Primary Mitochondrial Diseases Likely Require RCTs, But FDA Can Be Flexible On Endpoints And Designs, Agency Suggests During Reagan-Udall Workshop

Prevision Policy Clips | Onshoring Health Product Manufacturing Will Be Focus Of House Hearing June 11

Prevision Policy Clips | House FDA Appropriations Bill Follows White House Lead: $300 Million Cut

FDA Cell And Gene Therapy Roundtable Panelists Advocate For Onshoring Early Clinical Trials; OTP Leadership Praised Widely

FDA Opening The Archives: “Redacted” Documents Will Be Made Public, CBER’s Prasad Says; Real-Time Review Coming To More Classes?

AI And Generic Drugs: FDA Will Showcase Tool To Help Calculate Maximum Daily Dose; Agency’s Lionberger Stresses Big Returns From Investment In Generic Drug Science

Prevision Policy Clips | Sarepta Receives Platform Technology Designation For Gene Therapy Viral Vector

“ACNU” OTC Final Rule Highlighted In Senate Hearing: HELP Chair Cassidy Wants More Switches; Corrigan-Curay Handles First Hearing As Acting CDER Director

Prevision Policy Clips | Makary’s “Philosophy” For FDA Management Will Be Fleshed Out In Upcoming JAMA Article

Platform Technology Approvals Coming Soon? CBER’s Prasad Underscores FDA’s Continued Commitment To Program Under New FDA Leadership

The Case For Patient-Centered Artificial Intelligence: Former CDER Director Woodcock Sees Broad Uses For AI, If Models Are Aimed Correctly

FDA Deadline Tracker: Nucala COPD Approval Comes Two Weeks Late; Stealth Overdue Barth Drug Rejected

Prevision Policy Clips | FDA Rolls Out “Elsa” AI Tool: Industry Data Is Not Being Used To Train

CDER Staff Would Be Cut By 1,200 Under White House Budget, CBER Would Lose 260 Positions; Cuts Include User Fee Funded Slots

MAHA Report Directs FDA/NIH To “Independently Replicate” Industry Pediatric Trials; Kennedy Talking Points About “Overprescribing” Fleshed Out – But Undermined By Fictitious Citations

Prevision Policy Clips | CBER Appropriations Would Be Slashed 24%, CDER By 18% Under White House Budget Proposal

Repurposed Generics For Rare Diseases: Citizen Petition Process Could Jumpstart New FDA Pathway, Former CDER Director Woodcock Suggests

Prevision Policy Clips | Neuromuscular Disease Real World Data Collaboration Between Critical Path Institute and Citizen Health

Prevision Policy Clips | Stealth Elamipretide Rejected By FDA After Extended Review, But Accelerated Approval May Be Path Forward

Advisory Committee Tracker: Three Rare Disease Drugs Are Headed To Advisory Committees, But FDA Has Yet To Announce Any Dates; Capricor DMD Cell Therapy Could Be As Early As June

Prevision Policy Clips | COVID-19 Vaccination Removed From Vaccine Schedule For Healthy Children And Pregnant Women

COVID Vaccine 2025-2026 Formula Will Be JN.1 Lineage, With Preference for LP.8.1 Strain: VRBPAC Members Question Impact of New Framework on Availability; FDA Has No Answers

Prevision Policy Clips | Biopharma CEOs Invited To Engage With FDA Leadership

UroGen’s Bladder Cancer Mitomycin Gel Gets ODAC Split Vote; FDA Expresses Concern About Low-Bar Precedent From Single-Arm Trial

FDA Talc Talk: “Expert Roundtable” Urges Cautious Approach on Talc – And Illustrates Echo-Chamber Regulatory Approaches Under Makary; Pharmaceuticals Have Largely Moved to Alternative Excipients