Human Research Issues of Precision Medicine Study Explored

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By Jeannie Baumann

Feb. 29 — As the NIH works on enrolling a million participants in its precision medicine study, there's already one willing volunteer: the study's IRB chairwoman.

“If I can, I’m going to join the cohort,” Nancy Kass told Bloomberg BNA, adding that she is motivated both by her belief in the study as well as a desire to make sure the institutional review board is aware of any interaction she doesn't think is right. “I don’t mean to suggest that my experience represents the experience of a million people. But it’s something that I have decided to want to do.”

Kass is a bioethicist and deputy director for public health in the Johns Hopkins University Berman Institute of Bioethics in Baltimore. She also has been tapped by the National Institutes of Health to lead the central IRB for the Precision Medicine Cohort Program, a national, multiyear study designed to develop more effective ways to prolong health and treat disease. The cohort program is the largest arm of the White House Precision Medicine Initiative (PMI) to advance an emerging model of health-care delivery that targets treatments to patient subgroups identified by their genetic makeup.

NIH Director Francis S. Collins, who called the program an unprecedented effort, announced the establishment of the IRB Feb. 25 as part of a series of updates during a White House summit on precision medicine.

Carrie D. Wolinetz, who is the NIH's associate director for science policy, said that in establishing the IRB, the agency wanted to make sure that participants had a strong representation. She told Bloomberg BNA this is one of the principles of the PMI. While there are no actual participants on the IRB yet because the protocol hasn't started, the NIH wanted to ensure that the IRB members include “voices that represent the diversity of communities of potential participants.” The NIH also wanted to include specialized expertise that might be of particular use to precision medicine, such as bioinformaticists, health technology experts and other specialties that were closely related to the cohort program.

According to the PMI funding notice, “The PMI Cohort Program will have a single Institutional Review Board (IRB), to the extent permitted by law, constituted to ensure prompt and thoughtful consideration of the evolving protocols in the PMI Cohort Program and the central importance of participants as research partners. The PMI Cohort Program IRB will include significant representation by members of the public and representatives of the participant community.” Collins said in his Feb. 25 statement, the “NIH has been at the forefront in creating central IRBs to ensure consistent, thoughtful, and timely review of human research participant issues.”

Kass and Wolinetz spoke to Bloomberg BNA Feb. 29 in a joint interview about the IRB and human research protection issues related to the PMI program.

Same Fundamental Ethical Issues

The fundamental ethical responsibilities of the cohort IRB remain the same as for any other IRB, Kass said, but there are particular issues that require sensitivity.

First, Kass said, volunteers must clearly understand what they have agreed to do, specifically, that they will upload their medical record data and biological data, and they will need to be re-contacted during the course of the study.

“It’s not rocket science,” she said. “But it’s important for people to know that when they sign up for this, they’re giving permission to be recontacted because that’s how it works.”

Kass added, “It’s important for people to know that by virtue of agreeing to be part of the database, knowledge does not instantly happen, but they’re officially being asked to join a community that will then be kept for additional and very specific projects.”

“It’s really, really important to get this right,” Kass said, adding, “This is the largest research enterprise that our government has ever invested in, and in that way [the cohort] has so much visibility for the American public.”

The cohort IRB will need to invest more time than an institutional IRB in providing public information and transparency, going beyond the informed consent interaction between the one-on-one provider and participant, Kass said. There also needs to be a lot more public engagement about how these data will be used, and more accountability to ensure any findings are publicized “so the American public understands this is not only an enormous investment of their tax dollars and a big ask of the American public to give literally of their time and their blood but that there’s payoff that we’re learning things to improve the health of the public.”

“While not all of these tasks will be the sole responsibility of the IRB, I would imagine that it’s part of the IRB’s responsibility to make sure that each of these things is done responsibly,” Kass said.

Reliance Agreement, Local IRBs

Wolinetz said she expects the interaction between the central IRB and the local IRB to be the same as any single IRB situation, in which reliance agreements have been worked out between the central IRB and the local institution. She noted that the NIH has just announced that Vanderbilt University will run the pilot program for enrolling volunteers, which means a limited pool of institutions will be involved in the initial phase.

“We’re not at the point where we’ve got a resource in which we’ve got a lot of institutions and investigators coming in with research proposals,” Wolinetz said. “We have some time to lay the groundwork for the type of reliance agreements and those sorts of things to put in place. I think that’s not going to be a problem.”

Common Rule Changes

The NIH's goal is to enroll 79,000 cohort participants by the end of 2016 and reach a million by the end of 2019. In that time, the regulatory landscape for IRBs will change if the Department of Health and Human Services adopts the notice of proposed rulemaking to modernize the federal human research regulations (45 C.F.R. Part 46) known as the Common Rule . Those proposed rules include a number of possible changes to the consent requirements, including reclassifying the review for biospecimens.

“If the rules change, then what the IRB puts in place will change,” Kass said, adding that she supports the proposal to require all consent forms to be shorter and simpler. “The IRB is required by law to be consistent by whatever is required from a regulatory perspective.”

Next Steps

Wolinetz said the IRB will have its first meeting sometime in the next month.

“It will be an orientation for new members,” she said, which is pretty typical in setting up a new IRB and then moving into the first meeting.

To contact the reporter on this story: Jeannie Baumann in Washington at

To contact the editor responsible for this story: Lee Barnes at

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