PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-25DOI: 10.1007/s40273-024-01439-y
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
{"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":"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":"311-329"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825582/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-15DOI: 10.1007/s40273-024-01453-0
Xiaomo Xiong, Jeff Jianfei Guo
{"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":"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":"271-282"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","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}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-12-02DOI: 10.1007/s40273-024-01454-z
Jedidiah I Morton, Danny Liew, Gerald F Watts, Sophia Zoungas, Stephen J Nicholls, Christopher M Reid, Zanfina Ademi
{"title":"Immediate Versus 5-Year Risk-Guided Initiation of Treatment for Primary Prevention of Cardiovascular Disease for Australians Aged 40 Years: A Health Economic Analysis.","authors":"Jedidiah I Morton, Danny Liew, Gerald F Watts, Sophia Zoungas, Stephen J Nicholls, Christopher M Reid, Zanfina Ademi","doi":"10.1007/s40273-024-01454-z","DOIUrl":"10.1007/s40273-024-01454-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Current Australian cardiovascular disease (CVD) prevention guidelines calculate 5-year CVD risk and recommend treatment when risk crosses specific thresholds. This may leave risk factors untreated for people with a low short-term (i.e. 5 years), but high long-term (i.e. lifetime), risk of CVD. We aimed to evaluate the cost effectiveness of intervention for risk factor control at age 40 years (regardless of calculated risk) compared to intervention for risk factor control at the age recommended by contemporary Australian CVD prevention guidelines (when the 5-year CVD risk reaches 10%) across a range of individual risk factor profiles.</p><p><strong>Methods: </strong>We used a causal microsimulation model populated with 108 different risk factor profiles, each replicated 10,000 times. Model data were derived from the UK Biobank study and published sources. The primary causal relationships factored in were those of low-density lipoprotein-cholesterol and systolic blood pressure with CVD (defined as myocardial infarction or stroke). The model simulated the ageing of individuals from 40 to 85 years. We calculated years of life lived, quality-adjusted life-years gained, incremental healthcare costs and the incremental cost-effectiveness ratio when low-density lipoprotein-cholesterol and blood pressure were controlled from age 40 years compared to initiation of treatment as recommended by Australian guidelines. The main side effect in the model was an increased risk of type 2 diabetes mellitus from statin use. The trade-off between reduced CVD and increased type 2 diabetes was summarised via quality-adjust life-years. Incremental cost-effectiveness ratios were compared to the Australian willingness-to-pay threshold of AU$28,000 per quality-adjust life-year gained. We adopted a healthcare perspective (2022 AUD) and discounted results at 3% annually.</p><p><strong>Results: </strong>An earlier intervention meaningfully prevented CVD in all but the lowest risk individuals. Intervention at age 40 years versus age when the 5-year CVD risk reaches 10% led to an increase in quality-adjust life-years for 37/54 female individuals and 44/54 male individuals simulated and an increase in years of life lived (i.e. life expectancy) for 46/54 female individuals and 47/54 male individuals simulated. Earlier intervention was also cost effective in 5/54 female individuals and 17/54 male individuals.</p><p><strong>Conclusions: </strong>Current guidelines may result in certain individuals with a lower 5-year, but higher lifetime, risk of CVD being overlooked for earlier cost-effective interventions to prevent CVD.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"331-349"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771142","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}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-12-03DOI: 10.1007/s40273-024-01460-1
Sarah J Nevitt, David M Phillippo, Robert Hodgson, Nicky J Welton, Sofia Dias
{"title":"Application of Multi-level Network Meta-Regression in the NICE Technology Appraisal of Quizartinib for Induction, Consolidation and Maintenance Treatment of Newly Diagnosed FLT3-ITD-Positive Acute Myeloid Leukaemia: An External Assessment Group Perspective.","authors":"Sarah J Nevitt, David M Phillippo, Robert Hodgson, Nicky J Welton, Sofia Dias","doi":"10.1007/s40273-024-01460-1","DOIUrl":"10.1007/s40273-024-01460-1","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"243-247"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771140","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}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-25DOI: 10.1007/s40273-024-01457-w
Enoch Yi-Tung Chen, Paul W Dickman, Mark S Clements
{"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":"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":"297-310"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825556/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-02-01DOI: 10.1007/s40273-024-01461-0
Julie A Campbell, Glen J Henson, Valery Fuh Ngwa, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Andrew J Palmer
{"title":"Correction: Estimation of Transition Probabilities from a Large Cohort (> 6000) of Australians Living with Multiple Sclerosis (MS) for Changing Disability Severity Classifications, MS Phenotype, and Disease-Modifying Therapy Classifications.","authors":"Julie A Campbell, Glen J Henson, Valery Fuh Ngwa, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Andrew J Palmer","doi":"10.1007/s40273-024-01461-0","DOIUrl":"10.1007/s40273-024-01461-0","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"241"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782290/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142910135","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}
PharmacoEconomicsPub Date : 2025-02-01Epub Date: 2024-11-02DOI: 10.1007/s40273-024-01444-1
Maksat Jumamyradov, Benjamin M Craig
