Advancing Research by Giving Patients Greater Access to Their Health Data
We know we could do a better job of treating many diseases and conditions if we could make better use of health data. So I was very pleased to join my colleague Sharon Terry, head of the Genetic Alliance, and some 200 patients, clinicians, researchers, policy makers, and others at the White House recently for an important update on a bold effort to do just that.
The Precision Medicine Initiative, or PMI, seeks to gather a range of health information—including genetic data, bio-specimens, clinical records, and information reported by patients themselves—from more than 1 million people. Its goal is to create a research resource that can help develop treatments tailored to individual patients.
PCORI's work is a natural complement to efforts, like the PMI and Congress’s 21st Century Cures initiative, to speed the delivery of new, innovative, targeted treatments to patients. The research PCORI funds examines newly approved treatments as well as those already in wide use. After all, even if new treatments are tailored to individual patients’ genetic makeup, many questions will remain about how well those therapies work in real-world settings, where environment, patient preferences and behavior, and other non-genetic factors can influence outcomes.
That's why the research we fund is so important. It provides the opportunity to evaluate more broadly and over a longer term what works for whom, in different situations and care settings, focused squarely on outcomes that patients have told us are important to them. Our work also can identify those for whom currently available treatments don’t work well or might be harmful, information that can guide the search for new approaches.
Advancing Precision Medicine Through Innovative Use of Health Data
One of the more ambitious ways we’re supporting precision, or personalized, medicine is through PCORnet, the National Patient-Centered Clinical Research Network. This is our initiative to harness the power of health data and patient partnerships to conduct patient-centered outcomes research faster, with more power, and at lower cost than is possible now.
It was one of the unique aspects of PCORnet that brought Sharon and me to the White House—the active participation of patients and others who want to drive research to improve care and outcomes. Sharon founded the Genetic Alliance, a health advocacy group, after her two children were diagnosed with a rare genetic condition. As chair of the PCORnet Engagement Committee, Sharon is leading the Building Trustworthiness in PCORnet workshop, March 28-29 in Washington, DC. This is an opportunity that will bring together patients, caregivers, clinicians, industry, advocacy groups, the research community, and the broader public at large to begin a critical dialogue about ethics, trust, and engagement, not just for PCORnet, but for clinical research overall.
The PCORnet Patient-Powered Research Networks (PPRNs) comprise patient groups and partners focused on particular conditions or other characteristics of special interest to them. They’re dedicated to helping participants play an active role in patient-centered research. So, Sharon and all of us at PCORI are particularly attuned to helping find ways to allow people who wish to obtain and share their personal health data, with appropriate safeguards, to do so in ways that support research that could improve care for their conditions.
That's what we announced as part of the PMI summit—a pledge by our 20 PPRNs to help their participants more readily obtain information from their electronic health records, or EHRs. The PPRNs will also encourage their participant members to share their health information with researchers approved by the network’s members to support studies identified as highly relevant and useful. Because they are built to focus specifically on the needs of their participants, the PPRNs are uniquely positioned to facilitate the flow of health data and to collect and add patient-provided data for research and healthcare improvement.
Helping People Access and Share Their Health Data
The PPRNs will help participants retrieve their own EHR data through existing participant portals and offer them an immediate option to make these data available for research. With guidance and input from patients and other stakeholders, the PPRNS are evaluating and employing ways to help people acquire their EHR data. For example, nine PPRNs are gathering data electronically either directly from healthcare providers or through intermediaries, including several of the 13 health system-based Clinical Data Research Networks (CDRNs) that also are part of PCORnet.
Information shared by patients is crucial to meeting PCORI’s goal of enhancing the quality, efficiency, and usefulness of clinical research for patients and other end users of the results. We and our PCORnet colleagues believe that the research we support must be informed by the everyday experiences of ordinary patients, so it addresses the questions that matter most to patients and the results are useful and widely applicable. These are the kinds of issues the PCORnet Trustworthiness workshop is designed to address.
In encouraging patients to contribute their data to support research, we know that privacy and security safeguards are essential. I’m confident that, together, we can craft an approach that will balance critical privacy and security concerns with the tremendous opportunities that appropriate use of this kind of health information provide to improve care and patient outcomes.
Throughout our rewarding day at the PMI summit, Sharon and I heard stories from people who have benefited from early efforts to better tailor treatment to patients based on their individual characteristics. All of them are clearly dedicated to helping others and advancing the goals of this new approach to clinical research. It was especially heartening to hear President Obama say that individuals own their health data, and that the practice of others “hoarding” individuals’ data needs to change. That practice does not serve the interests of the majority of patients who would like to see their data used to improve their health status.
The meeting was a powerful reminder that we’re well on the way to a day when health research will no longer be the sole province of scientists, and patients will sit at the table with researchers from start to finish.