Precision Medicine Initiative Unveils National Collaborative Framework and Large-Scale Research Architecture

Background of “Precision Medicine”

On January 25, 2015, in his State of the Union Address to Congress, President Obama, drawing on recommendations from numerous scientists at the National Institutes of Health (NIH), proposed an initiative for the U.S. life sciences community—particularly in basic, clinical, and translational medical research—to embrace “Precision Medicine” (Precision
Medicine Initiative, PMI). It aims to tackle major diseases plaguing the American public, such as cancer and diabetes, enabling more families and individuals to enjoy health and a high quality of life. In the subsequent nearly 10 months of widespread debate, the NIH convened a series of relevant forums and workshops in an attempt to further define what precision medicine “actually does, how it is implemented, and what outcomes are expected.” As most people have heard and seen, nearly everyone interprets the term “precision medicine” through the lens of their own research fields and interests, yet, like blind men touching an elephant, none fully grasps its precise meaning.

This July, the NIH held a two-day workshop to discuss the aforementioned issues, aiming to clearly outline the collaborative framework for the U.S. National “Precision Medicine” initiative. Meanwhile, the NIH invited a group of experts to form the “Precision Medicine” Advisory Committee, tasked with specifically developing the collaborative framework for the U.S. National “Precision Medicine” initiative. The report released today explicitly provides the definition of “Precision Medicine,” stating: “Precision Medicine refers to a series of measures taken in the process of treating and preventing diseases, based on individual differences in genetics, social environment, and lifestyle, to seek the optimal approach.” “Precision Medicine guides us to gain new insights into the onset and progression of diseases, as well as various feedback and outcomes during treatment; this includes disease and health conditions arising at the molecular and genetic levels, under different environmental conditions, and influenced by diverse lifestyle and behavioral habits. Therefore, Precision Medicine enables us to achieve accurate disease diagnosis, develop more comprehensive disease prevention strategies, select more effective treatment options, and apply innovative therapies, among other benefits.”

Core Components of the National Precision Medicine Framework

First, starting in 2016, efforts will be made to recruit one million participants as research subjects for the “Precision Medicine” initiative within the next three to four years. The NIH believes that studying the health and disease status of this one-million-person cohort will significantly drive innovative research in precision medicine, particularly in precisely determining personalized treatment plans based on three key factors: individual genetic information, living environment, and lifestyle. (Notably, individual socioeconomic status is not emphasized here; yet in real life, financial constraints force many residents in impoverished or underdeveloped regions into difficult choices.)

The NIH envisions the “architecture of precision medicine” as a framework that will usher medical science into a new era by guiding patient participation in investigator-initiated research projects through basic and clinical research, the application of both innovative and mature technologies, and proactive policy direction. While precision medicine initially encompassed numerous core elements, these now require further clarification. Notably, the federal government has allocated $215 million to support the NIH in leading frontier research in precision medicine, including areas such as cancer genomics. Of this amount, $130 million will be dedicated to the NIH’s effort to recruit and study a cohort of one million participants.

Meanwhile, the NIH will establish the “Precision Medicine Collaborative Network” to create the largest population-based health cohort study sample. It will also encourage all participating scientists to share data, biospecimens, and other research resources, fostering collaborative efforts to understand the fundamental causes of diseases and the impact of environmental factors on health. Therefore, the newly announced precision medicine framework places particular emphasis on: how to establish collaborative mechanisms; how to engage participants in research; and how to share data, biospecimen information, management policies, and standards.

Advocating “Precision Medicine”: What Are the Expected Outcomes?

Master the interaction mechanisms involving integrated environmental factors, intrinsic individual genetic factors, and environment-directed gene mutations, to develop quantitative analyses for assessing disease risk.
Differentiate individual variations observed and manifested during routine pharmacotherapy, as well as differences at the genetic level;
Develop variables reflecting the increase or decrease of biomarkers during the onset and progression of diseases, and use them to determine individualized differences;
Apply digital and mobile health technologies to monitor and observe individual activities, physiological signs, and the impact of environmental factors on individual health status;
Develop and reformulate disease classifications, as well as the interrelationships among diseases;
Encourage participants in precision medicine research to utilize their own data to improve their health outcomes;
Establish a nationwide collaborative platform to test and validate targeted therapy trials and products.

NIH Identifies Leaders for the “Precision Medicine” Initiative

Despite the fervent enthusiasm for “Precision Medicine” displayed by the NIH and the U.S. life sciences community over the past nine months, it is only now that the NIH has formally appointed Dr. Josephine P. Briggs, former Director of the National Center for Complementary and Integrative Health (NCCIH), as the head of the U.S. National “Precision Medicine” Initiative. Her responsibilities extend beyond merely steering America’s “Precision Medicine” initiative; indeed, elites in the fields of biology and medicine from countries around the world are watching her leadership as a bellwether for the future of “Precision Medicine.”

To browse more of Dr. Shi Zhanxiang's featured articles, please clickTable of Contents for Dr. Shi Zhanxiang’s Column

Published by VCBeat with authorization from Professor Zhan Xiang. For reprints, please obtain permission from the author.

WeChat Official Account: sasctm