Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics


The Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research, in co-sponsorship with the American College of Gastroenterology (ACG), the American Gastroenterological Association (AGA), the Crohn’s and Colitis Foundation of America, Inc (CCFA), the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) and the Pediatric IBD Foundation, is announcing a 2-day public workshop entitled “Gastroenterology Regulatory Endpoints and the Advancement of Therapeutics (GREAT II).”  The purpose of this workshop is to provide a forum to consider issues related to endpoints that can support drug development in the following disease areas: pediatric and adult inflammatory bowel diseases.

This workshop was planned by The Division of Gastroenterology and Inborn Errors Products (DGIEP) in the Center for Drug Evaluation and Research (CDER) at the U.S. Food and Drug Administration (FDA).  This workshop follows in the success of the GREAT workshop held in September 2012, where a forum for stakeholders provided an opportunity to discuss issues related to endpoints supporting drug development in eosinophilic esophagitis, pediatric and inflammatory bowel disease, and parenteral nutrition-associated liver disease.  The momentum for change and collaboration created by GREAT remains essential to all stakeholders, including the FDA, to prevent delay in the development of more effective endpoints for regulatory filings.  The goal of this educational activity is to discuss issues encountered in clinical trials specific to drug development in inflammatory bowel disease (IBD) and to facilitate continued stakeholder collaboration on endpoint development.  The workshop will discuss candidate endpoints for IBD registration trials, disease activity indices that measure treatment outcome, and dose-finding strategies.

DATES: The public workshop will be held on October 21 & 22, 2013, from 8:30 am – 5 pm


LOCATION:  The public workshop will be held at the HILTON WASHINGTON DC NORTH/GAITHERSBURG  620 Perry Parkway, Gaithersburg, MD 20877



This workshop will address endpoints for registration trials.  Stakeholders, including industry sponsors, academia, patients and FDA, will be engaged to address challenging issues related to selection of endpoints and assessment methodologies in registration trials for products intended to treat adult and/or pediatric inflammatory bowel disease.   The definition and measurement of treatment benefit in Crohn’s disease registration trials, the role of existing and future clinical outcome assessments including development of patient reported outcome (PRO) measures, timing of endpoint assessments, and dose-finding strategies will be discussed. In addition, there will be a follow-up to previous workshop discussions of endpoints and clinical trial design for ulcerative colitis registration trials. Strategies and methods to overcome the challenges of developing drugs in pediatric populations and facilitate the collection of dosing, safety and efficacy information for drugs used “off-label” in children will be discussed. Strategies and methods to overcome the challenges of defining the safety,  efficacy and appropriate dose of drugs that are currently being used off label in pediatric populations and for which PREA and BPCA do not apply.  


Registration:  There is no fee to attend the public workshop, but attendees must register in advance.  Space is limited, and registration will be on a first-come, first-served basis.  Persons interested in attending this workshop must register online at  before October 01, 2013.