COMBINING ESTIMATES OF CONDITIONAL TREATMENT EFFECTS

2018 ◽  
Vol 35 (6) ◽  
pp. 1089-1110 ◽  
Author(s):  
Craig A. Rolling ◽  
Yuhong Yang ◽  
Dagmar Velez
Keyword(s):  
Treatment Effect ◽  
Treatment Effects ◽  
Direct Marketing ◽  
Skills Training ◽  
Accurate Estimate ◽  
Accurate Estimation ◽  
Model Combination ◽  
Using Data ◽  
Effect Estimation ◽  
Combining Estimates

Estimating a treatment’s effect on an outcome conditional on covariates is a primary goal of many empirical investigations. Accurate estimation of the treatment effect given covariates can enable the optimal treatment to be applied to each unit or guide the deployment of limited treatment resources for maximum program benefit. Applications of conditional treatment effect estimation are found in direct marketing, economic policy, and personalized medicine. When estimating conditional treatment effects, the typical practice is to select a statistical model or procedure based on sample data. However, combining estimates from the candidate procedures often provides a more accurate estimate than the selection of a single procedure. This article proposes a method of model combination that targets accurate estimation of the treatment effect conditional on covariates. We provide a risk bound for the resulting estimator under squared error loss and illustrate the method using data from a labor skills training program.

scholarly journals Dynamic borrowing in the presence of treatment effect heterogeneity

Biostatistics ◽  
2020 ◽  
Author(s):  
Ales Kotalik ◽  
David M Vock ◽  
Eric C Donny ◽  
Dorothy K Hatsukami ◽  
Joseph S Koopmeiners
Keyword(s):  
Clinical Trials ◽  
Treatment Effect ◽  
Treatment Effects ◽  
Randomized Clinical Trials ◽  
Potential Outcomes ◽  
Operating Characteristics ◽  
Treatment Effect Heterogeneity ◽  
Using Data ◽  
Effect Heterogeneity ◽  
Potential Outcomes Framework

Summary A number of statistical approaches have been proposed for incorporating supplemental information in randomized clinical trials. Existing methods often compare the marginal treatment effects to evaluate the degree of consistency between sources. Dissimilar marginal treatment effects would either lead to increased bias or down-weighting of the supplemental data. This represents a limitation in the presence of treatment effect heterogeneity, in which case the marginal treatment effect may differ between the sources solely due to differences between the study populations. We introduce the concept of covariate-adjusted exchangeability, in which differences in the marginal treatment effect can be explained by differences in the distributions of the effect modifiers. The potential outcomes framework is used to conceptualize covariate-adjusted and marginal exchangeability. We utilize a linear model and the existing multisource exchangeability models framework to facilitate borrowing when marginal treatment effects are dissimilar but covariate-adjusted exchangeability holds. We investigate the operating characteristics of our method using simulations. We also illustrate our method using data from two clinical trials of very low nicotine content cigarettes. Our method has the ability to incorporate supplemental information in a wider variety of situations than when only marginal exchangeability is considered.

Increasing Precision without Altering Treatment Effects: Repeated Measures Designs in Survey Experiments

2021 ◽  
pp. 1-18
Author(s):  
SCOTT CLIFFORD ◽  
GEOFFREY SHEAGLEY ◽  
SPENCER PISTON
Keyword(s):  
Treatment Effect ◽  
Repeated Measures ◽  
Treatment Effects ◽  
Repeated Measurements ◽  
Experimental Designs ◽  
Treatment Effect Size ◽  
Standard Design ◽  
Survey Experiments ◽  
Repeated Measures Designs ◽  
Alternative Designs

