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An Era Of Precision Medicine And Rapid Learning – Health Affairs Blog

An Era Of Precision Medicine And Rapid Learning – Health Affairs Blog

An Era Of Precision Medicine And Rapid Learning

February 20th, 2015 

At a recent White House event, President Obama presented his proposals for a Precision Medicine Initiative. The key elements include a national research system where 1 million or more volunteers can share their (privacy protected) electronic health records, genetics, and other data, and a national cancer initiative. The proposals will be developed in more detail based on meetings led by the National Institutes of Health (NIH) Director Francis Collins.
If national health policy adopts these proposals, much about today’s medical care system—including biomedical science, medical education, diagnostics, treatment options, comparative effectiveness research, quality metrics, payment systems, the role of patients, the personalization of medical care and prevention, and an understanding of the roles of environment, nutrition, culture, and many other factors—may greatly change.
The Obama administration proposes a highly collaborative, non-partisan public-private process. These proposals bring the era of “big data” to the center of the heath policy arena (see the July 2014 Health Affairs theme issue, “Using Big Data To Transform Care”). Many in the health system may want to take part in developing the proposals and being part of the implementation.

One Million Person Research System

In 2004, Francis Collins proposed a national research cohort, with at least several hundred thousand participants, for studies of genetics and the environment. With sharply declining costs of genetic sequencing, electronic health records for tens of millions of individuals, high-speed Internet, and high performance computing, this vision can now quickly be realized, at very large scale.
The National Research Council’s Precision Medicine report (2010) makes a compelling case for a new research system. It notes that most of our disease descriptions (ICDA codes) are out of date; what we now call one disease is often several diseases that share similar symptoms. Many treatments for chronic diseases, as now defined, show a few patients have many benefits, while many patients have few (or no) benefits. We need a precision medicine research system to advance medical knowledge about how and why we differ as individuals, to make new scientific discoveries and far more precise diagnoses, to develop targeted therapies, and to personalize medical care.
The Obama proposals are a foundation for a new era in medicine because they also go beyond the original genetic “biobank” concept to envision an “everything included” learning system, where computerized databases can potentially take account of anything that influences individual health. Such factors could include more physiological data, such as proteomics, environment, lifestyle, patient-generated information, and mobile device and sensor data.
Eric Topol’s book on The Creative Destruction of Medicine: How the Digital Revolution Will Create Better Health Care (2013) includes many insights about what may be possible and how it could change medicine. Once all the major influences on population health are “in the computer,” the speed at which scientists can generate new scientific knowledge will vastly accelerate. Today’s “petaflop” computers do a quadrillion operations per second. With the new data-rich system, much may be rapidly learned.
Many pieces of this new research system could come together within a few months. For example, 500,000 Kaiser Permanente members are volunteering their electronic health records and genetic data for a research database. More than 375,000 women have signed up for the Army of Women, started by Susan Love, to participate in breast cancer research.
The Million Veteran Program, at the Veterans Health Administration (VHA), has 350,000 veterans in its integrated genetics-electronic health records biobank. These examples show that Americans are willing to volunteer in large numbers for the kind of new national research system proposed by President Obama. They also provide important lessons for how such initiatives can be carried out at scale, with efficiency and privacy protections.
Filling coverage gaps. There are many gaps in the coverage of current biobanks that will need to be addressed. For example the Kaiser and VHA cohorts focus on older age groups (60+). A special effort will be needed to assure coverage of children and to address children’s health; the new National Pediatric Learning Health System, with leading child health research centers, could help to fill out that part of the new system.
Similarly, the Medicaid and Medicare disability and special needs populations are not adequately included in most research databases. The nation’s 25 million persons with more than 6,800 rare diseases, as well as minority populations, can benefit from national database initiatives. There is much work to be done to identify the most important new data types and sources that should be included in the new national research system.

Cancer Initiative

The second major piece of the Precision Medicine proposals is a cancer initiative to accelerate cures and create a national “cancer learning network” to guide new treatment decisions. Harold Varmus, the National Cancer Institute (NCI)’s director, will develop the initiative. He was awarded a Nobel Prize for cancer genetics research, and he headed the NIH and the Memorial Sloan Kettering Cancer Center.
There is consensus that it is time for a new national cancer strategy grounded in precision medicine. The Pulitzer Prize-winning book by Siddhartha Mukherjee, The Emperor of All Maladies (2010), documents the evolution of this new and promising way of understanding cancer and how it might be diagnosed, prevented, and treated in the future. Experts believe that breast cancer, for example, may be 10 or more different cancers, making it essential to have individualized diagnoses and treatments. In leading cancer centers, patients with breast, lung, and colorectal cancer, melanoma, and leukemia now routinely have molecular testing.
The cancer initiative could move ahead quickly. NCI has been prototyping a new Master Protocol system for cancer research that may sharply increase the number of cancer patients in clinical trials by offering treatment options that are predicted to be most effective for their cancers. It has also pioneered in international data-sharing and a cancer genome atlas.
National rapid-learning cancer systems have been proposed in Health Affairs (my 2009 paper on “Medicare’s Future: Cancer Care”) and in the 2010 Institute of Medicine report, “A Foundation for Evidence-Driven Practice: A Rapid-Learning System for Cancer Care.” The American Society of Clinical Oncology is rolling out a learning system for the nation’s oncologists (CancerLinQ) to accelerate precision cancer care and delivery of the best care for all cancer patients. The initiatives may prompt most cancer patients to request genetic testing, consultations, and personalized medicine.
The role of the Medicare program will need attention. Most cancer patients are in Medicare, so there will be many coverage and payment policies involved in widespread genetics testing and new personalized treatments. It is important to get this right, as the new era of precision medicine may soon involve genetic testing and new therapies for millions of Medicare patients.
The Center for Medicare and Medicaid Services (CMS) Innovation Center, with $10 billion to support better delivery systems, could be a useful player in the new cancer initiative. A worstcase scenario, however, could see Medicare expenses growing by billions of dollars to pay for new tests and treatments, while losing most of the potential learning opportunities, because of a failure to develop the Medicare patient data registries with the genomics needed for a rapid-learning cancer system.
As discussed in my recent Health Affairs article (“Rapid Learning: A Breakthrough Agenda”), the health system will need many upgrades for the new cancer and precision medicine initiatives to be fully successful.
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