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Accelerating Hope for Rare Disease Patients

U.S. launches platform to get medicine to patients sooner.

CSL Behring employee in the lab at Kankakee site

With a goal of accelerating potential treatments for people living with a rare disease, the U.S. government is backing the creation of a data and analytics platform to address knowledge gaps about rare conditions.

The Rare Disease Cures Accelerator-Data and Analytics Platform is a joint effort by the National Organization for Rare Disorders (NORD) and the Arizona-based Critical Path Institute that is being funded through a grant from the U.S. Food and Drug Administration. In announcing the effort, the organizations say they hope the platform “will accelerate the movement of therapies from bench to bedside for rare diseases.”

“Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding about how rare diseases progress or how to measure clinical improvements,” said Peter Saltonstall, President and Chief Executive Officer of NORD.

Organizers say the platform will integrate rare disease data from numerous sources, including clinical trials, observational studies, real world data and patient registries. The analytics component will allow developers of potential therapies to comb through the data to use in designing clinical trials and performing regulatory reviews.

Development of the Rare Disease Cures Accelerator-Data and Analytics Platform will formally launch later this month when organizers meet in Bethesda, Maryland on Sept. 17. 

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