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Multi-objective optimal designs in comparative clinical trials with covariates: The reinforced doubly adaptive biased coin design

含协变量临床试验的多目标最优设计:强化双重自适应偏币设计

The present paper deals with the problem of allocating patients to two competing treatments in the presence of covariates or prognostic factors in order to achieve a good trade-off among ethical concerns, inferential precision and randomness in the treatment allocations. In particular we suggest a multipurpose design methodology that combines efficiency and ethical gain when the linear homoscedastic model with both treatment/covariate interactions and interactions among…

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Quantile treatment effects in the regression discontinuity design

断点回归设计中的分位数处理效应

We introduce a nonparametric estimator for local quantile treatment effects in the regression discontinuity (RD) design. The procedure uses local distribution regression to estimate the marginal distributions of the potential outcomes. We illustrate the procedure through Monte Carlo simulations and an application on the distributional effects of a universal pre-K program in Oklahoma. We find that participation in a pre-K program significantly raises the lower end and the…

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Nonparametric identification in nonseparable panel data models with generalized fixed effects

具有广义固定效应的非可分面板模型的非参数识别

This paper extends the familiar notion of fixed effects to nonlinear structures with infinite-dimensional unobservables, like preferences. The main result is that a generalized version of differencing identifies local average responses (LARs) in nonseparable structures. In contrast to existing results, this does not require either substantial restrictions on functional form or independence between the persistent unobservables and the explanatory variables of interest, and it…

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Does Marriage Boost Men’s Wages?: Identification of Treatment Effects in Fixed Effects Regression Models for Panel Data

婚姻会提高男性工资吗?面板固定效应回归中的处理效应识别

Social scientists have generated a large and inconclusive literature on the effect(s) of marriage on men’s wages. Researchers have hypothesized that the wage premium enjoyed by married men may reflect both a tendency for more productive men to marry and an effect of marriage on productivity. To sort out these explanations, researchers have used fixed effects regression models for panel data to adjust for selection on unobserved time-invariant confounders, interpreting…

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Meta-Analysis of Rare Binary Adverse Event Data

罕见二元不良事件数据的元分析

We examine the use of fixed-effects and random-effects moment-based meta-analytic methods for analysis of binary adverse event data. Special attention is paid to the case of rare adverse events which are commonly encountered in routine practice. We study estimation of model parameters and between-study heterogeneity. In addition, we examine traditional approaches to hypothesis testing of the average treatment effect and detection of the heterogeneity of treatment effect…

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Evaluating the Effect of Early Versus Late ARV Regimen Change if Failure on an Initial Regimen: Results From the AIDS Clinical Trials Group Study A5095

初始抗逆转录病毒方案失败后早期与延迟换药的效果评估

The current goal of initial antiretroviral (ARV) therapy is suppression of plasma human immunodeficiency virus (HIV)-1 RNA levels to below 200 copies per milliliter. A proportion of HIV-infected patients who initiate antiretroviral therapy in clinical practice or antiretroviral clinical trials either fail to suppress HIV-1 RNA or have HIV-1 RNA levels rebound on therapy. Frequently, these patients have sustained CD4 cell counts responses and limited or no clinical symptoms…

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Evaluation of Viable Dynamic Treatment Regimes in a Sequentially Randomized Trial of Advanced Prostate Cancer

晚期前列腺癌序贯随机试验中可实施动态治疗策略的评估

We present new statistical analyses of data arising from a clinical trial designed to compare two-stage dynamic treatment regimes (DTRs) for advanced prostate cancer. The trial protocol mandated that patients were to be initially randomized among four chemotherapies, and that those who responded poorly were to be rerandomized to one of the remaining candidate therapies. The primary aim was to compare the DTRs' overall success rates, with success defined by the occurrence of…

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Clustering, Spatial Correlations, and Randomization Inference

