The collaborative project between the organizations will kick off at a launch meeting in September and will aim to reduce barriers for the development of new treatments and cures for rare diseases

The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD to formally launch development of a new rare disease data and analytics platform. Funded by a cooperative agreement through the Food and Drug Administration, [Critical Path Public-Private Partnerships Grant Number U18 FD005320  from the US Food and Drug Administration] the goal of the platform is to accelerate the movement of therapies from bench to bedside for rare diseases. The platform will provide the infrastructure for a sustainable, cooperative scientific approach to clinical trial readiness in rare diseases by addressing vast 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 (RDCA-DAP) will provide a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development. The robust integrated platform will include integrated rare disease data from various sources such as clinical trials, observational studies, real world data and patient registries — including those within NORD’s IAMRARE registry platform — and an analytics platform that will allow efficient and effective interrogation of that data to generate solutions to inform clinical trial design and regulatory review.

“For people living with rare diseases, time is of the essence,” said Joseph Scheeren, PharmD, C-Path President and Chief Executive Officer. “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.”

“Currently, more than 25 million people in the United States are affected by one or more of the over 7,000 rare diseases,” stated Peter L. Saltonstall, NORD President and Chief Executive Officer. “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. FDA-approved treatments exist for only 10% of rare diseases; with this collaboration we can change that statistic for the better for our rare community.”

Meeting attendees will include representatives from across the community including regulators, patient organizations, clinicians, researchers and pharmaceutical companies interested in rare disease drug development. Attendees will have the opportunity to learn about the goals of the RDCA-DAP and how to engage in the early stages of this effort.

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