Medical Decision Making最新文献

{"title":"The \"Stock of Time\" Method: A New Approach to Calculate Indirect Costs and Benefits in Economic Evaluations.","authors":"Lucy Kok, Carl Koopmans","doi":"10.1177/0272989X251333787","DOIUrl":"https://doi.org/10.1177/0272989X251333787","url":null,"abstract":"<p><p>BackgroundHealth interventions affect people's welfare directly by impacting people's health but also indirectly via a change in consumption and leisure time caused by the change in health. This study discusses 2 ongoing issues in the economic evaluation of health interventions. The first is how to value a change in the amount of leisure time of a patient. The second issue concerns the valuation of a change in production.MethodsWe present a theoretical model that assumes that individual utility depends on health, consumption, and leisure time. We assume that the total stock of time consists of 3 components: leisure time, working time, and recovery time. The model takes a societal perspective and assumes that individuals optimize their utility, conditional on time and budget restrictions.ResultsFor the first issue, the model indicates that the value of a change in the stock of time available for leisure and work has to be added to the direct effects of a health intervention, instead of only a change in work hours. For the second issue, the model indicates that in case of a change in longevity, only the change in taxes paid may be counted because the income change is included in the value of the change in quality-adjusted life-years. A numerical example shows that this approach may counterbalance the potential overestimation of the welfare effects of treatments with the human capital method and underestimation related to the friction cost method.ConclusionWe propose a new method that includes the welfare effects of health interventions due to a change in the amount of leisure time and avoids double counting of welfare changes, which are included in the direct effects.HighlightsWe present a theoretical model and use it to analyze 2 issues in the economic evaluation of health interventions: the inclusion of leisure time and the valuation of production.The model indicates that the effects of health changes on the amount of both work and leisure hours need to be taken into account in economic evaluation.As to the valuation of production, the model indicates that in case of a change in longevity, only the change in taxes may be counted.We propose the \"stock of time\" method to value changes in working hours and leisure hours, which may counterbalance potential overestimates and underestimates in established methods.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X251333787"},"PeriodicalIF":3.1,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036584","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":"Co-designing a Structured Expert Elicitation with Clinicians to Enhance Health Care Decision Making in Exercise Oncology.","authors":"Yufan Wang, Alexandra L McCarthy, Haitham Tuffaha","doi":"10.1177/0272989X251332967","DOIUrl":"https://doi.org/10.1177/0272989X251332967","url":null,"abstract":"<p><p>BackgroundWhile structured expert elicitation (SEE) is gaining traction in health technology assessment in situations in which data are scarce, its application in practice remains limited. Co-designing a practical and fit-for-purpose SEE with experts could enhance its acceptability and feasibility in clinical research.ObjectivesAn SEE was co-designed with clinicians to elicit expert opinions on 3 uncertain quantities of interest (QoIs) for a decision-analytic model in exercise oncology.MethodsA series of co-design meetings was convened to design 6 elicitation stages. Individual elicitation was conducted using the variable interval method (VIM), via videoconferencing. Linear pooling was adopted to generate group estimates. Semi-structured interviews were conducted after the elicitation exercise to gather the experts' first-hand experience of the elicitation process and to identify areas for improvement. Qualitative data were transcribed and content analyzed.ResultsTwelve experts participated in the co-designed SEE. Three beta distributions were derived and estimated from the experts' responses: the relative risk reduction of cardiovascular events of exercise for women who survived early-stage endometrial cancer (Mean: 0.362, SD: 0.15), the probability that a clinician would refer a patient to the exercise program (Mean: 0.457, SD: 0.218), and the probability that a cancer patient would use such a health service upon referral (Mean: 0.446, SD: 0.203). Most of the experts' first-hand experience of the co-designed SEE was positive. The qualitative feedback highlighted critical aspects of the elicitation process that should be designed and executed with caution when targeting clinicians with no prior experience of SEE.ConclusionsThis is the first expert elicitation conducted in exercise oncology. Engaging diverse stakeholders through co-design meetings and incorporating qualitative feedback proved effective and practical in introducing expert elicitation into clinical research.HighlightsRecent SEE guidelines aim to facilitate the conduct of expert elicitation in model-based economic evaluation, but its application in practice remains limited.Engaging experts in the design of SEE could enhance its acceptability and feasibility in clinical research.This is the first co-designed expert elicitation involving clinicians in the field of exercise oncology.This practical approach to conducting SEE could promote a wider adoption to inform health care policy decisions when the evidence is lacking or uncertain.