New RDCA-DAP program launches to accelerate rare disease treatment innovation

The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®) officially launched its next phase on Tuesday establishing itself as the leading platform to accelerate rare disease treatment innovation. The launch was part of an all-day virtual workshop and annual meeting of rare disease stakeholders, featuring a platform demonstration, industry expert presentations and attendance by over 400 patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists.

RDCA-DAP is an FDA-funded initiative that will serve as a centralized and standardized base to host and share de-identified rare diseases data and to support treatment innovation. It was created through a partnership between Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD®) and the U.S. Food and Drug Administration (FDA) and has grown to include many others throughout the rare disease community.

There is a lot of enthusiasm for seeing this platform become all it can be for patients and drug developers. It is not just about the data. This is a place to generate solutions in a highly collaborative way. 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."

Jeff Barrett, Ph.D., F.C.P., C-Path Senior Vice President and RDCA-DAP Lead

After a public premiere showcasing the functionality of the platform, presentations throughout the day emphasized how RDCA-DAP will curate data to break down silos, provided insight into the platform's development and testing and showcased the importance of RDCA-DAP from the perspective of critical stakeholders, including academic, clinical, regulatory and patient communities.

On the importance of incorporating patients' perspectives through RDCA-DAP, Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, Center for Drug Evaluation and Research (CDER), FDA, said, "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."

A total of 31 speakers and panelists from patient organizations and regulatory, industry and academic fields participated in the workshop. Five patient profiles underscoring the importance of data sharing were also highlighted. Those interested in viewing the workshop and accessing supplemental information can do so here.

"Patient owned data collected by patient groups are less likely to get stuck in a silo," said Ed Neilan, M.D., Ph.D., Chief Medical and Scientific Officer, NORD. "Of the over 7,000 estimated rare diseases, more 90% have no FDA approved treatment. And 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."

To learn more and request access to RDCA-DAP, apply directly on the platform at https://portal.rdca.c-path.org. To submit critical rare disease data, contact the project team at [email protected] or visit c-path.org/programs/rdca-dap and rarediseases.org/rdca-dap.

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