Regulatory Focus™ > News Articles > New FDA Guidance Establishes Pediatric Data Submission Standards for Devices

New FDA Guidance Establishes Pediatric Data Submission Standards for Devices

Posted 01 May 2014 | By Alexander Gaffney, RAC

US Food and Drug Administration (FDA) regulators have finalized a new guidance document that aims to make medical devices a little bit safer for children.


In 2007, Congress passed the Food and Drug Administration Amendments Act (FDAAA), a massive piece of legislation that channeled a significant amount of energy into commitments meant to make products safer for consumers, including pediatric patients, to use.

Notably, section 515A of FDAAA requires any sponsor of a premarket approval application (PMA), PMA supplement, humanitarian device exemption (HDE) or product development protocol (PDP) to submit to FDA information regarding how many children suffer from the disease or condition their product is intended to treat.

FDA defines pediatric populations as including neonates (0-28 days), infants (<2 years), children (2-12 years of age) and adolescents under 21 years of age. In other words, if a product can treat a person under the age of 21, it falls under section 515A of FDAAA.

The law states:

"The application or protocol described … shall include, with respect to the device for which approval is sought and 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 goal of section 515A is relatively straightforward: Federal regulators should have a good idea if a product is likely to be used in pediatric subpopulations, as those populations often have very specific needs that may limit their safe or effective use.

Those concerns have historically been primarily associated with pharmaceutical products due to their potential to be metabolized differently by children, but FDAAA expanded those concerns to devices as well, and called on regulators to develop plans to expand pediatric medical device research and development.

Section 515A calls on manufacturers to submit to FDA data that might be used to support its safety and efficacy in children.

"If the course of the disease or condition and the effects of the device are sufficiently similar in adults and pediatric patients, the FDA may conclude that adult data may be used to support a determination of a reasonable assurance of effectiveness in pediatric populations, as appropriate," section 515A explains. Additionally, data about subpopulations may be extrapolated to one another.

Guidance Clarifications

But Section 515A is ambiguous regarding at least one point: What does "if readily available" mean for companies? And which data should a company provide to FDA, and how?

FDA's final guidance, Providing Information about Pediatric Uses of Medical Devices, is intended to answer just that.

In the guidance, FDA explains that it takes the term "readily available" to mean, "Information that is available in the public domain … available through commonly used public resources for conducting biomedical, regulatory and medical product research."

These sources, it says, might include medical journals, statistical registries maintained by federal agencies, meta-analyses maintained by groups like Cochrane, information from, large surveys, patient registries and other clinical databases.

Unacceptable sources of data will include internal marketing surveys, sales records, R&D reports, private consultations with experts and unpublished presentations, FDA wrote. In other words, anything that isn't publicly available.

Submitting Data

FDA explained in the draft guidance that sponsors should collect all relevant information in a separate section of their application entitled "515A Pediatric Device Use Information."

That section will consist of two major elements: a description of the uses of the device and an estimate of the number of pediatric patients it affects in the US. This information should be contained in tabular form ("Pediatric Summary Table"), along with a brief narrative summary of the disease itself.

Example of Pediatric Summary Table

IndicationPediatric IncidencePediatric PrevalencePediatric Subpopulation / Age RangeSpecific Device / ComponentSource
This device is indicated for use as an adjunct to medical therapy in the management of pediatric and adult patients with the following clinical conditions: Congestive heart failure related to rare congenital disease, e.g., Duchenne's muscular dystrophy3,0001,200Adolescent 16-21Widget Model M-111

FDA will reject an application if it does not include pediatric data under Section 515A, and will require the information to be submitted through an amendment to the application.

While the data are necessary for FDA to approve a device, even for an adult-oriented device, disease incidence data is not sufficient to support a pediatric application on its own. Instead, FDA says it will use the information in reports to Congress and to identify age-specific warnings that may be required to be included in the device labeling.

"Ultimately, FDA would like to use this data to determine unmet pediatric needs in medical device development," the agency wrote. "Once unmet needs are identified, FDA will be better able to coordinate efforts of stakeholders, device manufacturers and FDA staff to promote new device development and proper labeling of existing medical devices for pediatric use."

The guidance follows the March 2014 publication of a similar guidance on developing pediatric medical devices, and the January 2014 release of the FDAAA final rule on which the new guidance is based.

Providing Information about Pediatric Uses of Medical Devices (FR)

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