Rare Disease Research Goes Viral

Laura Enderle, Associate Editor
Published Online: Monday, September 19, 2011
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Online patient communities are home to thriving discussions about unexplained symptoms, baffling test results, and the daily challenges of living with rare disease. A new study suggests the targeted social networks have another benefit—providing a steady stream of enthusiastic participants for clinical trials.

Recruiting patients for studies on rare diseases is notoriously difficult, but social networks are an untapped resource, a group of cardiologists reported in the September 2010 issue of the Mayo Clinic Proceedings. In a pilot project testing the novel method, the researchers were able to quickly enroll participants for a study about spontaneous coronary artery dissection, or SCAD.

Using social networks makes sense, both financially and strategically, said study coauthor Marysia Tweet, MD. “Patients with rare diseases tend to find one another and connect because they are searching for information and support,” she explained. “Studies of rare diseases often are underfunded, and people with these conditions are quite motivated.”

It was an especially motivated patient who helped launch the SCAD pilot. Frustrated by how little is known about the rare heart condition, SCAD survivor Katherine Leon spoke directly with Sharonne Hayes, MD, the study’s lead author and founder of the Mayo Clinic’s Women’s Heart Clinic. She asked Dr. Hayes what she could do to prompt more research on the disease.

Together, Leon and Dr. Hayes recruited patients through WomenHeart.org, a Web site that hosts a patient-directed support community for women living with heart disease. Within a week, 18 volunteers came forward—6 more than were needed to justify social media as a viable recruitment tool for the study’s next phase.

The test run was ordered to examine what is considered a radical departure from standard clinical trial procedures. “This is a completely different research model than Mayo Clinic is used to,” said Dr. Hayes. “Investigators here typically rely on the stores of patient information from the clinic. This was truly patient-initiated research.”
Dr. Hayes and her team plan to use social networks in addition to conventional methods to gather genetic data from up to 400 SCAD survivors and their relatives. The resulting biobank will be the first to contain enough information about SCAD to help researchers pinpoint its possible genetic origins.

Engaging the most motivated patients to kick-start trials recruitment is a promising tactic that could be applied to the roughly 7000 diseases that are currently tracked by the National Institute of Health’s Office of Rare Disease Research, according to Lee Aase, director of Mayo Clinic’s Center for Social Media.

“Patient leadership in this is huge,” he said. “Patients with rare diseases are highly motivated to see research happen.”



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