Rare Disease Drug Development
Over 350 million people in the world have one of more than 7,000 rare diseases. In the United States a rare disease is defined as one affecting fewer than 200,000 people. The Orphan Drug Act in the U.S., and similar laws in other countries, have created incentives for companies to develop treatments for these “orphan diseases,” because no drug companies were interested in developing treatments for such small populations of patients. Today, many new drug targets have been identified and new products are in development.
Nonetheless, 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. This means that the design of clinical trials that can reliably evaluate the efficacy and safety of a potential therapy is challenging. Developing a clear understanding of how each disease progresses, as measured by defined outcome measures and/or biomarkers, would allow development of clinical trial protocols that could efficiently determine if a new therapeutic was effective or not. This would accelerate clinical development, make it less expensive, and encourage new companies to develop rare disease drugs.
A New Data and Analytics Platform
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is an integrated database and analytics hub that is designed to be used in building novel tools to accelerate drug development across rare diseases. It is being developed by C-Path and the National Organization for Rare Disorders® (NORD) 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 will promote the sharing of existing patient-level data and encourage the standardization of new data collection. By integrating such data in a regulatory-grade format suitable for analytics, RDCA-DAP will accelerate the understanding of disease progression (including sources of variability to optimize the characterization of subpopulations), clinical outcome measures and biomarkers, and facilitate the development of mathematical models of disease and innovative clinical trial designs. RDCA-DAP will be positioned to generate solutions to drug development bottlenecks. As such, the utility of the patient-level data will be maximized and 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
The RDCA-DAP will house 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 will be 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 will partner with NORD to leverage their IAMRARE™ registry platform and extensive expertise to identify the data contributors, establish contacts with the contributing organizations and help to negotiate data contribution and use agreements to allow patient-level data to be transferred to the RDCA-DAP. C-Path will 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.
