Small Molecules, Big Price Tags: ICER Finds Doubling In Launch Prices For Novel Small Molecule Drugs Between 2022-2025; Reaction To IRA?

Deadline Tracker: November Approvals Could Bring FDA Near Record For NMEs; Etcamah, Lyrfigtu Approvals Showcase Different Themes For Accelerated Approvals In Oncology

Prevision Policy Clips | Drug Facility Listing Removal Process Streamlined By FDA

FDA Sticks To Cell & Gene Therapy Potency Assurance Recommendations In Final Guidance, Provides Case Examples To Help Guide Sponsors

Sydnexis Ophthalmic Atropine Appeal: Advisory Committee Sides With FDA On Issues With Evidence Of Efficacy; Compounding Use Not Relevant To Discussion, Agency Says

Prevision Policy Clips | UniQure Huntington’s Gene Therapy BLA Expected To Go To Advisory Committee

FDA Drug Inspection Operations Update Features More Involvement Of Drug Center; ConOps 2.0 Revision Includes Remote Regulatory Assessments

Prevision Policy Clips | CDER Associate Director For Research Is Scott Steele

Advisory Committee Tracker: Biohaven’s Troriluzole Will Be Next Reconsideration Of Makary-Era Rejection To Go To Committee; Date TBD

HELP Leadership Change In 2027 Will Complicate User Fee Reauthorization, Staffer Warns; Clinical Trial Reform, Country-of-Origin Labeling Measures Are Likely PDUFA Riders

Rare Disease Framework Needs Top-To-Bottom Implementation, Patient Advocates Tell FDA; Structural Changes For Review Teams Suggested

Prevision Policy Clips | FDA “Single Trial” Policy Change Explains “Full” Approval For Lyrfigtu

HHS Interim Policy Opens Up Market To Compound Six Peptides That Received Positive Votes At July PCAC: FDA Objections Overruled; Guardrails Include Ban On Combos, Complex Dosage Forms

“Now Is The Moment For Us To Step In”: FDA Center Directors Explain Drivers For Rare Disease Framework, Key Goals For Early Implementation

Sydnexis Atropine Ophthalmic Appeal Will Showcase New Era For Advisory Committees; FDA Has Long List Of Efficacy Concerns, And Is Not Asking For A Vote

Prevision Policy Clips | CMS Cell & Gene Therapy Access Model Will Expand Beyond Sickle Cell Disease: RFAs Coming Soon

Rare Disease Advocates Should Unite Behind “System Changes,” Ex-CDER Head Woodcock Urges; Gene Editing Guidance Is Type Of Change Woodcock Wants To See

Gene Editing Treatments For Monogenic Diseases: FDA Guidance Would Allow Multiple Variants In Same BLA; “Individualized Feature Space” Is Key Regulatory Concept

Prevision Policy Clips | Gene Editing “Platforms”: FDA Draft Guidance Describes Approach To Single BLA For Multiple Variants

CMC/Facility Prep Components Of BsUFA IV Agreement Praised By FDA And Industry During Public Review Of Commitment Letter; Changes Reflect “Maturing” Program

Prevision Policy Clips | Staff Numbers Are Stabilizing At FDA Biologics Center

Treasury Proposed Rule On COINS Act Adds Biotech To “Prohibited Technology” List, Final Reg Likely To Have Narrowed Definition; Waiver Process Provides Another Out

Prevision Policy Clips | Biohaven Troriluzole Resubmission Will Be Latest Reconsideration By FDA Of Makary-Era Rejections

CMS Sickle Cell Model Is Ramping Up: 31 Patients Have Completed Treatment, But “Over 100” Are In Pipeline; Can Model Be Replicated Beyond Medicaid?

Prevision Policy Clips | FDA Associate Chief Counsel Dinesh Kumar Leaves FDA