Press Release

RARE Hope Joins DATAcc by DiMe to Accelerate Development of Digital Measures for Pediatric Rare Disease

8 April 2025

RARE Hope, a nonprofit organization advancing research and innovation for rare pediatric neurological diseases, is proud to join DATAcc by DiMe’s newest project team. This multi-stakeholder collaboration brings together leaders from medicine, technology, pharma, regulatory science, advocacy, and research to develop a core set of digital measures for pediatric rare disease—a critical step in accelerating the delivery of life-saving therapies to children who simply cannot wait.

Children with rare diseases are in a race against time. Ninety-five percent of these conditions have no approved treatment, and the average time to diagnosis is 4.8 years. Tragically, 30% of affected children do not live to see their fifth birthday.

Digital measures developed through this initiative will provide researchers and developers with a robust, scalable platform to generate meaningful, regulator-ready clinical endpoints. These tools have already demonstrated their potential to reduce the time, cost, and risk associated with traditional drug development. They also streamline the creation of pediatric-focused digital health technologies and support decentralized trials—enabling high-quality, high-resolution data collection wherever children receive care.

This standardized framework will further reduce barriers to innovation in pediatric rare disease research, where time is a precious resource.

“By advancing the development and adoption of digital measures, researchers can leverage new technologies and solutions to speed the development of new therapies that will save the lives of children with rare disease," said Sarah Valentine, Associate Program Director, DiMe. "By collaborating with leaders across disciplines, we have the ability to fast track progres. We're proud to work with RARE Hope on our latest project and leverage their experience and expertise in this important work."

Why This Matters

The development of a unified core set of digital measures will:

  • Accelerate therapy development by enabling the use of validated digital endpoints that optimize pipeline decisions and reduce overall development costs
  • Facilitate the creation of pediatric-focused digital health technologies
  • Support decentralized clinical trials through real-time, high-quality data collection—bringing clinical research to children wherever they are

This effort provides a foundation for rapidly creating broadly accepted, high-quality digital endpoints tailored to rare pediatric diseases. These tools have already demonstrated the ability to reduce the risk, cost, and timelines associated with traditional development pathways, while expanding access to innovative therapies.

A Timely Opportunity

The digital health landscape is evolving swiftly. The U.S. Food and Drug Administration (FDA) issued final guidance on digital health technologies in December 2023 and qualified the first digital endpoint in May 2024, signaling strong regulatory support for advancing digital measurement frameworks.

Several recent developments underscore the need—and the opportunity—for a unified digital measure set for pediatric rare disease:

  • The Rare Disease Innovation Hub, a cross-FDA initiative featuring dedicated workstreams for novel endpoints, biomarker development, real-world evidence, and innovative trial design
  • Collaborative data networks, registries, and repositories, which are expanding access to shared data and enabling adaptive trial models
  • The maturation of digital sensors and derived measures, supported by the FDA’s Medical Device Development Tool (MDDT) program, including clinical outcome assessments and biomarker tests

“Establishing a core set of digital measures for children is a vital step toward building a scalable infrastructure for rare disease research,” said Nina Frost, President of RARE Hope. “This initiative is about creating the right tools—and using them to deliver therapies faster and more effectively.”

About RARE Hope

RARE Hope is a nonprofit organization dedicated to supporting research, innovation, and advocacy for children with rare neurological diseases.