聚类、空间相关与随机化推断

It is a standard practice in regression analyses to allow for clustering in the error covariance matrix if the explanatory variable of interest varies at a more aggregate level (e.g., the state level) than the units of observation (e.g., individuals). Often, however, the structure of the error covariance matrix is more complex, with correlations not vanishing for units in different clusters. Here, we explore the implications of such correlations for the actual and estimated…

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Efficient local IV estimation of an empirical auction model

经验拍卖模型的有效局部工具变量估计

In this paper we examine semiparametric efficiency bounds and efficient estimators for the case of a linear local instrument variable (LIV) model under the assumptions studied in Abadie et al. (2002). We apply the semiparametrically efficient estimation method to analyze the relation between bid dispersion and early bidding in an online auction dataset, which is collected from a natural experiment conducted in Nekipelov (2007). The results confirm the theoretical findings…

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Rerandomization to improve covariate balance in experiments

通过再随机化改善实验中的协变量平衡

Randomized experiments are the “gold standard” for estimating causal effects, yet often in practice, chance imbalances exist in covariate distributions between treatment groups. If covariate data are available before units are exposed to treatments, these chance imbalances can be mitigated by first checking covariate balance before the physical experiment takes place. Provided a precise definition of imbalance has been specified in advance, unbalanced randomizations can be…

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Confidence intervals for the quantile of treatment effects in randomized experiments

随机实验中处理效应分位数的置信区间

In this paper, we explore partial identification and inference for the quantile of treatment effects for randomized experiments. First, we propose nonparametric estimators of sharp bounds on the quantile of treatment effects and establish their asymptotic properties under general conditions. Second, we construct confidence intervals for the bounds and the true quantile by using the approach in Chernozhukov et al. (2009). Third, under additional conditions, we develop a new…

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Treatment effects in sample selection models and their nonparametric estimation

样本选择模型中的处理效应及其非参数估计

In a sample-selection model with the ‘selection’ variable Q and the ‘outcome’ variable Y∗, Y∗ is observed only when Q=1. For a treatment D affecting both Q and Y∗, three effects are of interest: ‘participation’ (i.e., the selection) effect of D on Q, ‘visible performance’ (i.e., the observed outcome) effect of D on Y≡QY∗, and ‘invisible performance’ (i.e., the latent outcome) effect of D on Y∗. This paper shows the conditions under which the three effects are identified,…

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Rich Dad, Smart Dad: Decomposing the Intergenerational Transmission of Income

富爸爸,聪明爸爸:收入代际传递的分解

We construct a simple model, consistent with Becker and Tomes, that decomposes the intergenerational income elasticity into the causal effect of financial resources, the mechanistic transmission of human capital, and the role that human capital plays in the determination of fathers' permanent incomes. We show how a particular set of instrumental variables could separately identify the money and human capital transmission effects. Using data from a 35 percent sample of…

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(Interstate) Banking and (interstate) trade: Does real integration follow financial integration?

跨州银行业与跨州贸易:实体一体化是否跟随金融一体化?

We conjecture that banks present in two regions charge the appropriate risk premiums for trade-related projects between these markets, whereas higher rates are charged for projects involving shipments to markets where they are absent. These differences affect regional trade flows. US interstate banking deregulation serves as a natural experiment to test our model's implication with the Commodity Flow Survey data. Difference-in-differences estimates suggest that the trade…

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The effect of board structure on firm value: A multiple identification strategies approach using Korean data

董事会结构对企业价值的影响:基于韩国数据的多重识别策略

Outside directors and audit committees are widely considered to be central elements of good corporate governance. We use a 1999 Korean law as an exogenous shock to assess whether and how board structure affects firm market value. The law mandates 50% outside directors and an audit committee for large public firms, but not smaller firms. We study this shock using event study, difference-in-differences, and instrumental variable methods, within an overall regression…

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Nonparametric estimation of the impact of taxes on female labor supply

税收对女性劳动供给影响的非参数估计

Abstract This paper proposes a simple extension of nonparametric estimation methods for nonlinear budget‐set models derived in Blomquist and Newey ( 2002 ) to censored dependent variables. The nonparametric method is applied to estimate female labor supply elasticities using data on married women from the 1985 and 1989 waves of the Panel Study of Income Dynamics, exploiting the substantial variation in budget sets caused by the Tax Reform Act of 1986 as a source of…

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Proportional likelihood ratio models for mean regression

用于均值回归的比例似然比模型

The proportional likelihood ratio model introduced in Luo & Tsai (2011) is adapted to explicitly model the means of observations. This is useful for the estimation of and inference on treatment effects, particularly in designed experiments, and allows the data analyst greater control over model specification and parameter interpretation.

