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While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…
Stroke is the second most frequent cause of death world wide with an annual mortality of around 5.5 million. Recurrence rates of stroke are between 5 and 25% in the first year. As mortality rates for relapses are extraordinarily high (40%)…
The primary object of a phase I clinical trial is to determine the maximum tolerated dose (MTD). Typically, the MTD is identified using a dose-escalation study, where initial subjects are treated at the lowest dose level and subsequent…
Longitudinal observational patient data can be used to investigate the causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for controlling for the time-dependent confounding that…
Introduction: There have been many warnings that inappropriate dichotomisation of results into positive or negative, high, or normal etc., during medical research could be very damaging. The aim of this paper is to argue that this is the…
Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly…
Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…
Machine learning (ML) has shown great promise for revolutionizing a number of areas, including healthcare. However, it is also facing a reproducibility crisis, especially in medicine. ML models that are carefully constructed from and…
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…
Advances in wearable technologies and health interventions delivered by smartphones have greatly increased the accessibility of mobile health (mHealth) interventions. Micro-randomized trials (MRTs) are designed to assess the effectiveness…
Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…
Treatment switching in a randomized controlled trial is said to occur when a patient randomized to one treatment arm switches to another treatment arm during follow-up. This can occur at the point of disease progression, whereby patients in…
Recursive learning -- where models are trained on data generated by previous versions of themselves -- is increasingly common in large language models, autonomous agents, and self-supervised systems. However, standard performance metrics…
Multiple sclerosis is a disease that affects the brain and spinal cord, it can lead to severe disability and has no known cure. The majority of prior work in machine learning for multiple sclerosis has been centered around using Magnetic…
Stereotactic radiotherapy (SRT) is one of the most important treatment for patients with brain metastases (BM). Conventionally, following SRT patients are monitored by serial imaging and receive salvage treatments in case of significant…
BACKGROUND: Randomized controlled trials (RCTs) are the gold standard design of clinical research to assess interventions. However, RCTs cannot always be applied for practical or ethical reasons. To investigate the current practices in rare…
Objective Alzheimer disease (AD) is the most common cause of dementia, a syndrome characterized by cognitive impairment severe enough to interfere with activities of daily life. We aimed to conduct a systematic literature review (SLR) of…
Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…
Patients with breast cancer tend to die from other diseases, so for studies that focus on breast cancer, a competing risks model is more appropriate. Considering subdistribution hazard ratio, which is used often, limited to model…
Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…