Simpson's paradox can invalidate the results of medical trials if an unobserved variable is considered.
problem The validity of medical trial results can be invalidated by an unobserved confounding variable.
method Simulated controlled trials to demonstrate the paradox.
result The results of a trial can be reversed by including an unobserved confounding variable.
Efficiently find near-optimal medical treatments with less trial and error.
problem Finding effective medical treatments through trial and error.
method Formalizes the problem, uses a causal inference framework, and proposes model-based dynamic programming and greedy algorithms.
result Our methods compare favorably to model-free reinforcement learning, offering a more transparent trade-off between search time and treatment efficacy.
Enhances clinical trial predictions by quantifying uncertainty.
problem Uncertainty in medical diagnosis and drug discovery predictions.
method Selective classification integrated with Hierarchical Interaction Network (HINT).
result Significant improvement in PR-AUC, F1, ROC-AUC, and overall accuracy.
Causal analysis reveals regional discrepancies in TOPCAT trial results.
problem Inconclusive results in TOPCAT trial for heart failure treatment.
method Causal discovery methods with domain knowledge integration.
result Significant causal effects shown for some subgroups globally.
C3T-Budget optimizes drug efficacy in dose-finding trials with budget and safety constraints.
problem Heterogeneous patient populations and budget constraints make dose-finding clinical trials challenging.
method Contextual constrained clinical trial algorithm that maximizes drug efficacy while learning subgroup responses.
result Demonstrates efficient budget usage and balanced learning-treatment trade-off in simulated trials.
The treatment effects of medications play a key role in guiding medical prescriptions. They are usually assessed with randomized controlled trials (RCTs), which are expensive. Recently, large-scale electronic health records (EHRs) have become available, opening up new opportunities for more cost-effective assessments. …
When devising a course of treatment for a patient, doctors often have little quantitative evidence on which to base their decisions, beyond their medical education and published clinical trials. Stanford Health Care alone has millions of electronic medical records (EMRs) that are only just recently being leveraged to i…
Mild cognitive impairment (MCI) is a prodromal phase in the progression from normal aging to dementia, especially Alzheimers disease. Even though there is mild cognitive decline in MCI patients, they have normal overall cognition and thus is challenging to distinguish from normal aging. Using transcribed data obtained …
New framework for adaptive clinical trials to address real-world challenges.
problem Real-world challenges in post-regulatory clinical trials.
method RFAN framework integrating regulatory constraints and treatment policy value.
result Empirical evaluation of RFAN's performance.
Deep neural network architectures have traditionally been designed and explored with human expertise in a long-lasting trial-and-error process. This process requires huge amount of time, expertise, and resources. To address this tedious problem, we propose a novel algorithm to optimally find hyperparameters of a deep n…
Novel strategy benchmarks observational studies against randomized trials.
problem Benchmarking observational studies for treatment effect bias.
method Statistical test for null hypothesis of treatment effect difference.
result Valid lower bound on maximum bias strength for any subgroup.
We consider the problem of constructing diffusion operators high dimensional data X to address counterfactual functions F, such as individualized treatment effectiveness. We propose and construct a new diffusion metric KF that captures both the local geometry of X and the directions of variance of F. The res…
TrialGraph uses graph machine learning to improve clinical trial design and predict side effects.
problem Complexity and cost in clinical trials hinder drug development.
method Curated clinical trial data set converted to graph-structured formats, applied graph machine learning algorithms.
result MetaPath2Vec algorithm performed exceptionally well, improving prediction accuracy.
Interventional cancer clinical trials are generally too restrictive, and some patients are often excluded on the basis of comorbidity, past or concomitant treatments, or the fact that they are over a certain age. The efficacy and safety of new treatments for patients with these characteristics are, therefore, not defin…
The paper evaluates index-based allocation policies using data from randomized control trials.
problem Evaluating index-based allocation policies in resource-scarce scenarios.
method Using data from randomized control trials, the paper introduces an efficient estimator and methods for computing asymptotically correct confidence intervals.
result Valid statistical conclusions can be drawn for index-based allocation policies.
Framework harmonizes EHR data across institutions for better analysis.
problem Heterogeneity of medical codes and terminologies hinder EHR data analysis.
method MASH (Multi-source Automated Structured Hierarchy) uses neural optimal transport and learned hyperbolic embeddings to align and structure EHR data.
result MASH generates interpretable hierarchical graphs for unstructured local laboratory codes.
