New draft guidance on extrapolating existing data to gain approval for pediatric indications applies to new devices as well as those already approved for adult use, an FDA official says.
“We certainly did not intend to limit the application of this guidance to only products that already have an approval for adult indications,” says Kathryn O’Callaghan, acting associate director for science and strategic partnership in the Center for Devices and Radiological Health. Companies that think they can leverage adult data or data from another pediatric population should speak with the agency early in the preapproval process about their plans and the rationale, she says.
Before extrapolating data, devicemakers should ensure that the existing adult response data or population characteristics are similar to the intended pediatric population and that the adult data is of good quality. They should also verify that the data can be used fairly and responsibly to determine the safety and effectiveness of the device, O’Callaghan 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, O’Callaghan says. She spoke at a recent FDA webinar on the guidance.
The draft guidance applies to PMA and HDE devices only because 510(k) devices fall outside the scope of the original regulation. However, the principles behind the guidance might apply to certain 510(k) devices as well, O’Callaghan says. She encourages sponsors of 510(k)s to contact the FDA during the presubmission process and file comments on the draft to alert the agency to concerns about the scope of the guidance.
CDRH issued the draft guidance to address ongoing challenges in pediatric device trials, such as small sample sizes and anatomy. For example, a reference draw may require more blood than is safe to take from a child.
The draft also applies to biologics-device combination products that are partially regulated by the Center for Biologics Evaluation and Research.