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

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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 naïve 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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Acknowledgements

I wish to thank the editorial board for their helpful comments and their encouragement to expand Section 1 to include more details of my transitions between academic fields.

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Authors and Affiliations

  1. Yau Mathematical Sciences Center, Tsinghua University, Beijing, China

    Donald B. Rubin

  2. Department of Statistical Science, Fox School of Business, Philadelphia, PA, US

    Donald B. Rubin

  3. Emeritus Professor, Department of Statistics, Harvard University, Cambridge, MA, US

    Donald B. Rubin

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  1. Donald B. Rubin
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Correspondence to Donald B. Rubin.

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Communicated by Maomi Ueno.

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Rubin, D.B. The practical importance of understanding placebo effects and their role when approving drugs and recommending doses for medical practice. Behaviormetrika 47, 5–18 (2020). https://doi.org/10.1007/s41237-019-00091-7

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  • DOI: https://doi.org/10.1007/s41237-019-00091-7

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