimumab Plus Durvalumab for Unresectable Hepatocellular Carcinoma in the USA.","authors":"Xiaomo Xiong, Jeff Jianfei Guo","doi":"10.1007/s40273-024-01453-0","DOIUrl":"https://doi.org/10.1007/s40273-024-01453-0","url":null,"abstract":"<p><strong>Background: </strong>Treating unresectable hepatocellular carcinoma (uHCC) is challenging. Clinical trials have shown that Single Tremelimumab Regular Interval Durvalumab (STRIDE) offers clinical benefits as a first-line treatment for uHCC, but its cost effectiveness remains unknown in the USA.</p><p><strong>Objective: </strong>We aimed to assess the cost effectiveness of STRIDE (tremelimumab plus durvalumab) versus sorafenib and durvalumab monotherapy as the first-line treatment for uHCC in the USA.</p><p><strong>Methods: </strong>A partitioned survival model was constructed to assess the cost effectiveness of STRIDE compared to sorafenib and durvalumab monotherapy as the first-line treatment for uHCC from the US societal perspective. The time horizon was 48 months with 1-month cycles. Seven parametric survival functions replicated survival curves from clinical trials, with the best-fitting model used to calculate survival probabilities. Costs, health utilities, and adverse events were included, with quality-adjusted life-years (QALYs) as the primary effectiveness measure. Both costs and effectiveness were discounted at 3%. In the base-case analysis, the incremental cost-effectiveness ratio was compared to a willingness-to-pay threshold of $150,000 per QALY gained. Deterministic and probabilistic sensitivity analyses were conducted to examine the uncertainty of the model.</p><p><strong>Results: </strong>In the base-case analysis, STRIDE was cost effective compared to sorafenib, with an incremental cost-effectiveness ratio of $97,995.51 per QALY gained, based on a willingness-to-pay threshold of $150,000 per QALY gained. However, STRIDE was not cost effective compared to durvalumab monotherapy at the same threshold, with an incremental cost-effectiveness ratio of $754,408.92 per QALY gained. Deterministic sensitivity analyses were consistent with the base-case analysis. A probabilistic sensitivity analysis indicated that STRIDE was more likely to be cost effective than sorafenib and durvalumab monotherapy when the willingness-to-pay exceeded $101,000 and $713,000, respectively.</p><p><strong>Conclusions: </strong>The STRIDE regimen appears to be cost effective compared to sorafenib but not compared to durvalumab for first-line uHCC treatment in the USA. However, durvalumab has not yet been approved for uHCC in the USA. Future research should focus on long-term data and economic evaluations of other recommended biologics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638768","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}

{"title":"Comparison of EQ-5D-Y-3L Utility Scores Using Nine Country-Specific Value Sets in Chinese Adolescents.","authors":"Ya'nan Wu, Yanjiao Xu, Zhao Shi, Junchao Feng, Zhihao Yang, Zhuxin Mao, Lei Dou, Shunping Li","doi":"10.1007/s40273-024-01451-2","DOIUrl":"https://doi.org/10.1007/s40273-024-01451-2","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to assess and compare the measurement properties of EQ-5D-Y-3L utilities derived from available countries' value sets (Chinese, Japanese, Slovenian, German, Spanish, Hungarian, Netherlandish, Belgian, and Indonesian), among Chinese adolescents.</p><p><strong>Methods: </strong>From July to September 2021, a large-scale cross-sectional survey was administered across 16 cities in Shandong, China, with the objective of assessing the health status of junior high school students aged 10-18 years. Supported by the educational authorities, quick response (QR) codes and questionnaire links were disseminated to schools. A total of 97,413 junior high school students completed the questionnaire. Agreement, convergent validity, and known-group validity were determined in the nine country-specific value sets.</p><p><strong>Results: </strong>The Indonesian value set demonstrated the highest mean health utility score (0.970), followed by the Japanese (0.961), Chinese (0.960), Netherlandish (0.948), Hungarian (0.942), German (0.938), Belgian (0.932), Slovenian (0.926), and Spanish (0.926) value sets, respectively. The utility scores derived from Asian value sets were higher than those from Europe. Good or excellent agreements (intraclass correlation coefficients > 0.7) were found between each paired value set. In Bland-Altman plots, the 95% limits of agreement for any two value sets were 0.046-0.348. A strong relationship (Spearman's correlation coefficients > 0.99) between any two value sets was found. The EQ-5D-Y-3L utility scores discriminated equally well for the nine value sets across three known groups. The effect size and the relative efficiency statistics showed the Chinese value sets were more sensitive in general. Referring to the Chinese value set, all the relative efficiency values in each value set were similar across three known groups, ranging from 0.9 to 1.0.</p><p><strong>Conclusions: </strong>A total of nine country-specific EQ-5D-Y-3L value sets showed an overall high level of agreement, strong correlation, and good known-group validity. However, the utility scores derived from nine EQ-5D-Y-3L value sets were different and the country-specific value sets were not interchangeable.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625477","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}

