Life Sciences Law & Industry Report connects the dots among the many disciplines that make up the burgeoning life sciences industry, with biweekly updates on current regulatory, legislative,...
Feb. 25— Advancing precision medicine is likely to require updates to regulatory frameworks both at the NIH and the FDA, President Barack Obama said Feb. 25.
The president said during a White House summit on precision medicine that such changes include realigning funding mechanisms at the National Institutes of Health to encourage data sharing as well as modernizing how the Food and Drug Administration reviews and approves new medical products.
“There’s going to be a whole range of areas where we may need new safeguards—for example, in terms of privacy and security of the data that’s being disseminated,” Obama said. “There may be other areas where we need to break down regulations that might have applied and made sense in another era of medicine but aren’t going to apply now. And that’s the kind of evaluation that we’re doing.”
The White House summit marked the one-year anniversary of the president's Precision Medicine Initiative (PMI) to advance an emerging model of health-care delivery that targets treatments to patient subgroups identified by their genetic makeup.
“So often what we label as a health-care system is actually more of a disease-care system,” Obama said. “One of the promises of precision medicine is not just identifying or giving researchers and medical practitioners tools to help cure people. It is also empowering individuals to monitor and take a more active role in their own health.”
He indicated plans to change the NIH system from one that heavily rewards scientists who get grants to one that instead fosters a culture of collaboration.
The president said the best researchers and universities often horde their samples because “that's how you get grants.” Research dollars flow in the direction of who gets credit for the science, Obama said, so rethinking how the NIH and other agencies design their grant-making processes will be important.
For the FDA, he said, regulating products by categorizing them as drugs and devices stemmed from a traditional thought process about protecting public health. Companion diagnostics are expected to be a critical component of precision medicine as devices will be used to screen patients to determine whether they'll benefit from specific drugs or therapeutics .
“When it comes to gathering data, disseminating data, making sure it’s accurate and valid, figuring out how it’s communicated to the patient or the individual who’s interested in it,” Obama said, “sometimes we’re fitting square pegs into round holes, and we may have to re-conceptualize how we think about this to open up this space.”
In conjunction with the summit, several federal agencies, including the NIH, announced updates to their work on the PMI.
The NIH is leading the largest part of the initiative, building a national research project that will involve at least a million volunteers. NIH Director Francis S. Collins said he'd like to enroll 79,000 cohort participants by the end of 2016 and reach a million by the end of 2019, in “the largest, most ambitious research project of this sort ever undertaken.”
“Building this national treasure will require innovative approaches and the engagement and trust of volunteers,” Collins said at the summit.
Vanderbilt University will develop the pilot project for building the million-person cohort, in collaboration with advisers from Verily (formerly Google Life Sciences), the University of Michigan and the Broad Institute of MIT and Harvard under a $1.2 million award from the NIH. Volunteers in the pilot will help the research team establish and test innovative methods and technologies for enabling robust participant engagement, as well as user-friendly data collection, which Collins said will lay the foundation for a “democratized, transformative research environment.”
Josh Denny, a bioinformatics professor at Vanderbilt and principal investigator for the pilot, called the precision medicine cohort, “a grand experiment on a scale that has never been tested before.”
“We’ll pilot how to authentically engage individuals to participate in the program and build the initial informatics and Web infrastructures to support it,” Denny said in a Feb. 25 statement.
Sen. Lamar Alexander (R-Tenn.), who is chairman of the Senate Health, Education, Labor and Pensions Committee and attended the summit, praised the pilot project announcement. The senator from Tennessee—where Vanderbilt is located—is leading a medical innovation initiative with Sen. Patty Murray (D-Wash.) as the Senate's companion effort to the House-passed 21st Century Cures bill (H.R. 6). A March 9 HELP markup that's part of the medical innovation legislative package is expected to include a bill on precision medicine.
“I’m committed to supporting the president’s Precision Medicine Initiative through our Senate health committee’s innovation agenda to ensure Americans can take advantage of this remarkable time in science,” Alexander said.
The NIH also has established its PMI Cohort Program Institutional Review Board (IRB)—to oversee and review of the program's research, Collins said. The IRB includes members with backgrounds in mobile health, bioinformatics, health disparities, epidemiology, genomics and environmental health.
The NIH has been at the forefront in creating central IRBs to ensure consistent, thoughtful and timely review of human research participant issues. Nancy Kass, director of the bioethics institute at Johns Hopkins University, will serve as chairwoman of the IRB.
Collins also announced:
“All of these actions represent enormous progress in the planning and implementation of this bold initiative,” Collins said, adding that in the coming months, the remaining foundation will be put into place to carry the program’s success over the next four to five years. “So, while I’m extremely gratified by the progress we’ve made over the last year, we have much work ahead of us.”
To contact the reporter on this story: Jeannie Baumann in Washington at email@example.com
To contact the editor responsible for this story: Randy Kubetin at firstname.lastname@example.org
More information about the summit is at https://www.whitehouse.gov/live/white-house-precision-medicine-initiative-summit.
Collins's full statement at the summit is at https://www.nih.gov/about-nih/who-we-are/nih-director/statements/preparing-launch-precision-medicine-initiative-cohort-program.
All Bloomberg BNA treatises are available on standing order, which ensures you will always receive the most current edition of the book or supplement of the title you have ordered from Bloomberg BNA’s book division. As soon as a new supplement or edition is published (usually annually) for a title you’ve previously purchased and requested to be placed on standing order, we’ll ship it to you to review for 30 days without any obligation. During this period, you can either (a) honor the invoice and receive a 5% discount (in addition to any other discounts you may qualify for) off the then-current price of the update, plus shipping and handling or (b) return the book(s), in which case, your invoice will be cancelled upon receipt of the book(s). Call us for a prepaid UPS label for your return. It’s as simple and easy as that. Most importantly, standing orders mean you will never have to worry about the timeliness of the information you’re relying on. And, you may discontinue standing orders at any time by contacting us at 1.800.960.1220 or by sending an email to email@example.com.
Put me on standing order at a 5% discount off list price of all future updates, in addition to any other discounts I may quality for. (Returnable within 30 days.)
Notify me when updates are available (No standing order will be created).
This Bloomberg BNA report is available on standing order, which ensures you will all receive the latest edition. This report is updated annually and we will send you the latest edition once it has been published. By signing up for standing order you will never have to worry about the timeliness of the information you need. And, you may discontinue standing orders at any time by contacting us at 1.800.372.1033, option 5, or by sending us an email to firstname.lastname@example.org.
Put me on standing order
Notify me when new releases are available (no standing order will be created)