We consider the optimal decision-making problem in a primary sample of interest with multiple auxiliary sources available. The outcome of interest is limited in the sense that it is only observed in the primary sample. In reality, such multiple data sources may belong to heterogeneous studies and thus cannot be combined directly. This paper proposes a new framework to handle heterogeneous samples and address the limited outcome simultaneously through a novel calibrated optimal decision-making method, by leveraging the common intermediate outcomes in multiple data sources. Specifically, our method allows the baseline covariates across different samples to have either homogeneous or heterogeneous distributions. Under the equal conditional means of intermediate outcomes in different samples given baseline covariates and the treatment information, we show that the proposed estimator of the conditional mean outcome is asymptotically normal and more efficient than using the primary sample solely. Extensive experiments on simulated datasets demonstrate empirical validity and improved efficiency using our approach, followed by a real application to electronic health records.
Consider sensitivity analysis for estimating average treatment effects under unmeasured confounding, assumed to satisfy a marginal sensitivity model. At the population level, we provide new representations for the sharp population bounds and doubly robust estimating functions, recently derived by Dorn, Guo, and Kallus. We also derive new, relaxed population bounds, depending on weighted linear outcome quantile regression. At the sample level, we develop new methods and theory for obtaining not only doubly robust point estimators for the relaxed population bounds with respect to misspecificatio…
We evaluated the implications of different approaches to characterize uncertainty of calibrated parameters of stochastic decision models (DMs) in the quantified value of such uncertainty in decision making. We used a microsimulation DM of colorectal cancer (CRC) screening to conduct a cost-effectiveness analysis (CEA) of a 10-year colonoscopy screening. We calibrated the natural history model of CRC to epidemiological data with different degrees of uncertainty and obtained the joint posterior distribution of the parameters using a Bayesian approach. We conducted a probabilistic sensitivity ana…
Faculty of Health Sciences, University of Maribor（马里博尔大学健康科学学院）
Machine Learning (ML) models are often complex and difficult to interpret due to their 'black-box' characteristics. Interpretability of a ML model is usually defined as the degree to which a human can understand the cause of decisions reached by a ML model. Interpretability is of extremely high importance in many fields of healthcare due to high levels of risk related to decisions based on ML models. Calibration of the ML model outputs is another issue often overlooked in the application of ML models in practice. This paper represents an early work in examination of prediction model calibratio…
Personalized optimal decision making, finding the optimal decision rule (ODR) based on individual characteristics, has attracted increasing attention recently in many fields, such as education, economics, and medicine. Current ODR methods usually require the primary outcome of interest in samples for assessing treatment effects, namely the experimental sample. However, in many studies, treatments may have a long-term effect, and as such the primary outcome of interest cannot be observed in the experimental sample due to the limited duration of experiments, which makes the estimation of ODR imp…
Which Regularized Propensity-Score and Doubly Robust Methods Are Best Calibrated When Exposures or Outcomes Are Rare? A Plasmode Study of Proxy-Based Confounding Adjustment
School of Population and Public Health, University of British Columbia（英属哥伦比亚大学公共卫生与人口健康学院）
University of California Irvine（加州大学欧文分校）；London School of Economics and Political Science（伦敦政治经济学院）；North Carolina State University（北卡罗来纳州立大学）
An individualized decision rule (IDR) is a decision function that assigns each individual a given treatment based on his/her observed characteristics. Most of the existing works in the literature consider settings with binary or finitely many treatment options. In this paper, we focus on the continuous treatment setting and propose a jump interval-learning to develop an individualized interval-valued decision rule (I2DR) that maximizes the expected outcome. Unlike IDRs that recommend a single treatment, the proposed I2DR yields an interval of treatment options for each individual, making it mo…
The George Washington University（乔治华盛顿大学）；University of Southern California（南加利福尼亚大学）；University of North Carolina（北卡罗来纳大学）
Recent exploration of optimal individualized decision rules (IDRs) for patients in precision medicine has attracted a lot of attention due to the heterogeneous responses of patients to different treatments. In the existing literature of precision medicine, an optimal IDR is defined as a decision function mapping from the patients' covariate space into the treatment space that maximizes the expected outcome of each individual. Motivated by the concept of Optimized Certainty Equivalent (OCE) introduced originally in \cite{ben1986expected} that includes the popular conditional-value-of risk (CVaR…
