Experimental design is an approach for selecting samples among a given set so as to obtain the best estimator for a given criterion. In the context of linear regression, several optimal designs have been derived, each associated with a different criterion: mean square error, robustness, \emph{etc}. Computing such designs is generally an NP-hard problem and one can instead rely on a convex relaxation that considers probability distributions over the samples. Although greedy strategies and rounding procedures have received a lot of attention, straightforward sampling from the optimal distribution has hardly been investigated. In this paper, we propose theoretical guarantees for randomized strategies on E and G-optimal design. To this end, we develop a new concentration inequality for the eigenvalues of random matrices using a refined version of the intrinsic dimension that enables us to quantify the performance of such randomized strategies. Finally, we evidence the validity of our analysis through experiments, with particular attention on the G-optimal design applied to the best arm identification problem for linear bandits.
In randomized controlled trials (RCTs), treatment is often assigned by stratified randomization. I show that among all stratified randomization schemes which treat all units with probability one half, a certain matched-pair design achieves the maximum statistical precision for estimating the average treatment effect (ATE). In an important special case, the optimal design pairs units according to the baseline outcome. In a simulation study based on datasets from 10 RCTs, this design lowers the standard error for the estimator of the ATE by 10% on average, and by up to 34%, relative to the origi…
We examine study designs for extending (generalizing or transporting) causal inferences from a randomized trial to a target population. Specifically, we consider nested trial designs, where randomized individuals are nested within a sample from the target population, and non-nested trial designs, including composite dataset designs, where a randomized trial is combined with a separately obtained sample of non-randomized individuals from the target population. We show that the causal quantities that can be identified in each study design depend on what is known about the probability of sampling…
Estimating within-cluster and between-cluster spillover effects in randomized saturation designs
Department of Statistics, University of California, Berkeley（加州大学伯克利分校统计学系）；Division of Biostatistics, University of California, Berkeley（加州大学伯克利分校生物统计学区）
Nonignorable missingness and noncompliance can occur even in well-designed randomized experiments making the intervention effect that the experiment was designed to estimate nonidentifiable. Nonparametric causal bounds provide a way to narrow the range of possible values for a nonidentifiable causal effect with minimal assumptions. We derive novel bounds for the causal risk difference for a binary outcome and intervention in randomized experiments with nonignorable missingness caused by a variety of mechanisms and with or without noncompliance. We illustrate the use of the proposed bounds in o…
We establish the inferential properties of the mean-difference estimator for the average treatment effect in randomized experiments where each unit in a population is randomized to one of two treatments and then units within treatment groups are randomly sampled. The properties of this estimator are well-understood in the experimental design scenario where first units are randomly sampled and then treatment is randomly assigned, but not for the aforementioned scenario where the sampling and treatment assignment stages are reversed. We find that the inferential properties of the mean-difference…
Inference of Sample Complier Average Causal Effects under Experiments with Completely Randomized Design and Computer Assisted Balance-Improving Designs
Numerous publications have now addressed the principles of designing, analyzing, and reporting the results of, stepped-wedge cluster randomized trials. In contrast, there is little research available pertaining to the design and analysis of multi-arm stepped-wedge cluster randomized trials, utilized to evaluate the effectiveness of multiple experimental interventions. In this paper, we address this by explaining how the required sample size in these multi-arm trials can be ascertained when data are to be analyzed using a linear mixed model. We then go on to describe how the design of such tria…
The injunction to `analyze the way you randomize' is well-known to statisticians since Fisher advocated for randomization as the basis of inference. Yet even those convinced by the merits of randomization-based inference seldom follow this injunction to the letter. Bernoulli randomized experiments are often analyzed as completely randomized experiments, and completely randomized experiments are analyzed as if they had been stratified; more generally, it is not uncommon to analyze an experiment as if it had been randomized differently. This paper examines the theoretical foundation behind this …
We consider a potential outcomes model in which interference may be present between any two units but the extent of interference diminishes with spatial distance. The causal estimand is the global average treatment effect, which compares outcomes under the counterfactuals that all or no units are treated. We study a class of designs in which space is partitioned into clusters that are randomized into treatment and control. For each design, we estimate the treatment effect using a Horvitz-Thompson estimator that compares the average outcomes of units with all or no neighbors treated, where the …
