Unleashing Innovators to Launch Precision Medicine

The White House has announced a large research initiative aimed at advancing “precision medicine” in the U.S. The term refers to an array of prevention strategies and medical treatments designed for the individual, rather than the average, patient.

The concept is not new. One common example that has been around for more than a century is blood typing, to guide blood transfusions. But the prospect of applying precision medicine much more broadly has been dramatically improved by the recent development of enormous biologic databases, powerful computational tools to analyze them, and technological advances in patient categorization. The latter group includes new fields of study such as proteomics, metabolomics and genomics.

The sequencing of the human genome provides a recent example of precision medicine at work: a small but reliable percentage of all cases of cystic fibrosis are caused by a specific mutation in one gene. A drug developed to target that mutation lets patients breathe easily.

This new way of looking at diseases is not limited to genes but examines environmental factors as well. Federal officials say researchers plan to collect data on a person’s diet, exercise, smoking history, exposure to toxins and populations of microorganisms in and on the body. They will be seeking medical records, laboratory test results, medication histories and even physiological monitoring by mobile phones. (The White House will seek advice from patients, bioethicists, civil libertarians and others on how to do this while preserving patient privacy.)

It’s clear that what is needed now is a broad research program to encourage creative approaches to precision medicine, test them rigorously, and ultimately use them to build the evidence base needed to guide clinical practice.

The new federal initiative has two main components: a near-term focus on cancers and a longer-term aim to generate knowledge applicable to the whole range of health and disease. The initiative taps into converging trends of increased connectivity, through social media and mobile devices, and Americans' growing desire to be active partners in medical research.

The 2016 budget will allot $215 million toward the following:

  • National Institutes of Health: assemble a national research cohort of at least one million volunteers who will contribute their health data (e.g., medical records, genetic profiles) "to propel our understanding of health and disease."

  • National Cancer Institute: ramp up efforts to identify genetic determinants of cancer and, in turn, develop better cancer therapies.

  • FDA: create high quality databases in an effort to "advance innovation in precision medicine and protect public health."

  • Office of the National Coordinator for Health Information Technology: promote "standards and requirements" to ensure that patient data can be shared safely.

Technology helps make precision medicine finally feasible. The cost of sequencing a person’s genome has fallen to around $1,000, far less than the $3 billion it cost to sequence the first human genome more than a decade ago. Electronic medical records allow quick access to a patient’s medical history, and data sharing technology will allow researchers to tap into the results of clinical trials, insurance plan information and other databanks. Precision medicine is an idea whose time has finally come, and the administration is prepared to invest in it. 

White House Fact Sheet, Precision Medicine Initiative, Jan. 2015, http://www.whitehouse.gov/the-press-office/2015/01/30/fact-sheet-president-obama-s-precision-medicine-initiative

NEJM, Perspective, Jan. 2015, http://www.nejm.org/doi/full/10.1056/NEJMp1500523

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