FDA To Launch “Real-Time Clinical Trial” Pilot In August; Commissioner Highlights Amgen, AZ Studies As Proof Of Concept

AstraZeneca’s Truqap sNDA Will Face Uphill Battle At ODAC: Magnitude Of Clinical Benefit – Plus Fatal Toxicities – Will Make For Tough Review

GDUFA Negotiations Wrap Up After Agreement On New Fee Structure; “America First” Changes To Promote Onshoring Will Be Included

AZ ctDNA-Based Strategy Leaves FDA “Uncertain” About Camizestrant Efficacy Heading Into Advisory Committee; First ODAC Post-Pazdur May Test FDA’s Ability To Shape A Vote

Prevision Policy Clips | FDA Formally Moves To Withdraw Amgen’s Tavneos Citing “Untrue Statements Of Material Fact”

FDA Quality Management Maturity Program: Pilot Work Continues, Plans For Scale-Up TBD; 2024 Cohort Results Shared At Generic Drugs Forum

Prevision Policy Clips | Compass, Usona And Transcend/Otsuka Receive CNPVs For “Psychedelic” Medications

FDA Launching OTC Switch Pilot: Agency Will Identify Drugs To Make Nonprescription, Work With Sponsors On Applications; ACNU Pathway Has “Great Potential,” FDA’s Murry Says

Prevision Policy Clips | Regeneron DB-OTO Deafness Gene Therapy Will Be Free In US After FDA Approval April 23

Prevision Policy Clips | CMS Drug Price Negotiation Town Hall: Formulary Access, Utilization Management Are Focus

Office Of Generic Drugs Paints Rosy Picture Of Performance At Annual Forum: ANDA Rejections, RTRs Declining; “Q1/Q2” Advice Having Positive Impact

CBER Urged To Devote More Effort To Cell & Gene Therapy Guidances To Share Approaches Discussed In One-on-One Sessions With Sponsors

Prevision Policy Clips | Medicare Weight Loss Demo: “GLP-1 Bridge” Will Run Through 2027, “BALANCE” On Hold

ANDA Application Fee Could Come Down Under GDUFA IV Proposals; Inspection Topics, “Data Fidelity” Dominate Recent Negotiations

Prevision Policy Clips | FDA Issues One-Year Progress Report On New Approach Methodologies To Reduce Animal Testing

PDUFA VIII Negotiations Run Behind Schedule, Meeting Minutes Indicate; Definition Of US-Based Company For Fee Waiver Still In Discussions

FDA, Industry Still Struggling To Define Threshold For Pediatric Gene Therapy Trials; Requirement For “Prospect Of Direct Benefit” Remains Key Challenge

Regulatory Flexibility Is “Consistent” For Ultra-Rare And Less Rare Diseases, FDA’s Rick Maintains; UniQure’s AMT-130 Receives Attention At NORD Meeting

FDA’s Summer Trip? Three CNPV Reviews Coming For Psychedelic Meds, Makary Says; Antidepressants Criticized At White House Event Promoting Psychedelic Therapies

Prevision Policy Clips | FDA To Issue Three CNPVs For Psychedelic Therapies “This Week”

“Plausible Mechanism” Framework Calls For Higher Data Quality Standards As Bespoke Therapies Move From Research To Approval, FDA’s Buracchio Says

Prevision Policy Clips | Kennedy Defends Replimune Rejection: “Marty Made The Correct Decision"

“Abbreviated BLA” Pathway Discussed At Senate HELP Hearing: MedPAC’s Miller Proposes Streamlined Biosimilar Requirements To Increase Competition

Prevision Policy Clips | FDA Planning Second Peptide Compounding Advisory Committee “Before The End Of February 2027”

CDER’s Innovative Trial Pilots Slow To Launch: Acceptance From Other Regulators Is One Reason, But Industry May Need To “Get Over” Risk Intolerance