Wendy Erler has worked in rare disease patient communities her entire career. She says one theme that has been consistent in every community is the question of patient data, who owns it, and how it can be used to advance science more broadly than a single research program or physician.
“When RARE-X created this moonshot mission to build federated data sets, I was all in,” said Erler, vice president and head of patient insights and advocacy for the rare disease drug developer Alexion Pharmaceuticals and a RARE-X board member. “I understood the value. I understood the need.”
RARE-X today announced that Alexion Pharmaceuticals is providing critical, early-stage funding to support the nonprofit’s 2021 priorities and pilot programs intended to demonstrate how RARE-X’s approach to patient-owned data collection and federated data sharing for biomedical research will accelerate disease understanding and therapy development.
Among the priorities that RARE-X announced is the launch of five structured data domains in 2021, beginning with neurology, and includes plans to launch 20 neurology-focused patient owned data collection portals during the year.
RARE-X will also initiate five collaborative demonstration projects that will leverage the entire technology platform supporting 21st-century data collection and federated data sharing globally.
The organization will also scope its first international patient owned data collection effort, with the release of a landscape report in collaboration with RARE-X founding partner, IndoUSrare.
“At Alexion, we have been on a journey to amplify and accelerate what it means to be patient centric,” Erler said. “As a company in the rare disease and devastating disease space, we develop drugs that are transformative. Our science is incredible, but how do we up our game and really deliver on the mission of patient centricity? RARE-X offered that opportunity. It’s really for the good of all rare disease patients.”
Erler said having open-source data and making data available will be critical components of the effort. Also important is educating patients and communities at large as to why that matters. There’s a lot of careful work that has to be done around issues such as patient privacy and protecting personal health information, but she said that we could really learn a lot by looking at and across rare disease data in the aggregate.
Right now, there are patient registries that house important natural history data and other information, but they’re set up in siloes. They don’t talk to each other. The registries also don’t necessarily collect data in a way that is usable for different purposes, Erler said. She thinks breaking down those walls and silos will solve some big problems specific to the natural history of disease and the true burden of disease work that is important to advancing research and science.
“We’re committed to the mission that is RARE-X, and we showed that commitment very early because we believe in the value of this work and the collaborative spirit with which it’s being built,” she said. “It speaks to a lot of our principles—patient-centric, collaborative, not competitive.”