RARE-X Exchange Patient Innovator Forum to Help Advocates Advance Data to Treatments

August 17, 2022

Rare disease advocates and organizations have learned that the path to a treatment begins with gathering data that’s needed to support the work of researchers and biopharmaceutical companies, but one of the critical aspects of this work is learning to think ahead and be prepared for what’s next in the process.

To help patient organizations advance their efforts to find treatments for their conditions, RARE-X this year is launching the Exchange – A Patient Innovator Forum. This is an opportunity for patient organizations to learn best practices for driving rare disease research and how to attract interest from researchers.

The inaugural RARE-X Exchange Patient Innovator Forum will be held from September 14 to 15 in San Diego. The event dovetails with Global Genes’ Rare Patient Advocacy Summit and will be held at the Town & Country Hotel in San Diego. Though the program was initially designed for RARE-X-affiliated patient organizations already using its collaborative global data sharing platform, RARE-X has opened the program up to others due to interest from organizations attending the Global Genes event. Although the event is free, spaces are limited.

“Organizations collect patient data and then wonder, ‘Now what?’” said Nicole Boice, founder and executive director of RARE-X. “We thought that this is an important opportunity to bring RARE-X partners, partner organizations, and collaborators together to help support patient community’s next steps in their research driving activities. We want those patient community partners we support through RARE-X to better understand the research funnel, where there are opportunities, who to collaborate with, and how to do it. This is no small task, but we want to help make this more manageable for patient advocacy leaders.”

The inaugural RARE-X event is intended to provide patient organizations with insights and introductions as they focus on building on their data collection efforts to advance drug development. The event will feature speakers including Morrie Ruffin, Co-Founder & Senior Advisor for Alliance for Artificial Intelligence in Healthcare (AAIH), moderating a panel on “When Rare Disease Becomes a BIG Data Opportunity,” with panelists Ryan Colburn a Pompe Patient and Innovator; Stacie Calad-Thompson of BioSymetrics; and Robert Deans of Synthego.

“One of the things that is unique to RARE-X is the fact that we’re building out this platform that is creating an opportunity for rare disease to be a Big Data play,” said Boice. “What if we could engage in rare disease challenges with AI companies that can look across disease, look at data and aggregate across phenotypes, and apply their model to a broader data set? We’re bringing to the table individuals who are thinking along those lines and who want to dive into rare diseases.”

Other speakers include RARE-X CEO Charlene Son Rigby, Third Rock Ventures Venture Partner Walt Kowtoniuk, and Harvard Medical School Associate Professor of Pediatrics and Boston Children’s Hospital Researcher Timothy Yu. In addition to several speakers and panels, attendees will have the opportunity to take deep dives in intimate roundtable discussions with innovators.

These 25 roundtable discussions will be limited to eight people at each table, and attendees will have a chance to participate in two back-to-back discussions each. The roundtables will cover broad topics including collaboration, drug repurposing, cell and gene therapy, mentoring, and other topics. Among the innovators leading round discussions will be n-Lorem chief development officer Sarah Glass, Mira’s Miracle Foundation founder and CEO Julia Vitarello, Columbus Children’s Foundation Executive Director Laura Hameed, Vice President of Clinical Development & Neuroscience/Rare Disease Recursion Glenn Morrison, for Healx, Chief Collaboration Officer Bruce Bloom, among many others.

“We want people to come ready to engage and learn. We want attendees to leave activated, with clear next steps on their research and scientific road mapping,” said Boice. “We recognize that our guests could be using this time spent with their family, which is why their time with us needs to be off-the-charts valuable.”

To learn more, view the event agenda, or register, go to the Exchange Forum page on the RARE-X website.

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