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U.S. Department of Health and Human Services

About FDA

Use of Extrapolation of Efficacy in Products Granted Pediatric Exclusivity

Description: Evaluate the use of Extrapolation of Efficacy in Pediatric Trials in products submitted in response to the WRs issued by FDA. OPT will summarize its experience with the use of extrapolation of efficacy. An analysis of the level of evidence and historical changes in approach will be discussed by the involved divisions. A draft paper describing the approach to pediatric extrapolation will be provided for further input. A final paper will be developed for publication.

Accomplishment: OPT evaluated the use of extrapolation of efficacy in pediatric trials in products submitted in response to the Written Requests (WRs) issued by FDA. A final paper was developed for publication describing the approach to pediatric extrapolation. Extrapolation is important because it minimizes the number of pediatric patients needed to demonstrate safety and efficacy, and may expedite drug development. In this regard, extrapolation of efficacy from the adult to the pediatric population has helped to maximize the use of existing information to increase the efficiency of pediatric drug development programs while maintaining the goals of increasing the number of safe and effective medicines approved for pediatric use based on scientifically robust data. Over the last decade, FDA has tested its assumptions about extrapolation and modified its approaches as knowledge and experience increased. The approaches ranged along a continuum and are still being refined. The data we published in Pediatrics on 166 products submitted in response to 159 Written Requests submitted to the Agency between 1998 and 2008 should lead to a more informed use of extrapolation of efficacy in pediatric drug development programs for those involved in pediatric trial design as well as for health professionals.

Extrapolation of Adult Data and Other Data in Pediatric Drug-Development Programs Pediatrics 2011;128;e1242; originally published online October 24, 2011; Julia Dunne, William J. Rodriguez, M. Dianne Murphy, B. Nhi Beasley, Gilbert J.Burckart, Jane D. Filie, Linda L. Lewis, Hari C. Sachs, Philip H. Sheridan, Peter Starke and Lynne P. Yao DOI: 10.1542/peds.2010-3487

The online version of this article, along with updated information and services, is located on the World Wide Web at: http://pediatrics.aappublications.org/content/128/5/e1242.full.html

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

A. Prepare and distribute draft paper for consideration by the Divisions which participated in the working group

3/3/2010

Completed 3/3/2010

B. Incorporate changes and have working group provide final review

8/1/2010

Completed 5/27/2010

C. Obtain internal clearance for submission to a peer reviewed journal

11/5/2010

Completed 6/2/2010

D. Manuscript developed for publication

2/1/2011

Completed 5/16/2011

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.

Glossary

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.