Cohort-based enrollment can slow down dose-finding trials since the outcomes of the previous cohort must be fully evaluated before the next cohort can be enrolled. This results in frequent suspension of patient enrollment. The issue is exacerbated in recent immune-oncology trials where toxicity outcomes can take a long time to observe. We propose a novel phase I design, the probability-of-decision toxicity probability interval (PoD-TPI) design, to accelerate phase I trials. PoD-TPI enables dose assignment in real-time in the presence of pending toxicity outcomes. With uncertain outcomes, the d…
A pressing challenge in medical research is to identify optimal treatments for individual patients. This is particularly challenging in mental health settings where mean responses are often similar across multiple treatments. For example, the mean longitudinal trajectories for patients treated with an active drug and placebo may be very similar but different treatments may exhibit distinctly different individual trajectory shapes. Most precision medicine approaches using longitudinal data often ignore information from the longitudinal data structure. This paper investigates a powerful precisio…
A Causal Inference Approach for Evaluating Diagnostic Tests and AI-Enabled Medical Devices: From Effect Modification to Information-Augmented Decision-Making
Division of Biostatistics, University of California, Berkeley（加州大学伯克利分校生物统计系）
Predicting risks of chronic diseases has become increasingly important in clinical practice. When a prediction model is developed in a given source cohort, there is often a great interest to apply the model to other cohorts. However, due to potential discrepancy in baseline disease incidences between different cohorts and shifts in patient composition, the risk predicted by the original model often under- or over-estimates the risk in the new cohort. The remedy of such a poorly calibrated prediction is needed for proper medical decision-making. In this article, we assume the relative risks of …
In many important applications of precision medicine, the outcome of interest is time to an event (e.g., death, relapse of disease) and the primary goal is to identify the optimal individualized decision rule (IDR) to prolong survival time. Existing work in this area have been mostly focused on estimating the optimal IDR to maximize the We propose a new robust framework for estimating an optimal static or dynamic IDR with time-to-event outcomes based on an easy-to-interpret quantile criterion. The new method does not need to specify an outcome regression model and is robust for heavy-tailed di…
North Carolina State University（北卡罗来纳州立大学）；University of Waterloo（滑铁卢大学）
Dynamic treatment regimes operationalize the clinical decision process as a sequence of functions, one for each clinical decision, where each function takes as input up-to-date patient information and gives as output a single recommended treatment. Current methods for estimating optimal dynamic treatment regimes, for example Q-learning, require the specification of a single outcome by which the `goodness' of competing dynamic treatment regimes are measured. However, this is an over-simplification of the goal of clinical decision making, which aims to balance several potentially competing outco…
Choosing with unknown causal information: Action-outcome probabilities for decision making can be grounded in causal models
FSG NeuroInformatics, Faculty of Computer Science, University of Vienna（维也纳大学计算机科学系神经信息学研究所）；Halicioglu Data Science Institute and Department of Philosophy, University of California, San Diego（加州大学圣地亚哥分校Halicioglu数据科学研究所和哲学系）
With the emergence of precision medicine, estimating optimal individualized decision rules (IDRs) has attracted tremendous attention in many scientific areas. Most existing literature has focused on finding optimal IDRs that can maximize the expected outcome for each individual. Motivated by complex individualized decision making procedures and the popular conditional value at risk (CVaR) measure, we propose a new robust criterion to estimate optimal IDRs in order to control the average lower tail of the individuals' outcomes. In addition to improving the individualized expected outcome, our p…
Conditional power calculations are frequently used to guide the decision whether or not to stop a trial for futility or to modify planned sample size. These ignore the information in short-term endpoints and baseline covariates, and thereby do not make fully efficient use of the information in the data. We therefore propose an interim decision procedure based on the conditional power approach which exploits the information contained in baseline covariates and short-term outcomes. We will realise this by considering the estimation of the treatment effect at the interim analysis as a missing dat…
Trial-based economic evaluations are typically performed on cross-sectional variables, derived from the responses for only the completers in the study, using methods that ignore the complexities of utility and cost data (e.g. skewness and spikes). We present an alternative and more efficient Bayesian parametric approach to handle missing longitudinal outcomes in economic evaluations, while accounting for the complexities of the data. We specify a flexible parametric model for the observed data and partially identify the distribution of the missing data with partial identifying restrictions and…
Impact of clinical decision support systems (cdss) on clinical outcomes and healthcare delivery in low- and middle-income countries: protocol for a systematic review and meta-analysis
ICMR National Institute for Research in Digital Health & Data Science（印度国家数字健康与数据科学研究所）；ICMR Headquarters（印度国家医学研究委员会总部）；ICMR-RMRC Bhubaneshwar（印度国家医学研究委员会比哈尔邦研究中心）
