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

Amanda Kowalski

arXiv 16 Aug 2019 · Statistics — Methodology · 2 citations (OpenAlex)

arXiv:1908.05810 · PDF · DOI · OpenAlex · Extracted main text

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.

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19
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Most heavily cited references

The works this paper leans on most, across its whole bibliography — not restricted to papers in our corpus. Ranked by composite intensity, which combines how often a work is mentioned, how many sections mention it, and how much of that falls in the main text rather than the appendix.

ReferenceIntensityMentionsSectionsMain text
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3US Food and Drug Administration and others (2011) FDA drug safety communication: voluntary market withdrawal of xigris due to failure to show a survival benefit0.40511100%
4Foot, P (1967) The problem of abortion and the doctrine of double effect0.40511100%
5Holland, P. W (1986) Statistics and causal inference0.40511100%
6Wolfram Research, Inc (2018) Mathematica, Version 11.30.40511100%
7The Mathworks, Inc (2016) Matlab, Version r2016a0.40511100%
8Rubin, D. B (1974) Estimating causal effects of treatments in randomized and nonrandomized studies0.40511100%
9Rubin, D. B (1977) Assignment to treatment group on the basis of a covariate0.40511100%
10Siegel, J. P (2002) Assessing the use of activated protein c in the treatment of severe sepsis0.40511100%

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arXiv econ.EM papers that cite this one, ranked by how heavily they lean on it.

Citing paperIntensityMentionsSections
12212.141050.58531
2Counting Defiers0.51121