The use and development of mobile interventions are experiencing rapid growth. In "just-in-time" mobile interventions, treatments are provided via a mobile device and they are intended to help an individual make healthy decisions "in the moment," and thus have a proximal, near future impact. Currently the development of mobile interventions is proceeding at a much faster pace than that of associated data science methods. A first step toward developing data-based methods is to provide an experimental design for testing the proximal effects of these just-in-time treatments. In this paper, we pro…
Cluster-based randomized experiments are popular designs for mitigating the bias of standard estimators when interference is present and classical causal inference and experimental design assumptions (such as SUTVA or ITR) do not hold. Without an exact knowledge of the interference structure, it can be challenging to understand which partitioning of the experimental units is optimal to minimize the estimation bias. In the paper, we introduce a monotonicity condition under which a novel two-stage experimental design allows us to determine which of two cluster-based designs yields the least bias…
Cluster randomized trials with measurements at baseline can improve power over post-test only designs by using difference in difference designs. However, subjects may be lost to follow-up between the baseline and follow-up periods. While equations for sample size and variance have been developed assuming no loss to follow-up ("cohort") and completely different subjects at baseline and follow-up ("cross-sectional") difference in difference designs, equations have yet to be developed when some subjects are observed in both periods ("mixture" designs). We present a general equation for calculatin…
Regression adjustment is widely used for the analysis of randomized experiments to improve the estimation efficiency of the treatment effect. This paper reexamines a weighted regression adjustment method termed as tyranny-of-the-minority (ToM), wherein units in the minority group are given greater weights. We demonstrate that the ToM regression adjustment is more robust than Lin 2013's regression adjustment with treatment-covariate interactions, even though these two regression adjustment methods are asymptotically equivalent in completely randomized experiments. Moreover, we extend ToM regres…
The micro-randomized trial (MRT) is a new experimental design which allows for the investigation of the proximal effects of a "just-in-time" treatment, often provided via a mobile device as part of a mobile health intervention. As with a traditional randomized controlled trial, computing the minimum required sample size to achieve a desired power is a crucial step in designing an MRT. We present MRT-SS Calculator, an online sample-size calculator for micro-randomized trials, built with R Shiny. MRT-SS Calculator requires specification of time-varying patterns for the proximal treatment effect …
The global average treatment effect (GATE) is a primary quantity of interest in the study of causal inference under network interference. With a correctly specified exposure model of the interference, the Horvitz-Thompson (HT) and Hájek estimators of the GATE are unbiased and consistent, respectively, yet known to exhibit extreme variance under many designs and in many settings of interest. With a fixed clustering of the interference graph, graph cluster randomization (GCR) designs have been shown to greatly reduce variance compared to node-level random assignment, but even so the variance is …
The original Finite Selection Model (FSM) was developed in the 1970s to enhance the design of the RAND Health Insurance Experiment (HIE; Newhouse et al. 1993). At the time of its development by Carl Morris (Morris 1979), there were fundamental computational limitations to make the method widely available for practitioners. Today, as randomized experiments increasingly become more common, there is a need for implementing experimental designs that are randomized, balanced, robust, and easily applicable to several treatment groups. To help address this problem, we revisit the original FSM under t…
We propose a class of subspace ascent methods for computing optimal approximate designs that covers both existing as well as new and more efficient algorithms. Within this class of methods, we construct a simple, randomized exchange algorithm (REX). Numerical comparisons suggest that the performance of REX is comparable or superior to the performance of state-of-the-art methods across a broad range of problem structures and sizes. We focus on the most commonly used criterion of D-optimality that also has applications beyond experimental design, such as the construction of the minimum volume el…
Just-in-time adaptive interventions (JITAIs) are time-varying adaptive interventions that use frequent opportunities for the intervention to be adapted such as weekly, daily, or even many times a day. This high intensity of adaptation is facilitated by the ability of digital technology to continuously collect information about an individual's current context and deliver treatments adapted to this information. The micro-randomized trial (MRT) has emerged for use in informing the construction of JITAIs. MRTs operate in, and take advantage of, the rapidly time-varying digital intervention environ…