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X251332967"},"PeriodicalIF":3.1,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019947","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":"Optimizing the Harms and Benefits of Cervical Screening in a Partially Vaccinated Population in Ontario, Canada: A Modeling Study.","authors":"Daniël D de Bondt, Erik E L Jansen, Christine Stogios, Bronwen R McCurdy, Rachel Kupets, Joan Murphy, Dustin Costescu, Linda Rabeneck, Rebecca Truscott, Jan A C Hontelez, Inge M C M de Kok","doi":"10.1177/0272989X251332597","DOIUrl":"https://doi.org/10.1177/0272989X251332597","url":null,"abstract":"<p><p>ObjectivesIn Ontario, Canada, the first cohorts who were offered school-based human papillomavirus (HPV) vaccination are now eligible for cervical screening. We determined which screening strategies for these populations would result in optimal harms-benefits ratios of screening.MethodsWe used the hybrid microsimulation model STDSIM- MISCAN-Cervix to determine the harms and cancers prevented of 309 different primary HPV screening strategies, varying by screening ages and triage methods. In addition, we performed an unstratified (i.e., uniform screening protocols) and stratified (i.e., screening protocols by vaccination status) analysis. Harms induced were quantified as a weighted combination of the number of primary HPV-based screens and colposcopy referrals at 1:10. A harms-benefit acceptability threshold of number of harms induced for each cancer prevented was set at the estimated ratio under current screening recommendations in unvaccinated cohorts in Ontario.ResultsFor the unstratified scenario, 5 lifetime screens with HPV16/18 genotyping was optimal. For the stratified scenario, the optimal scenario was 3 lifetime screens with HPV16/18/31/33/45/52/58 genotyping for vaccinated individuals versus 6 lifetime screens with HPV16/18 genotyping for unvaccinated individuals.ConclusionsWe determined the optimal cervical screening strategy in Ontario over the next decades. To maintain an optimal harms-benefits balance of screening, the Ontario Cervical Screening Program could adjust screening recommendations in the future to reduce the number of lifetime screens and extend screening intervals to account for vaccinated cohorts. Stratified screening by vaccination status could further improve this balance on an individual level.HighlightsPeople in cohorts who were offered HPV vaccination as part of Ontario's school-based program may achieve a better harms-benefits balance if cervical screening recommendations are updated to a less intensive protocol in future. This holds for the cohorts as a whole (i.e., unstratified screening) as well as for both vaccinated and unvaccinated individuals in these cohorts.Instead of using a cost-effectiveness threshold, it is possible to determine optimal screening protocols by calculating an acceptability threshold using alternative harms-benefits measures based on existing policy.Using univariate harms measures such as primary HPV screening tests or colposcopies per 1,000 people can yield biases in optimizing cervical screening programs. Alternatively, combining both primary screens and colposcopy referrals could provide a more accurate harms measure and result in optimal strategies with a better balance between harms and benefits.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"272989X251332597"},"PeriodicalIF":3.1,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144056317","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":"Segmenting the Population and Estimating Transition Probabilities Using Data on Health and Health-Related Social Service Needs from the US Health and Retirement Study.","authors":"Lize Duminy","doi":"10.1177/0272989X251320887","DOIUrl":"10.1177/0272989X251320887","url":null,"abstract":"<p><p>BackgroundSimulation modeling is a promising tool to help policy makers and providers make evidence-based decisions when evaluating integrated care programs. The functionality of such models, however, depends on 2 prerequisites: 1) the analytical segmentation of populations to capture both health and health-related social service (HASS) needs and 2) the precise estimation of transition probabilities among the various states of need.MethodsWe took a validated instrument for segmenting the population by HASS needs and adapted it to the Health and Retirement Study, a nationally representative survey dataset from the US population older than 50 y. We then estimated the transition probabilities across all 10 need states and death using multistate modeling. A need state was defined as a combination of any of the 5 ordinal global impression segments