Obama’s precision medicine initiative: an update

Despite significant challenges, President Obama’s ambitious initiative to lay the scientific foundation for precision medicine remains on track, say federal officials and outside experts.

Yet, the long-term effort to accelerate research aimed at helping clinicians tailor medical treatments to  individual patients must have funding to continue. Obama is asking Congress for $309 million to fund the Precision Medicine Initiative (PMI) in fiscal year 2017, up about $100 million from the previous year.

PritchardSome experts assert that PMI’s bipartisan support and enrollment of the first volunteers into its 1-million-person research cohort will also put pressure on Capitol Hill to keep the initiative going.

PMI is “a strong effort, a great commitment from the [Obama] administration, and I believe it will continue on ... regardless of the outcome of the elections,” Daryl Pritchard, PhD, vice president of science policy for the Personalized Medicine Coalition in Washington, D.C. told Managed Healthcare Executive earlier this fall. “... It’s not a controversial program.” The coalition's 225-plus members include Aetna and numerous academic health centers, community hospital systems and information management firms.

A complex initiative

The program, first announced by Obama during his 2015 State of the Union Address, has many moving parts. Funding for fiscal year 2016 provided $130 million to the National Institutes of Health (NIH) to create a 1-million-person research cohort (the PMI Cohort Program); $70 million to the National Cancer Institute (NCI) to ramp up efforts to identify genomic drivers in cancer; $10 million to the FDA to acquire more expertise and develop databases to support the regulatory framework for precision-medicine advances; and $5 million to the Office of the National Coordinator for Health Information Technology to develop interoperability standards and ensure privacy.

Anyone in the U.S. who is willing to share their medical information, take a health survey, get a baseline medical exam and provide a blood sample can volunteer to serve as part of the research cohort.

In February 2016, during a summit to discuss next steps, John Holdren, director of the White House Office of Science and Technology Policy, described PMI as "an all-hands-on-deck operation."

“Precision medicine holds incredible promise for the future of healthcare,” Holdren says. The initiative has brought the “relevant federal agencies to a new level of activity and collaboration in pursuit of that promise, and it’s drawing as well on the essential contributions of groups outside of government-providers, technologists, researchers, privacy and security experts, physicians, and, of course, patients.”

Next: Progress to date

Progress to date

• Infrastructure. NIH has made “significant headway” in establishing the infrastructure for the PMI Cohort

• Program, including a data and research support center, biobank, participant technologies center, and a network of healthcare provider organizations to help enroll and retain volunteers, collect samples and handle bio-banking, says Gwynne Jenkins, PhD, the program’s chief of staff at NIH. NIH's network of healthcare provider organizations includes regional medical centers (such as Columbia University Medical Center and  the University of Chicago).

• Jenkins

• Enrollment.NIH is not on track to recruit 79,000 individuals into the cohort by the end of 2016, federal officials say, but Jenkins describes that figure as an initial estimate. “We anticipate launching in phases, when systems are secure and ready to go and we can ensure a high-quality experience for our users,” says Jenkins. “When we are ready to enroll participants, we will spread the word.”

• Cancer research. As part of NCI’s contribution to the initiative, Jeff Abrams, MD, acting director for clinical research in NCI’s Division of Cancer Treatment and Diagnosis, says his division applied funding toward expanding its portfolio of genomic-based trials, improving the understanding of resistance to targeted agents and drug combinations, and developing a mechanistic understanding of immunotherapy.

The Precision Medicine Initiative in Oncology (PMI-O), which is part of the larger initiative, has also committed resources to improving pre-clinical models for evaluating targeted therapeutics, says Abrams, associate director of NCI’s Cancer Therapy Evaluation Program.

Finally, a major component of this overall effort was the launch of NCI’s Genomic Data Commons (GDC) in 2016. Abrams' NCI division intends to deposit molecular data from clinical trials into the GDC to foster secondary analyses and permit data mining by a broad range of investigators.

Next: From concept to integration

From concept to integration

Pritchard says his private-sector coalition is exploring strategies to rapidly integrate precision medicine into the delivery system, examining issues ranging from scientific discovery to regulation, reimbursement and clinical application. “I am seeing a rapid increase in the work we're making in the clinical adoption area now,” he says.

According to Pritchard, the federal effort still faces “considerable challenges, and they [i.e., federal officials] are aware of them.” He cites three primary hurdles:

• Patient empowerment. This includes data protection, consent, and ensuring volunteers for PMI's cohort are appropriately informed, he says. Information must get back to participants who are volunteering because of their interest in their particular genomic sequences as well as interest in the project per se.

• Information management. This will get the most attention because more information is being gathered for PMI than has likely been collected in any other research project, Pritchard says, “and you need to collect and manage that to create a learning health system.” Basically, he says, this means building a system that collects and stores outcomes data in a standardized way and informs participants in a timely manner.

• Significance determination. A third challenge is how to draw results and determine clinical significance, Pritchard says. Many of the genetic alterations studied will be relatively rare, he says, “but because you have 1 million patients, you can analyze rare occurrences statistically...and assess whether this truly makes a difference.”

ComiteAs for front-line challenges, Florence Comite, MD, an endocrinologist who runs a precision-medicine clinic in New York City and writes on the topic, describes “a rift between academic institutions and the practice of medicine.” Clinicians see the promise of precision medicine, she says, but the concept “doesn't yet have traction” as it does among academics.

Comite supports PMI, but views it as “a starting point.” Collecting a wealth of genomic data is exciting, she says, but it must be translated into actionable information for clinicians who often don't know how to make sense of “Fitbit” data, much less genomic information. “We're actually taught in medical school you don’t want to go on any fishing expeditions,” she notes. “Is one going to be actually able to take the scale of 1 million [participants in PMI’s research cohort] and extract it down to one human being? It's a complex universe we’ve entered into [that is] going to leave perhaps more questions than answers because of what we face in medicine.”

Judy Packer-Tursman is a writer in Washington, D.C.