Defining a core digital measures set for pediatric rare disease to accelerate research and transform care

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. Innovative approaches to accelerate access to lifesaving therapies are urgently needed to improve the care of the 200 million children worldwide affected by rare disease.

This project will develop a core digital measures set for pediatric rare diseases, to fill a crucial gap in pediatric research and care. Through this project, we aim to create mechanisms that substantially reduce the cost, time, and clinical trial burden associated with medical product development for children with rare diseases.

Join DATAcc by DiMe in this important endeavor, alongside pharmaceutical companies, the health tech industry, health systems, charitable foundations, payors, and pediatric rare disease advocacy groups, by expressing your interest here. Together, we can make a significant impact on the future of pediatric care.