President Barack Obama launched the "Precision Medicine Initiative” this past winter during the State of the Union address, and politicians on both sides of the aisle applauded the announcement. Broadly, precision medicine is meant to help diagnose individuals more accurately and better tailor treatment according to their physiology.

This approach has long been the goal of translational medicine, an allied field epitomized by the National Institutes of Health (NIH) Clinical and Translational Science Awards (CTSAs) program launched in 2006. The CTSAs were designed to fund the integration of basic and clinical science, so that discoveries in the laboratory could be more readily translated into new therapies, devices, or ways to predict and manage disease. 

Writing this week in Science Translational Medicine, Garret FitzGerald, MD, FRS, director of the Institute for Translational Medicine and Therapeutics (ITMAT) and chair of the department of Systems Pharmacology and Translational Therapeutics at the University of Pennsylvania's Perelman School of Medicine, stresses that to “influence emergence of the clinic of the future, one designed to practice precision medicine,” an NIH plan to establish large-scale collaborative clinical trials needs also to pay better attention to three areas of emerging practice. They include: 

• the application of adaptive trial designs, in which patients' early reactions  to drugs are used to modify trials' approaches  
• the integration of electronic medical records (EMRs) with biobank data to pinpoint potential new disease pathways
• the pursuit of human phenomic science (HPS), the cataloging of observable, detailed, evoked responses in small numbers of patients to find the significance of molecular pathways possibly involved in disease. 

FitzGerald’s comments were prompted by a shift in funding strategy by the National Center for Advancing Translational Science (NCATS). Roughly 20 percent of the current CTSA funding is being removed from university hubs to be used by NCATS, mostly to foster a network capable of large-scale, multicenter trials. 

“Even if a collaborative network of 62 CTSA institutes is a realistic near- or medium-term goal, it is highly unlikely to be as time or cost efficient as existing academic networks or CROs [commercial clinical research organizations] in the performance of large-scale clinical trials,” he writes. For now, the focus of the President’s Precision Medicine Initiative is on collecting and integrating vast amounts of clinical data from EMRs. But, this patient-record-based approach requires pairing with measures of actual physiology, pathology, or drug response, in small numbers of individuals to connect a person’s genetics and phenotype.

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