80_FR_26148 80 FR 26061 - Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability

80 FR 26061 - Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices; Draft Guidance for Industry and Food and Drug Administration Staff; Availability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

Page Range26061-26062
FR Document2015-10482

The Food and Drug Administration (FDA) is announcing the availability of the draft guidance entitled ``Leveraging Existing Clinical Data for Extrapolation to Pediatric Uses of Medical Devices.'' This draft guidance is being issued to explain the circumstances in which it may be appropriate to leverage existing clinical data to support pediatric device indications in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 The draft guidance also describes the approach that FDA would use to determine whether extrapolation is appropriate in medical devices, and the factors that would be considered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 This draft guidance is not final nor is it in effect at this time.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 Issue 87 (Wednesday, May 6, 2015) 
[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]


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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Uses of Medical Devices; Draft Guidance for Industry and Food and Drug 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.

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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availability of the draft guidance entitled ``Leveraging Existing 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This draft guidance is being issued to explain the circumstances in 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

Federal Register / Vol. 80, No. 87 / Wednesday, May 6, 2015 / Notices 26061 Draft guidance Docket No. Publication date 40. Recommended Warning for Surgeon’s Gloves and Patient Examination Gloves ........................ FDA–2011–D–0030 2/7/2011 41. Class II Special Controls Guidance Document: In Vitro Diagnostic Devices for Bacillus spp. FDA–2011–D–0102 5/18/2011 Detection. 42. Use of Antibiotic Resistance Marker Genes in Transgenic Plants ............................................... FDA–1998–N–0050 9/4/1998 43. Drugs, Biologics, and Medical Devices Derived from Bioengineered Plants for Use in Humans FDA–2002–D–0135 9/11/2002 and Animals. 44. Preliminary Timetable for the Review of Applications for Modified Risk Tobacco Products FDA–2009–D–0563 11/27/2009 under the Federal Food, Drug, and Cosmetic Act. 45. Guidance for Industry: Regulatory Procedures Manual—Chapter 9, Subchapter: Guidance FDA–1998–N–0050 11/5/2002 Concerning Recommending Customs’ Seizure and Destruction of Imported Human and Animal Food That has Not Been Reconditioned; Draft Guidance. 46. Submission of Laboratory Packages By Accredited Laboratories ................................................ FDA–2008–D–0510 1/2009 47. Guidance for the Public and FDA Staff on Convening Advisory Committee Meetings ................ FDA–2008–D–0417 8/1/2008 Dated: April 30, 2015. guidance before it begins work on the treatment (i.e., from birth through the Leslie Kux, final version of the guidance, submit 21st year of life, up to but not including Associate Commissioner for Policy. either electronic or written comments the 22d birthday). Pediatric [FR Doc. 2015–10477 Filed 5–5–15; 8:45 am] on the draft guidance by August 4, 2015. subpopulations are defined in section BILLING CODE 4164–01–P ADDRESSES: An electronic copy of the 520(m)(6)(E)(ii) (and adopted by guidance document is available for reference in section 515A(c) of the download from the Internet. See the FD&C Act (21 U.S.C. 360e)) to be DEPARTMENT OF HEALTH AND SUPPLEMENTARY INFORMATION section for neonates, infants, children, and HUMAN SERVICES information on electronic access to the adolescents. guidance. Submit written requests for a In an attempt to promote pediatric Food and Drug Administration single hard copy of the draft guidance medical device development, CDRH [Docket No. FDA–2015–D–1376] document entitled ‘‘Leveraging Existing published a final guidance document in Clinical Data for Extrapolation to 2004 entitled ‘‘Premarket Assessment