Yesterday I participated in the National Academy workshop, “Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development” (link here).  There were a number of great talks from leaders across academics, industry and government (agenda here).

I was struck, however, by a consistent theme: most think that “precision medicine” will improve delivery of approved therapies or those that are currently being developed, whether or not the therapies were developed originally with precision medicine explicitly in mind.  Many assume that the observation that ~90% medicines are effective in only 30% to 50% is the result of biological differences in people across populations (see recent Forbes blog here).  This hypothesis is very appealing, as there are many unique features to each of us.

An alternative explanation is that most medicines developed without precision medicine from the beginning only work in ~30% patients because the medicines don’t target the biological pathways that make each of us unique.

I believe the most likely application is in the discovery and development of new therapies.  That is, I believe that the greatest impact will come when precision medicine strategies are incorporated into the very beginning of drug discovery, and will only rarely have an impact on therapies that were not developed with precision medicine in mind from the start.…

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For our website, we have chosen the term “precision medicine” rather than “personalized medicine”.  A recent News article in Nature Medicine reinforces this concept (see here). 

I have had many of my non-genetic physician colleagues comment to me: “We practice personalized medicine every day.  It’s called basic patient care!”  Their point: physicians see patients and make decisions about the best course of treatment based on patient preferences.  For example, one RA patient may prefer to have a drug infusion once per month and another patient may prefer to take a pill each day. 

The Nature Medicine article emphasizes  “the idea that molecular information improves the precision with which patients are categorized and treated“.  While personalized medicine might say “patient X with disease Y should get drug Z”, precision medicine says “patient X has a subset of disease Y — actually, disease Y3, not disease Y1, Y2 or Y4 — and patients with disease Y tend to respond more favorably to drug Z”.  Said another way bt Charles Sawyers, an oncologist at the Memorial Sloan-Kettering Cancer Center in New York: “we are trying to convey a more precise classification of disease into subgroups that in the past have been lumped together because there wasn’t a clear way to discriminate between them“.…

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