FDA is clarifying how and when existing adult clinical data may be extrapolated to support marketing approval and labeling of medical devices for use in pediatric patients.
Sponsors are encouraged to engage FDA early on when considering whether existing clinical data might support pediatric claims.
The agency notes in final guidance released June 21 that there is a dearth of scientific evidence to substantiate submissions for devices that diagnose or treat pediatric patients.
The guidance provides sponsors a roadmap on:
The guidance notes that conducting device clinical trials in children has presented numerous challenges, and adult devices are often used in children off-label as a result.
The guidance clarifies that the appropriateness of extrapolation largely depends on the similarity of existing data and the characteristics to the intended pediatric sub-population.
Industry comments on the May 2015 draft version of the guidance prompted the FDA to clarify how and when extrapolation may be appropriate, and to include de novo requests in the final guidance.
Advamed supported the move to extrapolate data, and said in its comments that leveraging existing clinical data “will have the important added benefit of reducing the number of pediatric human subjects needed to participate in clinical trials to demonstrate safety and effectiveness in a particular device.”
Advamed and other industry stakeholders also sought clarification on the extent of extrapolation across pediatric subpopulations as well as what the agency meant by “borrowing strength.”
In response to those requests, the final guidance provides additional explanation of the concept of extrapolation, defines pediatric subpopulations and includes data collected for de novo applications.
The guidance also explains “borrowing strength” as a means to bolster data sources. The agency notes that the extent of leveraging depends on the similarity “between borrowed data and any pediatric data that will be collected.”
The guidance also notes that study design could be different for different endpoints, and there could be different considerations in the pediatric population for safety versus effectiveness. “Therefore, safety is considered independently from effectiveness in deciding whether or not extrapolation may be appropriate.”
Advamed and industry stakeholders also questioned why the guidance did not apply to 510(k) devices. The FDA reiterated that it only applies to devices subject to premarket approval applications, human device exemptions and de novo premarket requirements. The FDA noted that future guidance may address issues related to the 510(k) regulatory pathway.
Read the guidance here: www.fdanews.com/06-20-16-UsingExistingDataPediatricUsesofMedicalDevices.pdf. — Joya Patel