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Estimating treatment effects with treatment switching via semicompeting risks models: an application to a colorectal cancer study

利用半竞争风险模型估计治疗转换下的治疗效应:结直肠癌研究应用

Treatment switching is a frequent occurrence in clinical trials, where, during the course of the trial, patients who fail on the control treatment may change to the experimental treatment. Analysing the data without accounting for switching yields highly biased and inefficient estimates of the treatment effect. In this paper, we propose a novel class of semiparametric semicompeting risks transition survival models to accommodate treatment switches. Theoretical properties of…

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Semiparametric Tests for Sufficient Cause Interaction

充分原因交互作用的半参数检验

Summary A sufficient cause interaction between two exposures signals the presence of individuals for whom the outcome would occur only under certain values of the two exposures. When the outcome is dichotomous and all exposures are categorical, then, under certain no confounding assumptions, empirical conditions for sufficient cause interactions can be constructed on the basis of the sign of linear contrasts of conditional outcome probabilities between differently exposed…

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Inferring welfare maximizing treatment assignment under budget constraints

预算约束下福利最大化处理分配的推断

This paper concerns the problem of allocating a binary treatment among a target population based on observed covariates. The goal is to (i) maximize the mean social welfare arising from an eventual outcome distribution, when a budget constraint limits what fraction of the population can be treated and (ii) to infer the dual value, i.e. the minimum resources needed to attain a specific level of mean welfare via efficient treatment assignment. We consider a treatment…

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Semiparametric Double Balancing Score Estimation for Incomplete Data With Ignorable Missingness

可忽略缺失数据的半参数双平衡得分估计

When estimating the marginal mean response with missing observations, a critical issue is robustness to model misspecification. In this article, we propose a semiparametric estimation method with extended double robustness that attains the optimal efficiency under less stringent requirement for model specifications than the doubly robust estimators. In this semiparametric estimation, covariate information is collapsed into a two-dimensional score S, with one dimension for…

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Interim Design Modifications in Time-to-Event Studies

事件时间研究中的期中设计修改

We propose a flexible method for interim design modifications in time-to-event studies. With this method, it is possible to inspect the data at any time during the course of the study, without the need for prespecification of a learning phase, and to make certain types of design modifications depending on the interim data without compromising the Type I error risk. The method can be applied to studies designed with a conventional statistical test, fixed sample, or group…

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Partially Hidden Markov Model for Time-Varying Principal Stratification in HIV Prevention Trials

HIV预防试验中时变主分层的部分隐马尔可夫模型

It is frequently of interest to estimate the intervention effect that adjusts for post-randomization variables in clinical trials. In the recently completed HPTN 035 trial, there is differential condom use between the three microbicide gel arms and the No Gel control arm, so that intention to treat (ITT) analyses only assess the net treatment effect that includes the indirect treatment effect mediated through differential condom use. Various statistical methods in causal…

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Bayesian Inference for Dynamic Treatment Regimes: Mobility, Equity, and Efficiency in Student Tracking

动态处理方案的贝叶斯推断:学生分层中的流动、公平与效率

Policies in health, education, and economics often unfold sequentially and adapt to changing conditions. Such time-varying treatments pose problems for standard program evaluation methods because intermediate outcomes are simultaneously pretreatment confounders and posttreatment outcomes. This article extends the Bayesian perspective on causal inference and optimal treatment to these types of dynamic treatment regimes. A unifying idea remains ignorable treatment assignment,…

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