and a complicating factor status.ResultsKaplan-Meier survival curves, log-rank tests, and c-indices were used to assess predictive validity in relation to mortality. The Markov traces, using the estimated transition probability to replicate 2 closed cohorts, resembled the proportion of individuals per health state across subsequent waves well enough to indicate adequate fit of the estimated transition probabilities.ConclusionsThis article provides a population segmentation approach that incorporates HASS needs for the US population and 1-y transition probabilities across HASS need states and death. This is the first application of HASS segmentation that can estimate transitions between all 10 HASS need states, facilitating novel analysis of policy decisions related to integrated care.ImplicationsOur results will be used as input for a simulation model that performs scenario analysis on the long-term effects of various integrated care policies on population health.HighlightsWe took a validated tool for segmenting the population according to health and health-related social service (HASS) needs and adapted it to the Health and Retirement Study, a nationally representative survey dataset from the US population over the age of 50 y.We estimated the 1-y transition probabilities across all 10 HASS segments and death.This is the first application of a version of this HASS segmentation tool that includes HASSs in the various need states when estimating transition probabilities.Our results will be used as input for a simulation model that performs scenario analysis on the long-term effects of various integrated care policies on population health.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"286-301"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143484476","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":"Health Utilities in People with Hepatitis C Virus Infection: A Study Using Real-World Population-Level Data.","authors":"Yasmin A Saeed, Nicholas Mitsakakis, Jordan J Feld, Murray D Krahn, Jeffrey C Kwong, William W L Wong","doi":"10.1177/0272989X251319342","DOIUrl":"10.1177/0272989X251319342","url":null,"abstract":"<p><p>BackgroundHepatitis C virus (HCV) infection is associated with reduced quality of life and health utility. It is unclear whether this is primarily due to HCV infection itself or commonly co-occurring patient characteristics such as low income and mental health issues. This study aims to estimate and separate the effects of HCV infection on health utility from the effects of clinical and sociodemographic factors using real-world population-level data.MethodsWe conducted a cross-sectional retrospective cohort study to estimate health utilities in people with and without HCV infection in Ontario, Canada, from 2000 to 2014 using linked survey data from the Canadian Community Health Survey and health administrative data. Utilities were derived from the Health Utilities Index Mark 3 instrument. We used propensity score matching and multivariable linear regression to examine the impact of HCV infection on utility scores while adjusting for clinical and sociodemographic factors.ResultsThere were 7,102 individuals with hepatitis C status and health utility data available (506 HCV-positive, 6,596 HCV-negative). Factors associated with marginalization were more prevalent in the HCV-positive cohort (e.g., household income <$20,000: 36% versus 15%). Propensity score matching resulted in 454 matched pairs of HCV-positive and HCV-negative individuals. HCV-positive individuals had substantially lower unadjusted utilities than HCV-negative individuals did (mean ± standard error: 0.662 ± 0.016 versus 0.734 ± 0.015). The regression model showed that HCV positivity (coefficient: -0.066), age, comorbidity, mental health history, and household income had large impacts on health utility.ConclusionsHCV infection is associated with low health utility even after controlling for clinical and sociodemographic variables. Individuals with HCV infection may benefit from additional social services and supports alongside antiviral therapy to improve their quality of life.HighlightsHepatitis C virus (HCV) infection is associated with reduced quality of life and health utility. There is debate in the literature on whether this is primarily due to HCV infection itself or commonly co-occurring patient characteristics such as low income and mental health issues.We showed that individuals with HCV infection have substantially lower health utilities than uninfected individuals do even after controlling for clinical and sociodemographic variables, based on a large, real-world population-level dataset. Socioeconomically marginalized individuals with HCV infection had particularly low health utilities.In addition to improving access to HCV testing and treatment, it may be beneficial to provide social services such as mental health and financial supports to improve the quality of life and health utility of people living with HCV.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"332-343"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894892/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476923","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}

