Literature DB >> 27491758

Generalizing Treatment Effect Estimates From Sample to Population: A Case Study in the Difficulties of Finding Sufficient Data.

Elizabeth A Stuart1,2,3, Anna Rhodes4.   

Abstract

BACKGROUND: Given increasing concerns about the relevance of research to policy and practice, there is growing interest in assessing and enhancing the external validity of randomized trials: determining how useful a given randomized trial is for informing a policy question for a specific target population.
OBJECTIVES: This article highlights recent advances in assessing and enhancing external validity, with a focus on the data needed to make ex post statistical adjustments to enhance the applicability of experimental findings to populations potentially different from their study sample. RESEARCH
DESIGN: We use a case study to illustrate how to generalize treatment effect estimates from a randomized trial sample to a target population, in particular comparing the sample of children in a randomized trial of a supplemental program for Head Start centers (the Research-Based, Developmentally Informed study) to the national population of children eligible for Head Start, as represented in the Head Start Impact Study.
RESULTS: For this case study, common data elements between the trial sample and population were limited, making reliable generalization from the trial sample to the population challenging.
CONCLUSIONS: To answer important questions about external validity, more publicly available data are needed. In addition, future studies should make an effort to collect measures similar to those in other data sets. Measure comparability between population data sets and randomized trials that use samples of convenience will greatly enhance the range of research and policy relevant questions that can be answered.

Entities:  

Keywords:  Head Start Impact Study; REDI evaluation; causal inference; generalizability; transportability

Mesh:

Year:  2016        PMID: 27491758      PMCID: PMC8317069          DOI: 10.1177/0193841X16660663

Source DB:  PubMed          Journal:  Eval Rev        ISSN: 0193-841X


  25 in total

1.  External Validity in Policy Evaluations that Choose Sites Purposively.

Authors:  Robert B Olsen; Larry L Orr; Stephen H Bell; Elizabeth A Stuart
Journal:  J Policy Anal Manage       Date:  2013

2.  Propensity score estimation with boosted regression for evaluating causal effects in observational studies.

Authors:  Daniel F McCaffrey; Greg Ridgeway; Andrew R Morral
Journal:  Psychol Methods       Date:  2004-12

3.  Introduction to the special issue: subgroup analysis in prevention and intervention research.

Authors:  Lauren H Supplee; Brendan C Kelly; David P MacKinnon; David M Mackinnon; Meryl Yoches Barofsky
Journal:  Prev Sci       Date:  2013-04

4.  Improving propensity score weighting using machine learning.

Authors:  Brian K Lee; Justin Lessler; Elizabeth A Stuart
Journal:  Stat Med       Date:  2010-02-10       Impact factor: 2.373

5.  Psychometric approaches for developing commensurate measures across independent studies: traditional and new models.

Authors:  Daniel J Bauer; Andrea M Hussong
Journal:  Psychol Methods       Date:  2009-06

6.  Influence of subject eligibility criteria on compliance with National Institutes of Health guidelines for inclusion of women, minorities, and children in treatment research.

Authors:  Keith Humphreys; Kenneth R Weingardt; Alex H S Harris
Journal:  Alcohol Clin Exp Res       Date:  2007-04-11       Impact factor: 3.455

7.  Assessing the generalizability of randomized trial results to target populations.

Authors:  Elizabeth A Stuart; Catherine P Bradshaw; Philip J Leaf
Journal:  Prev Sci       Date:  2015-04

8.  Assessing sample representativeness in randomized controlled trials: application to the National Institute of Drug Abuse Clinical Trials Network.

Authors:  Ryoko Susukida; Rosa M Crum; Elizabeth A Stuart; Cyrus Ebnesajjad; Ramin Mojtabai
Journal:  Addiction       Date:  2016-04-01       Impact factor: 6.526

9.  Assessing and reporting heterogeneity in treatment effects in clinical trials: a proposal.

Authors:  David M Kent; Peter M Rothwell; John P A Ioannidis; Doug G Altman; Rodney A Hayward
Journal:  Trials       Date:  2010-08-12       Impact factor: 2.279

10.  The PhenX Toolkit: get the most from your measures.

Authors:  Carol M Hamilton; Lisa C Strader; Joseph G Pratt; Deborah Maiese; Tabitha Hendershot; Richard K Kwok; Jane A Hammond; Wayne Huggins; Dean Jackman; Huaqin Pan; Destiney S Nettles; Terri H Beaty; Lindsay A Farrer; Peter Kraft; Mary L Marazita; Jose M Ordovas; Carlos N Pato; Margaret R Spitz; Diane Wagener; Michelle Williams; Heather A Junkins; William R Harlan; Erin M Ramos; Jonathan Haines
Journal:  Am J Epidemiol       Date:  2011-07-11       Impact factor: 4.897

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  9 in total

1.  The Promise, and Challenges, of Methods to Enhance the External Validity of Randomized Trial Results.

Authors:  Elizabeth A Stuart; Catherine R Lesko
Journal:  Clin Pharmacol Ther       Date:  2020-08-08       Impact factor: 6.875

2.  Implementing statistical methods for generalizing randomized trial findings to a target population.

Authors:  Benjamin Ackerman; Ian Schmid; Kara E Rudolph; Marissa J Seamans; Ryoko Susukida; Ramin Mojtabai; Elizabeth A Stuart
Journal:  Addict Behav       Date:  2018-10-25       Impact factor: 3.913

3.  Improving pragmatic clinical trial design using real-world data.

Authors:  Susan M Shortreed; Carolyn M Rutter; Andrea J Cook; Gregory E Simon
Journal:  Clin Trials       Date:  2019-03-13       Impact factor: 2.486

4.  Generalizability of randomized trial results to target populations: Design and analysis possibilities.

Authors:  Elizabeth A Stuart; Benjamin Ackerman; Daniel Westreich
Journal:  Res Soc Work Pract       Date:  2017-07-27

5.  Target validity: Bringing treatment of external validity in line with internal validity.

Authors:  Catherine R Lesko; Benjamin Ackerman; Michael Webster-Clark; Jessie K Edwards
Journal:  Curr Epidemiol Rep       Date:  2020-06-30

6.  Generalizing randomized trial findings to a target population using complex survey population data.

Authors:  Benjamin Ackerman; Catherine R Lesko; Juned Siddique; Ryoko Susukida; Elizabeth A Stuart
Journal:  Stat Med       Date:  2020-11-26       Impact factor: 2.373

Review 7.  Clinical Trial Generalizability Assessment in the Big Data Era: A Review.

Authors:  Zhe He; Xiang Tang; Xi Yang; Yi Guo; Thomas J George; Neil Charness; Kelsa Bartley Quan Hem; William Hogan; Jiang Bian
Journal:  Clin Transl Sci       Date:  2020-04-10       Impact factor: 4.689

8.  Sensitivity analyses for effect modifiers not observed in the target population when generalizing treatment effects from a randomized controlled trial: Assumptions, models, effect scales, data scenarios, and implementation details.

Authors:  Trang Quynh Nguyen; Benjamin Ackerman; Ian Schmid; Stephen R Cole; Elizabeth A Stuart
Journal:  PLoS One       Date:  2018-12-11       Impact factor: 3.240

9.  Applicability of evidence from randomized controlled trials and systematic reviews to clinical practice: A conceptual review.

Authors:  Antti Malmivaara
Journal:  J Rehabil Med       Date:  2021-05-11       Impact factor: 2.912

  9 in total

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