Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices: A Summary of FDA Draft Guidance
By Molly Thomas
Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices: A Summary of FDA Draft Guidance
On May 6, 2015, the Food and Drug Administration (FDA) has released a draft guidance onLeveraging Existing Clinical Data for Extrapolation of Pediatric Uses of Medical Devices. This guidance is open for comments until August 4, 2015.
OVERVIEW
In the draft guidance, the FDA focuses its attention on the opportunity to leverage existing clinical data (adult and/or non-pediatric data) for use in pediatric indications. By leveraging the data, the FDA hopes to increase the number of pediatric medical devices introduced to the market. The objectives of this guidance are the following:
- Leverage adult and/or non-pediatric existing clinical data for use in pre-market approval applications (PMAs) and humanitarian device exemptions (HDEs) to provide safe and effective pediatric devices,
- Explain the varying factors where it is appropriate to increase clinical data to support pediatric device indications and labeling, and
- Provide a framework of how the FDA determines whether extrapolation is necessary and to what extent the data can be increased.
FRAMEWORK
The draft guidance furthers the information that is outlined in the final Premarket Assessment of Pediatric Medical Devices guidance (updated in 2014 from its original version published in 2004). Last year’s final guidance explained the necessary risks and considerations for pediatric medical devices.
In this proposed guidance, the FDA presents the set of challenges associated with clinical trials for pediatric populations (anyone under the age of 21). Common challenges include small pediatric populations, which lead to a small trial size and challenges with enrollment and consent of the procedures leading to increased time in determining the safety and effectiveness of the device. In addition, there are variations in pathophysiology, physiologic and anatomical structures in a child’s development compared to that of the adult or subpopulations. Required blood samples for studies may be too large to obtain from a child as well. Due to the challenges, associated studies had difficulties obtaining enrollment and consent from particular research subjects.
Additionally, due to these challenges, limited devices have pediatric-specific indications and labeling. Leveraging non –pediatric clinical data may reduce the need to develop large-scale clinical trials by using applicable data that ensures safety and effectiveness in a pediatric population.
