[Federal Register Volume 82, Number 234 (Thursday, December 7, 2017)]
[Notices]
[Pages 57759-57760]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-26357]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-6476]


Pediatric Rare Diseases--A Collaborative Approach for Drug 
Development Using Gaucher Disease as a Model; Draft Guidance for 
Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Pediatric 
Rare Diseases--A Collaborative Approach for Drug Development Using 
Gaucher Disease as a Model.'' This draft guidance focuses on drug 
development for pediatric patients with Gaucher disease. In particular, 
it proposes for consideration a novel approach to improve the 
efficiency of drug development in pediatric rare diseases using Gaucher 
disease as an example. The emergence of concomitant trials for multiple 
investigational drug products for the treatment of rare diseases can 
pose significant challenges to effective drug development, because 
there are limited numbers of patients for any given rare condition 
worldwide. This approach discusses the feasibility of the development 
of multiple drug products in a time-efficient manner while minimizing 
the number of patients necessary to be treated with placebo.

DATES: Submit either electronic or written comments on the draft 
guidance by February 5, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on https://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2017-N-6476 for ``Pediatric Rare Diseases--A Collaborative Approach 
for Drug Development Using Gaucher Disease as a Model; Draft Guidance 
for Industry; Availability''. Received comments will be placed in the 
docket and, except for those submitted as ``Confidential Submissions,'' 
publicly viewable at https://www.regulations.gov or at the Dockets 
Management Staff office between 9 a.m. and 4 p.m., Monday through 
Friday.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information

[[Page 57760]]

redacted/blacked out, will be available for public viewing and posted 
on https://www.regulations.gov. Submit both copies to the Dockets 
Management Staff. If you do not wish your name and contact information 
to be made publicly available, you can provide this information on the 
cover sheet and not in the body of your comments and you must identify 
this information as ``confidential.'' Any information marked as 
``confidential'' will not be disclosed except in accordance with 21 CFR 
10.20 and other applicable disclosure law. For more information about 
FDA's posting of comments to public dockets, see 80 FR 56469, September 
18, 2015, or access the information at: https://www.gpo.gov/fdsys/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    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 20993-0002. Send one 
self-addressed adhesive label to assist that office in processing your 
requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Hong Vu, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 22, Rm. 5345, Silver Spring, MD 20993-0002, 301-796-7401.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Pediatric Rare Diseases--A Collaborative Approach for Drug 
Development Using Gaucher Disease as a Model.'' The emergence of 
concomitant trials for multiple investigational drug products for the 
treatment of rare disease can pose significant challenges to effective 
drug development, given the limited number of patients worldwide with 
these diagnoses. This guidance discusses, among other things, a multi-
arm, multi-company clinical trial as a novel approach to enhance the 
efficiency of drug development in pediatric rare diseases using 
pediatric Gaucher disease as an example. The proposal applies only to 
systemic (i.e., non-neurological) manifestations of Gaucher disease 
(i.e., patients with Type I and Type III phenotypes).
    The purpose of this guidance is to facilitate drug development in 
pediatric rare diseases, with a focus on Gaucher disease. In this 
guidance, Gaucher disease is provided as a disease model. However, the 
principles underlying this proposal may be extended to other areas of 
drug development in rare diseases. The guidance was originally a 
document developed as a strategic collaboration between FDA and the 
European Medicines Agency to enhance the efficiency of drug development 
in Gaucher disease, which was released in 2014 for public comment. The 
draft guidance is an updated version of the document and has no 
fundamental changes to the original intent and content.
    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 current thinking of FDA on ``Pediatric 
Rare Diseases--A Collaborative Approach for Drug Development Using 
Gaucher Disease as a Model.'' It does not establish any rights for any 
person and is not binding on FDA or the public. You can use an 
alternative approach if it satisfies the requirements of the applicable 
statutes and regulations. This guidance is not subject to Executive 
Order 12866.

II. The 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 312 and 314 have been 
approved under OMB control numbers 0910-0014 and 0910-0001, 
respectively. The collections of information in 21 CFR 201.57 for the 
content and format of prescription drug labeling was approved under OMB 
control number 0910-0572.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or 
https://www.regulations.gov.

    Dated: December 1, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-26357 Filed 12-6-17; 8:45 am]
 BILLING CODE 4164-01-P