{"title":"Value-Based Healthcare in Practice: IDEATE, a Collaboration to Design and Test an Outcomes-Based Agreement for a Medicine in Wales.","authors":"Jessica R Burton, Kate Halsby, Graciela Sáinz de la Fuente, Jonathan Pearson-Stuttard, Rebecca Sloan, Thomas Porter, Gareth John, Andrew Warburton, Jennifer Selby, Gail Povey, Ruhe Chowdhury, Catherine Bale, Mark Davies, Emma Clifton-Brown, Hamish Laing","doi":"10.1007/s40273-024-01445-0","DOIUrl":"https://doi.org/10.1007/s40273-024-01445-0","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To develop a sustainable, scalable methodology for the design of outcome-based agreements (OBAs) that works on the ground and dynamically overcomes historical challenges.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;Project IDEATE co-created solutions to known (and emergent) challenges via iterative workshops and real-world data analysis to develop and refine a hypothetical model for an OBA in a trusted research environment. A cross-disciplinary collaboration between National Health Service (NHS) Wales, industry and academia was developed. Data were collected from Welsh national datasets and used to construct a novel linked dataset. OBA scenarios, with different contract parameters, were analysed to assess impact on the proportion of contract payment due and the volatility of payments.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;An approved, in market, locally advanced and metastatic breast cancer treatment was selected as the test case. The total number of patients in the treatment cohort (2017-2020) was n = 99, and 286 in the control cohort (2014-2016). The final outcome variables selected were: (1) 1-year survival,( 2) intolerance to treatment (deferral), and (3) the total days disrupted by care. The primary scenario included all three outcomes measured at the population level and used a linear payment model. Volatility analyses demonstrated contract parameters can dramatically alter the contract output with greatest risk from a single, binary outcome contract design.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;The design of an OBA is a complex process that requires a multi-disciplinary approach. By assessing solutions to data, outcomes and contracting challenges, IDEATE provides a strong foundation for future success of OBAs in the UK. Outcome-based agreements (OBAs) are a way to pay for medicines if they help patient health in a specific way over time. These agreements can make it faster for people to get new medicines, but they also have challenges, like needing a lot of time and effort to manage them. A team from the NHS Wales, life sciences, and Swansea University created Project IDEATE to find a better way to design OBAs and solve some of these problems. Welsh datasets were used to create a new breast cancer dataset to test different OBAs and see how payments would change. A breast cancer treatment was used for the project. The project had 99 patients who got the medicine (2017-2020) and 286 patients who had breast cancer but did not get the medicine (2014-2016). Three health outcomes were measured: (1) living for one year after treatment, (2) patients needing to stop the medicine, and (3) days spent in care. The main OBA option we tested used all three health outcomes; the more the outcomes improved, the more the payments could go up until they hit the highest amount agreed. The analysis showed that the way an OBA is designed can make a big difference in how stable or risky it is, especially if one of the health outcomes has only two opti","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625499","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}

{"title":"The Use of Real-World Data for Estimating Relative Treatment Effects in NICE Health Technology Assessment Submissions: A Review.","authors":"Yoojung Che, Stephen Duffield, Manuel Gomes","doi":"10.1007/s40273-024-01449-w","DOIUrl":"https://doi.org/10.1007/s40273-024-01449-w","url":null,"abstract":"<p><p>This paper investigates the current use of real-world data (RWD) for estimating relative treatment effects in National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) submissions. This review included 64 HTA submissions, which accounted for approximately 11% of the total NICE HTA submissions between January 2016 and December 2023. The main sources of RWD considered in the submissions were disease registries and electronic health records. RWD were primarily used to provide an external control arm to enable comparisons within single-arm trials and to inform long-term treatment effects when extrapolating survival data beyond the trial follow-up. Adjustments for potential systematic differences between treatment groups have improved over time; however, approximately one-third of the submissions still relied on unadjusted treatment comparisons. We found that approximately 10% of NICE HTA submissions used RWD to directly inform treatment effects estimation. Over one-third of the submissions relied on naïve and/or unadjusted treatment comparisons, which may have introduced biases. To ensure that RWD provide sound evidence for HTA, submissions should follow published guidelines, including the NICE real-world evidence (RWE) framework.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625480","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}

