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.
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 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.
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.
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 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.
Shared Keyboard design improves phase I clinical trials by borrowing information across doses.
problem Interim decisions based on current dose data may overlook signals from neighboring doses.
method Bayesian model-assisted design using Beta kernel process with kernel-weighted pseudo-counts.
result Significant improvements in identifying maximum tolerated dose and safety.
We study the problem of finding the optimal dosage in early stage clinical trials through the multi-armed bandit lens. We advocate the use of the Thompson Sampling principle, a flexible algorithm that can accommodate different types of monotonicity assumptions on the toxicity and efficacy of the doses. For the simplest…
Proposes a two-stage method for estimating heterogeneous treatment effects using gradient boosting trees.
problem Estimating heterogeneous treatment effects in randomized clinical trials with high-dimensional predictive markers.
method Two-stage statistical learning procedure using gradient boosting trees (XGBoost) to estimate main effects and HTE.
result Improves efficiency in estimating heterogeneous treatment effects through nonparametric function estimation.
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.
Accurately predicting drug responses to cancer is an important problem hindering oncologists' efforts to find the most effective drugs to treat cancer, which is a core goal in precision medicine. The scientific community has focused on improving this prediction based on genomic, epigenomic, and proteomic datasets measu…
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.
Different aspects of a clinical sample can be revealed by multiple types of omics data. Integrated analysis of multi-omics data provides a comprehensive view of patients, which has the potential to facilitate more accurate clinical decision making. However, omics data are normally high dimensional with large number of …
Proposes a new model to analyze CT scans for lung cancer patients.
problem Analyzing survival risks of lung cancer patients using CT scans.
method Penalized Deep Partially Linear Cox Model (Penalized DPLC) incorporating SCAD penalty and deep neural network.
result The model effectively selects important texture features and estimates nonparametric components.
SDF-Bayes finds safe drug combinations safely, balancing optimism and caution.
problem Finding safe drug combinations in clinical trials with multiple drugs and patient heterogeneity.
method SDF-Bayes uses Bayesian statistics to choose the most likely MTD while ensuring safety constraints.
result SDF-Bayes outperforms existing methods in both accuracy and safety for drug combination trials.
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.
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.
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 algorithms find all ε-good arms in stochastic bandits.
problem Finding all arms with means above a specified threshold in stochastic bandits.
method Two algorithms introduced to identify all ε-good arms.
result Demonstrated great empirical performance on large datasets.
Skin cancer is one of the most common types of cancer around the world. For this reason, over the past years, different approaches have been proposed to assist detect it. Nonetheless, most of them are based only on dermoscopy images and do not take into account the patient clinical information. In this work, first, we …
Study develops a new algorithm for assessing clinical trial abstracts.
problem Limited tools for assessing qualitative statements in clinical research.
method Developed a three-class sentiment classification algorithm using BERT model.
result Algorithm achieved 91.3% classification accuracy and 0.92 macro F1-Score.
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.
Discusses handling intercurrent events in clinical trials with time-to-event outcomes.
problem Handling intercurrent events in clinical trials with time-to-event outcomes.
method Defines estimands and six ICE handling strategies, including new competing-risk strategy.
result Novel methods for handling intercurrent events in clinical trials with time-to-event outcomes.
Copula-based fusion improves breast cancer risk stratification.
problem Combining clinical and genomic risk scores using simple rules fails to capture their joint relationship.
method Used copulas to model the joint relationship between clinical and genomic risk scores.
result Copula-based fusion improves risk stratification, identifying subgroups with the worst prognosis.
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 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.
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.
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 medical research facilitates to acquire a diverse type of data from the same individual for particular cancer. Recent studies show that utilizing such diverse data results in more accurate predictions. The major challenge faced is how to utilize such diverse data sets in an effective way. In this paper, we introduc…
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…
Doctor2Vec learns doctor representations from EHRs for better clinical trial recruitment.
problem Identifying the right doctors for clinical trials based on EHR data and trial descriptions.
method Dynamic Memory Network with attention mechanism to learn doctor and trial representations.
result Improved performance by up to 8.7% in PR-AUC on real-world trials and EHR data.
