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An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

Two useful strategies to speed up drug development are to increase the patient accrual rate and use novel adaptive designs. Unfortunately, these two strategies often conflict when the evaluation of the outcome cannot keep pace with the…

Methodology · Statistics 2018-07-24 Ruitao Lin , Ying Yuan

Existing strategies for determining the optimal treatment or monitoring strategy typically assume unlimited access to resources. However, when a health system has resource constraints, such as limited funds, access to medication, or…

Applications · Statistics 2019-03-18 Ellen C Caniglia , Eleanor J Murray , Miguel A Hernan , Zach Shahn

We propose a population model for TB-HIV/AIDS coinfection transmission dynamics, which considers antiretroviral therapy for HIV infection and treatments for latent and active tuberculosis. The HIV-only and TB-only sub-models are analyzed…

Optimization and Control · Mathematics 2015-04-16 Cristiana J. Silva , Delfim F. M. Torres

Recently a new experimental approach, the hybrid experimental design (HED), was introduced to enable investigators to answer scientific questions about building behavioral interventions in which human-delivered and digital components are…

Methodology · Statistics 2026-02-26 Mengbing Li , Inbal Nahum-Shani , Walter Dempsey

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

The two-stage preference design (TSPD) enables the inference for treatment efficacy while allowing for incorporation of patient preference to treatment. It can provide unbiased estimates for selection and preference effects, where a…

Methodology · Statistics 2023-11-28 Ruyi Liu , Fan Li , Denise Esserman , Mary M. Ryan

Cluster or group randomized trials (CRTs) are increasingly used for both behavioral and system-level interventions, where entire clusters are randomly assigned to a study condition or intervention. Apart from the assigned cluster-level…

Methodology · Statistics 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

Methodology · Statistics 2020-06-19 Jie Zhu , Blanca Gallego

Motivation: HIV is difficult to treat because its virus mutates at a high rate and mutated viruses easily develop resistance to existing drugs. If the relationships between mutations and drug resistances can be determined from historical…

Quantitative Methods · Quantitative Biology 2019-07-08 Li Xing , Mary Lesperance , Xuekui Zhang

To optimize mobile health interventions and advance domain knowledge on intervention design, it is critical to understand how the intervention effect varies over time and with contextual information. This study aims to assess how a push…

Applications · Statistics 2024-10-22 Jiaxin Yu , Tianchen Qian

Randomized control trials (RCTs) have been the gold standard to evaluate the effectiveness of a program, policy, or treatment on an outcome of interest. However, many RCTs assume that study participants are willing to share their…

Applications · Statistics 2021-12-07 Manjusha Kancharla , Hyunseung Kang

Randomized experiments ensure robust causal inference that are critical to effective learning analytics research and practice. However, traditional randomized experiments, like A/B tests, are limiting in large scale digital learning…

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Efficient screening and early diagnosis of HIV are critical for reducing onward transmission. Although large scale laboratory testing is not feasible, the widespread adoption of Electronic Health Records (EHRs) offers new opportunities to…

Background: trials to identify the minimal effective treatment duration are needed in different therapeutic areas, including bacterial infections, TB and Hepatitis--C. However, standard non-inferiority designs have several limitations,…

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

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Each year, there are nearly 4 million youth experiencing homelessness (YEH) in the United States with HIV prevalence ranging from 3 to 11.5%. Peer change agent (PCA) models for HIV prevention have been used successfully in many populations,…

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Understanding treatment effect heterogeneity has become an increasingly popular task in various fields, as it helps design personalized advertisements in e-commerce or targeted treatment in biomedical studies. However, most of the existing…

Methodology · Statistics 2024-07-12 Waverly Wei , Xinwei Ma , Jingshen Wang

We propose a population model for HIV-TB co-infection dynamics by considering treatments for HIV infection, active tuberculosis and co-infection. The HIV only and TB only models are analyzed separately, as well as full model. The basic…

Populations and Evolution · Quantitative Biology 2018-08-10 Sachin Kumar , Shikha Jain

Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. The main goal is to make the process of introducing new medical interventions to patients more efficient by reducing the cost and the…

Machine Learning · Computer Science 2014-11-17 Ognjen Arandjelovic