[Federal Register Volume 70, Number 172 (Wednesday, September 7, 2005)]
[Notices]
[Pages 53233-53235]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 05-17694]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. 2005D-0334]


Draft Guidance for Industry on the Pediatric Research Equity Act; 
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availability of a draft guidance for industry entitled ``How to Comply 
with the Pediatric Research Equity Act.'' This draft guidance provides 
recommendations on how to interpret the requirements of the Pediatric 
Research Equity Act (PREA), which requires pediatric studies of certain 
drugs and biological products to ensure that those products that are 
likely to be commonly used in children or that represent a meaningful 
therapeutic benefit over existing treatments contain adequate pediatric 
labeling for approved indications.

DATES: Submit written or electronic comments on the draft guidance by 
November 7, 2005. General comments on agency guidance documents are 
welcome at any time.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information (HFD-240), Center for Drug 
Evaluation and Research, Food and Drug Administration, 5600 Fishers 
Lane, Rockville, MD 20857; or to the Office of Communication, Training, 
and Manufacturers Assistance (HFM-40), Center for Biologics Evaluation 
and Research, Food and Drug Administration, 1401 Rockville Pike, 
Rockville, MD 20852-1448. Send one self-addressed adhesive label to 
assist that office in processing your requests. The draft guidance may 
also be obtained by mail by calling CBER at 1-800-835-4709 or 301-827-
1800. Submit written comments on the draft guidance to the Division of 
Dockets Management (HFA-305), Food and Drug Administration, 5630 
Fishers Lane, rm. 1061, Rockville, MD 20852. Submit electronic comments 
to http://www.fda.gov/dockets/ecomments. See the SUPPLEMENTARY 
INFORMATION section for electronic access to the draft guidance 
document.

FOR FURTHER INFORMATION CONTACT: 
     Grace Carmouze, Center for Drug Evaluation and Research (HFD-950), 
Food and Drug Administration,5600 Fishers Lane, Rockville, MD 20857, 
301-594-2041, or
    Leonard Wilson, Center for Biologics Evaluation and Research (HFM-
25), Food and Drug Administration, 1401 Rockville Pike, suite 200N 
Rockville, MD 20852-1448, 301-827-0373.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``How to Comply with the Pediatric Research Equity Act.'' On 
December 3, 2003, the Pediatric Research Equity Act was signed into 
law. PREA amends the Federal Food, Drug, and Cosmetic Act (the act) by 
adding section 505B (21 U.S.C. 355B). In PREA, Congress codified many 
of the elements of the Pediatric Rule, a final rule issued by FDA on 
December 2, 1998 (63 FR 66632), and suspended by court order on October 
17, 2002. Association of American Physicians, and Surgeons, Inc. v. 
FDA, 226 F. Supp. 2d 204 (D.D.C. 2002). Specifically, PREA, in adding 
section 505B(a) of the act, requires all applications (or supplements 
to an application) submitted under section 505 of the act (21 U.S.C. 
355) or section 351 of the Public Health Service Act (PHS Act) (42 
U.S.C. 262) for a new active ingredient, new indication, new dosage 
form, new dosing regimen, or

[[Page 53234]]

new route of administration to contain a pediatric assessment unless 
the applicant has obtained a waiver or deferral. PREA also authorizes 
FDA, under section 505B(b) of the act, to require holders of previously 
approved applications for marketed drugs and biological products to 
conduct pediatric studies under certain circumstances, even if the 
holders are not seeking one of the changes listed under section 505B(a) 
of the act. This draft guidance only provides recommendations related 
to studies required under section 505B(a) of the act.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 how to comply with PREA. 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 statutes 
and regulations.

II. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic comments on the draft guidance. 
Submit a single copy of electronic comments or two paper copies of any 
mailed comments, except that individuals may submit one paper copy. 
Comments are to be identified with the docket number found in brackets 
in the heading of this document. The draft guidance and received 
comments are available for public examination in the Division of 
Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.

