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FDA Expedited Programs Guidance: “Available Therapies” Depends On U.S. Standard Of Care

 
• By Sue Sutter

Draft guidance explains the four expedited drug development and approval pathways and offers the agency’s first formal advice on the type and amount of evidence needed for designation as a “breakthrough therapy” under FDASIA.

To ensure that “breakthrough therapies” provide a real advance over existing therapy, FDA is clarifying that to qualify for the designation established under FDASIA, a candidate generally should be compared to a current standard of care that is FDA-approved for the same indication.

The agency’s first official instructions on the breakthrough therapy designation created under the FDA Safety and Innovation Act come as...

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More from Approval Standards

Slowdown: US FDA’s 2025 Novel Approvals Are On Below-Average Pace

 
• By Bridget Silverman

US FDA's 25 novel approvals in 2025 fell short of the agency's five-year average count, driven by a drop in drugs center approvals; 45 user fee goals remain in second half of year.

US FDA Asking Questions About Commissioner’s Voucher Program With Details Scarce

 
• By Derrick Gingery

FDA staff are trying to plan for program implementation in the absence of critical eligibility and other details, although guidance may be coming soon.

Bayesian Statistics In EU Clinical Trials: EMA Discusses Balancing Efficiency With Rigor

 
• By Eliza Slawther

Bayesian statistics could help clinical trial sponsors to include external information in their analyses, but concerns around bias and incorrect conclusions remain, the European Medicines Agency said.

Drug Sponsors Can Help Shape Agenda For FDA/EMA Pediatric ‘Cluster’ Calls

 
• By Sue Sutter

Companies can propose specific issues for discussion during the regulators’ monthly teleconferences, which focus on finding areas of agreement and reasons for nonalignment on specific pediatric development plans or general issues.

More from Pathways & Standards

ANDA User Fees: Complex Generics, Transparency, Communications Are Early Negotiation Targets

 
• By Sue Sutter

Teva seeks a dedicated development program and first-cycle approval metrics for complex generics. Role of artificial intelligence in the generic drug review process is a talking point for both agency and industry at reauthorization kickoff meeting.

EMA’s PRIME Scheme Explored: Growing Designations - But Are Accelerated Assessments Slipping?

 
• By Neena Brizmohun

While the first six months of the year saw eight new drugs targeting a range of diseases enter the European Medicines Agency’s priority medicines scheme, and three PRIME-designated treatments go on to win EU marketing approval, use of the accelerated assessment mechanism appears to be limited.

US FDA Asking Questions About Commissioner’s Voucher Program With Details Scarce

 
• By Derrick Gingery

FDA staff are trying to plan for program implementation in the absence of critical eligibility and other details, although guidance may be coming soon.