Alexandra Moskalewicz, Sumit Gupta, Paul C Nathan, Petros Pechlivanoglou
{"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":"https://doi.org/10.1177/0272989X251314031","url":null,"abstract":"<p><strong>Background: </strong>Estimates 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.</p><p><strong>Methods: </strong>POSIM-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.</p><p><strong>Results: </strong>The 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.</p><p><strong>Conclusions: </strong>The 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.</p><p><strong>Highlights: </strong>This 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":"272989X251314031"},"PeriodicalIF":3.1,"publicationDate":"2025-02-04","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-25DOI: 10.1177/0272989X241302829
Brian L Egleston, Mary B Daly, Kaitlyn Lew, Lisa Bealin, Alexander D Husband, Jill E Stopfer, Pawel Przybysz, Olga Tchuvatkina, Yu-Ning Wong, Judy E Garber, Timothy R Rebbeck
{"title":"Changes in Risk Tolerance for Ovarian Cancer Prevention Strategies during the COVID-19 Pandemic: Results of a Discrete Choice Experiment.","authors":"Brian L Egleston, Mary B Daly, Kaitlyn Lew, Lisa Bealin, Alexander D Husband, Jill E Stopfer, Pawel Przybysz, Olga Tchuvatkina, Yu-Ning Wong, Judy E Garber, Timothy R Rebbeck","doi":"10.1177/0272989X241302829","DOIUrl":"10.1177/0272989X241302829","url":null,"abstract":"<p><strong>Background: </strong>Prior to COVID-19, little was known about how risks associated with such a pandemic would compete with and influence patient decision making regarding cancer risk reducing medical decision making. We investigated how the pandemic affected preferences for medical risk-reducing strategies among women at elevated risk of breast or ovarian cancer.</p><p><strong>Methods: </strong>We conducted a discrete choice experiment. Women about to undergo genetic testing and counseling at 2 medical centers participated. Enrollment occurred between 2019 and 2022, allowing us to investigate changes in preferences from before the pandemic to after the pandemic. Women chose from permuted scenarios that specified type of surgery, age of menopause, quality of menopausal symptoms, and risk of ovarian cancer, heart disease, or osteoporosis.</p><p><strong>Results: </strong>A total of 355 women, with a median age of 36 y, participated. In 2019, women were less likely to choose prevention scenarios with higher ovarian cancer risk (odds ratio [OR] = 0.42 per 10-point increase in risk, 95% confidence interval [CI] 0.22-0.61). In June 2020, the effect of higher ovarian cancer risk scenarios on choice was attenuated (OR = 0.86, 95% CI 0.68-1.04), with the effect becoming more salient again by July 2021 (OR = 0.59, 95% CI 0.52-0.67) (<i>P</i> = 0.039 for test of temporal interaction). No other attribute demonstrated a temporal trend.</p><p><strong>Conclusion: </strong>The risks associated with the COVID-19 pandemic may have attenuated the impact of risk of ovarian cancer on choice of risk-reducing prevention strategies for ovarian cancer. The maximum attenuation occurred at the beginning of the pandemic when access to risk-reducing surgery was most restricted. Our findings highlight how individuals evaluate competing health risks and adjust their uptake of cancer prevention strategies when faced with a future pandemic or similar global crisis.</p><p><strong>Highlights: </strong>In this discrete choice experiment, women were much less likely to choose prevention scenarios that had higher ovarian cancer risk prior to the COVID-19 pandemic than after the pandemic.The attenuation of preferences may have persisted through 2022.COVID-19 may have altered the relative importance of factors that motivate women to undergo risk-reducing surgeries.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"168-176"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142899970","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2025-01-05DOI: 10.1177/0272989X241308768
David U Garibay-Treviño, Hawre Jalal, Fernando Alarid-Escudero
{"title":"A Fast Nonparametric Sampling Method for Time to Event in Individual-Level Simulation Models.","authors":"David U Garibay-Treviño, Hawre Jalal, Fernando Alarid-Escudero","doi":"10.1177/0272989X241308768","DOIUrl":"10.1177/0272989X241308768","url":null,"abstract":"<p><strong>Highlights: </strong>The nonparametric sampling method is generic and can sample times to an event from any discrete (or discretizable) hazard without requiring any parametric assumption.The method is showcased with 5 commonly used distributions in discrete-event simulation models.The method produced very similar expected times to events, as well as their probability distribution, compared with analytical results.We provide a multivariate categorical sampling function for R and Python programming languages to sample times to events from processes with different hazards simultaneously.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"205-213"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11736974/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142933365","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-24DOI: 10.1177/0272989X241305142
