DATACC BY DIME PROJECT

Accelerating lifesaving therapies for pediatric rare diseases

Only 5% of rare diseases have an approved treatment, and small and geographically dispersed patient populations make traditional clinical trials impractical. The multifactorial nature of rare disease phenotypes means that single-domain endpoints often fail to capture the full impact of the disease and are therefore not fit for purpose for regulatory decision-making.

The result is a pipeline of promising therapies that stalls before it reaches patients.

Building on DiMe’s core digital measures for pediatric rare disease research and care, this project takes the next step. Now that we know what to measure, we will define how to combine those measures into composite and multimodal digital endpoints that are statistically robust, translatable across populations, and compatible with regulatory pathways.

Become a founding partner

If done right, composite and multimodal endpoints are not just trial-design improvements. They are the lever that makes pediatric rare disease research economically viable for the long term. As a founding partner, you will:

De-risk your pipeline by adopting endpoint strategies with a clear evidence base and a regulatory path.

Co-create the composite and multimodal endpoint framework and an integrated evidence library to define best practices across the rare disease community.

Earn early mover advantage by defining a set of composite and multimodal endpoints that will have an outsized impact on the field.

Participate in a formal FDA meeting to align on how real-world and patient-generated data can inform pivotal trial design.

Accelerate time-to-market with endpoints that translate across a broader range of disease phenotypes, enabling faster decisions and fewer detours.

DiMe invites life sciences, digital health technology developers, patient advocacy organizations, and regulators to join as founding partners.

Together, we will build the shared resources needed to accelerate pediatric rare disease trials and make them economically viable.