Mobile Health Gives Clinicians a Platform for Managing Rare Diseases

Healthcare providers who treat patients with Marfan syndrome are hoping a new mHealth platform will help them improve care management and coordination – and give researchers access to digital health data to study the rare disease.

The Marfan Foundation has unveiled a mobile health app, developed by Backpack Health, that allows people living with Marfan syndrome to collect health information from disparate mHealth devices and medical records platforms and create a personal health record. The app also helps providers manage care for patients who often see several specialists.

“It is difficult for families in our community to organize their medical records, track healthcare needs and keep communications seamless among the countless doctors needed to manage their condition,” Michael Weamer, President and CEO of the New York-based Marfan Foundation, said in a press release. “This partnership with Backpack Health enables our community to have a seat at the table when it comes to talking about their health and tracking their personal health records so that they can better navigate their complicated medical journey.”

Marfan syndrome is a life-threatening genetic disorder that damages the body’s connective tissue and affects roughly 200,000 Americans. Treatment involves medication and monitoring daily activities, as well as care coordination with specialists.

“Living with Marfan syndrome requires working with many specialists and, oftentimes, traveling among multiple hospitals to find skilled care. Having records transferred from one doctor to another is a huge pain,” Maya Brown-Zimmerman, who was diagnosed with Marfan syndrome as a child, added in the press release. Coordinating records through the mHealth app, she said, allows her “to manage my health and share information with all of my providers.”

People living with rare diseases – and the clinicians who treat them – are turning in increasing numbers to mHealth platforms to improve care management and coordination. Aside from gathering data on complex medical conditions, they’re using wearables and telehealth devices to gain better insight into daily activities that may affect the disease, and they’re collaborating with patients and providers across the globe on best practices and new forms of treatment.

Pharmaceutical companies are also interested in these mHealth interventions, seeing them as a means of gathering better information on a particular drug’s effectiveness. Those in the industry say mHealth platforms can help them develop better medications with fewer side effects.

One example of this is the partnership formed by the Lupus Research Alliance and Pfizer. Last June, they launched an app on Apple’s ResearchKit platform as part of its VALUE (VAlidation in LUpus of an Electronic Patient Reported Outcomes Tool) study, which looks to allow patients of the debilitating chronic disease to report this conditions through an iPhone.

“This is an important step in demonstrating that mobile technology can improve how and what patients report to their care teams about subjective but serious symptoms of Lupus, such as debilitating fatigue,” Kenneth M. Farber, the alliance’s co-CEO and co-president, said. “This app may enable more frequent and consistent reporting from patients, thus providing better information for care teams and empowering patients to take a larger role in developing future therapies.”

Also in 2017, Penn Medicine launched a new app on the ResearchKit platform to collaborate with providers treating sarcoidosis, an inflammatory condition that affects only a few thousand people around the world. Through the app, researchers are hoping to gain insights on treatment and gather data from patients that can be used to study the disease.

This new app has the potential to build up a larger cohort of more diverse patients in a shorter amount of time,” said Misha Rosenbach, MD, an assistant professor of dermatology in the Perelman School of Medicine at the University of Pennsylvania and one of the leaders of the ResearchKit study. “There’s a motivated and engaged group of sarcoidosis patients who are active online, but there’s a whole host of them out there we don’t know about. This app, which can securely, privately, and anonymously collect data, casts a wide net that may engage those people, and funnel valuable, much-needed information to researchers.”

“There’s a great opportunity that has never been done,” said Rosenbach, who’s collaborating on the project with Daniel O’Connor, a fourth-year Penn medical student, and Marc Judson, MD, of Albany Medical College and the Foundation for Sarcoidosis Research. “In traditional research, you can’t see patients every day, but in app-based research you can suddenly get all this information about the disease in real-time and over time, from many different patients all over the world. It gives us the power to do sarcoidosis research in a way that has never been done.”

The Marfan Foundation is hoping to do the same with its new app. They’re also launching the first-ever Marfan and Related Disorders International Patient Registry, which will gather de-identified data for studies “that advance understanding of Marfan syndrome and related disorders.”

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