FDA Issues Supplemental Proposal On Information About Pediatric Patients

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A Food and Drug Administration supplemental proposed rule (FDA-2009-N-0458) would require medical device manufacturers to submit information about pediatric patients who suffer from the disease or condition that the device is intended to treat, diagnose, or cure, even if the device is intended for adult use.

On April 1, 2010, the agency published a proposed rule (75 Fed. Reg. 16, 365) requiring submission of pediatric data, along with a companion direct final rule (75 Fed. Reg. 16,347) (4 MELR 239, 4/7/10). FDA said it received significant adverse comment and withdrew the direct final rule later in 2010 (4 MELR 533, 7/28/10).

The new proposal, which was published Feb. 19 in the Federal Register (78 Fed. Reg. 11,612), reflects the comments the agency received. Comments on the new proposal are due April 22, FDA said.

According to the agency, very few devices are developed or assessed specifically for use in pediatric patients--those 21 years or younger at the time of treatment or diagnosis. The effort will provide a better understanding of which devices developed for use in adults should be assessed or modified for use in pediatric populations, FDA said.

Under the proposed rule, manufacturers would need to include information on pediatric patients when submitting specific types of applications:

• any request for a humanitarian device exemption (HDE);

• any premarket approval application (PMA) or supplement to a PMA; and

• any product development protocol (PDP).

According to FDA, submitting pediatric information is required under Section 515A of the Federal Food, Drug, and Cosmetic Act, which was added by the FDA Amendments Act of 2007. The requirements also will improve the agency's ability to track the number of approved devices for which there is a pediatric subpopulation who could benefit and the number of approved devices labeled for use in pediatric patients, FDA said.

Under the 2007 law, manufacturers must provide certain pediatric information, if readily available, with each PMA application or supplement, HDE request, or PDP. Under the new requirement, manufacturers now must include a description of any pediatric subpopulations who suffer from the disease or condition. Manufacturers also must include the number of affected pediatric patients, FDA said.


FDA regulators may use “approvable” letters to tell device companies they will not approve a product until the necessary information is submitted.  


FDA said if the applicant does not submit the required information, the agency may not approve the application until the required information is submitted. FDA said it “intends to contact the applicant during the normal course of our review” to inform the applicant about the missing information. If the application has no other deficiencies and otherwise meets applicable statutory and regulatory requirements for approval, but still lacks the pediatric descriptions required by the proposed rule, FDA said, it intends to send the applicant an “approvable” letter saying the application will be approved after the required information is submitted.

Draft Guidance

FDA also issued a draft guidance to explain the meaning of readily available information and how to comply with the requirements in the proposed rule. The title of the document is Providing Information about Pediatric Uses of Medical Devices Under Section 515A of the Federal Food, Drug, and Cosmetic Act, and the document was announced in a Feb. 19 Federal Register notice (78 Fed. Reg. 11,654).

According to the draft guidance, “readily available information” is available in the public domain. All submitted pediatric device use information should be available through commonly used public resources for conducting biomedical, regulatory, and medical product research, such as bibliographic databases of life sciences and biomedical information (such as MEDLINE and PubMed) and online scientific and medical publishers.

Pediatric device use information should exclude proprietary, trade secret, and commercial confidential information about the device, because the information will routinely be made available to the public, FDA said.

“To put the pediatric use information into context, your submission should briefly describe in narrative form the natural history of the disease or condition which the device is intended to treat, diagnose, or cure. If readily available, it would be helpful for you to also explain whether adult and pediatric populations are affected similarly (e.g., course of disease, detectable symptoms, how the device is used) and, if they are not, how each subpopulation is affected,” FDA said in the guidance. “If the device has already been approved for use in a particular subpopulation, and you are seeking approval of the device for use in an additional subpopulation, you should indicate this in your submission.”

Also in the draft guidance, FDA reiterated the types of premarket submissions that would need pediatric information under the proposed rule. The draft clarified that 30-day notices are not considered PMA supplements for purposes of the rule, so they are not required to include readily-available pediatric information.

In addition to the requirements addressed in the proposed rule, FDA in the draft guidance said pediatric use information also may be provided for uses of the device outside the approved or proposed indication “if such uses are described or acknowledged in acceptable sources of readily available information.” The information submitted will allow FDA to identify pediatric uses outside their approved or proposed indication for use to determine areas where further pediatric device development could be useful, the agency said.

Comments on the draft guidance are due April 22, and the docket number is FDA-2013-D-0117.

By Nathaniel Weixel  

A copy of the proposed rule is at http://www.gpo.gov/fdsys/pkg/FR-2013-02-19/html/2013-03647.htm.

The draft guidance is at http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM339465.pdf.

The notice announcing the guidance is at http://www.gpo.gov/fdsys/pkg/FR-2013-02-19/html/2013-03652.htm.


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