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Register a new userOn Minimum Clinically Important Difference
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Tu Xu
Publication date
2013
fields
Mathematical Statistics
and research's language is
English
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In clinical trials, minimum clinically important difference (MCID) has attracted increasing interest as an important supportive clinical and statistical inference tool. Many estimation methods have been developed based on various intuitions, while little theoretical justification has been established. This paper proposes a new estimation framework of MCID using both diagnostic measurements and patient-reported outcomes (PROs). It first provides a precise definition of population-based MCID so that estimating such a MCID can be formulated as a large margin classification problem. The framework is then extended to personalized MCID to allow individualized thresholding value for patients whose clinical profiles may affect their PRO responses. More importantly, we show that the proposed estimation framework is asymptotically consistent, and a finite-sample upper bound is established for its prediction accuracy compared against the ideal MCID. The advantage of our proposed method is also demonstrated in a variety of simulated experiments as well as applications to two benchmark datasets and two phase-3 clinical trials.
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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 statistical problem where the parameter of interest lies in an individualized linear threshold. The goal of this paper is to develop a hypothesis testing procedure for the significance of a single element in this high-dimensional parameter as well as for the significance of a linear combination of this parameter. The difficulty dues to the high-dimensionality of the nuisance component in developing such a testing procedure, and also stems from the fact that this high-dimensional threshold model is nonregular and the limiting distribution of the corresponding estimator is nonstandard. To deal with these challenges, we construct a test statistic via a new bias corrected smoothed decorrelated score approach, and establish its asymptotic distributions under both the null and local alternative hypotheses. In addition, we propose a double-smoothing approach to select the optimal bandwidth parameter in our test statistic and provide theoretical guarantees for the selected bandwidth. We conduct comprehensive simulation studies to demonstrate how our proposed procedure can be applied in empirical studies. Finally, we apply the proposed method to a clinical trial where the scientific goal is to assess the clinical importance of a surgery procedure.
Modern multivariate machine learning and statistical methodologies estimate parameters of interest while leveraging prior knowledge of the association between outcome variables. The methods that do allow for estimation of relationships do so typically through an error covariance matrix in multivariate regression which does not scale to other types of models. In this article we proposed the MinPEN framework to simultaneously estimate regression coefficients associated with the multivariate regression model and the relationships between outcome variables using mild assumptions. The MinPen framework utilizes a novel penalty based on the minimum function to exploit detected relationships between responses. An iterative algorithm that generalizes current state of the art methods is proposed as a solution to the non-convex optimization that is required to obtain estimates. Theoretical results such as high dimensional convergence rates, model selection consistency, and a framework for post selection inference are provided. We extend the proposed MinPen framework to other exponential family loss functions, with a specific focus on multiple binomial responses. Tuning parameter selection is also addressed. Finally, simulations and two data examples are presented to show the finite sample properties of this framework.
Online A/B testing plays a critical role in the high-tech industry to guide product development and accelerate innovation. It performs a null hypothesis statistical test to determine which variant is better. However, a typical A/B test presents two problems: (i) a fixed-horizon framework inflates the false-positive errors under continuous monitoring; (ii) the homogeneous effects assumption fails to identify a subgroup with a beneficial treatment effect. In this paper, we propose a sequential test for subgroup treatment effects based on value difference, named SUBTLE, to address these two problems simultaneously. The SUBTLE allows the experimenters to peek at the results during the experiment without harming the statistical guarantees. It assumes heterogeneous treatment effects and aims to test if some subgroup of the population will benefit from the investigative treatment. If the testing result indicates the existence of such a subgroup, a subgroup will be identified using a readily available estimated optimal treatment rule. We examine the empirical performance of our proposed test on both simulations and a real dataset. The results show that the SUBTLE has high detection power with controlled type I error at any time, is more robust to noise covariates, and can achieve early stopping compared with the corresponding fixed-horizon test.
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