CDER Experts to Help Rare Disease Drugs Move Through ARC Approval Process

Specialized scientific liaisons will be key to moving rare disease drugs through the Accelerating Rare Disease Cures (ARC) Program, according to CDER Director Patrizia Cavazzoni.

The liaisons — which may be either individuals or teams — will be tasked with helping sponsors fortify the necessary deep foundation of translational research for these candidate drugs, Cavazzoni said during a media discussion of ARC’s first year. CDER launched the ARC program last spring.

While remaining nonspecific about the plan, Cavazzoni said CDER wants expert advisors who can work with both the sponsor and the product review team to help the sponsors be more prepared.

“We want to have some subject matter experts who talk to the teams and remind them of the things that need to be brought to the table,” she said, “to be able to have fully informed decisions about novel endpoints.”

Thus far, these liaisons have been drawn from in-house staff, but as ARC revs up, new experts may be onboarded.

“Additional staff could be focused on these activities,” she said, “identifying one or two programs that could really benefit from this intervention.”

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