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PCORI May 2014

AAMC Patient Centered Research Newsletter

As I See It - If You Build It

"The upcoming years will be a watershed period for the future of comparative effectiveness research in the US." This quote from a May 2 Health Affairs blog post on PCORI epitomizes my own opinion on the urgency and importance of this moment for the research community. One phenomenon in particular is poised to rapidly transform the way research is conducted: the largest coordinated, national-level investment in connecting and mining big data for clinical outcomes research in American history, PCORI's PCORnet initiative. Nature recently hailed PCORnet — which supports the development of infrastructure to link and query data from Academic Medical Centers, insurers and other key players — as nothing less than "a dream cohort" for researchers.

Ann Bonham., Ph.D.
Chief Scientific Officer, AAMC

If You Build It

While PCORnet may seem ambitious or abstract to some, the scope and possibility of the initiative has already entered the public imagination. In their April 15 article "Scientists embark on unprecedented effort to connect millions of patient medical records" for instance, the Washington Post paired exuberant praise of the initiative's novelty and value with a thoughtful and optimistic examination of the challenges left to overcome: in addition to efforts to successfully engage patients in the process, systems must uphold high standards for privacy, security and reliability.

Meanwhile, research demonstrating the vast potential of integrated, searchable and robust health data is taking off. On his NIH Director's blog, Francis Collins recently described this big data as "priceless raw material for the next era of biomedical research," referencing how researchers can develop and test hypotheses without assuming the cost and time resources for new data collection. For instance, in "Disease Risk Factors Identified Through Shared Genetic Architecture and Electronic Medical Records" (from April's Science Translational Medicine), a team led by Dr. Atul Butte at Stanford School of Medicine describe a correlation between serum magnesium levels and gastric cancer identified within a large database of genome-wide association studies.

The authors describe how this link was further confirmed using de-identified EHRs to compare the occurrence of gastric cancer among tens of thousands of patients whose blood serum had been tested for magnesium, finding a significantly higher rate of the disease in those whose serum levels tested higher than normal. If this work piques your interest, learn more from Atul Butte's 2012 TedMed.

The imperative for progress is clear. It is our collective and proper responsibility to make the most of the information that patients provide to establish an infrastructure and expectation that patient- and population centered care is inherently evidenced-based. Randomized controlled trials (RCTs), are no doubt a gold standard, yet their costs can be prohibitive and their need for restrictive inclusion and exclusion criteria can mean that the results are hard to generalize to real-world patients and care conditions.

RCTs will continue to provide important information for efficacy, safety, and effectiveness studies, but pragmatic trials and observational studies based on networked data will allow researchers to generate hypotheses and answer questions about the risks and benefits for a broad spectrum of patients in a potentially more rapid and less resource-intensive manner. I recommend this Viewpoint on overcoming the barriers to conducting large simple trials from the April 9 JAMA for a deeper treatment of these issues by Eapen, Lauer, and Temple.

PCORI's investments in PCORnet are a critical step in creating the infrastructure and large scale demonstrations to help comparative effectiveness research reach critical mass. This is the kickstart we need to accelerate towards our vision of learning health systems that integrate research into care and support evidence-based decision making for both clinicians and patients. It will also pay forward benefits to other important research efforts.

Analysts and historians looking back on this window of time may well label PCORnet as PCORI's most enduring legacy.

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