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Journal ArticleDOI

The practical importance of understanding placebo effects and their role when approving drugs and recommending doses for medical practice

Donald B. Rubin, +1 more
- 01 Jan 2020 - 
- Vol. 47, Iss: 1, pp 5-18
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TLDR
The general reliance on blinded placebo-controlled randomized trials, both to approve drugs and to set their recommended dosages, although statistically sound for some purposes, may be statistically naïve in the context of guiding general medical practice.
Abstract
The general reliance on blinded placebo-controlled randomized trials, both to approve drugs and to set their recommended dosages, although statistically sound for some purposes, may be statistically naive in the context of guiding general medical practice. Briefly, the reason is that medical prescriptions are unblinded, and so patients who receive drugs in practice receive both the active medical effect of the drug, as estimated in blinded trials, as well as any “placebo effects”, rarely carefully defined or estimated, but intuitively defined as the extra effect, on “you”, when you think you are being actively treated, even when in fact you may not be receiving anything that actually works.

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Journal ArticleDOI

Essential concepts of causal inference: a remarkable history and an intriguing future

TL;DR: Much of what is written about causal inference is found to be mathematically inapposite in one of these senses because the descriptions either include irrelevant clutter or omit conditions required for the correctness of the assertions.
References
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Journal ArticleDOI

Estimating causal effects of treatments in randomized and nonrandomized studies.

TL;DR: A discussion of matching, randomization, random sampling, and other methods of controlling extraneous variation is presented in this paper, where the objective is to specify the benefits of randomization in estimating causal effects of treatments.
Journal ArticleDOI

Inference and missing data

Donald B. Rubin
- 01 Dec 1976 - 
TL;DR: In this article, it was shown that ignoring the process that causes missing data when making sampling distribution inferences about the parameter of the data, θ, is generally appropriate if and only if the missing data are missing at random and the observed data are observed at random, and then such inferences are generally conditional on the observed pattern of missing data.
Journal ArticleDOI

Statistics and Causal Inference

TL;DR: In this article, the authors use a particular model for causal inference (Holland and Rubin 1983; Rubin 1974) to critique the discussions of other writers on causation and causal inference.
Journal Article

Identification of Causal effects Using Instrumental Variables

TL;DR: In this paper, a framework for causal inference in settings where assignment to a binary treatment is ignorable, but compliance with the assignment is not perfect so that the receipt of treatment is nonignorable.
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