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As the Covid-19 pandemic forces researchers to take a second look at how clinical trials are structured, some patient advocacy groups are creating their own programs around drug discovery. The KCNT1 Foundation, a nonprofit supporting patients with a severe form of childhood epilepsy, is working with health data platform LunaPBC and the Genetic Alliance to create a platform where families can share health data and help researchers design studies in a way that would make it easier for patients to participate.
KCNT1-related epilepsy is rare disease that causes severe seizures in young patients. The condition is associated with mutations in the KCNT1 gene, but relatively little is known about it. Through the partnership, Massachusetts-based biotechnology company Biogen will work with patients to study the cause of the disease and develop treatments.
The Genetic Alliance will help patients’ families develop an advocacy group and give them tools to design a registry and network with researchers. The Washington D.C.-based nonprofit struck a partnership with LunaPBC last year, developing a way for patients to specify how researchers may use the health data that they share.
“Parents are full collaborators in design and implementation in these studies,” Genetic Alliance CEO Sharon Terry said in a phone interview. “(The KCNT1 Foundation) has had an advocacy group for some time. What we’re doing is giving them the heavy lift tools — biobanking, registries and an institutional review board — so they don’t have to reinvent the wheel.”
LunaPBC, which was founded by a group of former Illumina executives, created a platform called LunaDNA where patients can contribute health data in exchange for company shares. Researchers can approach LunaPBC with questions or analytical models, but the data is never propagated outside of the system.
The KCNT1 Foundation will ask members to enroll in LunaDNA to participate in Biogen’s research. If they choose to enroll, they are then asked to take an initial survey and upload any medical data they are comfortable sharing, including health records and DNA test results.
Through these advocacy groups, patients’ families can share input on clinical trial design that will hopefully allow more patients to participate and ensure treatments meet their needs.
“We’ve always wanted to do trials in a more participant-centered way,” LunaPBC President Dawn Barry said in a phone interview. “It’s never been clearer that bringing trials to the home is much safer for people. Let’s talk in the case of pediatric epilepsy. They have many seizures per day. … Can you imagine as a parent putting these kids on long-distance rides or airplanes for a study? You don’t need Covid to see that’s a bad idea.”
Both Barry and Terry said they were hopeful that the pandemic would lead to a second look at how trials are designed.
“There’s a huge interest now with what are we going to do with all the clinical trials that have been stalled or supposed to start. Trying to talk companies and academic groups into studies at home has been difficult,” Terry said. “I don’t want (to say) anything good about this virus, but this is a huge benefit in that people are saying we do need to rethink this.”