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.
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.
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.
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.
Machine learning boosts RCT efficiency by controlling type I error and improving statistical power.
problem Improving statistical efficiency in RCTs with complex covariate adjustments.
method Machine learning-assisted adjustment under Rosenbaum's framework for exact tests.
result The proposed method robustly controls type I error and significantly boosts statistical efficiency.
Improves trial efficiency by adjusting for historical prognostic scores.
problem Reducing statistical uncertainty in randomized trial estimates.
method Linear covariate adjustment using a prognostic model trained on historical data.
result Prognostic covariate adjustment achieves minimum variance and reduces mean-squared error.
Adaptive Prespecification improves precision in randomized trials.
problem Selecting optimal covariates for precision in randomized trials.
method Adaptive Prespecification using V-fold cross-validation and influence curve-squared loss function.
result Substantial gains in precision, equivalent to 20-43% reductions in sample size for the same power.
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.
Study designs statistical inference for collaborative science teams.
problem Maintaining scientific rigor in distributed, collaborative research.
method Analyzes hypothesis testing with strategic agents and principals.
result Principal can design policies to control posterior probability of null.
New TTP framework fuses control arms while controlling Type-I error.
problem Bias in borrowing control data from previous trials.
method Kernel two-sample testing via MMD and equivalence testing.
result Higher power than standard TTP methods while maintaining error control.
We present the recent advances along with an error analysis of the IBM speaker recognition system for conversational speech. Some of the key advancements that contribute to our system include: a nearest-neighbor discriminant analysis (NDA) approach (as opposed to LDA) for intersession variability compensation in the i-…
WHOMP optimizes randomized controlled trials by minimizing subgroup bias.
problem Minimizing subgroup bias in randomized controlled trials.
method Wasserstein Homogeneity Partition (WHOMP) method.
result WHOMP optimally minimizes type I and type II errors in trials.
The change of two orders of magnitude in the 'new DCF' of NIST's SRE'10, relative to the 'old DCF' evaluation criterion, posed a difficult challenge for participants and evaluator alike. Initially, participants were at a loss as to how to calibrate their systems, while the evaluator underestimated the required number o…
We study the problem of using low computational cost to automate the choices of learners and hyperparameters for an ad-hoc training dataset and error metric, by conducting trials of different configurations on the given training data. We investigate the joint impact of multiple factors on both trial cost and model erro…
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.
We show how to compute the Bayes error-rate for speaker verifiers.
problem How many errors does a speaker verifier make in a hundred trials?
method We compute the Bayes error-rate using calibrated likelihood ratios and user-supplied prior probabilities.
result The Bayes error-rate is upper bounded by the minimum of EER, P, and 1-P.
Unified Bayesian framework improves clinical trial hypothesis testing.
problem Lack of transparency and inability to quantify evidence in traditional P-values.
method Interval null hypothesis framework combined with Bayes factor-based tests.
result Bayesian interval hypothesis testing ensures frequentist error control and interpretability.
Assessing heterogeneous treatment effects has become a growing interest in advancing precision medicine. Individualized treatment effects (ITE) play a critical role in such an endeavor. Concerning experimental data collected from randomized trials, we put forward a method, termed random forests of interaction trees (RF…
Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform randomization. We show that this procedure can be highly sub-optimal (in terms of learnin…
Privacy-preserving inference for clinical trials using differential privacy.
problem Balancing knowledge sharing and privacy in healthcare data.
method Differential privacy (DP) applied to log-linear belief updates in distributed settings.
result Differentially private, distributed inference methods outperform existing techniques.
Conventional inclusion criteria used in osteoarthritis clinical trials are not very effective in selecting patients who would benefit from a therapy being tested. Typically majority of selected patients show no or limited disease progression during a trial period. As a consequence, the effect of the tested treatment ca…
A-TMLE estimates ATE from RCT and RWD, achieving super-efficiency.
problem Estimating ATE from RCT and RWD data.
method Adaptive-TMLE framework for decomposing and estimating ATE.
result A-TMLE is root-n consistent and asymptotically normal, achieving super-efficiency.
Heart rate estimation from electrocardiogram signals is very important for the early detection of cardiovascular diseases. However, due to large individual differences and varying electrocardiogram signal quality, there does not exist a single reliable estimation algorithm that works well on all subjects. Every algorit…
Proposes a new Q-learning method for survival outcomes in clinical trials.
problem Incomplete follow-up data and nonlinear covariate effects in clinical trials.
method Combines Buckley-James boosting with flexible base learners for estimating optimal treatment regimes.
result Improves treatment decision accuracy and stability in longitudinal clinical trials.
New method calibrates heterogeneous treatment effect models.
problem Difficulty in estimating and calibrating heterogeneous treatment effects.
method Defined and proposed a robust estimator for HTE calibration, based on doubly robust treatment effect estimators.
result Proposed method evaluates calibration of learned HTE models, addressing overfitting and high-dimensionality.
Transforms any test into anytime-valid with sample savings.
problem Sequential data invalidates classical test guarantees.
method Predicts test outcomes to create anytime-valid stopping rules.
result Ensures Type-I error control and near-optimal power.
Suppose that we observe y∈Rf and X∈Rf×m in the following errors-in-variables model: \begin{eqnarray*} y & = & X_0 β^* + ε\\ X & = & X_0 + W \end{eqnarray*} where X0 is a f×m design matrix with independent subgaussian row vectors, ε∈Rf is a noise vector…
We present a novel method for variable selection in regression models when covariates are measured with error. The iterative algorithm we propose, MEBoost, follows a path defined by estimating equations that correct for covariate measurement error. Via simulation, we evaluated our method and compare its performance to …
Proposes a method to correct for covariate shift in meta-analysis of randomized trials.
problem Invalidation of standard IPD meta-analysis due to covariate shift across studies.
method Placebo-anchored transport framework that treats source-trial outcomes as proxy signals and target-trial placebo outcomes as gold labels.
result Yields target-identified effect estimates in connected targets and a principled screen--then--transport procedure in disconnected targets.
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.
Suppose that we observe y∈Rn and X∈Rn×m in the following errors-in-variables model: \begin{eqnarray*} y & = & X_0 β^* +ε\\ X & = & X_0 + W, \end{eqnarray*} where X0 is an n×m design matrix with independent subgaussian row vectors, ε∈Rn is a noise vecto…
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.
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…
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.
Neural networks have recently had a lot of success for many tasks. However, neural network architectures that perform well are still typically designed manually by experts in a cumbersome trial-and-error process. We propose a new method to automatically search for well-performing CNN architectures based on a simple hil…
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…
Observed events in recommendation are consequence of the decisions made by a policy, thus they are usually selectively labeled, namely the data are Missing Not At Random (MNAR), which often causes large bias to the estimate of true outcomes risk. A general approach to correct MNAR bias is performing small Randomized Co…
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…
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.
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.
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.
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…