Erika L Thompson, Justin Luningham, Sarah A Alkhatib, Jessica Grace, Idara N Akpan, Ellen M Daley, Gregory D Zimet, Christopher W Wheldon
{"title":"Testing an HPV Vaccine Decision Aid for 27- to 45-Year-Old Adults in the United States: A Randomized Trial.","authors":"Erika L Thompson, Justin Luningham, Sarah A Alkhatib, Jessica Grace, Idara N Akpan, Ellen M Daley, Gregory D Zimet, Christopher W Wheldon","doi":"10.1177/0272989X241305142","DOIUrl":"10.1177/0272989X241305142","url":null,"abstract":"<p><strong>Background: </strong>In the United States, human papillomavirus (HPV) vaccination among 27- to 45-y-olds (mid-adults) is recommended based on shared clinical decision making with a health care provider. We developed a patient decision aid tool to support the implementation of this mid-adult HPV vaccination guideline. The purpose of this study was to evaluate the effect of a patient decision aid tool for HPV vaccination, HPV DECIDE, compared with an information fact sheet among mid-adults who have not received the HPV vaccine.</p><p><strong>Method: </strong>Participants were recruited between December 2023 and January 2024. We used a randomized Solomon, 4-group, pretest/posttest design with mid-adults aged 27 to 45 y who were unvaccinated for HPV and balanced based on sex (<i>n</i> = 612). The primary outcome was decisional conflict. Intermediate outcomes included knowledge, behavioral expectancies, self-efficacy, and perceived risk. Variables were measured using validated scales. Pretest sensitization was not present; intervention and control groups were compared. Fixed-effects inverse-variance weighting was used to pool effect estimates and determine meta-analytic statistical significance across tests with and without pretest controls.</p><p><strong>Results: </strong>Participants in the intervention group had significantly lower total decisional conflict scores (B = -3.58, <i>P</i> = 0.007) compared with the control group. Compared with the control group, participants in the intervention group showed higher knowledge (B = 0.48, <i>P</i> = 0.020), greater intention to receive (B = 0.196, <i>P</i> = 0.049) and discuss the HPV vaccine (B = 0.324, <i>P</i> ≤ 0.001), and greater self-efficacy about HPV vaccine decision making (B = 3.28, <i>P</i> = 0.043). There were no statistically significant results for perceived risks of HPV infection.</p><p><strong>Conclusions: </strong>The HPV DECIDE tool for mid-adult HPV vaccination shows promise for immediate reductions in decisional conflict and improvement in knowledge, intentions, and self-efficacy about the HPV vaccine. Future studies are warranted to evaluate the effectiveness of this patient decision aid tool in real-world settings.</p><p><strong>Highlights: </strong>Shared clinical decision making is recommended for HPV vaccination with mid-adults.A patient decision aid for HPV vaccination reduced decisional conflict for mid-adults.The HPV vaccine patient decision aid was acceptable to mid-adults.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"192-204"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11736972/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142883617","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-14DOI: 10.1177/0272989X241302288
Romy Richter, Jesse Jansen, Josine van der Kraan, Wais Abbaspoor, Iris Bongaerts, Fleur Pouwels, Celine Vilters, Jany Rademakers, Trudy van der Weijden
{"title":"How Inclusive Are Patient Decision Aids for People with Limited Health Literacy? An Analysis of Understandability Criteria and the Communication about Options and Probabilities.","authors":"Romy Richter, Jesse Jansen, Josine van der Kraan, Wais Abbaspoor, Iris Bongaerts, Fleur Pouwels, Celine Vilters, Jany Rademakers, Trudy van der Weijden","doi":"10.1177/0272989X241302288","DOIUrl":"10.1177/0272989X241302288","url":null,"abstract":"<p><strong>Objective: </strong>Patient decision aids (PtDAs) can support shared decision making. We aimed to explore how inclusive PtDAs are for people with limited health literacy (LHL) by analyzing 1) the understandability of PtDAs using established criteria, 2) how options and probabilities of outcomes are communicated, and 3) the extent to which risk communication (RC) guidelines are followed.