of Leveraging Existing Clinical Data for Pediatric Uses of Medical Devices’’ to Pediatric Medical Devices’’ (Ref. 1). This Extrapolation to Pediatric Uses of the Office of the Center Director, 2004 document indicates that data can Medical Devices; Draft Guidance for Guidance and Policy Development, be extrapolated to support effectiveness Industry and Food and Drug Center for Devices and Radiological and, on a limited basis, safety for Administration Staff; Availability Health (CDRH), Food and Drug premarket approval applications (PMAs) AGENCY: Food and Drug Administration, Administration, 10903 New Hampshire when consistent with scientific HHS. Ave., Bldg. 66, Rm. 5431, Silver Spring, principles. Congress was aware of this MD 20993–0002. Send one self- 2004 document when it passed the Food ACTION: Notice. addressed adhesive label to assist that and Drug Administration Amendments SUMMARY: The Food and Drug office in processing your request. Act of 2007 (FDAAA). Title III of Administration (FDA) is announcing the Submit electronic comments on the FDAAA is the Pediatric Medical Device availability of the draft guidance draft guidance to http:// Safety and Improvement Act (PMDSIA). entitled ‘‘Leveraging Existing Clinical www.regulations.gov. Submit written The FDAAA specifically authorized the Data for Extrapolation to Pediatric Uses comments to the Division of Dockets use of adult data to demonstrate of Medical Devices.’’ This draft Management (HFA–305), Food and Drug pediatric effectiveness. While safety guidance is being issued to explain the Administration, 5630 Fishers Lane, Rm. exploration is not discussed in PMDSIA, circumstances in which it may be 1061, Rockville, MD 20852. Identify FDA believes that there are specific appropriate to leverage existing clinical comments with the docket number cases where it will be appropriate to data to support pediatric device found in brackets in the heading of this consider extrapolation of existing indications in premarket approval document. clinical safety data to support or applications (PMAs) and humanitarian FOR FURTHER INFORMATION CONTACT: enhance evidence for pediatric device exemptions (HDEs). The draft Jacqueline Francis, Center for Devices indications. FDA seeks comment on the guidance also describes the approach and Radiological Health, Food and Drug appropriateness of extrapolating from that FDA would use to determine Administration, 10903 New Hampshire adult clinical data to support medical whether extrapolation is appropriate in Ave., Bldg. 66, Silver Spring, MD device safety in pediatric patients. medical devices, and the factors that 20993–0002, 301–796–6405; or Stephen FDA aims to increase the availability would be considered within a statistical Ripley, Center for Biologics Evaluation of safe and effective pediatric devices model for extrapolation. Extrapolation and Research (CBER), Food and Drug while ensuring that the approval of may be appropriate when the course of Administration, 10903 New Hampshire these devices is based on valid scientific the disease or condition and the effects Ave., Bldg. 71, Rm. 7301, Silver Spring, evidence. Extrapolation of adult data for of the device are sufficiently similar in MD 20993–0002, 240–402–7911. pediatric use may benefit pediatric adults and pediatric patients and the SUPPLEMENTARY INFORMATION: patients by making it possible for tkelley on DSK3SPTVN1PROD with NOTICES adult data are of high quality for devices to be approved for pediatric- borrowing. This draft guidance is not I. Background specific indications and labeling, even final nor is it in effect at this time. Section 520(m)(6)(E)(i) of the Federal when there is little or no existing DATES: Although you can comment on Food, Drug, and Cosmetic Act (FD&C pediatric data. Extrapolation facilitates any guidance at any time (see 21 CFR Act) (21 U.S.C. 360j) defines pediatric the use of available relevant data by 10.115(g)(5)), to ensure that the Agency device patients as persons aged 21 or making optimal use of what is already considers your comment of this draft younger at the time of their diagnosis or known about device effects in other VerDate Sep<11>2014 18:43 May 05, 2015 Jkt 235001 PO 00000 Frm 00067 Fmt 4703 Sfmt 4703 E:\FR\FM\06MYN1.SGM 06MYN1

