The Senate Committee on Health Education Labor and Pensions (HELP) May 5 held a hearing on continuing America's leadership realizing the promise of precision medicine for patients.
Much of the hearing focused on the role of the electronic health record (EHR) in precision medicine. HELP Committee Chair Lamar Alexander (R-Tenn.) noted his concerns with the electronic health care records system, saying “We spent $28 billion, it's a great idea, it holds great promise, but it is not working the way it is supposed to. The current standards for meaningful use are not clear. Upgrades are expensive. The systems do not work well enough to share the data. We hear it is expensive to share the data because of the relationships between vendors and doctors.”
He said that he and the committee’s ranking member Patty Murray (D-Wash.) “have begun a working group to identify the five or six things we can do to help make the failed promise of electronic health records something that physicians and providers look forward to, instead of something they endure.”
Noting that “protecting privacy will be an important challenge throughout this process,” Senator Murray cautioned, “As researchers and providers and patients gather and use more health information, we need to be aware that data is being created that cyber criminals will want to exploit, and that means we will need to develop strategies to protect privacy that meet today's challenges.”
She added, “Chairman Alexander and I are investigating the current state of cyber security in the health sector, and it's clear this needs to be an all hands on deck effort, with providers, and insurers, and government working together.”
National Institutes of Health (NIH) Director Francis Collins, M.D., Ph.D., opened the hearing by saying that the concept of precision medicine is not entirely new. “But the prospect of applying this concept broadly has been dramatically improved by the development of powerful and affordable methods for characterizing personal, biological information,” he told the committee.
“That includes genomics, the widespread adoption of electronic health records, the recent revolution in mobile health technologies, and the emergence of computational tools for analyzing large biomedical data sets. Furthermore, patients are increasingly interested in taking part in research. All of these developments will help make possible the dream of personalizing a wide range of health applications,” he added.
Dr. Collins described plans to launch “a National Research Cohort of one million or more volunteers, who will play an active role in how their medical, genetic, and environmental information is used to prevent and manage a broad array of diseases.” He added, “Participants, some recruited from existing NIH supported cohorts and some new volunteers, will be centrally involved in the design and implementation of this process.”
Karen DeSalvo, M.D., M.P.H., M.Sc., National Coordinator, Office of the National Coordinate for Health Information Technology at HHS, told the committee that health information technology “is foundational to the president’s Precision Medicine Initiative.”
“The data in electronic health records, married with advanced analytics, information for mobile health devices, and other sources of data, including patient preferences, will provide the fulsome picture of a person's health and needs,” Dr. DeSalvo explained, adding, “This comprehensive data picture is necessary to identify the right prevention and treatment that is not only the most effective, but also most desired by the patient.”
But Dr. DeSalvo advised, “To get there, we will need to stay the course in adoption to see that every American has an electronic health record. We also need to go beyond the pockets of data exchange, and achieve true interoperability as described in our nationwide road map. We will need to establish standards for the most fundamental clinical information that are shared by all. We will also need to establish standards for new data necessary for Precision Medicine, including genomics, but also environmental exposures and patient-generated information. We will build a trust framework that respects individual privacy and establishes strong security protections.”
Jeffrey Shuren, M.D., J.D., director, Center for Devices and Radiological Health at FDA, told the committee, “The success of Precision Medicine depends upon having accurate, reliable, and clinically meaningful tests.”
He explained that as a result of next generation sequencing “we are moving away from the model of one test, one disease, but a test that can identify one of many different diseases, or even the risk of developing that disease. But today there are significant barriers in place for advancing technology, and it affects research, and affects development, and it affects our ability to use this optimally in health care.”
Dr. Shuren said in December the agency “proposed an entirely different framework for the oversight of next generation sequencing tests…. For analytical validity, we need to have reference standards, essentially subsets of genetic variance, that if you can show accuracy in measuring those, it is reasonable to infer you are good measuring the other variants. And in fact, we gave $2 million to the National Institute for Standards and Technology (NIST) to work with the scientific community to come up with the very first reference standard for the genome, and they just released that last week.”
In response to a question from Senator Michael Bennet (D-Colo.) on FDA’s draft framework to regulate laboratory developed tests (LDTs) and whether congressional action would be helpful, Dr, Shuren acknowledged the important role the tests play in the health care system and said, “Our goal here is not shutting down laboratory developed tests, but in fact making sure that we are both facilitating innovation and that these tests are accurate, reliable, and clinically meaningful. Under our proposal, we try to strike that balance.”
He also expressed uncertainty if the FDA would receive thousands of tests to review “because what we've heard from the lab community is that a lot of the tests they make are to address unmet needs,” which would not require review under the proposed guidance.
Responding to a question from Senator Elizabeth Warren (D-Mass.), Dr. Collins said, “To get back on a stable [funding] trajectory that would result in a healthy biomedical research ecosystem … I would estimate, in my professional judgment, we would need to be at the pace of inflation, plus 4 or 5 percent per year.”
Dr. Collins also commented on the proposal announced last week as part of the latest 21st Century Cures discussion draft to create an NIH Innovation Fund supported by $10 billion over five years, saying, “[W]e were thrilled also to see what's in the 21st Century Cures, the $2 billion a year mandatory gave a great jolt of excitement, and some relief to a community that has been really quite stressed over the past 12 years as we have been losing ground.”
Senator Warren observed, “$2 billion a year for five years is certainly better than nothing. But let's not pretend that a small temporary investment that falls billions of dollars short of what we're going to need to do the job, there is a gaping hole in our NIH budget, and we need a serious plan to fix it. There are many ways to make that happen, I have a Medical Innovation Act, for example that could add another $6 billion a year, wouldn't cost taxpayers a dime. But whatever we do, this committee has to get serious about medical innovation, and that means we have to do better than the House proposal on this.”
Committee member questions included recommendations on how to improve the EHR, the role of children in the National Research Cohort, the impact of precision medicine on lowering health care costs, the steps ONC is taking to address cybersecurity, and interoperability between the VA and Department of Defense EHR systems, among other issues.
Chairman Alexander closed the hearing saying, “[I]t is my hope that we can finish our work on our innovation initiative this year, and report it to the full Senate early next year so it can be acted on."