</p><p><strong>Methods: </strong>In a descriptive document analysis, we analyzed Dutch PtDAs available in 2021 that met the International Patient Decision Aid Standards. We developed and pilot tested a data extraction form based on key RC and health literacy literature.</p><p><strong>Results: </strong>Most PtDAs (151/198) met most of the understandability criteria on layout (7-8 out of 8 items) such as font size but not on content aspects (121/198 PtDAs scored 5-7 out of 12 items) such as defining medical terms. Only 31 of 198 PtDAs used a short and simple sentence structure. Most PtDAs presented 2 to 4 treatment options. Many followed RC recommendations such as the use of numerical RC strategies such as percentages or natural frequencies (160/198) and visual formats such as icon arrays (91/198). Only 10 used neutral framing (10/198). When presented, uncertainty was presented verbally (134/198) or in ranges (58/198). Four PtDAs were co-created together with patients with LHL and used only verbal RC or no RC.</p><p><strong>Conclusion: </strong>Most PtDAs met most of the understandability criteria on layout, but content aspects and adherence to RC strategies can be improved. Many PtDAs used long sentences and mostly verbal RC and are therefore likely to be inappropriate for patients with LHL. Further research is needed on PtDA characteristics and RC strategies suitable for people with LHL.</p><p><strong>Highlights: </strong>Despite meeting most criteria for understandability, many of the Dutch PtDAs use long sentences, which likely impede comprehension for patients with LHL.Most of the Dutch PtDAs follow established recommendations for risk communication, with room for improvement for some strategies such as framing and a clear reference to the time frame.Overall, more research is needed to tailor PtDAs to the needs of people with LHL.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"143-155"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11736975/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824535","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-21DOI: 10.1177/0272989X241305119
Ronald Scott Braithwaite
{"title":"A Parsimonious Approach to Remediate Concerns about QALY-Based Discrimination.","authors":"Ronald Scott Braithwaite","doi":"10.1177/0272989X241305119","DOIUrl":"10.1177/0272989X241305119","url":null,"abstract":"<p><strong>Highlights: </strong>Important barriers to the use of QALYs in the United States include concerns about disability and age discrimination.Modifications to the utility function underlying QALYs have been proposed to mitigate these concerns, but some find them challenging to consider and/or to apply.Unrelated to these concerns, QALYs have been adapted within the framework of distributional cost-effectiveness analysis to allow consideration of inequality as well as efficiency.I outline how this framework can also remediate concerns about disability and age discrimination.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"214-219"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142873327","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-21DOI: 10.1177/0272989X241302096
Sanket S Dhruva, Aaron S Kesselheim, Steven Woloshin, Robin Z Ji, Zhigang Lu, Jonathan J Darrow, Rita F Redberg
{"title":"Physician-Patient Communication about Novel Drugs and High-Risk Medical Devices.","authors":"Sanket S Dhruva, Aaron S Kesselheim, Steven Woloshin, Robin Z Ji, Zhigang Lu, Jonathan J Darrow, Rita F Redberg","doi":"10.1177/0272989X241302096","DOIUrl":"10.1177/0272989X241302096","url":null,"abstract":"<p><strong>Background: </strong>After a new drug or medical device is approved by the US Food and Drug Administration (FDA), physician-patient communication about benefits and risks is critical, including whether the product was approved through an expedited pathway based on limited evidence. In addition, physician reporting of drug- and device-related adverse events in real-world use is necessary to have a complete safety profile. We studied physician-reported communication and safety-reporting practices related to drugs and devices.</p><p><strong>Methods: </strong>We surveyed a random national sample of American Board of Internal Medicine-certified internists, cardiologists, and oncologists between October 2021 and September 2022 about the sources of information used to prescribe a drug or medical device, details of communication with patients, and reporting of adverse events.