[Federal Register Volume 79, Number 236 (Tuesday, December 9, 2014)]
[Notices]
[Pages 73079-73080]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-28716]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-1275]


General Clinical Pharmacology Considerations for Pediatric 
Studies for Drugs and Biological Products; Draft Guidance for Industry; 
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availability of a draft guidance for industry entitled ``General 
Clinical Pharmacology Considerations for Pediatric Studies for Drugs 
and Biological Products.'' The draft guidance is intended to assist 
those sponsors of new drug applications (NDAs), biologics license 
applications (BLAs) for therapeutic biologics, and supplements to such 
applications who are planning to conduct clinical studies in pediatric 
populations. Effectiveness, safety, or dose finding studies in 
pediatric patients involve gathering clinical pharmacology information, 
such as information regarding a product's pharmacokinetics and 
pharmacodynamics pertaining to dose selection and individualization. 
This draft guidance addresses general clinical pharmacology 
considerations for conducting studies so that the dosing and safety 
information for drugs and biologic products can be sufficiently 
characterized, leading to well-designed trials to evaluate 
effectiveness.

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 on 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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by February 9, 2015.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10001 New 
Hampshire Ave., Hillandale Building, 4th Floor, Silver Spring, MD 
20993. Send one self-addressed adhesive label to assist those offices 
in processing your requests. See the SUPPLEMENTARY INFORMATION section 
for electronic access to the draft guidance document.
    Submit electronic comments on the draft guidance to http://www.regulations.gov.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.

FOR FURTHER INFORMATION CONTACT: Gilbert J. Burckart, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 3184, Silver Spring, MD 20993-0002, 301-
796-2065.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``General Clinical Pharmacology Considerations for Pediatric 
Studies for Drugs and Biological Products.'' During the past two 
decades, FDA has worked to address the problem of inadequate pediatric 
testing and inadequate pediatric use information in drug and biological 
product labeling. The Food and Drug Administration Modernization Act of 
1997 (Pub. L. 105-115) addressed the need for improved information 
about drug use in the pediatric population (codified at 21 U.S.C. 355a) 
by establishing incentives for conducting pediatric studies on drugs 
while exclusivity or patent protection exists. Congress subsequently 
passed the Best Pharmaceuticals for Children Act (BPCA) in 2002 and the 
Pediatric Research Equity Act (PREA) in 2003. Both BPCA and PREA were 
reauthorized in 2007 and were made permanent under Title V of the Food 
and Drug Administration Safety and Innovation Act of 2012 (Public Law 
112-144).
    Under BPCA, sponsors of certain applications and supplements filed 
under section 505 of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 355) (the FD&C Act) can obtain an additional 6 months of 
exclusivity if, in accordance with the requirements of the statute, the 
sponsor submits information responding to a written request from the 
Secretary relating to the use of a drug in the pediatric population.
    Under PREA, sponsors of certain applications and supplements filed 
under section 505 of the FD&C Act or section 351 of the Public Health 
Service Act are required to submit pediatric assessments, unless they 
receive an applicable waiver or deferral of this requirement. If 
applicable, sponsors must submit a request for a deferral or waiver as 
part of an initial pediatric study plan.
    This draft guidance focuses on the clinical pharmacology 
information (e.g., exposure-response, pharmacokinetics, and 
pharmacodynamics) needed to

[[Page 73080]]

support findings of effectiveness and safety and helps identify 
appropriate doses in pediatric populations. The draft guidance also 
describes the use of quantitative approaches (i.e., pharmacometrics) to 
employ disease and exposure-response knowledge from relevant prior 
clinical studies to design and evaluate future pediatric studies. The 
draft guidance does not describe: (1) Standards for approval of drugs 
and biological products in the pediatric population, (2) criteria to 
allow a determination that the course of a disease and the effects of a 
drug or a biologic are the same in adults and pediatric populations, or 
(3) clinical pharmacology studies for vaccine therapy, blood products, 
or other products not regulated by the Center for Drug Evaluation and 
Research.
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance 
represents the Agency's current thinking on the general clinical 
pharmacology considerations for pediatric studies for drugs and 
biological products. 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 
requirement of the applicable statutes and regulations.

II. Paperwork Reduction Act of 1995

    This draft guidance includes information collection provisions that 
are subject to review by the Office of Management and Budget (OMB) 
under the Paperwork Reduction Act (PRA) of 1995 (44 U.S.C. 3501-3520) 
(PRA). The collections of information referenced in this draft guidance 
that are related to the burden for the submission of investigational 
new drug applications are covered under 21 CFR part 312 and have been 
approved under OMB control number 0910-0014. The collections of 
information referenced in this draft guidance that are related to the 
burden for the submission of new drug applications are covered under 21 
CFR part 314 and have been approved under OMB control number 0910-0001. 
The submission of prescription drug product labeling under 21 CFR 
201.56 and 201.57 is approved under OMB control number 0910-0572.
    In accordance with the PRA, prior to publication of any final 
guidance document, FDA intends to solicit public comment and obtain OMB 
approval for any information collections recommended in this guidance 
that are new or that would represent material modifications to those 
previously approved collections of information found in FDA regulations 
or guidances.

III. 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.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at 
http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances or http://www.regulations.gov.

    Dated: December 2, 2014.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2014-28716 Filed 12-8-14; 8:45 am]
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