The use of survey experiments has surged in political science. The most common design is the between-subjects design in which the outcome is only measured posttreatment. This design relies heavily on recruiting a large number of subjects to precisely estimate treatment effects. Alternative designs that involve repeated measurements of the dependent variable promise greater precision, but they are rarely used out of fears that these designs will yield different results than a standard design (e.g., due to consistency pressures). Across six studies, we assess this conventional wisdom by testing experimental designs against each other. Contrary to common fears, repeated measures designs tend to yield the same results as more common designs while substantially increasing precision. These designs also offer new insights into treatment effect size and heterogeneity. We conclude by encouraging researchers to adopt repeated measures designs and providing guidelines for when and how to use them.

scholarly journals Estimating and reporting treatment effects in clinical trials for weight management: using estimands to interpret effects of intercurrent events and missing data

2021 ◽  
Author(s):  
Sean Wharton ◽  
Arne Astrup ◽  
Lars Endahl ◽  
Michael E. J. Lean ◽  
Altynai Satylganova ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Missing Data ◽  
Weight Management ◽  
Treatment Effect ◽  
Repeated Measures ◽  
Treatment Effects ◽  
Randomized Clinical Trials ◽  
Weight Losses ◽  
The Difference ◽  
Treatment Of Obesity

AbstractIn the approval process for new weight management therapies, regulators typically require estimates of effect size. Usually, as with other drug evaluations, the placebo-adjusted treatment effect (i.e., the difference between weight losses with pharmacotherapy and placebo, when given as an adjunct to lifestyle intervention) is provided from data in randomized clinical trials (RCTs). At first glance, this may seem appropriate and straightforward. However, weight loss is not a simple direct drug effect, but is also mediated by other factors such as changes in diet and physical activity. Interpreting observed differences between treatment arms in weight management RCTs can be challenging; intercurrent events that occur after treatment initiation may affect the interpretation of results at the end of treatment. Utilizing estimands helps to address these uncertainties and improve transparency in clinical trial reporting by better matching the treatment-effect estimates to the scientific and/or clinical questions of interest. Estimands aim to provide an indication of trial outcomes that might be expected in the same patients under different conditions. This article reviews how intercurrent events during weight management trials can influence placebo-adjusted treatment effects, depending on how they are accounted for and how missing data are handled. The most appropriate method for statistical analysis is also discussed, including assessment of the last observation carried forward approach, and more recent methods, such as multiple imputation and mixed models for repeated measures. The use of each of these approaches, and that of estimands, is discussed in the context of the SCALE phase 3a and 3b RCTs evaluating the effect of liraglutide 3.0 mg for the treatment of obesity.

scholarly journals Long-Term Development of Urban Agriculture: Resilience and Sustainability of Farmers Facing the Covid-19 Pandemic in Japan

2021 ◽  
Vol 13 (8) ◽  
pp. 4316
Author(s):  
Shingo Yoshida ◽  
Hironori Yagi
Keyword(s):  
Urban Agriculture ◽  
Food Systems ◽  
Direct Marketing ◽  
Farming System ◽  
Long Term Effects ◽  
Short Term ◽  
Short Supply ◽  
Using Data ◽  
Urban People

The coronavirus disease 2019 (Covid-19) pandemic has forced global food systems to face unprecedented uncertain shocks even in terms of human health. Urban agriculture is expected to be more resilient because of its short supply chain for urban people and diversified farming activities. However, the short-and long-term effects of the Covid-19 pandemic on urban farms remain unclear. This study aims to reveal the conditions for farm resilience to the Covid-19 pandemic in 2020 and the relationship between short-term farm resilience and long-term farm development using data from a survey of 74 farms located in Tokyo. The results are as follows. First, more than half of the sample farms increased their farm sales during this period. This resilience can be called the “persistence” approach. Second, short-term farm resilience and other sustainable farm activities contributed to improving farmers’ intentions for long-term farm development and farmland preservation. Third, the most important resilience attributes were the direct marketing, entrepreneurship, and social networks of farmers. We discussed the necessity of building farmers’ transformative capabilities for a more resilient urban farming system. These results imply that support to enhance the short-term resilience of urban farms is worth more than the short-term profit of the farms.

