Is it possible to eliminate the groups of placebos in all those clinical trials that have a precision medicine nature? Since if it is precision medicine, it is supposed to cure 100% of those affected by the disease, and what you have to do is compare with the common statistics associated with those who suffer from the disease. After all, if everyone is cured by the placebo effect, the substance (in terms of its psychological effect) is a success in treating the disease.

At minute 6 in this https://youtu.be/IWu6XWzbPBs?t=360 video there are some examples.

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    You usually compare either treatment vs placebo or new treatment vs current treatment. The difference in outcome is the difference between the two. So if a new drug decreases mortality by 4%, it means mortality by 4% in comparison to the placebo or control group.// I'm not aware of anything that is able to cure 100% of a disease. The interactions are much too complicated to guarantee a 100% success rate, and complications may always arise.
    – Narusan
    Commented Aug 11, 2020 at 8:27
  • As you point out, a new precision medicine that's 100% effective is completely uninteresting if the placebo is also 100% effective. If the placebo is 90% effective, it's still not that exciting, but if the placebo is 0% effective, you have a new useful drug. Without the control arm, you'll never know how effective the placebo is. I don't see any rationale here for eliminating control arms. Commented Aug 18, 2020 at 15:46

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The National Research Council has explained that:

“Precision Medicine refers to the tailoring of medical treatment to the individual characteristics of each patient. It does not literally mean the creation of drugs or medical devices that are unique to a patient, but rather the ability to classify individuals into subpopulations that differ in their susceptibility to a particular disease, in the biology or prognosis of those diseases they may develop, or in their response to a specific treatment.”

WIKIPEDIA accessed 8/17/2020

National Research Council (US) Committee on A Framework for Developing a New Taxonomy of Disease. Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease. Washington (DC): National Academies Press (US); 2011. PMID: 22536618. Appendix E. Glossary. Precision Medicine

“Precision” treatments—ones that might work better in a particular subpopulation--still need to be studied to see if they do work better in a particular subpopulation. Some of these studies might involve use of a placebo.

There are, however, some (a few) “precision” treatments that have been developed to treat a single patient. A good account of the development of a precision treatment for a single patient is given in a lay journal.


The case is also described in publication in the medical literature. https://www.nejm.org/doi/10.1056/NEJMoa1813279

In this instance, the drug to treat a rare condition was identified and tested in this patient in a study called an N-of-1 study. That is, the drug was tested in this and only this patient. The patient improved on the treatment. This N-of-1 study did not involve use of a placebo.

So the answer to your question is “Yes, it is possible (sometimes) to eliminate the use of a placebo when evaluating a treatment that has been developed based on the principles of precision medicine.”

Note that this patient improved but was not cured by this truly precision treatment. Precision medicine does not always cure a patient.

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