NIH Framework Points The Way Forward For Developing The President’s Precision Medicine Initiative
News Sep 21, 2015
Dr. Collins embraced the design recommendations made by the ACD, noting the need to remain nimble and adaptable as the Initiative progresses. He also thanked the Committee for their recommendations on policy issues and welcomed the opportunity to review them. NIH plans to move quickly to build the infrastructure so that participants can begin enrolling in the cohort in 2016, with a goal of enrolling at least 1 million participants in three to four years.
“We have an incredible opportunity to advance research and make new medical breakthroughs through precision medicine, which tailors disease prevention and treatment to individuals based on genetics, environment and lifestyle,” said Department of Health and Human Services Secretary Sylvia M. Burwell.
In order to advance this approach to medicine, President Obama proposed the Precision Medicine Initiative, which aims to enable a new era of medicine through research, technology and policies that empower patients, researchers and providers to work together toward development of individualized care. The Initiative includes many components with efforts from across the federal government and was budgeted at $215 million in fiscal year 2016 by the President. NIH will lead efforts in cancer genomics, as well as the development of the participant cohort. Of the total proposed in FY16, $130 million was allocated to NIH to build the research cohort.
Dr. Collins established the Precision Medicine Initiative Working Group of his ACD and tasked the group to develop a plan for creating and managing a large research cohort, with data and specimens that can be accessed by all researchers, for studies to understand the variables that contribute to health and disease, with the ultimate goal of developing more effective treatments tailored to individuals. The report drafted by the working group makes numerous recommendations on cohort assembly, participant engagement, data, biological specimens, policy and governance. The recommendations are based on a set of high-value scientific opportunities that were identified by the working group following extensive stakeholder engagement, including four public workshops and two requests for information. Among the scientific opportunities presented by this cohort is the ability to:
• develop quantitative estimates of risk for a range of diseases by integrating environmental exposures, genetic factors and gene-environment interactions;
• identify the causes of individual variation in response to commonly used therapeutics (commonly referred to as pharmacogenomics);
• discover biological markers that signal increased or decreased risk of developing common diseases;
• use mobile health (mHealth) technologies to correlate activity, physiological measures and environmental exposures with health outcomes;
• develop new disease classifications and relationships;
• empower study participants with data and information to improve their own health; and
• create a platform to enable trials of targeted therapies.
“Many factors have converged to make now the right time to begin this ambitious project,” said Dr. Collins. “Americans are engaging in improving their health and participating in health research more than ever before, electronic health records have been widely adopted, genomic analysis costs have dropped significantly, data science has become increasingly sophisticated and health technologies have become mobile. We have to seize this moment to invest in these promising scientific opportunities to help Americans live healthier lives.”