FDA Issues Final Guidance for Providing Information on Pediatric Uses of Medical DevicesMay 8, 2014
By Allyson B. Mullen –
On May 1, 2014, FDA issues a final version of the guidance document “Providing Information about Pediatric Uses of Medical Devices” (the “Final Guidance”). The draft of this guidance document was originally issued on February 19, 2013, at the same time FDA issued a supplemental notice of proposed rulemaking to implement the pediatric submission requirements from the Food and Drug Administration Amendments Act of 2007 (“FDAAA”), which was finalized earlier this year (see our earlier posts here and here).
Section 515A of the Federal Food, Drug, and Cosmetic Act (FD&C Act) requires that PMAs, HDEs and PDPs (PDP stands for Product Development Protocol, a premarket pathway that essentially exists only on paper) include “if readily available – (A) a description of any pediatric subpopulations that suffer from the disease or condition that the device is intended to treat, diagnose, or cure; and (B) the number of affected pediatric patients.” The Final Guidance provides useful information for applicants regarding the pediatric information requirement in a question and answer format. Such information includes what is meant by “readily available,” definitions of the pediatric populations, some acceptable sources that could address the pediatric information requirements, and where and how to include this information in a submission.
The Final Guidance explains that the applicant should provide in narrative form “(1) a description of the uses of the device (proposed indication for the device) and (2) an estimate of the number of affected pediatric patients in the United States that suffer from the disease or condition that the device is intended to treat, diagnose or cure (including an estimate of the affected pediatric patient population size as a whole and according to the pediatric subpopulations).” Final Guidance at 6. The Final Guidance also provides a suggested table format for providing FDA with the required pediatric information. In addition, the Final Guidance provides examples of acceptable and unacceptable data sources for obtaining the required pediatric information. Generally speaking, acceptable sources are reliable, publicly available sources, and unacceptable sources are internal sources, including marketing and sales data.
Most notably, the Final Guidance explains that if the submitted pediatric information suggests that there is a potential for harm from off-label use in pediatric populations, FDA may require limitations, warnings or contraindications in the device labeling. Labeling limitations seems like a logical result if there is a potential for harm to pediatric patients from off-label use of the device, but what if the effect of the device on pediatric populations is simply unknown or what if the pediatric information shows that the disease the device is intending to treat is widely present in pediatric populations, but the applicant is only seeking approval for an adult indication? It remains to be seen whether FDA will require labeling limitations or something greater (e.g., data to support a pediatric indication).
Finally, the Final Guidance explains that the required pediatric information (discussed above) is not alone sufficient to establish the safety and effectiveness of a device for a new pediatric indication for use. The requirement to include pediatric information in PMAs, HDEs and PDPs took effect on April 10, 2014 for all newly submitted applications.