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.

DATAcc by DiMe’s newest project has brought together leaders from industry, advocacy, research, and regulatory sectors to define a core set of digital measures for pediatric rare diseases. This collaborative effort will equip the field with tools to accelerate the development of life-saving therapies, improve prioritization in drug pipelines, reduce development costs, and streamline digital health solutions designed specifically for children.

DATAcc’s core set of digital measures for pediatric rare disease will help you to:

Use digital endpoints proven to speed the development of life-saving therapies, optimize and accelerate pipeline prioritization, and reduce cost

Streamline the development of digital health technologies designed for pediatric patients

Decentralize trials with high-quality data collection to ensure lifesaving therapies are delivered to children where and when they need it

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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.