{"title":"Measuring Effectiveness Based on Patient Experience (Instead of QALYs) in US Value Assessments.","authors":"Maksat Jumamyradov, Benjamin M Craig","doi":"10.1007/s40273-024-01444-1","DOIUrl":"10.1007/s40273-024-01444-1","url":null,"abstract":"<p><strong>Background: </strong>A key challenge in value assessment is how to summarize effectiveness, particularly the impact of interventions on patient health-related quality of life (HRQoL). One approach is to quantify the gains in HRQoL and life expectancy together as quality-adjusted life years (QALYs); however, this approach has faced various criticisms regarding its potential discriminatory aspects toward persons with disabilities, older adults, and the most vulnerable individuals in society.</p><p><strong>Methods: </strong>Instead of QALYs, we provide an alternative approach that summarizes HRQoL gains from the perspective of its stakeholders (e.g., patients, parents, and caregivers) using an \"experience\" scale. On an experience scale, a positive value signifies an experience better than having no experience at all, while a negative value indicates an experience worse than having no experience. To illustrate the merits of this approach, we examine US preferences on the relief of child health problems, namely a discrete choice experiment (DCE) with kaizen tasks and alternatives described using the EQ-5D-Y-3L.</p><p><strong>Results: </strong>Using this approach, we demonstrate the differences in perspectives between parents (N = 179), mothers (N = 99), and fathers (N = 80) of children younger than 18 years of age, as well as the feasibility of this patient-centered approach using a brief DCE survey of less than 100 respondents each (and without QALYs). Specifically, we found that mothers place a higher value on the child's feelings than fathers. The results also suggest other differences between the perspectives of mothers and fathers, but these differences were not statistically significant (p-values < .05).</p><p><strong>Conclusions: </strong>We put forth that future value assessments may summarize gains in HRQoL on a patient experience scale (i.e., experience scale from the patient perspective) to inform decision-making.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"171-176"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782394/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142564477","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}
PharmacoEconomicsPub Date : 2025-02-01Epub Date: 2024-08-02DOI: 10.1007/s40273-024-01417-4
Julie A Campbell, Glen J Henson, Valery Fuh Ngwa, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Andrew J Palmer
{"title":"Estimation of Transition Probabilities from a Large Cohort (> 6000) of Australians Living with Multiple Sclerosis (MS) for Changing Disability Severity Classifications, MS Phenotype, and Disease-Modifying Therapy Classifications.","authors":"Julie A Campbell, Glen J Henson, Valery Fuh Ngwa, Hasnat Ahmad, Bruce V Taylor, Ingrid van der Mei, Andrew J Palmer","doi":"10.1007/s40273-024-01417-4","DOIUrl":"10.1007/s40273-024-01417-4","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) is a chronic autoimmune/neurodegenerative disease associated with progressing disability affecting mostly women. We aim to estimate transition probabilities describing MS-related disability progression from no disability to severe disability. Transition probabilities are a vital input for health economics models. In MS, this is particularly relevant for pharmaceutical agency reimbursement decisions for disease-modifying therapies (DMTs).</p><p><strong>Methods: </strong>Data were obtained from Australian participants of the MSBase registry. We used a four-state continuous-time Markov model to describe how people with MS transition between disability milestones defined by the Expanded Disability Status Scale (scale 0-10): no disability (EDSS of 0.0), mild (EDSS of 1.0-3.5), moderate (EDSS of 4.0-6.0), and severe (EDSS of 6.5-9.5). Model covariates included sex, DMT usage, MS-phenotype, and disease duration, and analysis of covariate groups were also conducted. All data were recorded by the treating neurologist.</p><p><strong>Results: </strong>A total of N = 6369 participants (mean age 42.5 years, 75.00% female) with 38,837 person-years of follow-up and 54,570 clinical reviews were identified for the study. Annual transition probabilities included: remaining in the no, mild, moderate, and severe states (54.24%, 82.02%, 69.86%, 77.83% respectively) and transitioning from no to mild (42.31%), mild to moderate (11.38%), and moderate to severe (9.41%). Secondary-progressive MS was associated with a 150.9% increase in the hazard of disability progression versus relapsing-remitting MS.</p><p><strong>Conclusions: </strong>People with MS have an approximately 45% probability of transitioning from the no disability state after one year, with people with progressive MS transitioning from this health state at a much higher rate. These transition probabilities will be applied in a publicly available health economics simulation model for Australia and similar populations, intended to support reimbursement of a plethora of existing and upcoming interventions including medications to reduce progression of MS.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"223-239"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782298/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879169","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}
PharmacoEconomicsPub Date : 2025-02-01Epub Date: 2024-11-04DOI: 10.1007/s40273-024-01440-5
Alexandra Starry, Nils Picker, Jonathan Galduf, Ulf Maywald, Axel Dittmar, Stefan G Spitzer
{"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":"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":"177-189"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","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}
PharmacoEconomicsPub Date : 2025-02-01Epub Date: 2024-11-03DOI: 10.1007/s40273-024-01447-y
Sietse van Mossel, Rafael Emilio de Feria Cardet, Lioe-Fee de Geus-Oei, Dennis Vriens, Hendrik Koffijberg, Sopany Saing
{"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":"10.1007/s40273-024-01447-y","url":null,"abstract":"<p><strong>Background and objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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":"133-151"},"PeriodicalIF":4.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782410/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}