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U.S. Department of Health and Human Services

About FDA

Use of Alternative Endpoints in Pediatric Clinical Trials

Description: OPT will conduct a study to identify and evaluate alternate endpoints which have and/or can be used in pediatric clinical trials. The Institute of Medicine (IOM) also will be conducting such an analysis using publicly available data as mandated by FDAAA 2007. OPT will evaluate this same question using all data available to FDA, focusing on products in one therapeutic area of importance to pediatrics (e.g., neurology, cardiovascular, or metabolic/endocrine). The results of this study will greatly enhance FDA's ability to identify critical improvements in the path for pediatric product development.

Accomplishment: OPT conducted a study to identify and evaluate alternate endpoints which have and/or could be used in pediatric clinical trials in collaboration with the Division of Gastroenterology and Inborn Error Products (DGIEP). An alternate endpoint was defined as a primary (or secondary) outcome specifically designed for the pediatric population and used to assess the impact of study treatment, where the endpoint was different from the one used in adult clinical trials. The primary objective was to evaluate the alternate endpoints and activity indices in the registration trials of the approved therapies for pediatric Ulcerative Colitis (UC). The results demonstrated that alternate endpoints were used as primary endpoints in the ColazalĀ® pediatric trial and used as secondary endpoints for the RemicadeĀ® pediatric trial. There was also no consistency in the definition of primary efficacy endpoints and the instruments in pediatric UC registration trials. These results have been published as an abstract at the 2012 Pediatric Academic Societies (PAS) and at the 2012 North American Society for Pediatric Gastroenterology Hepatology, and Nutrition (NASPGHAN. The study has also been submitted to the Journal of Pediatric Gastroenterology and Nutrition (JPGN) for publication.

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

A. Establish Task Order with the Institute of Medicine


Completed 9/1/2010

B. Identify Area of Therapeutic Focus


Completed 4/15/2011

C. Establish Working Group and Identify Tasks


Completed 5/31/2011

D. Preliminary Analysis and Decision on Timeline


Completed 10/31/2011

E. First Draft of Assessments/Recommendations


Completed 12/31/2011

Key Projects Legend

Milestone StatusDefinition
Not Yet StartedWork for specific milestone has not yet been started.
CompletedMilestone and/or overall project is completed.
On TrackMilestone - On track for completion by milestone deadline. Quarter status - Project is on track for completion based on overall milestone status.
On HoldMilestone - On hold, but deadline for completion has not passed. Quarter status - Project is on hold, based on overall milestone status.
DelayedMilestone - Delayed as it has not been completed and deadline has passed. Quarter status - Project is delayed based on overall milestone status.


Note: The data provided on this website is produced on an ongoing basis for performance management purposes and is subject to change due to updates of preliminary estimates, corrections, or other reasons. In addition, FDA may change the type or amount of data provided on this website at any time. Information marked as "Completed" may include measures and/or key projects for which activities are ongoing but no longer tracked as part of FDA-TRACK.