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We intend to create a new risk assessment methodology that combines the best characteristics of both risk score and machine learning models. More specifically, we aim to develop a method that, besides having a good performance, offers a…

Machine Learning · Computer Science 2021-10-19 Francisco Valente , Jorge Henriques , Simão Paredes , Teresa Rocha , Paulo de Carvalho , João Morais

Clinical trials are critical for drug development. Constructing the appropriate eligibility criteria (i.e., the inclusion/exclusion criteria for patient recruitment) is essential for the trial's success. Proper design of clinical trial…

Computation and Language · Computer Science 2023-10-10 Zifeng Wang , Cao Xiao , Jimeng Sun

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

Artificial Intelligence · Computer Science 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Clinical trials are an integral component of medical research. Trials require careful design to, for example, maintain the safety of participants, use resources efficiently and allow clinically meaningful conclusions to be drawn. Adaptive…

Methodology · Statistics 2025-07-17 James M. McGree , Antony M. Overstall , Mark Jones , Robert K. Mahar

Achieving fault-tolerance will require a strong relationship between the hardware and the protocols used. Different approaches will therefore naturally have tailored proof-of-principle experiments to benchmark progress. Nevertheless,…

Quantum Physics · Physics 2024-05-28 Milan Liepelt , Tommaso Peduzzi , James R. Wootton

Clinical trials assessing neurological treatment are challenging due to the diversity of brain function, and the difficulty in quantifying it. Traditional treatment studies in epilepsy use seizure frequency as the primary outcome measure,…

Applications · Statistics 2025-12-02 Ian Miller , Ann Hyslop , Colin Decker

Clinical trials are an instrument for making informed decisions based on evidence from well-designed experiments. Here we consider adaptive designs mainly from the perspective of multi-arm Phase II clinical trials, in which one or more…

Methodology · Statistics 2021-08-31 Elja Arjas , Dario Gasbarra

Clinical trials are typically run in order to understand the effects of a new treatment on a given population of patients. However, patients in large populations rarely respond the same way to the same treatment. This heterogeneity in…

Machine Learning · Statistics 2024-02-12 Alihan Hüyük , Zhaozhi Qian , Mihaela van der Schaar

Externally controlled trials (ECTs) are increasingly used when randomized controls are infeasible, unethical, or insufficient, including applications in rare diseases, oncology, pediatrics, and post-approval effectiveness research. Although…

Randomized controlled trials (RCTs) in oncology often allow control group participants to crossover to experimental treatments, a practice that, while often ethically necessary, complicates the accurate estimation of long-term treatment…

Methodology · Statistics 2025-02-12 Harlan Campbell , Nicholas Latimer , Jeroen P Jansen , Shannon Cope

Considerations regarding clinical effectiveness and cost are essential in comparing the overall value of two treatments. There has been growing interest in methodology to integrate cost and effectiveness measures in order to inform policy…

Methodology · Statistics 2019-12-04 Andrew J. Spieker , Nicholas Illenberger , Jason A. Roy , Nandita Mitra

Adaptive experiment designs can dramatically improve statistical efficiency in randomized trials, but they also complicate statistical inference. For example, it is now well known that the sample mean is biased in adaptive trials.…

Machine Learning · Statistics 2021-02-16 Vitor Hadad , David A. Hirshberg , Ruohan Zhan , Stefan Wager , Susan Athey

Phase III randomized clinical trials play a monumentally critical role in the evaluation of new medical products. Because of the intrinsic nature of uncertainty embedded in our capability in assessing the efficacy of a medical product,…

Methodology · Statistics 2019-02-25 Changyu Shen , Xiaochun Li

In adaptive clinical trials, the conventional confidence interval (CI) for a treatment effect is prone to undesirable properties such as undercoverage and potential inconsistency with the final hypothesis testing decision. Accordingly, as…

The best evidence concerning comparative treatment effectiveness comes from clinical trials, the results of which are reported in unstructured articles. Medical experts must manually extract information from articles to inform…

Computation and Language · Computer Science 2022-01-11 Benjamin E. Nye , Jay DeYoung , Eric Lehman , Ani Nenkova , Iain J. Marshall , Byron C. Wallace

The ability to generalize experimental results from randomized control trials (RCTs) across locations is crucial for informing policy decisions in targeted regions. Such generalization is often hindered by the lack of identifiability due to…

Econometrics · Economics 2021-12-10 Xinkun Nie , Guido Imbens , Stefan Wager

We seek to understand the probability an individual benefits from treatment (PIBT), an inestimable quantity that must be bounded in practice. Given the innate uncertainty in the population-level bounds on PIBT, we seek to better understand…

Methodology · Statistics 2024-04-04 Gabriel Ruiz , Oscar Hernan Madrid Padilla

Platform trials are multi-arm designs that simultaneously evaluate multiple treatments for a single disease within the same overall trial structure. Unlike traditional randomized controlled trials, they allow treatment arms to enter and…

Methodology · Statistics 2024-12-16 Michele Santacatterina , Federico Macchiavelli Giron , Xinyi Zhang , Ivan Diaz

Matching patients to clinical trials demands a systematic and reasoned interpretation of documents which require significant expert-level background knowledge, over a complex set of well-defined eligibility criteria. Moreover, this…

Computation and Language · Computer Science 2024-10-01 Mael Jullien , Alex Bogatu , Harriet Unsworth , Andre Freitas

Clinical trials provide essential guidance for practicing Evidence-Based Medicine, though often accompanying with unendurable costs and risks. To optimize the design of clinical trials, we introduce a novel Clinical Trial Result Prediction…

Computation and Language · Computer Science 2020-10-13 Qiao Jin , Chuanqi Tan , Mosha Chen , Xiaozhong Liu , Songfang Huang