Published
ARPA-H is betting on a future where a child’s chance of surviving cancer—or any rare, complex disease—doesn’t depend on the hospital they happen to live near, or how many binders of medical records their parents can carry. Making this future a reality demands grit, passion, creativity, and cutting-edge science.
Moreover, it demands thoughtful engagement with families, clinicians, and advocates, so they can help shape practical answers from day one. That's why ARPA-H's Pediatric Care eXpansion (PCX) is partnering directly with them to transform care for America’s kids.
Energizing community leaders across advocacy, industry, and research
PCX builds on the Biomedical Data Fabric (BDF) Toolbox program. In this program, ARPA-H-funded research teams demonstrated that an interoperable pediatric cancer data exchange across two large hospitals could successfully harmonize and map more than 90% of unstructured clinical data, electronic health records, and genetic data. In January, we announced a $50 million commitment to double down on that achievement and bring PCX to more than 200 pediatric care centers nationwide.
From a packed room in Arlington, VA on Feb. 18, ARPA-H kicked off the first stop of its PCX roadshow, convening stakeholders from across the country. Cancer researchers, clinicians, technologists, data specialists, caregivers, and patient advocates shared candid insights about what it’s like to navigate care for rare and complex pediatric conditions.
“Rare disease in general is underserved, and it has the highest unmet need... Having been through the lived experience of navigating a pediatric cancer diagnosis, this [PCX] is a system I wish we had when we were in the fight.”
— Misha Mehta, Patient Advocate, Senior Director of Research, Pediatric Brain Tumor Foundation
Too often, the burden of compiling a child’s health information falls to caregivers in crisis. Families travel long distances to find experts with the right knowledge and kids lose critical time waiting for accurate diagnoses.
“We have [patient] data sitting in desperate silos across the country. To be able to go get it and actually assemble that data to then inform models and drive clinical insights and actually affect the care of a life, that connectivity and communication will finally allow for the assembly of all this data. And rather than a billion points of data sitting on somebody’s desk to try and cipher through, they’ll be able to move from a billion points of data to a point of view.”
— Travis White, Senior Vice President, Kno2
Technical demonstrations of PCX tools showed how secure, interoperable data sharing and AI-powered tools could help alleviate patient data pain points, and PCX leaders welcomed community input in Q&A sessions.
Building on community momentum to meet patient needs
The following week, on Feb. 24, PCX Program Manager Dr. Erika Kim spoke at the Alliance for Childhood Cancer Action Days, where more than 300 advocates from across the country gathered to champion policies and programs that help kids beat cancer and look forward to healthy futures.
Dr. Kim outlined how PCX aims to connect key health data across hospitals, specialists, and services so that children can have the best possible chance at better outcomes—no matter their zip code. The vision resonated strongly with the audience, who welcomed PCX’s commitment to community-informed design.
On the eve of Rare Disease Day, ARPA-H hosted a PCX Community Conversation with leaders from professional associations, patient advocacy organizations, and hospital systems. Dr. Kim and other ARPA-H staff led a discussion focused on how PCX could best serve patients, caregivers, and clinicians in real-world settings.
Participants brainstormed key issues that PCX must address to ensure tools are trusted, useful, and optimized for systems with a wide range of resources and families with a wide range of experiences. The insights shared are directly shaping PCX program design, from how funded projects are selected to how future solutions will be tested and deployed in real-world settings.
These touchpoints ensure that the tools PCX develops are deeply informed by families’ lived experiences in the fight against serious childhood illness. At each event, one message came through again and again: community engagement is not a box to check—it is core to how ARPA-H operates.
By involving families, clinicians, advocates, and health system leaders early and often, PCX is building solutions that are trusted by patients and caregivers, practical and sustainable for providers and health systems, and ready to scale across the country.
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