Rare-disease initiative collaborators work to advance research innovation

By Jenni Spinner contact

- Last updated on GMT

(elenabs/iStock via Getty Images Plus)
(elenabs/iStock via Getty Images Plus)

Related tags: Rare disease, orphan disease, Research, CDER, Fda

Stakeholders in the Rare Disease Cures Accelerator-Data and Analytics Platform initiative are partnering in hopes of bringing treatments to patients faster.

The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP) gathered rare disease stakeholders this week at an all-day workshop and meeting. The event included a demonstration of its platform, presentations by industry experts, and attendance by more than 400 researchers, patients, care providers, clinicians, pharma industry representatives, and regulatory reviewers.

Funded by the US Food and Drug Administration (FDA), the RDCA-DAP initiative is designed to act as a centralized, standardized base for stakeholders to share de-identified data on rare diseases and to support treatment innovation. It originated via a partnership among the Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD), and the FDA.

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

After a demonstration outlining the functionality of the RDCA-DAP platform, various stakeholders offered presentations on the organization’s work. These include efforts to curate data to break down silos, insight into the platform's development and testing, and perspective on why the RDCA-DAP initiative is important.

Theresa Mullin, associate director for strategic initiatives with the Center for Drug Evaluation and Research (CDER) of the 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​."

"Patient-owned data collected by patient groups are less likely to get stuck in a silo​," said Ed Neilan, NORD’s chief medical and scientific officer. “Of the over 7,000 estimated rare diseases, more than 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​."

Visit here for more information or to request RDCA-DAP access​. 

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