Collaboration Between C-Path and NORD will Help Standardize Data and Accelerate Research for Rare Diseases

Critical Path Institute

The Critical Path Institute, or C-Path is a nonprofit organization which was established in 2005. It has a US branch, headquartered in Tucson, Arizona as well as an EU branch headquartered in Dublin, Ireland. The mission of C-Path is to help accelerate new innovative approaches to medicine. To accomplish this goal, they have established global consortia, which include patient organizations, regulatory agencies, scientists, academia, disease foundations, as well as pharmaceutical companies. In total there are over 1,600 scientists involved in the organization.

C-Path has recently collaborated with the National Organization for Rare Disorders (NORD) to accelerate research for rare diseases.


NORD started as just a small group of patient advocates. The initial goal was to pass the Orphan Drug Act (passed in 1983). Now, it is the leading advocacy organization for rare diseases, supporting the identification of rare diseases and research for cures. NORD has 275 member organizations which are disease specific. Since the passage of the Orphan Drug Act their goals have become more broad. They aim to voice the needs of the rare community, accelerate research, initiate supportive policies, provide resources for rare families, and educate the public.

The Rare Disease Cures Accelerator-Data and Analytics Platform

The collaboration of these two organizations could significantly improve research for rare diseases. Considering the fact that there are over 25 million rare disease patients in the US alone and 90% of rare conditions still do not have a single FDA approved therapy, there is clearly an unmet need.

Thanks to funding from the FDA, NORD and C-Path will work together to establish The Rare Diseases Cures Accelerator-Data and Analytics Platform, or RDCA-DAP. This platform will officially be launched on September 17th, 2019 in Bethesda, Maryland. There, patient organizations, regulators, researchers, clinicians, and pharmaceutical companies will meet together to learn about the platform, how to engage with it, and how it could aid in the facilitation of their goals.

Ultimately, the goal of this platform is to-

“Accelerate the movement of therapies from bench to bedside.”

The data included in this platform will range from observational studies, clinical trials, and patient registries (including the IAMRARETM registry platform created by NORD). This data will then be utilized to improve rare disease research and get new therapies to patients faster. The platform will essentially serve as a standardized and centralized program that will encourage collaboration and minimize knowledge gaps. Importantly, the data in this platform will also include the patient perspective.

There are high hopes for the RDCA-DAP because it will be able to leverage the data that NORD has collected over its 35 years of existence, as well as the capabilities of C-Path to curate, aggregate, and govern advanced data analytics. This platform could help to accelerate drug development by making quality data accessible to researchers.

You can read more about this new platform here.

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