[Disclaimer: I am an employee of Celgene. The views reported here are my own.]
I presented at the PharmacoGenomics Research Network (PGRN) portion of the 2018 ASHG meeting (link to my slides here). A major theme from my talk was that precision medicine holds promise for advancing novel therapies, but that implementation of pharmacogenomics (PGx) will happen by design not by accident. Here is what I mean – and why our health care systems need to build for this future state today.
PGx by design – PGx by design starts at the very beginning of the drug discovery journey, when the choice is made to develop a therapeutic molecule against a target or a pathway. A precision medicine hypothesis is carried forward into the design of a therapeutic molecule (“matching modality with mechanism”), pre-clinical biomarkers to measure pharmacodynamic responses, and early proof-of-concept clinical studies in defined patient subsets. Late-stage clinical development is performed in these patient subsets, and regulatory approval is obtained with a label that defines this patient subset. Health care systems will essentially be required to incorporate precision medicine into patient care.
There are emerging examples of PGx by design. Indeed, there are an increasing number of FDA approvals that fit with the PGx by design model (see figure below).…