This workshop assisted the FDA in identifying current knowledge gaps for using NITs as diagnostic biomarkers and reasonably likely surrogates, as well as provided a framework for additional data that are needed to fill these knowledge gaps. The primary focus of this workshop was “non-cirrhotic NASH/MASH population with advanced (i.e., Stage 2 or Stage 3) liver fibrosis.”

-Session One: Considerations for Surrogate Endpoint Development and Approval Pathways.
-Session Two: Identify Knowledge Gaps for Current Endpoints in NASH/MASH Clinical Trials.

Timestamps

00:12 – Opening Remarks

08:28 – Biomarkers and Surrogate Endpoints

25:39 – Recent Example of Reasonably Likely Surrogate Endpoint Accepted by the FDA - Reduction in Amyloid Beta Plaques Measured by PET in Alzheimer’s Disease

37:54 – Lesson Learned from Makena Drug Development

54:28 – Approval Pathways and NASH/MASH Drug Development

01:13:28 – Advancing Endpoint Development

01:25:43 – Session Two Introductions

01:28:06 – One Stage Reversal of Fibrosis – How Do Hepatologists View This Change?

01:46:00 – One Stage Reversal of Fibrosis – How Do Pathologists View This Change?

02:04:33 – NASH Resolution – How Do Hepatologists View This Change?

02:18:52 – NASH Resolution – How Do Pathologists View This Change?

02:35:13 – The Value of Completing Clinical Benefit Trial for Validating Surrogate Endpoint – Clinician’s View

02:56:59 – Session Two Q&A Discussion Panel

Speakers:

Jeffrey Siegel, MD
Office Director
Office of Drug Evaluation Sciences (ODES)
Office of New Drugs (OND)
Center for drug Evaluation and Research (CDER) | FDA

Peter Stein, MD
Director
OND | CDER | FDA

Kevin Krudys, PhD
Associate Director
Office of Neuroscience (ON)
OND | CDER | FDA

Christina Chang, MD, MPH
Division Director
Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM)
Division of Urology, Obstetrics and Gynecology (DUOG)
OND | CDER | FDA

George Makar, MD, MSCE
(Acting) Deputy Director
Division of Hepatology and Nutrition (DHN)
Office of Immunology and Inflammation (OII)
OND | CDER | FDA

Rebecca Hager, PhD
Lead Mathematical Statistician
Division of Biometrics III (DBIII)
Office of Biostatistics (OB)
Office of Translational Sciences (OTS) | CDER | FDA

Laura Lee Johnson, PhD
Division Director
DBIII | OB | OTS | CDER | FDA

Don C. Rockey, MD
Professor of Medicine
Specialties: Gastroenterology and Hepatology
College of Medicine
Medical University of South Carolina

David E. Kleiner, MD, PhD
Senior Research Physician
Director, Laboratory Information System
Chief, Post-mortem Section
Laboratory of Pathology
National Cancer Institute (NCI)
National Institutes for Health (NIH)

Naga Chalasani, MD
David W Crabb Professor of Gastroenterology and Hepatology
Vice President for Academic Affairs
Indiana University School of Medicine &
Indiana University Health

Cynthia Behling, MD, PhD
Pathologist
University of California, San Diego and
Pacific Rim Pathology Lab

Theo Heller, MD
Section Chief: Translational Hepatology Section, Liver Diseases Branch
Senior Investigator: Clinical Research Section, Liver Diseases Branch
National Institute of Diabetes and Digestive and Kidney Diseases (NIKDDK)
National Institute for Health (NIH)

Panelists:

Naga Chalasani, Laura Lee Johnson, Don C. Rockey, David E. Kleiner, Cynthia Behling, Theo Heller

and

Scott Friedman, MD
Dean for Therapeutic Discovery
Fishberg Professor of Medicine
Professor of Pharmacologic Sciences
Chief, Division of Liver Diseases
Icahn School of Medicine at Mount Sinai

Mary Rinella, MD
Director of the Metabolic and Fatty Liver Program
Professor of Medicine at the University of Chicago Pritzker School of Medicine

Gregory Levin, PhD
Associate Director for Statistical Science and Policy
OB | OTS | CDER | FDA

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