Rare Daily Staff

The Critical Path Institute and the National Organization for Rare Disorders said they are launching the Rare Disease Cures Accelerator-Data and Analytics Platform, an effort to create a centralized and standardized infrastructure to support and accelerate the characterization of rare diseases with the goal of accelerating therapy development.

“For people living with rare diseases, time is of the essence,” said Joseph Scheeren, president and CEO of the Critical Path Institute, a nonprofit public and private partnership created to spur medical innovation. “By leveraging the rare disease community access and data of NORD and the data curation, aggregation, governance and advanced analytics expertise of C-Path, we are poised to make a significant impact on rare disease drug development by providing quality data that will inform clinical trial design and accelerate the development of therapies.”

The platform, which will be formally launched at a public meeting September 17 in Maryland, will provide the infrastructure for a sustainable, cooperative scientific approach to clinical trial readiness in rare diseases by addressing knowledge gaps about the natural course of disease, the clinical evaluation of new treatments, and patients’ perspective on disease and treatment.

It will include data from various sources including clinical trials, observational studies, real world data and patient registries, as well as an analytics platform that will allow interrogation of that data to generate solutions to inform clinical trial design and regulatory review.

The U.S. Food and Drug Administration is providing funding for the data and analytics platform through a cooperative agreement.

 “Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding of how rare diseases progress or how to measure clinical improvements,” said Peter Saltonstall, president and CEO of NORD. FDA-approved treatments exist for only 10 percent of rare diseases; with this collaboration we can change that statistic for the better for our rare community.”

Photo: Joseph Scheeren, president and CEO of the Critical Path Institute

X