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Sep 4

Cost-effectiveness analysis for therapy sequence in advanced cancer: A microsimulation approach with application to metastatic prostate cancer

Purpose. Patients with advanced cancer may undergo multiple lines of treatment, switching therapies as their disease progresses. Motivated by a study of metastatic prostate cancer, we develop a microsimulation framework to study therapy sequence. Methods. We propose a discrete-time state transition model to study two lines of anti-cancer therapy. Based on digitized published progression-free survival (PFS) and overall survival (OS) curves, we infer event types (progression or death), and estimate transition probabilities using cumulative incidence functions with competing risks. Our model incorporates within-patient dependence over time, such that response to first-line therapy informs subsequent event probabilities. Parameters governing the degree of within-patient dependence can be used to calibrate the model-based results to those of a target trial. We demonstrate these methods in a study of two therapy sequences for metastatic prostate cancer, where Docetaxel (DCT) and Abiraterone Acetate (AA) are both appropriate for use in either first or second line treatment. We assess costs, Quality-Adjusted Life Years (QALYs) and Incremental Cost Effectiveness Ratio (ICER) for two treatment strategies: DCT then AA vs AA then DCT. Results. Using digitized survival curves from relevant clinical trials, we identified 8.6-13.9% of PFS times that should be categorized as deaths, allowing for estimation of cumulative incidence functions. Models assuming within-patient independence overestimated OS time, corrected with our calibration approach. Correction resulted in meaningful changes in the difference in QALYs between treatment strategies (0.07 vs 0.15) and the ICER (-\76,836/QALY vs -21,030/QALY). Conclusions. Microsimulation models can be successfully used to study cost-effectiveness of therapy sequences, taking care to account correctly for within-patient dependence.

  • 5 authors
·
Oct 10, 2022

How life-table right-censoring affected the Brazilian Social Security Factor: an application of the gamma-Gompertz-Makeham model

Automatic Adjustment Mechanisms (AAM) are legal instruments that help social security systems respond to demographic and economic changes. In Brazil, the Social Security Factor (SSF) was introduced in the late 1990s as an AAM to link retirement benefits to life expectancy at the retirement age, with the hope of promoting contributory justice and discouraging early retirement. Recent research has highlighted the limitations of right-censored life tables, such as those used in Brazil. It has recommended using the gamma-Gompertz-Makeham (GGM) model to estimate adult and old-age mortality. This study investigated the impact of right-censoring on the SSF by comparing the official SSF and other social security metrics with a counterfactual scenario computed based on fitted GGM models. The results indicate that from 2004 to 2012, official life tables may have negatively impacted retirees' income, particularly for those who delayed their retirement. Furthermore, the GGM-fitted models' life expectancies had more stable paths over time, which could have helped with long-term planning. This study's findings are significant for policymakers as they highlight the importance of using appropriate mortality metrics in AAMs to ensure accurate retirement benefit payments. They also underscore the need to consider the potential impacts of seemingly innocuous hypotheses on public action outcomes. Overall, this study provides valuable insights for public planners and policymakers looking to enhance the effectiveness and fairness of social security systems.

  • 3 authors
·
Sep 11, 2023

Predicting life satisfaction using machine learning and explainable AI

Life satisfaction is a crucial facet of human well-being. Hence, research on life satisfaction is incumbent for understanding how individuals experience their lives and influencing interventions targeted at enhancing mental health and well-being. Life satisfaction has traditionally been measured using analog, complicated, and frequently error-prone methods. These methods raise questions concerning validation and propagation. However, this study demonstrates the potential for machine learning algorithms to predict life satisfaction with a high accuracy of 93.80% and a 73.00% macro F1-score. The dataset comes from a government survey of 19000 people aged 16-64 years in Denmark. Using feature learning techniques, 27 significant questions for assessing contentment were extracted, making the study highly reproducible, simple, and easily interpretable. Furthermore, clinical and biomedical large language models (LLMs) were explored for predicting life satisfaction by converting tabular data into natural language sentences through mapping and adding meaningful counterparts, achieving an accuracy of 93.74% and macro F1-score of 73.21%. It was found that life satisfaction prediction is more closely related to the biomedical domain than the clinical domain. Ablation studies were also conducted to understand the impact of data resampling and feature selection techniques on model performance. Moreover, the correlation between primary determinants with different age brackets was analyzed, and it was found that health condition is the most important determinant across all ages. This study demonstrates how machine learning, large language models and XAI can jointly contribute to building trust and understanding in using AI to investigate human behavior, with significant ramifications for academics and professionals working to quantify and comprehend subjective well-being.

