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A Model of a Randomized Experiment with an Application to the PROWESS Clinical Trial

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  • Amanda E. Kowalski

Abstract

I develop a model of a randomized experiment with a binary intervention and a binary outcome. Potential outcomes in the intervention and control groups give rise to four types of participants. Fixing ideas such that the outcome is mortality, some participants would live regardless, others would be saved, others would be killed, and others would die regardless. These potential outcome types are not observable. However, I use the model to develop estimators of the number of participants of each type. The model relies on the randomization within the experiment and on deductive reasoning. I apply the model to an important clinical trial, the PROWESS trial, and I perform a Monte Carlo simulation calibrated to estimates from the trial. The reduced form from the trial shows a reduction in mortality, which provided a rationale for FDA approval. However, I find that the intervention killed two participants for every three it saved. [This paper has been combined with “Counting Defiers” (www.nber.org/ papers/w25671) and superseded by “Counting Defiers: Examples from Health Care” (https://arxiv.org/abs/1912.06739) as of July 17, 2020.]

Suggested Citation

  • Amanda E. Kowalski, 2019. "A Model of a Randomized Experiment with an Application to the PROWESS Clinical Trial," NBER Working Papers 25670, National Bureau of Economic Research, Inc.
  • Handle: RePEc:nbr:nberwo:25670
    Note: AG EH LS TWP
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    References listed on IDEAS

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    1. Donald B. Rubin, 1977. "Assignment to Treatment Group on the Basis of a Covariate," Journal of Educational and Behavioral Statistics, , vol. 2(1), pages 1-26, March.
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    Cited by:

    1. Amanda Kowalski, 2019. "Counting Defiers," Papers 1908.05811, arXiv.org, revised Jul 2020.

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    More about this item

    JEL classification:

    • C1 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods and Methodology: General
    • H0 - Public Economics - - General
    • I1 - Health, Education, and Welfare - - Health

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