Devicemakers looking to market their products for use in children based on adult clinical trials should limit their use of that data to situations in which the course of the disease or condition and the device’s effects are “sufficiently similar” in both populations, the U.S. FDA says.
Partial data extrapolation, combined through a statistical model with pediatric data sources to demonstrate a reasonable assurance of effectiveness, is preferred over full extrapolation of data from adult studies, the agency says in draft guidance published Wednesday.
The guidance addresses the use of adult clinical trial data to increase the availability of devices for pediatric patients. It also discusses how devices should be labeled to support their safe and effective use in patients 21 and younger.
The FDA lists three main factors for partial extrapolation of data: the similarity of the existing adult response data and/or population characteristics to the intended pediatric population; the quality of the adult data; and whether the data can be used fairly and responsibly to determine the safety and effectiveness of the device.
Dealing With Uncertainty
The FDA cautions devicemakers that using extrapolated data adds a degree of uncertainty to safety and effectiveness assessments. The extent of that uncertainty depends on the differences between adult and pediatric patients and the quality of the data. Manufacturers should consult the FDA’s benefit-risk guidance to understand how the agency weighs extrapolated data during the course of premarket reviews, the guidance says.
The guidance is intended to address the pitfalls sponsors often face with pediatric trials. Among these are small and scattered populations leading to small trial sizes, challenges in enrollment and consent procedures and variations in physiology, anatomy and human factors in pediatric patients.