regulatory

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US lawmakers take FDA to task over rare diseases record

Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.

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FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.

R&D

Navigating development in a complex molecule world

For decades, the pharmaceutical industry has relied on accelerated development and manufacturing timelines to bring new therapies to patients quickly.

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FDA starts review of Regeneron's drug for rare disease FOP

Can Regeneron's drug for ultra-rare bone disease FOP pick up the baton from Ipsen's Sohonos, whose growth has stalled?

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Scrabble lettering of Two and One on grass

Makary says FDA will "end two-trial dogma" for approvals

The FDA plans to adopt a new policy that will do away with the default requirement to base regulatory filings for new medicines on two pivotal trials.

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