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Targeted Therapies On FDA’s Orphan Designation Radar

 
• By Derrick Gingery

As personalized medicine continues to grow, FDA is watching to see if there is a rise in orphan designations for products targeting a “smaller slice” of a common disease.

FDA is on the lookout for the effects of personalized medicine on the orphan drug program and the potential for all diseases to be defined in such a way that they become eligible for orphan status.

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More from United States

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• By Cathy Kelly

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The hub’s goals of efficiency, centralization and coordination fit with the Trump Administration’s current focus for the FDA, Amy Comstock Rick, director of strategic coalitions, told the Biopharma Congress.

Pharma Still Nervous While Riding Trump’s Tariff Rollercoaster

 
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US FDA Finds ‘Significant’ Data Integrity Breaches In CRO Raptim Studies

 
• By Urtė Fultinavičiūtė

The US FDA has deemed certain in vitro bioequivalence studies conducted by CRO Raptim Research “not acceptable” and raised concerns over its in vivo study methods.

More from North America

US Pharma Tariff Reprieve May Be Ending

 
• By Jessica Merrill

Sector-specific tariffs, including on pharmaceuticals, could be announced as early as this week.

Missing BsUFA Revenue Trigger Could Open US FDA Staff To Criminal Penalty

 
• By Derrick Gingery

Massive layoffs and the biosimilar user fee program’s historic flirtation with the non-user fee spending requirements raise concerns that the FDA could lose its ability to collect BsUFA revenue, which could mean a fine or jail time for agency staff.

Trump’s US FDA Takeover Similar To Flat Earth Proponents Running NASA, Woodcock Says

 
• By Sue Sutter

The former principal deputy commissioner said the “decapitation” of senior leadership will make resolving internal disputes more difficult, which will slow application reviews. Woodcock also called the FDA layoffs a slow-moving catastrophe.