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,
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:
request for a humanitarian device exemption (HDE);
premarket approval application (PMA) or supplement to a PMA; and
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
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.
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
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
By Nathaniel Weixel
A copy of the proposed rule is at
The draft guidance is at http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM339465.pdf.
The notice announcing the guidance is at