Bayesian optimisation outperforms standard ML-II in small samples.
problem Standard ML-II fails in small-sample trials.
method Adopting fully Bayesian optimisation (FBO) as an alternative.
result FBO is more robust and practical than ML-II.
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48 results for “small-sample trials”
G-computation improves clinical trial power with machine learning.
problem Balancing prognostic factors in randomized trials to prevent near-confounders.
method G-computation with penalized models (Lasso, Elasticnet) and algorithm-based methods (neural network, SVM, super learner).
result G-computation with Elasticnet and splines reduces variance and increases power in RCTs.
Generative AI models improve clinical trial data by generating survival outcomes.
problem Generating valid survival outcomes for clinical trials with synthetic data.
method A variational autoencoder (VAE) that jointly generates mixed-type covariates and survival outcomes.
result The method outperforms GAN baselines on fidelity, utility, and privacy metrics.
Is Your Imitation Learning Policy Better than Mine? Policy Comparison with Near-Optimal Stoppingcs.RO
Paper proposes a sequential statistical test for comparing imitation learning policies with near-optimal stopping.
problem Challenges in rigorously comparing imitation learning policies due to small sample sizes and potential p-hacking.
method Sequential statistical test that adapts the number of trials based on intermediate results, achieving near-optimal stopping.
result Reduces the number of evaluation trials by up to 32% compared to state-of-the-art baselines, saving significant time and effort.
For machine learning task, lacking sufficient samples mean the trained model has low confidence to approach the ground truth function. Until recently, after the generative adversarial networks (GAN) had been proposed, we see the hope of small samples data augmentation (DA) with realistic fake data, and many works valid…
Personalized medicine seeks to identify the causal effect of treatment for a particular patient as opposed to a clinical population at large. Most investigators estimate such personalized treatment effects by regressing the outcome of a randomized clinical trial (RCT) on patient covariates. The realized value of the ou…
The diagnosis of Alzheimer's disease (AD) in routine clinical practice is most commonly based on subjective clinical interpretations. Quantitative electroencephalography (QEEG) measures have been shown to reflect neurodegenerative processes in AD and might qualify as affordable and thereby widely available markers to f…
Novel approach for SEM in small samples with .
problem Small sample size and issues in factor-based SEM.
method Reformulates covariance structure into self-covariance and cross-covariance, defines a feasible set with relative error constraint.
result Improved stability and directional information in small-sample settings.
Unified Bayesian model for multi-modal, small sample size biomedical data classification.
problem Classifying high-dimensional, multi-modal biomedical data with small sample sizes.
method Combines multi-modal data views into a latent space, prunes irrelevant features, and uses dual kernels for small sample size scenarios.
result Outperforms state-of-the-art models and identifies features aligned with existing markers.
FedFaiREE addresses fairness in decentralized learning with small samples.
problem Ensuring fairness in decentralized federated learning with limited data.
method FedFaiREE is a post-processing algorithm for distribution-free fair learning in decentralized settings with small samples.
result FedFaiREE provides theoretical guarantees for both fairness and accuracy in decentralized environments.
Generative Latent Implicit Conditional Optimization (GLICO) learns from small samples.
problem Learning from small labeled datasets.
method Generative Latent Implicit Conditional Optimization (GLICO) learns a latent space and generator from small labeled data.
result GLICO synthesizes new samples for every class using as few as 10 examples per class.
Algorithm recovers large causal tree from small samples.
problem Determining causal structure in large gene networks.
method Algorithm that recovers tree with high accuracy under mild conditions.
result High accuracy in recovering causal tree from small samples.
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.
Simple private estimators for mean and covariance outperform existing methods.
problem Private estimation of mean and covariance at small sample sizes.
method Differentially private estimators for multivariate sub-Gaussian data.
result Asymptotic error rates match theoretical bounds and outperform previous methods.
Proposes a new signal model for high-dimensional, small-sample-size data.
problem Signal detection in high-dimensional, small-sample-size datasets.
method Intrinsic signal model based on dynamical system assumption.
result Taguchi method effectively detects signals in the proposed model.
