New Data-sharing Program Aims to Speed Innovation in Rare Diseases

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by Shayna Korol |

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A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection.

Launched during a virtual workshop in September, the U.S. Food and Drug Administration (FDA)-funded initiative includes rare disease data from clinical trials, observational studies, patient registries, and real-world data.

It also provides workspaces with advanced analytics for data interrogation, through which researchers are expected to advance the understanding of disease progression in rare diseases, develop mathematical models of disease, and improve clinical trial design.

Created through a partnership between the National Organization for Rare Disorders or NORD, the Critical Path Institute, known as C-Path, and the FDA, the initiative is intended to find solutions to bottlenecks in drug development.

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

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

“We expect RDCA-DAP to be a place to promote candid dialogue, optimize patient selection and trial design, and co-develop the tools to further de-risk decision making for rare disease drug development,” he added.

It can be challenging to create and run effective clinical trials to test potential therapies for rare diseases because of small patient populations and a lack of understanding of each condition’s causes, symptoms, and progression.

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

“Of the over 7,000 estimated rare diseases, more 90% have no FDA-approved treatment,” said Ed Neilan, MD, PhD, the chief medical and scientific officer at NORD.

“And,” Neilan said, “currently the attention of the pharmaceutical industry is on 100 or 200 of these rare diseases. RDCA-DAP may bring attention to rare diseases that might otherwise remain relatively neglected.”

The platform was publicly introduced and demonstrated for the first time at the all-day RDCA-DAP 2021 Virtual Workshop. More than 400 rare disease stakeholders attended the online session, with a total of 31 speakers and panelists from patient groups, industry, and academia.

Workshop presentations showed how RDCA-DAP will curate data to find solutions to bottlenecks, and demonstrated the platform’s importance to academic, clinical, regulatory, and patient groups. Five patient profiles were featured that highlighted 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 constitute a meaningful change, can provide insight on the clinical context for regulatory review and potentially more direct evidence of drug benefits and risks when collected using valid and reliable measures and tools,” said Theresa Mullin, PhD, the associate director for strategic initiatives at the FDA’s Center for Drug Evaluation and Research.

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