Inference for treatment effect parameters in potentially misspecified high-dimensional models

Biometrika ◽  
2020 ◽  
Author(s):  
Oliver Dukes ◽  
Stijn Vansteelandt
Keyword(s):  
Sample Size ◽  
Confidence Intervals ◽  
Treatment Effect ◽  
Observational Studies ◽  
Treatment Effects ◽  
Treatment Selection ◽  
High Dimensional ◽  
Selection Mechanism ◽  
Dimensional Models ◽  
Effect Parameter

Summary Eliminating the effect of confounding in observational studies typically involves fitting a model for an outcome adjusted for covariates. When, as often, these covariates are high-dimensional, this necessitates the use of sparse estimators, such as the lasso, or other regularization approaches. Naïve use of such estimators yields confidence intervals for the conditional treatment effect parameter that are not uniformly valid. Moreover, as the number of covariates grows with the sample size, correctly specifying a model for the outcome is nontrivial. In this article we deal with both of these concerns simultaneously, obtaining confidence intervals for conditional treatment effects that are uniformly valid, regardless of whether the outcome model is correct. This is done by incorporating an additional model for the treatment selection mechanism. When both models are correctly specified, we can weaken the standard conditions on model sparsity. Our procedure extends to multivariate treatment effect parameters and complex longitudinal settings.

Estimating Heterogeneous Treatment Effects and the Effects of Heterogeneous Treatments with Ensemble Methods

2017 ◽  
Vol 25 (4) ◽  
pp. 413-434 ◽  
Author(s):  
Justin Grimmer ◽  
Solomon Messing ◽  
Sean J. Westwood
Keyword(s):  
Treatment Effects ◽  
Ensemble Methods ◽  
Ensemble Method ◽  
Superior Performance ◽  
Accurate Estimation ◽  
Randomized Experiments ◽  
Heterogeneous Treatment Effects ◽  
Data Set ◽  
Heterogeneous Effects ◽  
The Ensemble Method

Randomized experiments are increasingly used to study political phenomena because they can credibly estimate the average effect of a treatment on a population of interest. But political scientists are often interested in how effects vary across subpopulations—heterogeneous treatment effects—and how differences in the content of the treatment affects responses—the response to heterogeneous treatments. Several new methods have been introduced to estimate heterogeneous effects, but it is difficult to know if a method will perform well for a particular data set. Rather than using only one method, we show how an ensemble of methods—weighted averages of estimates from individual models increasingly used in machine learning—accurately measure heterogeneous effects. Building on a large literature on ensemble methods, we show how the weighting of methods can contribute to accurate estimation of heterogeneous treatment effects and demonstrate how pooling models lead to superior performance to individual methods across diverse problems. We apply the ensemble method to two experiments, illuminating how the ensemble method for heterogeneous treatment effects facilitates exploratory analysis of treatment effects.

scholarly journals Blinding, sham, and treatment effects in randomized controlled trials for back pain in 2000–2019: A review and meta-analytic approach

2021 ◽  
pp. 174077452098487
Author(s):  
Brian Freed ◽  
Brian Williams ◽  
Xiaolu Situ ◽  
Victoria Landsman ◽  
Jeehyoung Kim ◽  
...  
Keyword(s):  
Back Pain ◽  
Active Treatment ◽  
Treatment Effect ◽  
Effect Size ◽  
Treatment Effects ◽  
Effect Sizes ◽  
Controlled Trials ◽  
Sham Treatment ◽  
Randomized Controlled ◽  
Random Guess