III. Paperwork Reduction Act of 1995

    Under the Paperwork Reduction Act (44 U.S.C. 3501-3520) (the PRA), 
Federal agencies must obtain approval from the Office of Management and 
Budget (OMB) for each collection of information they conduct or 
sponsor. ``Collection of information'' is defined in 44 U.S.C. 3502(3) 
and 5 CFR 1320.3(c) and includes agency requests or requirements that 
members of the public submit reports, keep records, or provide 
information to a third party. Section 3506(c)(2)(A) of the PRA (44 
U.S.C. 3506(c)(2)(A)) requires Federal agencies to provide a 60-day 
notice in the Federal Register concerning each proposed collection of 
information before submitting the collection to OMB for approval. To 
comply with this requirement, FDA is publishing notice of the proposed 
collection of information set forth in this document.
    With respect to the following collection of information, FDA 
invites comment on: (1) Whether the proposed collection of information 
is necessary for the proper performance of FDA's functions, including 
whether the information will have practical utility; (2) the accuracy 
of FDA's estimate of the burden of the proposed collection of 
information, including the validity of the methodology and assumptions 
used; (3) ways to enhance the quality, utility, and clarity of the 
information to be collected; and (4) ways to minimize the burden of the 
collection on respondents, including through the use of automated 
collection techniques, when appropriate, and other forms of information 
technology.
    Title: Draft Guidance for Industry: How to Comply with the 
Pediatric Research Equity Act.
    Description: The draft guidance provides recommendations to 
sponsors on how to interpret the requirements of PREA. PREA requires 
new drug applications (NDAs) and biologics licensing applications 
(BLAs) (or supplements to an applications) for a new active ingredient, 
new indication, new dosage form, new dosing regimen, or new route of 
administration to contain a pediatric assessment unless the applicant 
has obtained a waiver or deferral. Although PREA applies to both new 
applications (or supplements to an application) and currently marketed 
drugs and biological products for which a sponsor is not seeking one of 
the enumerated changes, the guidance only provides recommendations 
related to new applications or supplements to applications for drugs 
and biological products.
    Description of Respondents: Sponsors of NDAs or BLAs for human 
drugs and biological products.
    Burden Estimate: FDA is requesting public comments on estimates of 
annual submissions expected in 2005 (based on the number of submissions 
received in 2003 and 2004 unless otherwise indicated) as required by 
the following PREA requirements described in the draft guidance:
    Section 505B(a)(1) and (a)(2)--The draft guidance provides 
recommendations for submitting pediatric studies with applications (or 
supplements to an application) for a new active ingredient, new 
indication, new dosage form, new dosing regimen, or new route of 
administration under section 505 of the act or section 351 of the PHS 
Act. These assessments are required to contain data that are adequate 
to assess the safety and effectiveness of the drug product for the 
claimed indications in the relevant pediatric subpopulations and to 
support dosing and administration for each subpopulation for which the 
product is safe and effective. FDA estimates that 106 pediatric use 
assessments will be submitted from 78 applicants and it will take 50 
hours to prepare each assessment.
    Section 505B(a)(3)--The draft guidance makes recommendations on how 
to request a deferral of some or all assessments of safety and 
effectiveness required under PREA. FDA estimates that it will receive 
160 requests to defer assessments from 54 applicants and it will take 
24 hours to prepare each request.
    Section 505B(a)(4)--The draft guidance provides recommendations on 
how to request a full or partial waiver of the pediatric study 
requirements. Based on its 2003 and 2004 experience, FDA anticipates 
that it will receive approximately 110 requests annually from 
approximately 80 applicants and estimates it will take approximately 8 
hours to prepare each request.
    Section 505B(e)--The draft guidance makes recommendations for 
applicants to meet at appropriate times with FDA to discuss plans and 
timelines for pediatric studies and any planned requests for deferral 
or waiver of pediatric studies. FDA estimates it will receive 160 
submissions associated with meetings to discuss pediatric plans from 95 
applicants at 16 hours per meeting submission.
    FDA estimates that the collection of information resulting from 
this draft guidance is as follows:

                                   Table 1.--Estimated Annual Reporting Burden
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     PREA                                  Number of Responses     Total Annual    Hours per
  Provision      Number of Respondents       per Respondent         Responses      Response       Total Hours
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505B(a)(1)                       78                        1.4           106            50              5,300
 and (a)(2)
 Submission
 of pediatric
 assessments
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[[Page 53235]]

 
505B(a)(3)                       54                        3.0           160            24              3,840
 Deferrals
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505B(a)(4)                       80                        1.4           110             8                880
 Full and
 partial
 waivers
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505B(e)                          95                        1.7           160            16              2,560
 Meetings
----------------------------------------------------------------------------------------------------------------
Total          .......................  .......................  ..............  ............          12,580
----------------------------------------------------------------------------------------------------------------

    In addition, the draft guidance discusses when sponsors may need to 
report on the status of postmarketing study commitments as part of 
annual reports submitted under 21 CFR 314.81(b) and 21 CFR 601.70. The 
burdens associated with the annual reporting requirements were 
previously accounted for under OMB number 0910-0001 (expires 5/31/08) 
(for 21 CFR 314.81(b) and OMB number 0910-0433 (expires 3/31/07) (for 
21 CFR 601.70). Furthermore, although labeling submissions are required 
under certain PREA provisions (e.g., section 505B(a)(4)(D) of the act), 
the draft guidance does not provide recommendations on these 
requirements and therefore FDA has not estimated associated burdens.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at 
http://www.fda.gov/cder/guidance/index.htm, http://www.fda.gov/cber/guidelines.htm, or http://www.fda.gov/ohrms/dockets/default.htm.

    Dated: August 29, 2005.
Jeffrey Shuren,
Assistant Commissioner for Policy.
[FR Doc. 05-17694 Filed 9-6-05; 8:45 am]
BILLING CODE 4160-01-S