Where Are The Bespoke Breakthroughs? FDA Not Seeing Dramatic Efficacy From “N-Of-1” Treatments To Date; “Baby KJ” Is The Exception, Not The Rule

ICER Panel Reaffirms Cost-Effectiveness Of GLP-1 Weight Loss Agents; Budgetary Impact Still An Issue, Even At New Medicare Pricing, ICER Staff Suggests

“N-Of-1” Taking Off: CDER Has Cleared 49 Bespoke INDs, With Number Likely To Soar In 2026; CDER/CBER Coordinating, But Differences Will Remain For Gene-Editing Vs. ASOs

Prevision Policy Clips | FDA “Plausible Mechanism” Pathway Is Still “Under Construction”

Prevision Policy Clips | CDER “Meeting Minute Clarification Opportunity” Pilot Program

AI In Regenerative Medicine: Lack Of Training Data Is Obstacle To Advancing AI Use For Drug Development, CBER’s Oh Suggests; Manufacturing Is Focus For Early Adopters

Prevision Policy Clips | Where Are The IRA Prices? Senate Democrats Push For Publication Of Negotiated Prices

Prevision Policy Clips | US Generic Firms Will Testify At Senate Supply Chain Hearing November 19

Prevision Policy Clips | Sarepta Elevidys Relabeling Removes Non-Ambulatory Indication, Adds Black Box And Medication Guide

OCE Model May Expand To Other Areas, FDA’s Prasad Says During “Praise Pazdur” Edition Of Leadership Podcast; Makary, Prasad Affirm Support For Surrogate Endpoints

Prevision Policy Clips | Liver Safety Biomarker GLDH Qualified By FDA

Pazdur’s Welcome Letter To CDER Staff: “I Am Not A Micromanager”

Prevision Policy Clips | “Plausible Mechanism” Pathway Outlined By FDA Leadership

“Plausible Mechanism” Pathway Could Use “Regular” Approval Based On Evidence; RWE Will Be Key To Post-Market Surveillance, FDA NEJM Piece Says

FDA’s Makary Cites “New Workflow” Of CNPVs For Choosing Already Approved Products For Second Batch Of Pilot Program

CDER Director Pazdur Will Bring Immediate Boost To FDA Morale And Credibility – But Long-Term Issues Remain Unsettled; OCE Transition Will Be Next Big Change At Agency

Prevision Policy Clips | CDER Director Will Be Pazdur After All

FDA Requests Menopausal Hormone Therapy Label Updates: Regulatory Feedback Loop From Makary-Selected “Expert” Panel To Agency Action

Prevision Policy Clips | FDA Full-Year Funding Is Part Of Deal To End Shutdown

Medicaid “Most Favored Nation” Policy Fleshed Out: Demo Sets “GENEROUS” Terms For Manufacturers; Reference Price Is Not As Low As It Could Be, 340B Pricing Unaffected

Prevision Policy Clips | Still No CDER Director Announcement From FDA

GSK Jemperli Is Among Second Batch Of Commissioner’s National Priority Voucher Winners; Big Pharma Gets More This Time Around

Lilly, Novo GLP-1 Price Cuts For Current Indications Will Fund “Budget-Neutral” Expansion To Broader Weight Loss Coverage, CMS Says; CNPVs Included In Deal

Prevision Policy Clips | Senate Aging Committee Hearing Will Look at FDA Rare Disease Review Process

CMS Final Part B Physician Rule Drops Plan To Redefine “Bona Fide Service Fees” For Now; Manufacturers Will Still Be Expected To Document “Fair Market Value” Calculation