Background: Blinding aims to minimize biases from what participants and investigators know or believe. Randomized controlled trials, despite being the gold standard to evaluate treatment effect, do not generally assess the success of blinding. We investigated the extent of blinding in back pain trials and the associations between participant guesses and treatment effects. Methods: We did a review with PubMed/OvidMedline, 2000–2019. Eligibility criteria were back pain trials with data available on treatment effect and participants’ guess of treatment. For blinding, blinding index was used as chance-corrected measure of excessive correct guess (0 for random guess). For treatment effects, within- or between-arm effect sizes were used. Analyses of investigators’ guess/blinding or by treatment modality were performed exploratorily. Results: Forty trials (3899 participants) were included. Active and sham treatment groups had mean blinding index of 0.26 (95% confidence interval: 0.12, 0.41) and 0.01 (−0.11, 0.14), respectively, meaning 26% of participants in active treatment believed they received active treatment, whereas only 1% in sham believed they received sham treatment, beyond chance, that is, random guess. A greater belief of receiving active treatment was associated with a larger within-arm effect size in both arms, and ideal blinding (namely, “random guess,” and “wishful thinking” that signifies both groups believing they received active treatment) showed smaller effect sizes, with correlation of effect size and summary blinding indexes of 0.35 ( p = 0.028) for between-arm comparison. We observed uniformly large sham treatment effects for all modalities, and larger correlation for investigator’s (un)blinding, 0.53 ( p = 0.046). Conclusion: Participants in active treatments in back pain trials guessed treatment identity more correctly, while those in sham treatments tended to display successful blinding. Excessive correct guesses (that could reflect weaker blinding and/or noticeable effects) by participants and investigators demonstrated larger effect sizes. Blinding and sham treatment effects on back pain need due consideration in individual trials and meta-analyses.

Effecten van een sociale responsiviteitstraining op jongeren met ASS

2021 ◽  
Vol 20 (4) ◽  
Author(s):  
Sakinah Binti Idris ◽  
Jorieke Duvekot ◽  
Jan van der Ende ◽  
Kirstin Greaves Lord ◽  
Neeltje van Haren ◽  
...  
Keyword(s):  
Randomized Controlled Trial ◽  
Social Skills ◽  
Active Control ◽  
Treatment Effects ◽  
Controlled Trial ◽  
Skills Training ◽  
Parent Reports ◽  
Randomized Controlled ◽  
Age Appropriate ◽  
Relational Skills

The PEERS® (Program for Education and Enrichment of Relational Skills) training targets age-appropriate social skills. By the use of homework assignments and parallel parent sessions the generalization of skills enhanced. The results of this randomized controlled trial show that social skills significantly improved, as indicated by both selfand parent reports. However, the significant increase in observed social skills as observed in the PEERS® condition was as large as the increase observed in the active control condition. Further research will target mediators and moderators of treatment effects.

The Effect of Extension Services and Credit on Agricultural Production in Bolivia, Peru, and Colombia

2021 ◽  
Author(s):  
Mateus C. R. Neves ◽  
Felipe De Figueiredo Silva ◽  
Carlos Otávio Freitas
Keyword(s):  
Travel Time ◽  
Treatment Effect ◽  
Agricultural Production ◽  
Treatment Effects ◽  
Geographic Information ◽  
Average Treatment Effect ◽  
Extension Services ◽  
Average Treatment ◽  
Andean Countries ◽  
Average Treatment Effects

In this paper we estimate the average treatment effect from access to extension services and credit on agricultural production in selected Andean countries (Bolivia, Peru, and Colombia). More specifically, we want to identify the effect of accessibility, here represented as travel time to the nearest area with 1,500 or more inhabitants per square kilometer or at least 50,000 inhabitants, on the likelihood of accessing extension and credit. To estimate the treatment effect and identify the effect of accessibility on these variables, we use data from the Colombian and Bolivian Agricultural Censuses of 2013 and 2014, respectively; a national agricultural survey from 2017 for Peru; and geographic information on travel time. We find that the average treatment effect for extension is higher compared to that of credit for farms in Bolivia and Peru, and lower for Colombia. The average treatment effects of extension and credit for Peruvian farms are $2,387.45 and $3,583.42 respectively. The average treatment effect for extension and credit are $941.92 and $668.69, respectively, while in Colombia are $1,365.98 and $1,192.51, respectively. We also find that accessibility and the likelihood of accessing these services are nonlinearly related. Results indicate that higher likelihood is associated with lower travel time, especially in the analysis of credit.

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