80 FR 26061 - Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 80, Issue 87 (May 6, 2015)

The Food and Drug Administration (FDA) is announcing the availability of the draft guidance entitled ``Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices.'' This draft guidance is being issued to explain the circumstances in which it may be appropriate to leverage existing clinical data to support pediatric device indications in premarket approval applications (PMAs) and humanitarian device exemptions (HDEs). The draft guidance also describes the approach that FDA would use to determine whether extrapolation is appropriate in medical devices, and the factors that would be considered within a statistical model for extrapolation. Extrapolation may be appropriate when the course of the disease or condition and the effects of the device are sufficiently similar in adults and pediatric patients and the adult data are of high quality for borrowing. This draft guidance is not final nor is it in effect at this time.

[Federal Register Volume 80, Number 87 (Wednesday, May 6, 2015)]
[Notices]
[Pages 26061-26062]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2015-10482]


-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2015-D-1376]


Leveraging Existing Clinical Data for Extrapolation to Pediatric 
Uses of Medical Devices; Draft Guidance for Industry and Food and Drug 
Administration Staff; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of the draft guidance entitled ``Leveraging Existing 
Clinical Data for Extrapolation to Pediatric Uses of Medical Devices.'' 
This draft guidance is being issued to explain the circumstances in 
which it may be appropriate to leverage existing clinical data to 
support pediatric device indications in premarket approval applications 
(PMAs) and humanitarian device exemptions (HDEs). The draft guidance 
also describes the approach that FDA would use to determine whether 
extrapolation is appropriate in medical devices, and the factors that 
would be considered within a statistical model for extrapolation. 
Extrapolation may be appropriate when the course of the disease or 
condition and the effects of the device are sufficiently similar in 
adults and pediatric patients and the adult data are of high quality 
for borrowing. This draft guidance is not final nor is it in effect at 
this time.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment of this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by August 4, 2015.

ADDRESSES: An electronic copy of the guidance document is available for 
download from the Internet. See the SUPPLEMENTARY INFORMATION section 
for information on electronic access to the guidance. Submit written 
requests for a single hard copy of the draft guidance document entitled 
``Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses 
of Medical Devices'' to the Office of the Center Director, Guidance and 
Policy Development, Center for Devices and Radiological Health (CDRH), 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 
5431, Silver Spring, MD 20993-0002. Send one self-addressed adhesive 
label to assist that office in processing your request.
    Submit electronic comments on the draft guidance to http://www.regulations.gov. Submit written comments to the Division of Dockets 
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852. Identify comments with the docket number 
found in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Jacqueline Francis, Center for Devices 
and Radiological Health, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 66, Silver Spring, MD 20993-0002, 301-796-6405; 
or Stephen Ripley, Center for Biologics Evaluation and Research (CBER), 
Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 
7301, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    Section 520(m)(6)(E)(i) of the Federal Food, Drug, and Cosmetic Act 
(FD&C Act) (21 U.S.C. 360j) defines pediatric device patients as 
persons aged 21 or younger at the time of their diagnosis or treatment 
(i.e., from birth through the 21st year of life, up to but not 
including the 22d birthday). Pediatric subpopulations are defined in 
section 520(m)(6)(E)(ii) (and adopted by reference in section 515A(c) 
of the FD&C Act (21 U.S.C. 360e)) to be neonates, infants, children, 
and adolescents.
    In an attempt to promote pediatric medical device development, CDRH 
published a final guidance document in 2004 entitled ``Premarket 
Assessment of Pediatric Medical Devices'' (Ref. 1). This 2004 document 
indicates that data can be extrapolated to support effectiveness and, 
on a limited basis, safety for premarket approval applications (PMAs) 
when consistent with scientific principles. Congress was aware of this 
2004 document when it passed the Food and Drug Administration 
Amendments Act of 2007 (FDAAA). Title III of FDAAA is the Pediatric 
Medical Device Safety and Improvement Act (PMDSIA). The FDAAA 
specifically authorized the use of adult data to demonstrate pediatric 
effectiveness. While safety exploration is not discussed in PMDSIA, FDA 
believes that there are specific cases where it will be appropriate to 
consider extrapolation of existing clinical safety data to support or 
enhance evidence for pediatric indications. FDA seeks comment on the 
appropriateness of extrapolating from adult clinical data to support 
medical device safety in pediatric patients.
    FDA aims to increase the availability of safe and effective 
pediatric devices while ensuring that the approval of these devices is 
based on valid scientific evidence. Extrapolation of adult data for 
pediatric use may benefit pediatric patients by making it possible for 
devices to be approved for pediatric-specific indications and labeling, 
even when there is little or no existing pediatric data. Extrapolation 
facilitates the use of available relevant data by making optimal use of 
what is already known about device effects in other

[[Page 26062]]

populations to support indications in the pediatric population. If 
extrapolation is found to be appropriate, FDA believes that statistical 
modeling and methods can be used to increase the precision of pediatric 
inferences.
    This guidance should be used in conjunction with other device-
specific guidances to help ensure that medical devices intended for use 
in pediatric population provide reasonable assurance of safety and 
effectiveness.

II. Significance of Guidance

    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the Agency's current thinking on 
extrapolation of data for pediatric uses. It does not create or confer 
any rights for or on any person and does not operate to bind FDA or the 
public. An alternative approach may be used if such approach satisfies 
the requirements of the applicable statute and regulations.

III. Electronic Access

    Persons interested in obtaining a copy of the draft guidance may do 
so by downloading an electronic copy from the Internet. A search 
capability for all Center for Devices and Radiological Health guidance 
documents is available at http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/default.htm. Guidance 
documents are also available at http://www.regulations.gov. Persons 
unable to download an electronic copy of ``Leveraging Existing Clinical 
Data for Extrapolation to Pediatric Uses of Medical Devices'' may send 
an email request to [email protected] to receive an electronic 
copy of the document. Please use the document number 1827 to identify 
the guidance you are requesting.

IV. Paperwork Reduction Act of 1995

    This draft guidance refers to previously approved collections of 
information found in FDA regulations. These collections of information 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The 
collections of information in 21 CFR parts 801 and 809 have been 
approved under OMB control number 0910-0485 (medical device labeling); 
the collections of information in 21 CFR part 812 have been approved 
under OMB control number 0910-0078 (investigational device exemptions); 
the collections of information in 21 CFR part 814 have been approved 
under OMB control number 0910-0231 (subparts A through E, premarket 
approval).

V. Reference

    The following reference have been placed on display in the Division 
of Dockets Management (see ADDRESSES), and may be seen by interested 
persons between 9 a.m. and 4 p.m., Monday through Friday, and are 
available electronically at http://www.regulations.gov. (FDA has 
verified the Web site address, but we are not responsible for any 
subsequent changes to the Web sites after this document publishes in 
the Federal Register.)

1. FDA guidance entitled ``Premarket Assessment of Pediatric Medical 
Devices,'' March 24, 2014, available at http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm089740.htm.

VI. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of comments. Identify comments with the docket number 
found in brackets in the heading of this document. Received comments 
may be seen in the Division of Dockets Management between 9 a.m. and 4 
p.m., Monday through Friday, and will be posted to the docket at http://www.regulations.gov.

    Dated: April 30, 2015.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2015-10482 Filed 5-5-15; 8:45 am]
BILLING CODE 4164-01-P