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FDA moves to tackle orphan designation backlog

Some 200 requests are awaiting review by the US regulator


The US Food and Drug Administration (FDA) has unveiled its plans to eliminate a backlog of orphan drug designation requests that currently numbers around the 200 mark.

The US regulator's Orphan Drug Modernisation Plan aims to eliminate the waiting list, and is the first development under the FDA's new Medical Innovation Development Plan.

The move comes just one week after FDA commissioner Scott Gottlieb vowed to eradicate the backlog within 90 days (giving his agency a mid-September target), and said he wanted all new requests for designation to have a response within 90 days of receipt.

To ensure this, the agency’s latest plan will take a multifaceted approach and these efforts will include a re-organisation of review staff, better leveraging the experience across medical product centres and establishing a new FDA Orphan Products Council, which will address scientific and regulatory issues.

“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” said Gottlieb.

“Congress gave us tools to incentivise the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”

There are currently about 200 orphan drug designation requests that are pending review and as result, the agency plans to deploy a backlog team compromised of senior reviewers with experience in orphan drug designation to solely focus on the backlogged applications, starting with the oldest requests.

A designation review template will also be implemented to increase consistency and efficiency on the agency’s reviews and will look to collaborate within the FDA’s medical product centres.

Article by
Gemma Jones

3rd July 2017

From: Regulatory



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