Study examines impact of missing data on multi-armed bandit algorithms.
problem Impact of missing data on performance of multi-armed bandit algorithms.
method Extensive simulation study of two-armed bandit algorithms with binary outcomes, considering different probabilities of missingness.
result Impact on performance varies depending on the balance between exploration and exploitation.
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.
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…
We present the expected values from p-value hacking as a choice of the minimum p-value among independents tests, which can be considerably lower than the "true" p-value, even with a single trial, owing to the extreme skewness of the meta-distribution. We first present an exact probability distribution (meta-distrib…
Algorithme EM régulariséstat.ML
Regularized EM algorithm improves clustering performance with small sample sizes.
problem Performance reduction in EM algorithm due to small sample size and poorly conditioned covariance matrices.
method Regularized EM algorithm that uses prior knowledge to ensure positive definiteness of covariance matrices.
result The regularized EM algorithm outperforms standard EM in clustering tasks with small sample sizes.
Adversarial training can hurt robust accuracy in small sample size scenarios.
problem Adversarial training improves test accuracy but may degrade robustness in limited data settings.
method Analyzes high-dimensional linear classification with noiseless observations, and observes perceptible attacks on image datasets.
result Adversarial training can negatively impact robust generalization in small sample size regimes.
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…
Robust estimation of heterogeneous treatment effects in randomized trials leveraging external datastat.ML
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.
Causal Analysis of the TOPCAT Trial: Spironolactone for Preserved Cardiac Function Heart Failurestat.ML
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.
Clinical trials in the medical domain are constrained by budgets. The number of patients that can be recruited is therefore limited. When a patient population is heterogeneous, this creates difficulties in learning subgroup specific responses to a particular drug and especially for a variety of dosages. In addition, pa…
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.
Towards Regulatory-Confirmed Adaptive Clinical Trials: Machine Learning Opportunities and Solutionsstat.ML
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.
Machine learning improves trial analysis precision by adjusting for prognostic variables.
problem Improving precision in randomized trial analyses using covariate adjustment.
method Targeted machine learning estimation (TMLE) with adaptive pre-specification.
result Maximized empirical efficiency through cross-validated variance minimization.
New methods improve causal inference generalization using trial and observational data.
problem Limited trial data makes generalizing causal inferences to target populations statistically infeasible.
method Develops algorithms that combine trial and observational data to estimate complex nuisance functions.
result Improves generalization of causal inferences when the additional observational study is high-quality.
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.
New algorithm improves source separation with multi-trial supervision.
problem Non-convex optimization and interpretability of independent components.
method Proximal gradient-type algorithm in invertible matrices with backpropagation for joint learning.
result Increased success rate of non-convex optimization and improved interpretability.
Study evaluates synthetic data augmentation for small datasets, highlighting inconsistencies in traditional metrics.
problem Inconsistent validation of synthetic data generated for small sample sizes.
method Proposes a normalized Bottleneck distance metric to evaluate synthetic tabular data.
result Common metrics like propensity scoring and MMD fail for small datasets, showing instability and high variability.
A novel dose-finding design for cancer clinical trials using level set estimation.
problem Finding the maximum tolerated dose (MTD) in phase I cancer clinical trials.
method Proposes a novel dose-finding design based on level set estimation (LSE) to determine the next dose.
result The proposed LSE design achieves higher accuracy in estimating the MTD and lower risk of overdosing compared to existing designs.
Automated machine learning aims to automate the whole process of machine learning, including model configuration. In this paper, we focus on automated hyperparameter optimization (HPO) based on sequential model-based optimization (SMBO). Though conventional SMBO algorithms work well when abundant HPO trials are availab…
Develops a method for manifold learning with small sample size datasets.
problem Improving manifold learning performance for multiple tasks with limited samples.
method Uses instance and model transfer to integrate manifold models from similar tasks.
result Successfully estimates manifolds with tiny sample sizes across multiple tasks.
Detection of interactions between treatment effects and patient descriptors in clinical trials is critical for optimizing the drug development process. The increasing volume of data accumulated in clinical trials provides a unique opportunity to discover new biomarkers and further the goal of personalized medicine, but…