Rare Disease Research Goes Viral
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.”
Insulin Pump Hacking Unlikely, Says Medtronic
Medtronic’s insulin pumps were the subject of heated debate in August, when a computer security researcher took the company to task for a flaw in its pumps that could allow hackers to control the devices remotely, causing them to release incorrect doses of insulin or shut down completely.
Type 1 diabetes patient Jay Radcliffe performed the hack on his own pump before an audience of experts at the Black Hat security conference in Las Vegas, Nevada. News outlets picked up on the story, prompting a statement from Medtronic insisting that the risk of malicious insulin pump hacking is “extremely low,” and that the next generation of pumps will feature added protection.
The spectacle caught the attention of 2 members of Congress, who raised concerns about similar vulnerabilities in other medical devices. Representatives Anna G. Eshoo (D, CA) and Edward J. Markey (D, MA) urged the Government Accountability Office to investigate medical devices with wireless capabilities to ensure that they are “safe, reliable, and secure.”
Not satisfied with Medtronic’s response, Radcliffe urged patients to be vocal and vigilant about the issue. “If you are a customer, demand that [Medtronic] take this situation seriously and be truthful,” he told Reuters.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.
Hospital Pharmacies Need Better Automated Tools
Pharmacy automation and technology are critical to managing pharmacy inventory and can improve health systems’ bottom lines, but hospitals need more refined tools, according to an industry report released August 10, 2011, by the health research firm KLAS.
The analysis found that most providers use central pharmacy automation software as their primary inventory management tool, and many have realized dramatic savings by switching from manual to automated tracking. “Providers doing enterprise inventory management report it strengthens their buying power and allows them to further consolidate inventory and reduce waste,” the authors wrote.
Despite the noted benefits, however, providers consistently expressed dissatisfaction with the available systems’ performance in 3 key areas: formulary database integration, tracking medication expiration dates and lot numbers, and reporting. Technology vendors should focus on designing integrated solutions to meet these needs, according to the report.
“Automation may not be able to solve all problems,” said lead author Coray Tate, “but it can be a big part of the solution and needs to do better.”
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