Extending (generalizing or transporting) causal inferences from a randomized trial to a target population requires ``generalizability'' or ``transportability'' assumptions, which state that randomized and non-randomized individuals are exchangeable conditional on baseline covariates. These assumptions are made on the basis of background knowledge, which is often uncertain or controversial, and need to be subjected to sensitivity analysis. We present simple methods for sensitivity analyses that do not require detailed background knowledge about specific unknown or unmeasured determinants of the…
Trials enroll a large number of subjects in order to attain power, making them expensive and time-consuming. Sample size calculations are often performed with the assumption of an unadjusted analysis, even if the trial analysis plan specifies a more efficient estimator (e.g. ANCOVA). This leads to conservative estimates of required sample sizes and an opportunity for savings. Here we show that a relatively simple formula can be used to estimate the power of any two-arm, single-timepoint trial analyzed with a semiparametric efficient estimator, regardless of the domain of the outcome or kind of…
Instrumental variable methods are widely used in medical and social science research to draw causal conclusions when the treatment and outcome are confounded by unmeasured confounding variables. One important feature of such studies is that the instrumental variable is often applied at the cluster level, e.g., hospitals' or physicians' preference for a certain treatment where each hospital or physician naturally defines a cluster. This paper proposes to embed such observational instrumental variable data into a cluster-randomized encouragement experiment using statistical matching. Potential o…
Researchers are often interested in analyzing conditional treatment effects. One variant of this is "causal moderation," which implies that intervention upon a third (moderator) variable would alter the treatment effect. This study considers the conditions under which causal moderation can be identified and presents a generalized framework for estimating causal moderation effects given randomized treatments and non-randomized moderators. As part of the estimation process, it allows researchers to implement their preferred method of covariate adjustment, including parametric and non-parametric …
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 (potentially sensitive) data, specifically their response to treatment. This assumption, while trivial at first, is becoming difficult to satisfy in the modern era, especially in online settings where there are more regulations to protect individuals' data. The paper presents a new, simple experimental design that is differentially private, one of the strongest notion…
The usual development cycles are too slow for the development of vaccines, diagnostics and treatments in pandemics such as the ongoing SARS-CoV-2 pandemic. Given the pressure in such a situation, there is a risk that findings of early clinical trials are overinterpreted despite their limitations in terms of size and design. Motivated by a non-randomized open-label study investigating the efficacy of hydroxychloroquine in patients with COVID-19, we describe in a unified fashion various alternative approaches to the analysis of non-randomized studies. A widely used tool to reduce the impact of t…
Just-in-time adaptive interventions (JITAIs) are time-varying adaptive interventions that use frequent opportunities for the intervention to be adapted--weekly, daily, or even many times a day. The micro-randomized trial (MRT) has emerged for use in informing the construction of JITAIs. MRTs can be used to address research questions about whether and under what circumstances JITAI components are effective, with the ultimate objective of developing effective and efficient JITAI. The purpose of this article is to clarify why, when, and how to use MRTs; to highlight elements that must be consider…
Randomized pre-post designs, with outcomes measured at baseline and follow-ups, have been commonly used to compare the clinical effectiveness of two competing treatments. There are vast, but often conflicting, amount of information in current literature about the best analytic methods for pre-post design. It is challenging for applied researchers to make an informed choice. We discuss six methods commonly used in literature: one way analysis of variance (ANOVA), analysis of covariance main effect and interaction models on post-treatment measurement (ANCOVA I and II), ANOVA on change score betw…
Although there is much excitement surrounding the use of mobile and wearable technology for the purposes of delivering interventions as people go through their day-to-day lives, data analysis methods for constructing and optimizing digital interventions lag behind. Here, we elucidate data analysis methods for primary and secondary analyses of micro-randomized trials (MRTs), an experimental design to optimize digital just-in-time adaptive interventions. We provide a definition of causal "excursion" effects suitable for use in digital intervention development. We introduce the weighted and cente…
