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Event Title
Pediatric Trial Design and Modeling: Moving into the Next Decade

Public Workshop:

Date: Friday, September 8, 2017
Time: 8:30 a.m. to 4:30 p.m.
Location: Great Room, US FDA White Oak

The Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, US Food and Drug Administration is announcing a public workshop entitled “Pediatric Trial design and Modeling: Moving into the next decade”.
The purpose of this one day workshop is to:

  1. Review the current best practices in designing pediatric drug development trials using the knowledge and tools available;
  2. Discuss problems and potential solutions presently encountered with pediatric drug development trials;
  3. Discuss strategies related to design and evaluation that have the best chance of facilitating and optimizing the use of pediatric drug development trials to achieve the labeling of products for pediatric indications.

The Pediatric Research Equity Act and the Best Pharmaceuticals for Children Act have combined to facilitate the conduct of well over 1,000 pediatric clinical trials which have been submitted to the US FDA. An early evaluation of this program demonstrated that over 40% of these trials failed to achieve the intended outcome, with those products not having a labeled indication for pediatric patients.

This large number of pediatric studies has provided sufficient data for evaluation by industry, by academicians and by the FDA.  Numerous examples of both failed and successful programs have demonstrated the usefulness of modeling and simulation, and the use of trial design techniques using patient enrichment and qualified trial endpoints. Other techniques that have improved the efficiency of pediatric drug development include the extrapolation of efficacy from adults to pediatric patients, and clinical trial simulation.

At the same time, multiple challenges in pediatric drug development still exist. For many diseases, sponsors still struggle to recruit sufficient numbers of pediatric patients for drug development studies. Another challenge is the desire to include pediatric populations, such as newborn infants, who have not previously been routinely included in drug development trials.

New techniques such as physiologically-based pharmacokinetics and such as the application of Bayesian theory to designing pediatric drug development trials may provide an effective means of dealing with the challenges currently facing pediatrics.

Therefore the goal of this workshop is to look forward to the coming decade in the hope that pediatric drug development can now be optimized. This goal can be achieved through the cooperation of industry sponsors, the pediatric clinical community and regulatory agencies.


 Workshop Materials:


For questions regarding the workshop, please contact:

Gilbert J. Burckart, Pharm.D.
Associate Director for Pediatrics
Office of Clinical Pharmacology
10903 New Hampshire Ave.
Silver Spring, MD 20993

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