相关论文: On Minimum Clinically Important Difference

IIt is known that a statistically significant treatment may not be clinically significant. A quantity that can be used to assess clinical significance is called the minimum clinically important difference (MCID), and inference on the MCID…

Inference on the minimum clinically important difference, or MCID, is an important practical problem in medicine. The basic idea is that a treatment being statistically significant may not lead to an improvement in the patients' well-being.…

In clinical research, the effect of a treatment or intervention is widely assessed through clinical importance, instead of statistical significance. In this paper, we propose a principled statistical inference framework to learning the…

This work is motivated by learning the individualized minimal clinically important difference, a vital concept to assess clinical importance in various biomedical studies. We formulate the scientific question into a high-dimensional…

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

Personalized medicine has become an important part of medicine, for instance predicting individual drug responses based on genomic information. However, many current statistical methods are not tailored to this task, because they overlook…

Small sample sizes in clinical studies arises from factors such as reduced costs, limited subject availability, and the rarity of studied conditions. This creates challenges for accurately calculating confidence intervals (CIs) using the…

The estimand framework is increasingly established to pose research questions in confirmatory clinical trials. In evidence synthesis, the uptake of estimands has been modest, and the PICO (Population, Intervention, Comparator, Outcome)…

Manifold learning using deep neural networks been shown to be an effective tool for building sophisticated prior image models that can be applied to noise reduction in low-dose CT. We propose a new iterative CT reconstruction algorithm,…

Early diagnosis of diseases holds the potential for deep transformation in healthcare by enabling better treatment options, improving long-term survival and quality of life, and reducing overall cost. With the advent of medical big data,…

The Minimum Covariance Determinant (MCD) approach robustly estimates the location and scatter matrix using the subset of given size with lowest sample covariance determinant. Its main drawback is that it cannot be applied when the dimension…

Estimating individualized treatment effects from longitudinal observational data is central to data-driven medicine, yet existing methods face a fundamental limitation: reducing confounding bias often suppresses clinically informative…

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

When training a predictive model over medical data, the goal is sometimes to gain insights about a certain disease. In such cases, it is common to use feature importance as a tool to highlight significant factors contributing to that…

Unmeasured confounding is a key threat to reliable causal inference based on observational studies. Motivated from two powerful natural experiment devices, the instrumental variables and difference-in-differences, we propose a new method…

Effectively representing medical concepts and patients is important for healthcare analytical applications. Representing medical concepts for healthcare analytical tasks requires incorporating medical domain knowledge and prior information…

Background: Clinical prediction models are increasingly used to inform healthcare decisions, but determining the minimum sample size for their development remains a critical and unresolved challenge. Inadequate sample sizes can lead to…

Difference-in-differences (DiD) is the most popular observational causal inference method in health policy, employed to evaluate the real-world impact of policies and programs. To estimate treatment effects, DiD relies on the "parallel…

Clinical prediction models must be developed using sufficiently large datasets to minimise overfitting and ensure robust predictive performance. Existing sample size calculations assume complete predictor data for all included participants,…

Model informed precision dosing (MIPD) is a Bayesian framework to individualize drug therapy based on prior knowledge and patient-specific monitoring data. Typically, prior knowledge results from controlled clinical trials with a more…