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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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In brief SOTU remarks, Trump asks Congress to "codify" MFN

In a speech at least nominally focused on the economy and affordability, President Trump put surprisingly little emphasis on his drug pricing efforts.

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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.

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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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