  • 5 authors
·
Oct 17, 2025

A Flexible Parametric Modelling Framework for Survival Analysis

We introduce a general, flexible, parametric survival modelling framework which encompasses key shapes of hazard function (constant, increasing, decreasing, up-then-down, down-then-up), various common survival distributions (log-logistic, Burr type XII, Weibull, Gompertz), and includes defective distributions (i.e., cure models). This generality is achieved using four basic distributional parameters: two scale-type parameters and two shape parameters. Generalising to covariate dependence, the scale-type regression components correspond to accelerated failure time (AFT) and proportional hazards (PH) models. Therefore, this general formulation unifies the most popular survival models which allows us to consider the practical value of possible modelling choices for survival data. Furthermore, in line with our proposed flexible baseline distribution, we advocate the use of multi-parameter regression in which more than one distributional parameter depends on covariates - rather than the usual convention of having a single covariate-dependent (scale) parameter. While many choices are available, we suggest introducing covariates through just one or other of the two scale parameters, which covers AFT and PH models, in combination with a `power' shape parameter, which allows for more complex non-AFT/non-PH effects, while the other shape parameter remains covariate-independent, and handles automatic selection of the baseline distribution. We explore inferential issues in simulations, both with and without a covariate, with particular focus on evidence concerning the need, or otherwise, to include both AFT and PH parameters. We illustrate the efficacy of our modelling framework by investigating differences between treatment groups using data from a lung cancer study and a melanoma study. Censoring is accommodated throughout.

  • 3 authors
·
Jan 10, 2019

The Gini-Bayes Connection: The CAP Slope as Bayes' Theorem, with Applications to Weight of Evidence, Somers' D, and Calibration

The probabilistic reading of the cumulative accuracy profile (CAP) has a long industry lineage. Falkenstein, Boral and Carty (2000) state, in discrete form, that the default rate at a score percentile equals the portfolio average rate times the local slope of the power curve; van der Burgt (2008, 2019) formalizes this as the continuous identity p(Dmid x) = p_D, dy/dx and imports the continuous form as a working fact; Tasche (2009) analyzes the resulting calibration method; Voloshyn and Voloshyn (2023) substitute Bayes' theorem, f(xmid D)=p(Dmid x) f(x)/p_D, into the area integral and write the Gini as a functional of the calibration curve. The slope itself is already in the lineage (van der Burgt's dy/dx is the ratio of the two cumulative differentials), but it enters as a cited working fact, never as Bayes' theorem. We make that identification explicit and draw out its consequences. First, the CAP slope is Bayes' theorem in cumulative coordinates: the standardized PD it recovers is the posterior probability rescaled by the prior. The weight of the paper then falls on two results this reading unlocks. The odds form places the weight of evidence (the log of the likelihood ratio, i.e. the Bayes factor) and the information value inside one geometry (the weight of evidence at a point is the log of the ratio of the "bad" and "good" CAP slopes). The accuracy ratio, Somers' D_{xy}, and the Gini (2A-1)/(1-p_D) are revealed as one number computed three ways. Run in comparison mode (realized outcomes against model claims), the same identity recovers the reliability diagram in cumulative coordinates, with the sign of the gap between the empirical and model-implied Gini coefficients as a calibration diagnostic. A worked five-band example carries every identity in discrete form, and a kernel-density example extends them to the continuous case.

  • 1 authors
·
Jun 15

Temporal-spatial Correlation Attention Network for Clinical Data Analysis in Intensive Care Unit

In recent years, medical information technology has made it possible for electronic health record (EHR) to store fairly complete clinical data. This has brought health care into the era of "big data". However, medical data are often sparse and strongly correlated, which means that medical problems cannot be solved effectively. With the rapid development of deep learning in recent years, it has provided opportunities for the use of big data in healthcare. In this paper, we propose a temporal-saptial correlation attention network (TSCAN) to handle some clinical characteristic prediction problems, such as predicting death, predicting length of stay, detecting physiologic decline, and classifying phenotypes. Based on the design of the attention mechanism model, our approach can effectively remove irrelevant items in clinical data and irrelevant nodes in time according to different tasks, so as to obtain more accurate prediction results. Our method can also find key clinical indicators of important outcomes that can be used to improve treatment options. Our experiments use information from the Medical Information Mart for Intensive Care (MIMIC-IV) database, which is open to the public. Finally, we have achieved significant performance benefits of 2.0\% (metric) compared to other SOTA prediction methods. We achieved a staggering 90.7\% on mortality rate, 45.1\% on length of stay. The source code can be find: https://github.com/yuyuheintju/TSCAN.

  • 6 authors
·
Jun 2, 2023