Skip to main content

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

Launched at 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 querying data, through which researchers are expected to advance understanding of disease progression in rare diseases, develop mathematical models of disease, and improve design. clinical trials.

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 aims to find solutions to bottlenecks in the drug development.

“There’s a lot of excitement to see this platform become all it can be for patients and drug developers,” said Jeff Barrett, PhD, senior vice president of C-Path and head of RDCA-DAP, in a press release.

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

“We expect RDCA-DAP to be a place to promote frank dialogue, optimize patient selection and trial design, and co-develop the tools to further reduce decision-making risks for drug development. against rare diseases,” he added.

Creating and running effective clinical trials to test potential therapies for rare diseases can be challenging due to small patient populations and a lack of understanding of the causes, symptoms, and progression of each condition.

Drawing on the expertise of databases on a number of rare diseases, the RDCA-DAP will integrate disease data from several organizations and companies around the world and make it 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 more than 7,000 estimated rare diseases, more than 90% have no FDA-approved treatment,” said Ed Neilan, MD, PhD, medical and scientific director of NORD.

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

The platform was publicly presented and demonstrated for the first time during the one-day RDCA-DAP 2021 virtual workshop. Over 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 organize data to find solutions to bottlenecks and demonstrated the importance of the platform for academic, clinical, regulatory and patient groups. Five patient profiles were presented which highlighted the importance of data sharing for rare diseases.

“Identifying the burdens of disease and the most important current therapies, what can be measured, and what would constitute a meaningful change, can provide insight into the clinical context for regulatory review and potentially more direct evidence. benefits and risks of drugs when collected using valid data and reliable metrics and tools,” said Theresa Mullin, PhD, associate director of strategic initiatives at the Center for Drug Evaluation and Research at the FDA.

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 c-path.org/programs/rdca-dap and rarediseases.org/rdca-dap.