Develops a two-stage conformal prediction method for Parkinson's disease medication needs.
problem Heterogeneous disease progression and treatment response in Parkinson's Disease.
method Two-stage conformal prediction framework with statistical guarantees.
result Quantifies uncertainty in medication needs predictions, improving clinical trust and quality of life.
Estimates causal effects from patient trajectories using DeepACE model.
problem Estimating causal effects from observational data in medical practice.
method DeepACE model using iterative G-computation formula and sequential targeting procedure.
result DeepACE achieves state-of-the-art performance in estimating time-varying ACEs.
We present PubMed 200k RCT, a new dataset based on PubMed for sequential sentence classification. The dataset consists of approximately 200,000 abstracts of randomized controlled trials, totaling 2.3 million sentences. Each sentence of each abstract is labeled with their role in the abstract using one of the following …
Confidentiality of patient information is an essential part of Electronic Health Record System. Patient information, if exposed, can cause a serious damage to the privacy of individuals receiving healthcare. Hence it is important to remove such details from physician notes. A system is proposed which consists of a deep…
New disease drug trials use ML to predict best therapy.
problem Developing new drugs faster than usual clinical trials.
method Use Neural Network to predict drug effects from patient data.
result Neural Network outperforms traditional placebo-controlled trials.
Treatment recommendations within Clinical Practice Guidelines (CPGs) are largely based on findings from clinical trials and case studies, referred to here as research studies, that are often based on highly selective clinical populations, referred to here as study cohorts. When medical practitioners apply CPG recommend…
MetaStackVis aids in choosing better metamodels for stacking ensembles.
problem Difficulty in selecting optimal metamodels for stacking ensembles.
method Interactive visualization tool to explore and compare different metamodels.
result Alternative metamodels significantly improve stacking ensemble performance.
Machine learning predicts patient recruitment for clinical trials.
problem Improving patient recruitment prediction for clinical trials.
method Machine learning methods applied to historical clinical trial data.
result Reduced prediction error compared to current industry standards.
Despite significant advances in artificial intelligence (AI) for computer vision, its application in medical imaging has been limited by the burden and limits of expert-generated labels. We used images from optical coherence tomography angiography (OCTA), a relatively new imaging modality that measures perfusion of the…
A novel validation method improves feature importance analysis in subject-specific ML models.
problem Limited effectiveness of general ML models trained on large datasets for individual patient outcomes.
method A novel validation approach using a general ML model with repeated trials and random seed variation.
result Consistent identification of key features at the subject level and improved group-level feature importance analysis.
AdaptiveNet tackles disease progression prediction in rheumatoid arthritis using deep neural networks.
problem Predicting disease progression in rheumatoid arthritis using clinical data.
method AdaptiveNet, a novel recurrent neural network architecture, that handles multiple lists of different events and missing data.
result AdaptiveNet outperforms classical baselines in disease progression prediction.
Proposes an interpretable machine learning framework for multi-arm HTE estimation.
problem Challenges in estimating heterogeneous treatment effects in multi-arm settings.
method Rule-based ensemble approach for HTE estimation in multi-arm trials.
result Achieved lower bias and higher estimation accuracy compared to existing methods.
New neural model improves uplift modeling accuracy.
problem Improving accuracy in uplift modeling for specific treatments.
method Developed a twin neural network architecture with a new loss function.
result Proposed model outperforms existing methods in simulations and real data.
Combines trial and observational data to improve policy evaluation.
problem External validity of randomized trial results in target populations.
method Uses covariate data to model trial sampling and certifies policy evaluations.
result Valid trial-based policy evaluations under model miscalibration.
Precision medicine is becoming a focus in medical research recently, as its implementation brings values to all stakeholders in the healthcare system. Various statistical methodologies have been developed tackling problems in different aspects of this field, e.g., assessing treatment heterogeneity, identifying patient …
MILCCI integrates labels across categories for better understanding of multi-trial data.
problem Understanding how labels encode multi-trial observations and disentangling their effects.
method Sparse per-trial decomposition leveraging label similarities within each category.
result MILCCI identifies interpretable components and integrates label information.
