New data sharing program aims to accelerate innovation in rare diseases


A new US initiative called Rare Disease Cures Accelerator-Data and Analytics Platform – called RDCA-DAP – aims to accelerate rare disease treatment innovation by sharing existing patient data and promoting the standardization of new data collections.

The US Food and Drug Administration (FDA) funded initiative was launched during a virtual workshop in September and includes rare disease data from clinical trials, observational studies, patient registries, and real-world data.

It also provides workspaces with advanced analytics for data retrieval, through which researchers should improve understanding of disease progression in rare diseases, develop mathematical disease models, and improve clinical trial design.

The initiative, which emerged from a partnership between the National Organization for Rare Disorders (NORD), the Critical Path Institute, or C-Path for short, and the FDA aims to find solutions to bottlenecks in drug development.

“There is a lot of enthusiasm that this platform will be all it can be for patients and drug developers,” said Jeff Barrett, PhD, C-Path senior vice president and head of RDCA-DAP in a press release.

“It’s not just about the data. This is a place to generate solutions in a very collaborative way, ”said Barrett.

“We expect that RDCA-DAP will be a place to foster open dialogue, optimize patient selection and study design, and help develop the tools to further reduce risk in decision making in drug development for orphan diseases,” he added.

Designing and conducting effective clinical trials to test potential therapies for rare diseases can be challenging because the patient population is small and the causes, symptoms, and progression of each disease are unknown.

Building on the database expertise on a range of rare diseases, the RDCA-DAP will integrate disease data from multiple organizations and companies around the world and make them more accessible. The hope is that the new platform will lead to successful attempts to cut costs and encourage more companies and researchers to get involved in rare disease research.

“Of the over 7,000 estimated rare diseases, more than 90% do not have an FDA cleared treatment,” said Ed Neilan, MD, PhD, chief medical and scientific officer at NORD.

“And,” said Neilan, “right now the pharmaceutical industry’s attention is on 100 or 200 of these rare diseases. RDCA-DAP can draw attention to rare diseases that could otherwise be relatively neglected. “

The platform was first publicly presented and demonstrated at the all-day virtual workshop RDCA-DAP 2021. More than 400 rare disease stakeholders attended the online session with a total of 31 speakers and panelists from patient groups, industry and science.

Workshop presentations demonstrated how RDCA-DAP curates data to find solutions to bottlenecks and demonstrated the platform’s importance for academic, clinical, regulatory and patient groups. Five patient profiles were presented, highlighting the importance of data sharing for rare diseases.

“Identifying the burdens of disease and current therapies that matter most, what can be measured and what would represent a meaningful change can provide insights into the clinical context for regulatory review and potentially more direct evidence of the benefits and risks of drugs, when they are collected using valid data and reliable measures and tools, ”said Theresa Mullin, PhD, assistant director of strategic initiatives at the FDA’s Center for Drug Evaluation and Research.

Those interested in accessing rare disease data can find data sets of interest and apply for access through the platform. To submit data on rare diseases to the initiative, contact the project team at [email protected] or visit and

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