NIH Director Francis S. Collins, M.D., Ph.D., has accepted an advisory panel’s recommendations for implementing a key component of President Obama’s Precision Medicine Initiative (PMI)—creating a research cohort of 1 million or more Americans from which to glean data.

NIH said it plans to move quickly to build the cohort’s infrastructure so that participants can start enrolling next year. The agency has set a goal of enrolling at least 1 million participants in three to four years.

The Advisory Committee to the Director voted unanimously to accept policy recommendations developed by its Precision Medicine Initiative Working Group and contained in a report released yesterday. Dr. Collins, in turn, said in a tweet he accepted the report and its recommendations.

“The Precision Medicine Initiative Cohort Program will be a broad, powerful resource for researchers working on a variety of important health questions,” Dr. Collins said in a statement. “It will seek to extend precision medicine’s success with certain types of cancers to many other diseases, including common diseases such as diabetes, heart disease, Alzheimer’s, obesity, and mental illnesses like depression, bipolar disorder, and schizophrenia, as well as rare diseases.

“Importantly, the cohort will focus not just on disease, but also on ways to increase an individual’s chances of remaining healthy throughout the lifespan,” Dr. Collins added.

In February, Obama requested $215 million for PMI for the federal fiscal year that begins October 1. The NIH’s share of that proposed spending is $130 million, which is envisioned to fund the research cohort as well as lead efforts in cancer genomics. Obama unveiled PMI in his State of the Union Address on January 20.

The working group was tasked with developing a plan for creating and managing a large research cohort—with data and specimens that can be accessed by all researchers—for studies on the variables that contribute to health and disease. The ultimate goal, according to NIH, is to develop more effective treatments tailored to individuals.

The Precision Medicine Initiative Cohort Program—Building a Research Foundation for 21st Century Medicine recommended allowing any person living in the U.S. to voluntarily enroll in the cohort directly or through participating healthcare providers. Enrollees would volunteer to share core data—including their electronic health records (EHRs), health survey information, and mobile health data on lifestyle habits and environmental exposures. They would also undergo a standard baseline exam for vital signs, medication assessment, and past medical history and provide a blood sample.

“All potential participants in the PMI cohort must agree to share their health data, provide a biospecimen, and be recontacted for future research,” the report said. “The Working Group also recommends that the PMI cohort reflect the diversity of the U.S.”

In return, the report added, participants will have access to their study results, along with aggregated results from all study participants, and will be provided with tools to interpret the results. The NIH insists that data gathered by the cohort would be privacy-protected and secure.

The working group also called for centralized collection and storage of a diverse set of data: “The Working Group recognizes a number of types of biospecimens that could be collected, including blood, microbiome specimens, and nail and hair clippings, among others.”

In addition, the working group recommended, internal policies will need to be developed to address participant inclusion. Those policies, the group said, should address  institutional review board review and consent; privacy, misuse of information, and security; sharing of data and specimens with researchers; and sharing of data and research results with participants.

The Working Group identified several “scientific opportunities” presented by the cohort, including:

  • Developing quantitative estimates of risk for a range of diseases by integrating environmental exposures, genetic factors and gene-environment interactions;
  • Identifying the causes of individual variation in response to commonly used therapeutics;
  • Discovering biomarkers that signal increased or decreased risk of developing common diseases;
  • Using mobile health technologies to correlate activity, physiological measures and environmental exposures with health outcomes;
  • Developing new disease classifications and relationships;
  • Empowering study participants to improve their own health with cohort data and information; and
  • Creating a platform to enable trials of targeted therapies.

NIH also named Josephine P. Briggs, M.D., as interim director of the NIH Precision Medicine Initiative Cohort Program, and “will immediately launch a vigorous national search” for a permanent director.

Dr. Briggs is the director of NIH’s National Center for Complementary and Integrative Health. She is a nephrologist whose research has focused on mechanisms of diabetic kidney disease, and “brings extensive experience in oversight of clinical research to interim leadership of this program,” the agency added.

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