The Precision Medicine Initiative (PMI) Cohort Program is an ambitious observational study backed by the National Institutes of Health (NIH) that seeks to enroll at least one million Americans by 2020. Because of its massive scale, the Cohort Program is often compared to the 1948 Framingham Heart Study. Yet a crucial differentiator is that the Cohort Program aims to recruit participants with diverse backgrounds and ethnicities to better understand the root cause of disease for all Americans.

The NIH’s Cohort Program will become “a template for the future” of clinical research, said Dr. Eric Topol, director, Scripps Translational Science Institute, and one of the principal investigators. “The Framingham Heart Study enrolled nearly all Caucasians, and it taught us more about heart conditions that we had ever known,” Topol said. Because the PMI Cohort Program specifically seeks to enroll people of varying ancestries, it will provide “an immense amount of medical knowledge” on the wide variety of factors underlying diseases, Topol continued.

“I think they’re getting it right this time,” agreed Dr. David Goldstein, director of the Institute for Genomic Medicine, Columbia University, and one of the principal investigators. Because of issues of access and historically rooted mistrust issues, some groups have been more willing and able than others to participate in medical research, Goldstein explained.

Even today, clinical trials do not capture the diversity of American demographics. More than 95% of all trial participants are Caucasian, according to a 2014 study by the University of California Davis Comprehensive Cancer Center. However, the NIH has developed a robust strategy to recruit a wide range of volunteers in the Cohort Program, and these efforts will pave the way for broader changes in recruitment strategies in the clinical trial industry overall, experts agreed.

The NIH has announced a $55 million award and shared details about its two-pronged enrollment strategy. A bulk of the one million volunteers will be recruited through traditional hospital networks, or Healthcare Provider Organizations (HPOs). The awardees include the Columbia University Medical Center, New York City; Northwestern University, Chicago; the University of Arizona, Tucson; and the University of Pittsburgh.

Because of the diversity of cities such as Chicago, New York and Tucson, the HPOs should be able to enroll patients representing a wide range of ethnic groups, said Dr. Philip Greenland, Harry W. Dingman Professor, Northwestern University Feinberg, School of Medicine, and a principal investigator on the Cohort Program. The NIH also launched a pilot program with six community-based HPOs that target underserved populations, and partnered with the Veterans Administration to ensure veterans have a chance to participate.

Yet the NIH also aims to enroll people without any explicit ties to the healthcare system. This second approach, called “direct enrollment,” is an innovative way to reach volunteers outside major hospitals and care centers, Greenland said. The NIH is supporting this new, and still somewhat experimental, method of enrollment because “[HPOs will only cover] a small population of the U.S., and they want to have this accessible to everybody,” Greenland said. The task of orchestrating direct enrollment falls to Scripps Research Institute in La Jolla, California, and Vibrent Health in Fairfax, Virginia, as well as a number of sub-awardees such as Walgreens, PatientsLikeMe and Sage BioNetworks.

Collectively, these groups comprise the Participant Technologies Center, which is responsible for enrolling at least 350,000 volunteers. Scripps is spearheading the enrollment efforts, while Vibrent is in charge of building the secure technology volunteers will use to enroll, consent and share their health data. Because 70% of Americans use smartphones, many volunteers will likely engage with the Cohort Program through mobile apps, said Vibrent CEO Praduman Jain. But volunteers without smartphones will be able to participate using more rudimentary feature phones through web applications or by dialing to an interactive voice response program, Jain said.

Scripps, meanwhile, has “formed quite a powerful network that touches every American in different ways,” Topol said. The Institute has teamed up with Apple, Verizon, Blue Cross Blue Shield, the National Blood Collaborative and many other entities with large consumer followings.

Regardless of where they are enrolled, all participants will be asked to share rich health information such as genetic sequences, medical history, biological samples and survey questions. As time goes on and some participants develop health conditions, this baseline information will provide crucial clues on the underlying cause of disease, allowing researchers to better predict risk factors and tailor new targeted treatments. Enrollment opens in November, Toprol said. Patients will ideally be followed for a minimum of 10 years, Goldstein added.

One of the main design principles of the Cohort Program is to identify “new ways of appealing to people participating in human research,” Greenland said. “They will clearly have to be different techniques than we’ve ever used before, because the scale of this program is never-before-seen,” he said.

Enrolling a diverse cohort is essential to the efforts to find new therapies and risk factors for all Americans, Goldstein added. “I think efforts like [the Cohort Program] will help build trust so that a broader range of people are willing and comfortable participating in research studies, clinical trials and the whole range of clinical research,” Goldstein said.  

This article was reprinted from Volume 20, Issue 29, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »