The Problem:

Over 350 million people in the world have been diagnosed with a rare disease. In the United States, a rare disease is defined as affecting fewer than 200,000 people, and there are over 10,000 classified rare diseases. Only about 600, or 10%, of rare diseases have an FDA-approved treatment available, and drug development is frequently slowed by the low numbers of patients and limited understanding of the variability and progression of each disease.

The Solution:

Critical Path Institute’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is an FDA-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating the development of treatments and cures for rare diseases. RDCA-DAP creates the collaborative, non-competitive space to share existing patient-level data and encourages the standardization of new data collection. RDCA-DAP accelerates the understanding of disease progression (including sources of variability to optimize the characterization of subpopulations), clinical outcome measures and biomarkers, and facilitates the development of mathematical models of disease and innovative clinical trial designs. RDCA-DAP is positioned to generate solutions to drug development bottlenecks through program-led solutions and facilitation of C-Path Rare and Orphan diseases consortia activities.

The Impact:

The RDCA-DAP platform continues to expand since going live September 2021, and now contains data for 30 different rare diseases, including Polycystic Kidney Disease, Duchenne Muscular Dystrophy, mitochondrial diseases, neurodevelopmental disorders, rare epilepsies and rare neurodegenerative disorders, including Friedreich ataxia. More data will be added and made accessible as outreach efforts continue.

Since the platform’s launch, we’ve seen engagement from 325 approved platform requests and 35 approved workspaces for external users and research.

A New Data and Analytics Platform

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is an FDA-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating therapy development across rare diseases. This platform is made possible through a collaborative grant from the FDA [Critical Path Public-Private Partnerships Grant Number U18 FD005320 from the U.S. Food and Drug Administration].

RDCA-DAP promotes the sharing of existing patient-level data and encourages the standardization of new data collection. By integrating such data in a regulatory-grade format suitable for analytics, RDCA-DAP accelerates the understanding of disease progression (including sources of variability to optimize the characterization of subpopulations), clinical outcome measures and biomarkers, and facilitates the development of mathematical models of disease and innovative clinical trial designs. RDCA-DAP is positioned to generate solutions to drug development bottlenecks. As such, the utility of the patient-level data is maximized and data may be used to develop tools that will be accessible to the community in order to optimize and accelerate drug development across rare diseases.

How RDCA-DAP Works

RDCA-DAP houses integrated patient-level data from diverse sources, including clinical trials, longitudinal observational studies, patient registries and real-world data (e.g. electronic health records) across a multitude of rare diseases. Data are contributed from different organizations and companies around the world. C-Path has extensive experience in building such integrated databases for many diseases, including existing rare disease databases (in Duchenne muscular dystrophy, Huntington’s disease, Friedreich’s ataxia and polycystic kidney disease). C-Path has partnered with the National Organization for Rare Disorders (NORD) to leverage its IAMRARE® registry platform and extensive expertise to help identify data contributors and establish contacts with the contributing organizations. C-Path will negotiate data contribution and use agreements to allow patient-level data to be transferred to the RDCA-DAP, standardize and integrate the data with other contributed data, and make it available to the degree agreed to by the data contributors.

For questions or additional information about participating in RDCA-DAP, please email rdcadap@c-path.org.

If you’re a patient or a patient organization looking to start a registry, visit https://rarediseases.org/rdca-dap/.

Learn more about RDCA-DAP and stay connected by subscribing to our updates here.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 54% funded by the FDA/HHS, totaling $19,436,549, and 46% funded by non-government source(s), totaling $16,373,368. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.