</p><p><strong>Results: </strong>Among 509 respondents (39% response rate), 387 (76%) reported that FDA approval influenced their decision \"a lot\" to prescribe a new drug or recommend use of a medical device. Half (122; 50%) of the 244 physicians randomized to receive a question about their own communication of trial endpoints reported \"usually\" telling patients when products were approved based on surrogate measures and 126 (52%) \"usually\" reported telling patients if a postapproval trial was required to evaluate safety and effectiveness. Two-thirds (165) said they were likely to report drug- or device-related adverse events to FDA.</p><p><strong>Conclusions: </strong>Physician self-reporting of communication with patients about drugs and devices suggests that half include characteristics of the pivotal trials such as use of clinically meaningful endpoints or continued requirement for evidence generation.</p><p><strong>Implications: </strong>More consistent discussions with patients about the quality of evidence supporting new drugs and devices and increased reporting of adverse events could ensure optimal use of these products in clinical practice.</p><p><strong>Highlights: </strong>Among 509 board-certified internists, cardiologists, and oncologists, half reported telling patients when drugs or medical devices were approved based on surrogate measures and when there was an FDA-mandated postapproval trial to further evaluate safety and effectiveness.As drugs and medical devices are increasingly approved by the FDA through expedited pathways based on data with lingering uncertainties, discussion with patients about issues such as the nature of the endpoints assessed and existence of postapproval testing requirements can help inform patient decision making.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"156-167"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11736971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142873328","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-18DOI: 10.1177/0272989X241303876
Lucia Araujo Chaveron, Jonathan Sicsic, Cyril Olivier, Gerard Pellissier, Elisabeth Bouvet, Judith E Mueller
{"title":"Communicating on Vaccine Benefit-Risk Ratios: A Discrete-Choice Experiment among Health Care Professionals and the General Population in France.","authors":"Lucia Araujo Chaveron, Jonathan Sicsic, Cyril Olivier, Gerard Pellissier, Elisabeth Bouvet, Judith E Mueller","doi":"10.1177/0272989X241303876","DOIUrl":"10.1177/0272989X241303876","url":null,"abstract":"<p><strong>Background: </strong>We explored preferences around the benefit-risk ratio (BRR) of vaccination among the general adult population and health care sector workers (HCSWs). We estimated preference weights and expected vaccine uptake for different BRR levels for a vaccine recommended during an infectious disease emergence. In addition, we explored how far qualitative information about disease severity, epidemiological context, and indirect protection interacts with these preferences.</p><p><strong>Methodology: </strong>This was a cross-sectional study, using a self-administered online questionnaire containing a single-profile discrete choice experiment among HCSWs and the general population in France (quasi-representative sample). The questionnaire was available from January 12 to April 27, 2023, for HCSWs and from April 17 to May 3, 2023, for the general population. BRR is represented as the number of vaccine-prevented disease events for 1 event related to a vaccine side effect. Results are reported in 4 groups: general population sample, non-HCSWs, non-university-degree HCSWs, and university-degree HCSWs.</p><p><strong>Results: </strong>Among the 1,869 participants, 1,038 (55.5%) varied their vaccine decision among the different vaccine scenarios. Hypothetical vaccine acceptance among university-degree HCSWs increased when the vaccination BRR was 100:1, while non-university-degree HCSWs and non-HCSWs were more sensitive to qualitative information about the vaccine BRR than quantitative indicators. Among participants in the general population sample with varied decisions, expected acceptance increased by 40% sample if disease risk was high. Among serial vaccine nondemanders, high disease risk decreased their certitude to refuse hypothetical vaccination.</p><p><strong>Conclusion: </strong>Our results suggest that only university-degree HCSWs are sensitive to the notion of BRR, but not the general public. Given that previous research found speaking about BRR might reduce vaccine acceptance, this notion should be avoided in vaccine promotion.