{"title":"Recurrence of Cardiovascular Events After an Acute Myocardial Infarction in Patients with Multivessel Disease and Associated Healthcare Costs: A German Claims Data Analysis.","authors":"Alexandra Starry, Nils Picker, Jonathan Galduf, Ulf Maywald, Axel Dittmar, Stefan G Spitzer","doi":"10.1007/s40273-024-01440-5","DOIUrl":"https://doi.org/10.1007/s40273-024-01440-5","url":null,"abstract":"<p><strong>Aim: </strong>This study sought to quantify the healthcare costs of multivessel disease (MVD) and determine the prevalence and incidence of recurrent major adverse cardiovascular events (MACE) in high-risk patients diagnosed with MVD following an acute myocardial infarction (MI).</p><p><strong>Methods: </strong>This retrospective study utilized German claims data (AOK PLUS), between 01/01/2010 and 31/12/2020. Patients were included if they (1) had an inpatient diagnosis of an MI between 01/01/2012 and 31/12/2019 (index date), (2) were ≥ 18 years of age at date of MI diagnosis, and (3) had diabetes or met two of the following criteria: ≥ 65 years old, prior MI, peripheral arterial disease. MACE was defined as (1) MI, (2) stroke, or (3) death with a cardiovascular diagnosis within 30 days prior. To measure the burden of MVD, patients were identified during the index hospitalization by presence of MVD. Healthcare resource use and costs were compared after adjustment based on propensity score matching (PSM).</p><p><strong>Results: </strong>A total of 5158 patients with evidence for MVD were included in the main analysis. 31.17% experienced a MACE within 365 days following the incident MI. After PSM adjustment, 33.22% of the MVD cohort experienced a MACE versus 36.48% of non-MVD patients. MVD patients had a higher rate of recurrent MI (14.22% vs. 9.81%). Additionally, public healthcare costs were about €4 million higher in the total MVD cohort than in the non-MVD cohort in the first year after an MI (€47,896,012.32 vs. €43,718,713.75, respectively), reflecting the MVD cohort's higher use of the public healthcare system. More MVD patients were prescribed guideline-recommended medication (61.4% vs. 46.0%).</p><p><strong>Conclusion: </strong>This study found that presence of MVD contributed to higher rates of recurrent MI. Patients with MVD experienced higher rates of recurrent MI despite a higher proportion of patients receiving guideline-directed medication therapy compared to non-MVD patients. Conversely, there was a higher mortality rate observed in the non-MVD cohort.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142575779","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}

{"title":"A Systematic Literature Review of Modelling Approaches to Evaluate the Cost Effectiveness of PET/CT for Therapy Response Monitoring in Oncology.","authors":"Sietse van Mossel, Rafael Emilio de Feria Cardet, Lioe-Fee de Geus-Oei, Dennis Vriens, Hendrik Koffijberg, Sopany Saing","doi":"10.1007/s40273-024-01447-y","DOIUrl":"https://doi.org/10.1007/s40273-024-01447-y","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objective: &lt;/strong&gt;This systematic literature review addresses model-based cost-effectiveness studies for therapy response monitoring with positron emission tomography (PET) generally combined with low-dose computed tomography (CT) for various cancer types. Given the known heterogeneity in therapy response events, studies should consider patient-level modelling rather than cohort-based modelling because of its flexibility in handling these events and the time to events. This review aims to identify the modelling methods used and includes a systematic assessment of the assumptions made in the current literature.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This study was conducted and reported following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 statement. Information sources included electronic bibliographic databases, reference lists of review articles and contact with experts in the fields of nuclear medicine, health technology assessment and health economics. Eligibility criteria included peer-reviewed scientific publications and published grey literature. Literature searches, screening and critical appraisal were conducted by two reviewers independently. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) were used to assess the methodological quality. The Bias in Economic Evaluation (ECOBIAS) checklist was used to determine the risk of bias in the included publications.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;The search results included 2959 publications. The number of publications included for data extraction and synthesis was ten, representing eight unique studies. These studies addressed patients with lymphoma, advanced head and neck cancers, brain tumours, non-small cell lung cancer and cervical cancer. All studies addressed response to chemotherapy. No study evaluated response to immunotherapy. Most studies positioned PET/CT as an add-on modality and one study positioned PET/CT as a replacement for conventional imaging (X-ray and contrast-enhanced CT). Three studies reported decision-tree structures, four studies reported cohort-level state-transition models and one study reported a partitioned survival model. No patient-level models were reported. The simulation horizons adopted ranged from 1 year to lifetime. Most studies reported a probabilistic analysis, whereas two studies reported a deterministic analysis only. Two studies conducted a value of information analysis. Multiple studies did not adequately discuss model-specific aspects of bias. Most importantly and regularly observed were a high risk of structural assumptions bias, limited simulation horizon bias and wrong model bias.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;Model-based cost-effectiveness analysis for therapy response monitoring with PET/CT was based on cohorts of patients instead of individual patients in the current literature. Therefore, the heterogeneity in therapy response events was commonly not addr","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2024-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564433","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}