{"title":"Directed Acyclic Graphs in Decision-Analytic Modeling: Bridging Causal Inference and Effective Model Design in Medical Decision Making.","authors":"Stijntje W Dijk, Maurice Korf, Jeremy A Labrecque, Ankur Pandya, Bart S Ferket, Lára R Hallsson, John B Wong, Uwe Siebert, M G Myriam Hunink","doi":"10.1177/0272989X241310898","DOIUrl":"10.1177/0272989X241310898","url":null,"abstract":"<p><p>Decision-analytic models (DAMs) are essentially informative yet complex tools for solving questions in medical decision making. When their complexity grows, the need for causal inference techniques becomes evident as causal relationships between variables become unclear. In this methodological commentary, we argue that graphical representations of assumptions on such relationships, directed acyclic graphs (DAGs), can enhance the transparency of decision models and aid in parameter selection and estimation through visually specifying backdoor paths (i.e., potential biases in parameter estimates) and visually clarifying structural modeling choices of frontdoor paths (i.e., the effect of the model structure on the outcome). This commentary discusses the benefit of integrating DAGs and DAMs in medical decision making and in particular health economics with 2 applications: the first examines statin use for prevention of cardiovascular disease, and the second considers mindfulness-based interventions for students' stress. Despite the potential application of DAGs in the decision science framework, challenges remain, including simplicity, defining the scope of a DAG, unmeasured confounding, noncausal aspects, and limited data availability or quality. Broader adoption of DAGs in decision science requires full-model applications and further debate.HighlightsOur commentary proposes the application of directed acyclic graphs (DAGs) in the design of decision-analytic models, offering researchers a valuable and structured tool to enhance transparency and accuracy by bridging the gap between causal inference and model design in medical decision making.The practical examples in this article showcase the transformative effect DAGs can have on model structure, parameter selection, and the resulting conclusions on effectiveness and cost-effectiveness.This methodological article invites a broader conversation on decision-modeling choices grounded in causal assumptions.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"223-231"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894903/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143025164","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}

{"title":"Recalibrating an Established Microsimulation Model to Capture Trends and Projections of Colorectal Cancer Incidence and Mortality.","authors":"Jie-Bin Lew, Qingwei Luo, Joachim Worthington, Han Ge, Emily He, Julia Steinberg, Michael Caruana, Dianne L O'Connell, Eleonora Feletto, Karen Canfell","doi":"10.1177/0272989X251314050","DOIUrl":"10.1177/0272989X251314050","url":null,"abstract":"<p><p>BackgroundChanging colorectal cancer (CRC) incidence rates, including recent increases for people younger than 50 y, need to be considered in planning for future cancer control and screening initiatives. Reliable estimates of the impact of changing CRC trends on the National Bowel Cancer Screening Program (NBCSP) are essential for programmatic planning in Australia. An existing microsimulation model of CRC, <i>Policy1-Bowel</i>, was updated to reproduce Australian CRC trends data and provide updated projections of CRC- and screening-related outcomes to inform clinical practice guidelines for the prevention of CRC.Methods<i>Policy1-Bowel</i> was recalibrated to reproduce statistical age-period-cohort model trends and projections of CRC incidence for 1995-2045 in the absence of the NBCSP as well as published data on CRC incidence trends, stage distribution, and survival in 1995-2020 in Australia. The recalibrated <i>Policy1-Bowel</i> predictions were validated by comparison with published Australian CRC mortality trends for 1995-2015 and statistical projections to 2040. Metamodels were developed to aid the calibration process and significantly reduce the computational burden.Results<i>Policy1-Bowel</i> was recalibrated, and best-fit parameter sets were identified for lesion incidence, CRC stage progression rates, detection rates, and survival rates by age, sex, bowel location, cancer stage, and birth year. The recalibrated model was validated and successfully reproduced observed CRC mortality rates for 1995-2015 and statistical projections for 2016-2030.ConclusionThe recalibrated <i>Policy1-Bowel</i> model captures significant additional detail on the future incidence and mortality burden of CRC in Australia. This is particularly relevant as younger cohorts with higher CRC incidence rates approach screening ages to inform decision making for these groups. The metamodeling approach allows fast recalibration and makes regular updates to incorporate new evidence feasible.HighlightsIn Australia, colorectal cancer incidence rates are increasing for people younger than 50 y but decreasing for people older than 50 y, and colorectal cancer survival is improving as new treatment technologies emerge.To evaluate the future health and economic impact of screening and inform policy, modeling must include detailed trends and projections of colorectal cancer incidence, mortality, and diagnosis stage.We used novel techniques including integrative age-period cohort projections and metamodel calibration to update <i>Policy1-Bowel</i>, a detailed microsimulation of colorectal cancer and screening in Australia.