The paper compares methods for estimating heterogeneous treatment effects using multiple randomized trials.
problem Estimating heterogeneous treatment effects reliably and precisely with a single dataset is challenging.
method Non-parametric approaches for estimating heterogeneous treatment effects using data from multiple trials.
result Methods that directly allow for heterogeneity of the treatment effect across trials perform better than those that do not.
Massive electronic health records (EHRs) enable the success of learning accurate patient representations to support various predictive health applications. In contrast, doctor representation was not well studied despite that doctors play pivotal roles in healthcare. How to construct the right doctor representations? Ho…
New study shows non-adaptive trials can be outperformed by adaptive designs in treatment selection.
problem Determining the best allocation of resources in clinical trials.
method Analysis of batched arm elimination designs and comparison with completely randomized trials.
result Simple adaptive designs universally and strictly dominate non-adaptive completely randomized trials for at least three treatment arms.
New method detects biomarker-treatment interactions in clinical trials.
problem Detecting interactions between high-dimensional biomarkers and treatments in randomized trials.
method Two-stage penalized regression screening using ridge regression for multivariate screening.
result Ridge regression screening provides greater power than traditional methods in correlated data.
The personalization of treatment via bio-markers and other risk categories has drawn increasing interest among clinical scientists. Personalized treatment strategies can be learned using data from clinical trials, but such trials are very costly to run. This paper explores the use of active learning techniques to desig…
QR-learner estimates individual treatment effects using external data.
problem Limited power to detect individual treatment effects in randomized trials.
method Model-agnostic learner that estimates conditional average treatment effects (CATE) using external data.
result QR-learner reduces mean squared error and can recover true CATE.
Bayesian BIC for multi-trial data improves VAR model order selection.
problem Optimal VAR model order selection for multi-trial event-based data.
method Derive and apply Bayesian Information Criterion (BIC) for multi-trial ensemble data.
result Multi-trial BIC successfully recovers real model order and estimates small model order.
Optimum in Convex Hulls (OCH) generalizes clinical trial results to broader populations.
problem Clinical trials exclude confounding but limit recruitment; observational data are more inclusive but suffer from confounding.
method OCH uses convex hulls of conditional expectations or densities to approximate the true treatment effect from both observational and trial data.
result OCH estimates the treatment effect with state-of-the-art accuracy in terms of both expectations and densities.
The uncertainty measurement of classifiers' predictions is especially important in applications such as medical diagnoses that need to ensure limited human resources can focus on the most uncertain predictions returned by machine learning models. However, few existing uncertainty models attempt to improve overall predi…
The paper explores using historical data to improve clinical trial analysis by optimizing covariate weights.
problem Limited covariates in small clinical trials reduce the effectiveness of analysis.
method Leverage historical data to pre-specify covariate weights as a composite covariate.
result A composite covariate improves the cost/benefit ratio and reduces overfitting in small clinical trials.
SEEDA optimizes dose allocation in clinical trials to balance efficacy and safety.
problem Complex relationships between efficacy and toxicity in new drug trials.
method Adaptive clinical trial methodology that maximizes cumulative efficacy while ensuring safety constraints.
result SEEDA outperforms existing methods in finding optimal doses with higher success rates and fewer patients.
New methods improve subgroup analysis in trials with limited data.
problem Limited sample sizes in subgroup analyses of randomized controlled trials.
method Two TMLEs that borrow information from non-subgroup participants.
result Improved precision in subgroup-specific treatment effect estimates.
Syntax designs adaptive trials for subpopulations with potential benefits.
problem Identifying subpopulations with positive treatment effects in diverse patient populations.
method Adaptive patient recruitment and synthetic control estimation.
result Syntax outperforms conventional trial designs in identifying beneficial subpopulations.
Bayesian method improves clinical trial efficiency.
problem Increase treatment effect estimates in clinical trials.
method Combines prognostic covariate adjustment with a Bayesian framework.
result Substantial increase in statistical power with controlled type I error.
The P300 event-related potential (ERP), evoked in scalp-recorded electroencephalography (EEG) by external stimuli, has proven to be a reliable response for controlling a BCI. The P300 component of an event related potential is thus widely used in brain-computer interfaces to translate the subjects' intent by mere thoug…
The study optimizes free trial lengths to boost subscriptions and consumer loyalty.
problem Optimizing free trial lengths to maximize customer acquisition and retention.
method A large-scale field experiment with personalized policy design and evaluation.
result Personalized free trial policies outperform uniform trial lengths.