</p><p><strong>Highlights: </strong>The notion of benefit-risk ratio (BRR) of vaccination appears to be taken into account in vaccine decisions by university-degree HCSWs, but not by the general public. Mentioning a favorable BRR could imply that the vaccine is not safe and reduce vaccine motivation.Mentioning qualitative attributes of BRR surrounding disease frequency and severity, and indirect protection effects, strongly affected theoretical vaccine decisions in all participants, irrespective of professional categories.Expected vaccine acceptance increased by 40% among the general population sample if disease risk was presented as high, and expected vaccine coverage exceeded 50% in scenarios with high disease risk.Among those refusing vaccination in all vaccine scenarios, only a high risk of developing the disease decreased their certitude to refuse vaccination. This further underlines the importance","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"177-191"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848271","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}
Medical Decision MakingPub Date : 2025-02-01Epub Date: 2024-12-25DOI: 10.1177/0272989X241305414
Jeremy D Goldhaber-Fiebert, Hawre Jalal, Fernando Alarid-Escudero
{"title":"Microsimulation Estimates of Decision Uncertainty and Value of Information Are Biased but Consistent.","authors":"Jeremy D Goldhaber-Fiebert, Hawre Jalal, Fernando Alarid-Escudero","doi":"10.1177/0272989X241305414","DOIUrl":"10.1177/0272989X241305414","url":null,"abstract":"<p><strong>Purpose: </strong>Individual-level state-transition microsimulations (iSTMs) have proliferated for economic evaluations in place of cohort state transition models (cSTMs). Probabilistic economic evaluations quantify decision uncertainty and value of information (VOI). Previous studies show that iSTMs provide unbiased estimates of expected incremental net monetary benefits (EINMB), but statistical properties of iSTM-produced estimates of decision uncertainty and VOI remain uncharacterized.</p><p><strong>Methods: </strong>We compare iSTM-produced estimates of decision uncertainty and VOI to corresponding cSTMs. For a 2-alternative decision and normally distributed incremental costs and benefits, we derive analytical expressions for the probability of being cost-effective and the expected value of perfect information (EVPI) for cSTMs and iSTMs, accounting for correlations in incremental outcomes at the population and individual levels. We use numerical simulations to illustrate our findings and explore the impact of relaxing normality assumptions or having >2 decision alternatives.</p><p><strong>Results: </strong>iSTM estimates of decision uncertainty and VOI are biased but asymptotically consistent (i.e., bias approaches 0 as number of microsimulated individuals approaches infinity). Decision uncertainty depends on 1 tail of the INMB distribution (e.g., P[INMB <0]), which depends on estimated variance (larger with iSTMs given first-order noise). While iSTMs overestimate EVPI, their direction of bias for the probability of being cost-effective is ambiguous. Bias is larger when uncertainties in incremental costs and effects are negatively correlated since this increases INMB variance.</p><p><strong>Conclusions: </strong>iSTMs are useful for probabilistic economic evaluations. While more samples at the population uncertainty level are interchangeable with more microsimulations for estimating EINMB, minimizing iSTM bias in estimating decision uncertainty and VOI depends on sufficient microsimulations. Analysts should account for this when allocating their computational budgets and, at minimum, characterize such bias in their reported results.</p><p><strong>Highlights: </strong>Individual-level state-transition microsimulation models (iSTMs) produce biased but consistent estimates of the probability that interventions are cost-effective.iSTMs also produce biased but consistent estimates of the expected value of perfect information.The biases in these decision uncertainty and value-of-information measures are not reduced by more parameter sets being sampled from their population-level uncertainty distribution but rather by more individuals being microsimulated for each parameter set sampled.Analysts using iSTMs to quantify decision uncertainty and value of information should account for these biases when allocating their computational budgets and, at minimum, characterize such bias in their reported results.</p>","PeriodicalId":49839,"journal":{"name":"Medical Decision Making","volume":" ","pages":"127-142"},"PeriodicalIF":3.1,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142886378","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}
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
{"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":"https://doi.org/10.1177/0272989X241310898","url":null,"abstract":"<p><strong>Highlights: </strong>Our 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":"272989X241310898"},"PeriodicalIF":3.1,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}