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Totality Of ‘Breakthrough’ Evidence Guides FDA Designation Decisions

 
• By Sue Sutter

Sponsors should think about more than just a single efficacy data point when assembling their designation requests because the agency looks at the ‘entire puzzle’ in deciding whether ‘breakthrough’ status is warranted.

FDA’s Center for Drug Evaluation and Research decides whether to grant or deny “breakthrough therapy” designation requests based on the totality of evidence available for a product, not any one efficacy data point, agency officials said April 24.

At a meeting convened by the Brookings Institution’s Center for Health Policy, FDA officials said looking at all of the available evidence gives them more confidence that early clinical data...

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US FDA’s ‘Good Reputation’ For Science May Mean Loper Bright Not ‘Cataclysmic,’ Attorney Says

 
• By Malcolm Spicer

Bridget Dooling, a law school professor who reviewed draft regulations from the FDA and other agencies as an OMB attorney, said prior federal court decisions suggest judges typically defer to agency decisions based in science.

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What Information Does US FDA Need For Stealth’s Elamipretide After CRL?

 
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UK And US Regulators In Sync On RWD External Control Arms

 
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More from North America

What Information Does US FDA Need For Stealth’s Elamipretide After CRL?

 
• By Sue Sutter

The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.

UK And US Regulators In Sync On RWD External Control Arms

 
• By Vibha Sharma

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Rocky Rollout Spells Trouble for Medicare Prescription Payment Plan

 
• By Lauren Flynn Kelly

Enrollment in the new Medicare Prescription Payment Plan was lower than expected in the first year and well below CMS expectations, and invoicing data indicates members are slow to pay.