Single-Arm Rare Disease Trials “Work Only When Very Lucky”: FDA Maintains Strong Counsel To Rare Drug Sponsors To Randomize From First Patient

Prevision Policy Clips | FDA Unannounced Inspections Have Commenced In India, “Hundreds” Of Visits Expected

FDA “Enterprise Transformation” Starts With Inspections; Deputy Woodcock’s Latest (And Last?) Legacy Project

Avoiding Shortages: FDA Risk Management Plan Guidance For Manufacturers Follows ICH Template; Appendix Describes Specific Risk Factors

VALID Inclusion In Senate User Fee Bill Is Likely Best Chance For Passage Of LDT Oversight; Tiered Regulatory System Revives “Moderate-Risk” Test Category

Prevision Policy Clips | FDA Launches “Enterprise Modernization” Effort As Deputy Woodcock’s New Role Takes Shape

ARPA-H Coordination With FDA And CMS Added To Authorizing Legislation; FDA “May” Be Reimbursed – But Will It Be?

House User Fee Bill Breezes Through Full Committee: Accelerated Approval Rules Won’t Delay Pending Applications; Pediatric Cancer Combo Provision Added

Prevision Policy Clips | Senate FDA User Fee Bill Starts Small On Drug/Biologic Reforms, But Big On Other Areas Like LDTs

Senate FDA User Fee Bill Has Limited Drug/Biologic Reforms, But Ambitious Changes To LDT, Cosmetic And Supplement Authorities

Complex Generics At FDA: COVID Impact Felt “Across The Board” From Inspections To Fewer ANDA Filings

ARPA-H Will Need Early Communication With FDA To Bring Discoveries Through Development, Woodcock Says; Workload Capacity Will Be Issue

Prevision Policy Clips | House FDA User Fee Bill Heads To Full Committee May 18; “Cures” Hiring Authority Will Be Expanded

Prevision Policy Clips | Lilly Mounjaro (Tirzepatide) Type-2 Diabetes Approval Accompanied By FDA Reference To Superior Weight Loss

CBER Staffing Remains Challenging: Permanent Virtual Work May Help Offset Expected Retirements, Director Marks Says

Prevision Policy Clips | House Appropriations FDA Budget Hearing Rescheduled For May 19

Pediatric Study Waivers In Oncology: FDA Developing Framework To Consider Requests; ODAC Supports Flexible Approach In Crowded Classes

CBER Exploring “Intermediate Pathways” For Some Cell Therapies; House User Fee Bill Has One Starting Point

Prevision Policy Clips | FDA User Fee Bill Moving Fast In House: Full Committee Mark-Up Planned For Week Of May 16

House User Fee Bill On Fast Track To Passage: Minor Tweaks May Come Before Full Committee Mark-Up; CURES 2.0 On Separate Track For Now, But ARPA-H May Be Added

Prevision Policy Clips | CDER’s Accelerating Rare Disease Cures (ARC) Program Will Coordinate Development Of Policy

CBER Cell/Gene Therapy Enhanced Communication Pilot Program Floated As Way To (Eventually) Justify Funding For Additional Staff

FDA Inspection Backlog Could Be Greatly Eased By User Fee Bill Provisions; Foreign Manufacturer Focus Carries Forward In Bipartisan House Bill

FDA Oncology Dose Optimization Guidance Is Coming – But No Timeline Ready; Key Features Of Acceptable Dose Studies Suggested At FDA-ASCO Workshop

Prevision Policy Clips | House FDA User Fee Bill Mark-Up Set For May 11; ARPA-H Act Also On Agenda