Clinical Pharmacology Corner: FDA Announces Pediatric Trial Design and Modeling Workshop

FDA Announces Pediatric Trial Design and Modeling Workshop will be held on September 8, 2017 

The Office of Clinical Pharmacology, Office of Translational Sciences, Center for Drug Evaluation and Research, U.S. Food and Drug Administration (FDA) will hold a public workshop entitled “Pediatric Trial Design and Modeling: Moving into the Next Decade” on Friday, September 8, 2017. The purpose of this one day workshop is to:

• Review the current best practices in designing pediatric drug development trials using the knowledge and tools available.
• Discuss problems and potential solutions presently encountered with pediatric drug development trials.
• 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 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. New and unique approaches to pediatric drug development are needed given the multiple challenges in conducting pediatric clinical trials (e.g., insufficient numbers of pediatric patients and the desire to include pediatric populations, such as newborn infants, who have not previously been routinely included in drug development trials).

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. Approaches such as physiologically-based pharmacokinetics and 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 and optimize pediatric drug development using these techniques. This goal can be achieved through the cooperation of industry sponsors, the pediatric clinical community, and regulatory agencies.

The date, time, and location of the workshop are as follows:

Date: Friday, September 8, 2017
Time: 8:30 a.m. to 4:30 p.m.
Location: Great Room, FDA White Oak, 10903 New Hampshire Avenue, Silver Spring, MD 20993

The agenda for the workshop is available at: https://www.fda.gov/downloads/Drugs/NewsEvents/UCM565061.pdf

Registration is free and can be accessed at: https://www.eventbrite.com/e/pediatric-trial-design-and-modeling-moving-into-the-next-decade-tickets-35977814612. You may register to attend at FDA White Oak or via webcast.

FOR FURTHER INFORMATION CONTACT: Gilbert J. Burckart, Pharm.D., Associate Director for Pediatrics, Office of Clinical Pharmacology, 10903 New Hampshire Avenue, Silver Spring, MD 20993, 301-796-2065, gilbert.burckart@fda.hhs.gov or visit https://www.fda.gov/Drugs/NewsEvents/ucm564111.htm.

---

We always welcome your thoughts regarding the format, content, and utility of information you receive via this Burst email initiative. Comments may be sent via email to ocp@fda.hhs.gov.

This communication was prepared by Office of Clinical Pharmacology, Office of Translational Sciences, CDER, FDA.

---

FDA/Center for Drug Evaluation and Research (CDER)
Office of Translational Sciences
Office of Clinical Pharmacology
Email: ocp@fda.hhs.gov