New method uses probabilistic independence to discover disease signatures from medical records.
problem Insufficiently precise diagnosis of clinical disease leading to treatment failures.
method Unsupervised machine learning using probabilistic independence to disentangle disease patterns.
result Inferred 2000 clinical disease signatures from medical records, improving cancer prediction.
Study adaptive clinical trial methods for identifying patient subpopulations with treatment benefit.
problem Adaptive identification of patient subpopulations with treatment benefit in clinical trials.
method Proposes AdaGGI and AdaGCPI meta-algorithms for subpopulation construction.
result Empirical investigation of AdaGGI and AdaGCPI performance across various simulation scenarios.
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.
DWTS uses observational data to improve clinical trial efficiency.
problem Lack of definitive conclusions from randomized clinical trials due to insufficient patient cohorts and confounding biases.
method DWTS combines observational data with randomized clinical trials using Doubly Debiased LASSO (DDL) to identify reliable covariates.
result DWTS reduces cumulative regret in clinical trials compared to standard methods.
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…
Biotech IPOs in Q1 2021: advanced degrees, clinical trials, and IP key.
problem Identifying traits of biotech startups that go public.
method Database of biotech IPOs, analysis of leadership, technology, clinical trials, and financing.
result Advanced degrees, clinical trials, and IP are important for biotech startups.
The incidence of malignant melanoma continues to increase worldwide. This cancer can strike at any age; it is one of the leading causes of loss of life in young persons. Since this cancer is visible on the skin, it is potentially detectable at a very early stage when it is curable. New developments have converged to ma…
New method targets relative risk heterogeneity in clinical trials.
problem Identifying treatment effects across subgroups with absolute risk differences.
method Modified causal forests using a novel node-splitting procedure based on relative risk.
result Relative risk causal forests can capture heterogeneity not detected by absolute risk methods.
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.
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.
We analyze the sample complexity of the thresholding bandit problem, with and without the assumption that the mean values of the arms are increasing. In each case, we provide a lower bound valid for any risk δ and any δ-correct algorithm; in addition, we propose an algorithm whose sample complexity is of the same o…
Develops a TL framework for estimating RMST difference in clinical trials.
problem Estimating RMST difference in clinical trials with time-to-event outcomes.
method Targeted learning (TL) framework using pseudo-observations and copy reference (CR) approach for sensitivity analysis.
result Demonstrated the effectiveness of the TL framework using real data.
The consistency of doubly robust estimators relies on consistent estimation of at least one of two nuisance regression parameters. In moderate to large dimensions, the use of flexible data-adaptive regression estimators may aid in achieving this consistency. However, n1/2-consistency of doubly robust estimators is…
The stochastic multi-armed bandit problem is a well-known model for studying the exploration-exploitation trade-off. It has significant possible applications in adaptive clinical trials, which allow for dynamic changes in the treatment allocation probabilities of patients. However, most bandit learning algorithms are d…
CRBM generates digital twins for MS patients, aiding in disease progression analysis.
problem Characterizing and analyzing disease progression in MS patients.
method Unsupervised machine learning with Conditional Restricted Boltzmann Machines (CRBMs).
result Generated digital twins are statistically indistinguishable from actual subjects.
FRESH combines patient-level and aggregate-level data for better clinical decision making.
problem Combining patient-level and aggregate-level data for clinical decision making.
method FRESH method that re-calibrates a patient-level model to match specified aggregate statistics.
result Unified data-efficient model for clinical decision making.
Proposes dynamic borrowing method for historical data in clinical trials.
problem Insufficient statistical power in rare and pediatric disease clinical trials.
method Dynamic borrowing method based on frequentist approach using similarity measures.
result Demonstrates usefulness of dynamic borrowing in reanalyzing clinical trial data.