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U.S. Department of Health and Human Services

About FDA

Public Workshop on Cell and Gene Therapy Clinical Trials in Pediatric Populations

Description: OCTGT is collaborating with the FDA’s Office of Good Clinical Practice and Office of Pediatric Therapeutics, the National Institutes of Health’s Office of Biotechnology Activities, the American Society of Gene and Cell Therapy, the International Society for Stem Cell Research, the American Academy of Pediatrics and the Public Responsibility in Medicine and Research, a public interest group, to sponsor the workshop. The purpose of the workshop is to gather information from Institutional Review Boards (IRB), gene and cellular therapy clinical researchers, and other stakeholders on best practices in planning cell and gene therapy trials in pediatric populations. Specifically, the best practices will focus in three areas: evaluating novel therapeutic products prior to initiating pediatric clinical studies; identifying and minimizing risks associated with the administration of cell and gene therapy products in pediatric studies; and determining the data submitted to the IRB and FDA prior to initiating pediatric clinical trials.

Accomplishment: OCTGT collaborated with FDA’s Office of Good Clinical Practice and Office of Pediatric Therapeutics, the National Institutes of Health’s Office of Biotechnology Activities, the American Society of Gene and Cell Therapy, the International Society for Stem Cell Research, the American Academy of Pediatrics and the Public Responsibility in Medicine and Research, a public interest group to sponsor the public workshop. The purpose of the workshop was to gather information from Institutional Review Boards (IRB), gene and cellular therapy clinical researchers, and other stakeholders on best practices in planning cell and gene therapy trials in pediatric populations. Specifically, the best practices focused on three areas: evaluating novel therapeutic products prior to initiating pediatric clinical studies; identifying and minimizing risks associated with the administration of cell and gene therapy products in pediatric studies; and determining what data need to be submitted to the IRB and FDA prior to initiating pediatric clinical trials. The workshop provided education for FDA staff as well as IRBs and investigators. As a result of the workshop, OCTGT developed a training program for staff regarding best practices of informed consent review and develop internal policy regarding informed consent documents.

Milestone DescriptionMilestone DateMilestone StatusMilestone Completion Date

A. Invite speakers for workshop

7/23/2010

Completed 7/20/2010

B. Publish Federal Register notice

9/3/2010

Completed 9/7/2010

C. Finalize FDA presentations

10/27/2010

Completed 10/22/2010

D. Hold joint public workshop

11/2/2010

Completed 11/2/2010

E. Hold post-workshop meeting to evaluate possible output for educational programs

1/31/2011

Completed 12/21/2010

F. Provide webcast and publish transcript of the workshop

3/23/2011

Completed 12/15/2010

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.