26062 Federal Register / Vol. 80, No. 87 / Wednesday, May 6, 2015 / Notices populations to support indications in number 0910–0231 (subparts A through drug development, and an the pediatric population. If E, premarket approval). ‘‘administrative’’ new animal drug extrapolation is found to be appropriate, application (NADA) or abbreviated new V. Reference FDA believes that statistical modeling animal drug application (ANADA), the and methods can be used to increase the The following reference have been content, the procedures a sponsor precision of pediatric inferences. placed on display in the Division of should follow to submit such an This guidance should be used in Dockets Management (see ADDRESSES), application, and the intended time conjunction with other device-specific and may be seen by interested persons frame for its review. guidances to help ensure that medical between 9 a.m. and 4 p.m., Monday DATES: Submit either electronic or devices intended for use in pediatric through Friday, and are available written comments on Agency guidances population provide reasonable electronically at http:// at any time. assurance of safety and effectiveness. www.regulations.gov. (FDA has verified the Web site address, but we are not ADDRESSES: Submit written requests for II. Significance of Guidance single copies of the guidance to the responsible for any subsequent changes This draft guidance is being issued to the Web sites after this document Policy and Regulations Staff (HFV–6), consistent with FDA’s good guidance publishes in the Federal Register.) Center for Veterinary Medicine, Food practices regulation (21 CFR 10.115). 1. FDA guidance entitled ‘‘Premarket and Drug Administration, 7519 Standish The draft guidance, when finalized, will Assessment of Pediatric Medical Pl., Rockville, MD 20855. Send one self- represent the Agency’s current thinking Devices,’’ March 24, 2014, available at addressed adhesive label to assist that on extrapolation of data for pediatric http://www.fda.gov/MedicalDevices/ office in processing your request. See uses. It does not create or confer any DeviceRegulationandGuidance/ the SUPPLEMENTARY INFORMATION section rights for or on any person and does not GuidanceDocuments/ucm089740.htm. for electronic access to the guidance operate to bind FDA or the public. An document. alternative approach may be used if VI. Comments Submit electronic comments on the such approach satisfies the Interested persons may submit either guidance to http://www.regulations.gov. requirements of the applicable statute electronic comments regarding this Submit written comments to the and regulations. document to http://www.regulations.gov Division of Dockets Management (HFA– or written comments to the Division of 305), Food and Drug Administration, III. Electronic Access Dockets Management (see ADDRESSES). It 5630 Fishers Lane, Rm. 1061, Rockville, Persons interested in obtaining a copy is only necessary to send one set of MD 20852. of the draft guidance may do so by comments. Identify comments with the FOR FURTHER INFORMATION CONTACT: downloading an electronic copy from docket number found in brackets in the Katherine Weld, Center for Veterinary the Internet. A search capability for all heading of this document. Received Medicine (HFV–108), Food and Drug Center for Devices and Radiological comments may be seen in the Division Administration, 7500 Standish Pl., Health guidance documents is available of Dockets Management between 9 a.m. Rockville, MD 20855, 240–402–0846, at http://www.fda.gov/MedicalDevices/ and 4 p.m., Monday through Friday, and Katherine.Weld@fda.hhs.gov. DeviceRegulationandGuidance/ will be posted to the docket at http:// GuidanceDocuments/default.htm. www.regulations.gov. SUPPLEMENTARY INFORMATION: Guidance documents are also available Dated: April 30, 2015. I. Background at http://www.regulations.gov. Persons Leslie Kux, In the Federal Register of November unable to download an electronic copy of ‘‘Leveraging Existing Clinical Data for Associate Commissioner for Policy. 6, 2002 (67 FR 67631), FDA published Extrapolation to Pediatric Uses of [FR Doc. 2015–10482 Filed 5–5–15; 8:45 am] the notice of availability for a draft Medical Devices’’ may send an email BILLING CODE 4164–01–P guidance entitled ‘‘The Administrative request to CDRH-Guidance@fda.hhs.gov New Animal Drug Application Process’’ to receive an electronic copy of the giving interested persons until January document. Please use the document DEPARTMENT OF HEALTH AND 21, 2003, to comment on the draft number 1827 to identify the guidance HUMAN SERVICES guidance. FDA received several you are requesting. comments on the draft guidance and Food and Drug Administration those comments were considered as the IV. Paperwork Reduction Act of 1995 [Docket No. FDA–2002–D–0147 (formerly guidance was finalized. The guidance This draft guidance refers to Docket No. 2002D–0449)] was updated to clarify current processes previously approved collections of and include information about generic information found in FDA regulations. Administrative Applications and the new animal drugs. The guidance These collections of information are Phased Review Process; Guidance for announced in this notice finalizes the subject to review by the Office of Industry; Availability draft guidance dated November 6, 2002. Management and Budget (OMB) under AGENCY: Food and Drug Administration, To be legally marketed, a new animal the Paperwork Reduction Act of 1995 HHS. drug must be the subject of either an (44 U.S.C. 3501–3520). The collections ACTION: Notice. approved application under section of information in 21 CFR parts 801 and 512(b) of the Federal Food, Drug, and 809 have been approved under OMB SUMMARY: The Food and Drug Cosmetic Act (the FD&C Act) (21 U.S.C. control number 0910–0485 (medical Administration (FDA) is announcing the 360b), a conditional approval under tkelley on DSK3SPTVN1PROD with NOTICES device labeling); the collections of availability of a guidance for industry section 571 of the FD&C Act (21 U.S.C. information in 21 CFR part 812 have (GFI) #132 entitled ‘‘Administrative 360ccc), or an index listing under been approved under OMB control Applications and the Phased Review section 572 of the FD&C Act (21 U.S.C. number 0910–0078 (investigational Process.’’ This guidance defines the 360ccc–1). Sections 512(b)(1) and device exemptions); the collections of ‘‘phased review process’’ for reviewing 512(n)(1) of the FD&C Act describes the information in 21 CFR part 814 have application-level information during the information that must be submitted to been approved under OMB control investigational period of new animal FDA, specifically the Center for VerDate Sep<11>2014 18:43 May 05, 2015 Jkt 235001 PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 E:\FR\FM\06MYN1.SGM 06MYN1


Document Created: 2015-12-16 07:41:27
Document Modified: 2015-12-16 07:41:27
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice.
DatesAlthough 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.
ContactJacqueline 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.
FR Citation80 FR 26061 
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