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FDA Funds Health IT Infrastructure for Rare Disease Research

August 08, 2019 / Fred Donovan

The FDA is providing funding to the Critical Path Institute (C-Path) and the Organization for Rare Disorders (NORD) for a centralized and standardized health IT infrastructure to support rare disease research with the goal of accelerating therapy development. The IT infrastructure will include a platform for a 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. The Rare Disease Cures Accelerator-Data and Analytics Platform includes integrated rare disease data from various sources such as clinical trials, observational studies, real world data, and patient registries and an analytics platform that will enable interrogation of that data to generate solutions for clinical trial design and regulatory review.