Hemophilia Gene Therapy Guidance Updates Endpoint Discussion But Not Conclusion: FDA Sees Accelerated Approval Based On Factor Activity, ABR As Confirmatory Endpoint

Gene Therapy Guidance “Suite” Finalized In 18 Months; Rapid Turnaround For FDA Also Underscores Limits Of Guidance Pathway For Fast-Moving Science

FDA Gene Therapy “Sameness” Policy Leans Toward Finding Differences, Enabling Competition Under Orphan Drug Act

Prevision Policy Clips | FDA Finalizes “Suite” Of Gene Therapy Guidances; Issues Draft On “Sameness” For Orphan Drug Purposes

FDA Commissioner Hahn’s Inner Circle Takes Shape: Incoming Deputy Anand Shah Seen As Potential Successor To Abernethy – And Future Commissioner?

Prevision Policy Clips | House FDA Hearing January 29 Features J&J Exec, Friends of Cancer Research CEO

Antibiotic Subscription Model: Duke-Margolis Proposal Blends Government Value Assessments With Private Purchasing/Distribution Entities

Prevision Policy Clips | Minimal Residual Disease Biomarker Use Final Guidance

Prevision Policy Clips | ODAC Gets Two Reviews In February: Steba Biotech’s Padeliporfin for “Low-Risk” Prostate Cancer, Lilly’s Cyramza For NSCLC

CDER Drug Risk Management Board Will Integrate Quality With Clinical Safety Leaders; First Public Output Of Drug Safety Overhaul Under Deputy Cavazzoni

Golodirsen Approval Package Includes Commitment By Sarepta To Withdraw Vyondys 53 If Confirmatory Trial Does Not Show “Sufficient” Evidence Of Benefit

Prevision Policy Clips | OND Reorg: Phase III Launches January 21 With Placeholder Leadership In OTC

Early Approvals At FDA: Nearly One-Quarter Of NMEs Beat The User Fee Goal Date By More Than Two Weeks In 2018-2019; New Trend Gaining Steam

Prevision Policy Clips | Trump Impatience On Drug Pricing Complicates Finance/IPI Agreement

Part D Overhaul Endorsement From MedPAC Coming Soon: Could Boost Pharma-Friendly Legislation For Drug Pricing

FDA Committee Splits Over Durect’s Posimir For Post-Op Pain Relief; Agency-Sponsor Dispute Leaves Committee Confused

Prevision Policy Clips | FDA Invites Broader Stakeholder Input For Rare Disease Day 2020

When To Call ODAC? FDA’s Pazdur Gives Three Reasons

Part B Billion Dollar Club: Biosimilar Price Erosion For Infliximab And Growth Of Checkpoint Inhibitors Are Trends In 2018 Medicare Dashboard Data

Oncology Pediatric Study Plan Q&A Guidance Tries To Settle Confusion On FDARA Requirement

OxyContin “Real World” Abuse Data Again Under Review At FDA: Future Of Opioid Development May Hinge On Outcast Company

IPC’s Aximris XR Oxycodone Formula Voted Down By Advisory Committee; Members Implore FDA To Better Define “Abuse-Deterrent” Claims

Prevision Policy Clips | FDA Adds Four NDAs To Be “Deemed” BLAs In March

Nektar Ends Oxycodegol Program After Historic Rejection By Joint FDA Advisory Committee; Single Efficacy Trial and Unclear Abuse Liability Drive Negative Vote

Esteve Tramadol/Celecoxib Combo: Industry Reps Salvage Split Vote By FDA Committee, Preserving Hope That Novel Opioids May Still Be Approvable