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"257-275"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143366553","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":"Development of a Microsimulation Model to Project the Future Prevalence of Childhood Cancer in Ontario, Canada.","authors":"Alexandra Moskalewicz, Sumit Gupta, Paul C Nathan, Petros Pechlivanoglou","doi":"10.1177/0272989X251314031","DOIUrl":"10.1177/0272989X251314031","url":null,"abstract":"<p><p>BackgroundEstimates of the future prevalence of childhood cancer are informative for health system planning but are underutilized. We describe the development of a pediatric oncology microsimulation model for prevalence (POSIM-Prev) and illustrate its application to produce projections of incidence, survival, and limited-duration prevalence of childhood cancer in Ontario, Canada, until 2040.MethodsPOSIM-Prev is a population-based, open-cohort, discrete-time microsimulation model. The model population was updated annually from 1970 to 2040 to account for births, deaths, net migration, and incident cases of childhood cancer. Prevalent individuals were followed until death, emigration, or the last year of simulation. Median population-based outcomes with 95% credible intervals (CrIs) were generated using Monte Carlo simulation. The methodology to derive model inputs included generalized additive modeling of cancer incidence, parametric survival modeling, and stochastic population forecasting. Individual-level data from provincial cancer registries for years 1970 to 2019 informed cancer-related model inputs and internal validation.ResultsThe number of children (aged 0-14 y) diagnosed with cancer in Ontario is projected to rise from 414 (95% CrI: 353-486) in 2020 to 561 (95% CrI: 481-653) in 2039. The 5-y overall survival rate for 2030-2034 is estimated to reach 90% (95% CrI: 88%-92%). By 2040, 24,088 (95% CrI: 22,764-25,648) individuals with a history of childhood cancer (diagnosed in Ontario or elsewhere) are projected to reside in the province. The model accurately reproduced historical trends in incidence, survival, and prevalence when validated.ConclusionsThe rising incidence and prevalence of childhood cancer will create increased demand for both acute cancer care and long-term follow-up services in Ontario. The POSIM-Prev model can be used to support long-range health system planning and future health technology assessments in jurisdictions that have access to similar model inputs.HighlightsThis article describes the development of a population-based, discrete-time microsimulation model that can simulate incident and prevalent cases of childhood cancer in Ontario, Canada, until 2040.Use of an open cohort framework allowed for estimation of the potential impact of net migration on childhood cancer prevalence.In addition to supporting long-term health system planning, this model can be used in future health technology assessments, by providing a demographic profile of incident and prevalent cases for model conceptualization and budget impact purposes.This modeling framework is adaptable to other jurisdictions and disease areas where individual-level data for incidence and survival are available.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"245-256"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143191036","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":"Changing Time Representation in Microsimulation Models.","authors":"Eric Kai-Chung Wong, Wanrudee Isaranuwatchai, Joanna E M Sale, Andrea C Tricco, Sharon E Straus, David M J Naimark","doi":"10.1177/0272989X251319808","DOIUrl":"10.1177/0272989X251319808","url":null,"abstract":"<p><p>BackgroundIn microsimulation models of diseases with an early, acute phase requiring short cycle lengths followed by a chronic phase, fixed short cycles may lead to computational inefficiency. Examples include epidemic or resource constraint models with early short cycles where long-term economic consequences are of interest for individuals surviving the epidemic or ultimately obtaining the resource. In this article, we demonstrate methods to improve efficiency in such scenarios. Furthermore, we show that care must be taken when applying these methods to epidemic or resource constraint models to avoid bias.MethodsTo demonstrate efficiency, we compared the model runtime among 3 versions of a microsimulation model: with short fixed cycles for all states (FCL), with dynamic cycle length (DCL) defined locally for each state, and with DCL features plus a discrete-event-like hybrid component. To demonstrate bias mitigation, we compared discounted lifetime costs for 3 versions of a resource constraint model: with a fixed horizon where simulation stops, with a fixed entry horizon beyond which new individuals could not enter the model, and with a fixed entry horizon plus a mechanism to maintain a constant level of competition for the resource after the horizon.ResultsThe 3 versions of the microsimulation model had average runtimes of 515 (95% credible interval [CI]: 477 to 545; FCL), 2.70 (95% CI: 1.48 to 2.92; DCL), and 1.45 (95% CI: 1.26 to 2.61; DCL-pseudo discrete event simulation) seconds, respectively. The first 2 resource constraint versions underestimated costs relative to the constant competition version: $20,055 (95% CI: $19,000 to $21,120), $27,030 (95% CI: $24,680 to $29,412), and $33,424 (95% CI: $27,510 to $44,484), respectively.LimitationsThe magnitude of improvements in efficiency and reduction in bias may be model specific.ConclusionChanging time representation in microsimulation may offer computational advantages.HighlightsShort cycle lengths may be required to model the acute phase of an illness but lead to computational inefficiency in a subsequent chronic phase in microsimulation models.A solution is to create state-specific cycle lengths so that cycle lengths change dynamically as the simulation progresses.Computational efficiency can be enhanced further by using a hybrid model containing discrete-event-simulation-like features.Hybrid models can efficiently handle events subsequent to exit from an epidemic or resource constraint model provided steps are taken to mitigate potential bias.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"276-285"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894904/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143494530","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}

{"title":"Development of a Decision Model to Estimate the Outcomes of Treatment Sequences in Advanced Melanoma.","authors":"Saskia de Groot, Hedwig M Blommestein, Brenda Leeneman, Carin A Uyl-de Groot, John B A G Haanen, Michel W J M Wouters, Maureen J B Aarts, Franchette W P J van den Berkmortel, Willeke A M Blokx, Marye J Boers-Sonderen, Alfons J M van den Eertwegh, Jan Willem B de Groot, Geke A P Hospers, Ellen Kapiteijn, Olivier J van Not, Astrid A M van der Veldt, Karijn P M Suijkerbuijk, Pieter H M van Baal","doi":"10.1177/0272989X251319338","DOIUrl":"10.1177/0272989X251319338","url":null,"abstract":"<p><p>BackgroundA decision model for patients with advanced melanoma to estimate outcomes of a wide range of treatment sequences is lacking.ObjectivesTo develop a decision model for advanced melanoma to estimate outcomes of treatment sequences in clinical practice with the aim of supporting decision making. The article focuses on methodology and long-term health benefits.MethodsA semi-Markov model with a lifetime horizon was developed. Transitions describing disease progression, time to next treatment, and mortality were estimated from real-world data (RWD) as a function of time since starting treatment or disease progression and patient characteristics. Transitions were estimated separately for melanoma with and without a BRAF mutation and for patients with favorable and intermediate prognostic factors. All transitions can be adjusted using relative effectiveness of treatments derived from a network meta-analysis of randomized controlled trials (RCTs). The duration of treatment effect can be adjusted to obtain outcomes under different assumptions.ResultsThe model distinguishes 3 lines of systemic treatment for melanoma with a BRAF mutation and 2 lines of systemic treatment for melanoma without a BRAF mutation. Life expectancy ranged from 7.8 to 12.0 years in patients with favorable prognostic factors and from 5.1 to 8.7 years in patients with intermediate prognostic factors when treated with sequences consisting of targeted therapies and immunotherapies. Scenario analyses illustrate how estimates of life expectancy depend on the duration of treatment effect.ConclusionThe model is flexible because it can accommodate different treatments and treatment sequences, and the duration of treatment effects and the transitions influenced by treatment can be adjusted. We show how using RWD and data from RCTs can harness advantages of both data sources, guiding the development of future decision models.HighlightsThe model is flexible because it can accommodate different treatments and treatment sequences, and the duration of treatment effects as well as the transitions that are influenced by treatment can be adjusted.The long-term health benefits of treatment sequences depend on the place of different therapies within a treatment sequence.Assumptions about the duration of relative treatment effects influence the estimates of long-term health benefits.We show how the use of real-world data and data from randomized controlled trials harness the advantages of both data sources, guiding the development of future decision models.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"302-317"},"PeriodicalIF":3.1,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11894896/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143476920","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}