PharmacoEconomicsPub Date : 2025-03-29DOI: 10.1007/s40273-025-01485-0
Bart Heeg, Dawn Lee, Jane Adam, Maarten Postma, Mario Ouwens
{"title":"Defining Biological and Clinical Plausibility: The DICSA Framework for Protocolized Assessment in Survival Extrapolations Across Therapeutic Areas.","authors":"Bart Heeg, Dawn Lee, Jane Adam, Maarten Postma, Mario Ouwens","doi":"10.1007/s40273-025-01485-0","DOIUrl":"https://doi.org/10.1007/s40273-025-01485-0","url":null,"abstract":"<p><strong>Background: </strong>Numerous health technology assessment guidance documents emphasize the importance of biological/clinical plausibility of modeled lifetime incremental survival without clearly defining it.</p><p><strong>Objectives: </strong>This paper defines biologically and clinically plausible lifetime survival extrapolations and proposes a framework to systematically assess this by comparing survival expectations estimated premodeling, with the final modeled survival extrapolations. This framework is embedded in a survival extrapolation protocol template, which ensures that both the expectations and extrapolations are based on unified, comprehensive evidence.</p><p><strong>Methods: </strong>A targeted review was conducted of 29 guidance documents from National Institute for Health and Care Excellence, Pharmaceutical Benefits Advisory Committee, Haute Autorité de Santé, Canada's Drug Agency, and European joint clinical assessment, focusing on survival analysis, evidence synthesis, cost-effectiveness modeling methods, and use of observational data.</p><p><strong>Results: </strong>Survival extrapolations are biologically/clinically plausible when \"predicted survival estimates that fall within the range considered plausible a-priori, obtained using a-priori justified methodology.\" These a priori expectations should utilize the totality of evidence available and take into account local target setting (i.e., survival-influencing aspects such as patient population, treatment pathway, and country). Pre-protocolized biologically/clinically plausible survival extrapolation was operationalized in a five-step DICSA approach: (1) Describe the target setting as defined by all relevant treatment and disease aspects that influence survival; (2) collect Information from relevant sources; (3) Compare survival-influencing aspects across information sources; (4) Set pre-protocolized survival expectations and plausible ranges; and (5) Assess how trial-based extrapolations align with the set expectations by comparing modeled survival extrapolations to the range of values a priori considered to be plausible.</p><p><strong>Conclusion: </strong>The definition of plausibility of survival extrapolations, the operationalization of its assessment, and the corresponding extrapolation protocol template can contribute to the transparent development of biologically/clinically plausible survival extrapolations.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143743541","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: 2024-12-04DOI: 10.1007/s40273-024-01456-x
Trang T H Nguyen, Shweta Mital
{"title":"Cost-Effectiveness of Capivasertib as a Second-Line Therapy for Advanced Breast Cancer.","authors":"Trang T H Nguyen, Shweta Mital","doi":"10.1007/s40273-024-01456-x","DOIUrl":"10.1007/s40273-024-01456-x","url":null,"abstract":"<p><strong>Background: </strong>Capivasertib, a first-in-class AKT inhibitor, was recently approved as a second-line treatment for advanced breast cancer. However, capivasertib is expensive, raising questions over its economic value. This study provides the first evidence on the cost effectiveness of adding capivasertib to endocrine therapy (fulvestrant) for patients with PIK3CA/AKT1/PTEN-altered, hormone receptor-positive (HR<sup>+</sup>) human epidermal growth factor receptor 2-negative (HER2<sup>-</sup>) advanced breast cancer.</p><p><strong>Methods: </strong>A Markov model was built to compare the costs and effectiveness of three treatment strategies. The first strategy involved adding capivasertib to fulvestrant for all patients, while the second strategy involved adding it for only postmenopausal women. The third strategy involved treatment with fulvestrant alone. Analyses were conducted from a US payer perspective over a lifetime horizon. Costs were measured in 2023 US dollars, and effectiveness was measured in life years (LYs) and quality adjusted life years (QALYs), discounted at 3% per year. One-way sensitivity analyses, probabilistic sensitivity analyses, and scenario analyses were conducted to assess the robustness of results.</p><p><strong>Results: </strong>The addition of capivasertib to fulvestrant for all patients was associated with $410,765 higher costs and 1.46 additional quality adjusted life years (QALYs) compared with fulvestrant alone, resulting in an incremental cost effectiveness ratio of $280,854/QALY. The strategy of adding capivasertib for only patients who are postmenopausal was extended dominated, i.e., yielded fewer QALYs at a higher cost per QALY than if capivasertib was added for all patients. These results were found to be robust in sensitivity and scenario analyses.</p><p><strong>Conclusions: </strong>At its current price, our analysis suggests that the addition of capivasertib to fulvestrant as a second line treatment is not cost effective versus fulvestrant alone at a willingness-to-pay threshold of $100,000/QALY. The price of capivasertib will need to be reduced by nearly 70% (to $7000 per cycle) for it to become cost effective.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"351-361"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771141","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: 2024-11-21DOI: 10.1007/s40273-024-01455-y
Anton Pak, Madeline O'Grady, Gerald Holtmann, Ayesha Shah, Haitham Tuffaha
{"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":"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":"249-269"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","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":"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":"283-296"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825629/pdf/","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-25DOI: 10.1007/s40273-024-01439-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}