PMI Stands Up, Takes First Steps

The NIH plan to implement the Precision Medicine Initiative Cohort Program drew wide support and applause at the ACD’s Dec. 10 meeting.

Ushering in a “new era of medicine through research, technology and policies,” the Precision Medicine Initiative aims to “empower patients, researchers and providers to work together toward development of individualized treatments.” The initiative has been on a fast track to fruition since President Obama announced it in his State of the Union address in January 2015.

NIH deputy director for science, outreach and policy Dr. Kathy Hudson, who served as cochair of the ACD’s working group on PMI, tag-teamed with interim cohort program director NCCIH director Dr. Josephine Briggs on a progress report.

Summarizing the working group’s recommendations from September, Hudson pointed out the importance of keeping NIH’s portion of PMI in context with the much broader scope of both the initiative and the global pursuit of precision medicine in general. PMI has two components at NIH—one at NCI and the cohort program. The latter was the focus of discussion at this ACD meeting.

PMI’s initial task, Hudson explained, is to assemble a generational unit, or “cohort,” of one million or more volunteers who reflect the diversity of the U.S., with a strong focus on underrepresented communities. The cohort will be longitudinal, with continuing interactions and opportunities built in for recontacting participants for secondary studies. Initial recruitment of a million participants is estimated to take 3 to 4 years using two methods: direct volunteer enrollment and via partnership with health care provider organizations.

Hudson said a driving issue that planners kept top of mind while devising the PMI cohort was how to maintain participant engagement and enthusiasm over such a long period of time.

“So what are the questions we want to be able to answer and how can we make sure that there is value for the research establishment, for health care providers, for the participants in the cohort and most important for the individual volunteers, early in the short term, in the medium term and in the long term?” Hudson asked, recalling deliberations to develop the cohort. “There’s a whole range of scientific opportunities that cover the waterfront—some that we can realize in the short term and some in the long term that we can’t even imagine today.”

She urged people who are interested in keeping up with cohort progress to use the dedicated web site www.nih.gov/precision-medicine-initiative-cohort-program and to follow #PMINetwork on Twitter.

Explaining the implementation steps already under way, Briggs described how the program will operate, its governance, enrollment targets and early budgetary expectations, announcement of the first funding opportunities and a proposed timeline.

“Translating the [myriad ideas and recommendations for the cohort] into a true, effective plan has been the work of many people,” she pointed out. “What has made the complex project manageable is the relatively high and meticulous level of detail from the working group report. Many key elements were clearly specified…building on the success of the BRAIN Initiative.”

ACD member Dr. Harlan Krumholz of Yale School of Medicine, heartily endorsed PMI’s fledgling steps.

“This is an extraordinary accomplishment,” he said. “There are a couple of revolutionary—not merely evolutionary—things about this that go far beyond the idea of being able to accumulate a million [participants]. One is this commitment to secure data fluidity and access, really going all in on the idea of open science…You are extolling values that will have ripple effects. The example and the principles you’re setting forth from the very outset of this, I believe, are going to have a fundamental impact on the way we see science going forward.

“The second revolutionary thing,” he continued, “is that you call them ‘participants.’ They’re not ‘patients’ and they’re not ‘subjects.’ The notion of partnership with the people who are going to be involved in this…It took leadership from the top—Francis Collins, I commend you—and it took courage to take this leap” from the traditional structure of medical science wherein scientists conduct the research and consult only each other about the results to this “adaptive, agile model of PMI,” wherein the power and knowledge that stem from science are shared from the beginning with the people.