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Accelerating pediatric rare disease research with a core set of digital measures

Children with a rare disease, their families, and care providers are in a race against time. 95% of rare diseases lack approved treatments, while the average time to diagnosis is 4.8 years, resulting in as many as 30% of children with a rare disease dying before their 5th birthday.

Imagine a world where pediatric rare disease research is faster, more efficient, and brings lifesaving therapies to children in time. Now imagine you are part of the initiative making this a reality.

This collaborative, multi-stakeholder effort will:

  • Drive impactful innovation by defining high-quality digital measures to accelerate pediatric rare disease research and care
  • Convene industry leaders across medical, pharma, tech, regulatory, and patient advocacy groups to build and implement a roadmap for the pediatric community
  • Build a global resource for researchers and developers to use to speed the delivery of new drug therapies and mitigate risk, improving the lives of the 200 million children with rare disease

We are no longer recruiting project team members, but DiMe has many important projects on the horizon and we need your help! Learn more and join a project here. 

No family or caregiver with a child facing a rare disease should be left waiting years for a diagnosis and even longer for treatment—join us!

The digital health field is evolving rapidly, and so should the digital tools that are available to treat children. With the US Food and Drug Administration (FDA) issuing final guidance on digital measures in December 2023 and qualifying the first digital endpoint in May 2024, now is the time to build a unified core digital measures set for pediatric rare disease. Together, we will build resources that reduce the time, cost and burden of clinical trials to ensure lifesaving therapies reach the 200 million children suffering from a rare disease.