82 FR 57759 - Pediatric Rare Diseases-A Collaborative Approach for Drug Development Using Gaucher Disease as a Model; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 82, Issue 234 (December 7, 2017)
Food and Drug Administration
Federal Register Volume 82, Issue 234 (December 7, 2017)
| Page Range | 57759-57760 | |
| FR Document | 2017-26357 |
[Federal Register Volume 82, Number 234 (Thursday, December 7, 2017)] [Notices] [Pages 57759-57760] From the Federal Register Online [www.thefederalregister.org] [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Pediatric Rare Diseases--A Collaborative Approach for Drug Development Using Gaucher Disease as a Model; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS.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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Pediatric Rare Diseases--A Collaborative Approach for Drug Development Using Gaucher Disease as a Model.'' This draft guidance focuses on drug development for pediatric patients with Gaucher disease.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 DATES: Submit either electronic or written comments on the draft guidance by February 5, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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.thefederalregister.org/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
![Federal Register / Vol. 82, No. 234 / Thursday, December 7, 2017 / Notices 57759
elementary and secondary students. components of State Human Service Respondents: The 52 respondents
Respondents include various agencies. include the 50 States, the District of
Columbia, and Puerto Rico.
ANNUAL BURDEN ESTIMATES
Number of Average
Number of Total burden
Instrument responses per burden hours
respondents hours
respondent per response
Annual Statistical Report on Children in Foster Homes and Children Receiv-
ing Payments in Excess of the Poverty Level From a State Program
Funded Under Part A of Title IV of the Social Security Act ........................ 52 1 264.35 13,746.20
Estimated Total Annual Burden Rare Diseases—A Collaborative comments, that information will be
Hours: 13,746.20. Approach for Drug Development Using posted on https://www.regulations.gov.
Additional Information: Copies of the Gaucher Disease as a Model.’’ This draft • If you want to submit a comment
proposed collection may be obtained by guidance focuses on drug development with confidential information that you
writing to the Administration for for pediatric patients with Gaucher do not wish to be made available to the
Children and Families, Office of disease. In particular, it proposes for public, submit the comment as a
Planning, Research and Evaluation, 330 consideration a novel approach to written/paper submission and in the
C Street SW., Washington, DC 20201. improve the efficiency of drug manner detailed (see ‘‘Written/Paper
Attention Reports Clearance Officer. All development in pediatric rare diseases Submissions’’ and ‘‘Instructions’’).
requests should be identified by the title using Gaucher disease as an example. Written/Paper Submissions
of the information collection. Email The emergence of concomitant trials for
address: infocollection@acf.hhs.gov. multiple investigational drug products Submit written/paper submissions as
OMB Comment: OMB is required to for the treatment of rare diseases can follows:
make a decision concerning the pose significant challenges to effective • Mail/Hand delivery/Courier (for
collection of information between 30 written/paper submissions): Dockets
drug development, because there are
and 60 days after publication of this Management Staff (HFA–305), Food and
limited numbers of patients for any
document in the Federal Register. Drug Administration, 5630 Fishers
given rare condition worldwide. This
Therefore, a comment is best assured of Lane, Rm. 1061, Rockville, MD 20852.
approach discusses the feasibility of the • For written/paper comments
having its full effect if OMB receives it development of multiple drug products submitted to the Dockets Management
within 30 days of publication. Written in a time-efficient manner while Staff, FDA will post your comment, as
comments and recommendations for the minimizing the number of patients well as any attachments, except for
proposed information collection should necessary to be treated with placebo. information submitted, marked and
be sent directly to the following: Office identified, as confidential, if submitted
DATES: Submit either electronic or
of Management and Budget, Paperwork as detailed in ‘‘Instructions.’’
written comments on the draft guidance
Reduction Project, Email: OIRA_ Instructions: All submissions received
by February 5, 2018 to ensure that the
SUBMISSION@OMB.EOP.GOV, Attn: must include the Docket No. FDA–
Agency considers your comment on this
Desk Officer for the Administration for 2017–N–6476 for ‘‘Pediatric Rare
draft guidance before it begins work on
Children and Families. Diseases—A Collaborative Approach for
the final version of the guidance.
Robert Sargis, Drug Development Using Gaucher
ADDRESSES: You may submit comments Disease as a Model; Draft Guidance for
Reports Clearance Officer. on any guidance at any time as follows:
[FR Doc. 2017–26353 Filed 12–6–17; 8:45 am]
Industry; Availability’’. Received
Electronic Submissions comments will be placed in the docket
BILLING CODE 4184–01–P
and, except for those submitted as
Submit electronic comments in the ‘‘Confidential Submissions,’’ publicly
following way: viewable at https://www.regulations.gov
DEPARTMENT OF HEALTH AND
• Federal eRulemaking Portal: or at the Dockets Management Staff
HUMAN SERVICES
https://www.regulations.gov. Follow the office between 9 a.m. and 4 p.m.,
Food and Drug Administration instructions for submitting comments. Monday through Friday.
Comments submitted electronically, • Confidential Submissions—To
[Docket No. FDA–2017–N–6476] including attachments, to https:// submit a comment with confidential
www.regulations.gov will be posted to information that you do not wish to be
Pediatric Rare Diseases—A
the docket unchanged. Because your made publicly available, submit your
Collaborative Approach for Drug
comment will be made public, you are comments only as a written/paper
Development Using Gaucher Disease
solely responsible for ensuring that your submission. You should submit two
as a Model; Draft Guidance for
comment does not include any copies total. One copy will include the
Industry; Availability
confidential information that you or a information you claim to be confidential
third party may not wish to be posted, with a heading or cover note that states
sradovich on DSK3GMQ082PROD with NOTICES
AGENCY: Food and Drug Administration,
HHS. such as medical information, your or ‘‘THIS DOCUMENT CONTAINS
ACTION: Notice of availability. anyone else’s Social Security number, or CONFIDENTIAL INFORMATION.’’ The
confidential business information, such Agency will review this copy, including
SUMMARY: The Food and Drug as a manufacturing process. Please note the claimed confidential information, in
Administration (FDA or Agency) is that if you include your name, contact its consideration of comments. The
announcing the availability of a draft information, or other information that second copy, which will have the
guidance for industry entitled ‘‘Pediatric identifies you in the body of your claimed confidential information
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![57760 Federal Register / Vol. 82, No. 234 / Thursday, December 7, 2017 / Notices
redacted/blacked out, will be available among other things, a multi-arm, multi- Guidances/default.htm or https://
for public viewing and posted on company clinical trial as a novel www.regulations.gov.
https://www.regulations.gov. Submit approach to enhance the efficiency of Dated: December 1, 2017.
both copies to the Dockets Management drug development in pediatric rare Leslie Kux,
Staff. If you do not wish your name and diseases using pediatric Gaucher disease
Associate Commissioner for Policy.
contact information to be made publicly as an example. The proposal applies
only to systemic (i.e., non-neurological) [FR Doc. 2017–26357 Filed 12–6–17; 8:45 am]
available, you can provide this
information on the cover sheet and not manifestations of Gaucher disease (i.e., BILLING CODE 4164–01–P
in the body of your comments and you patients with Type I and Type III
must identify this information as phenotypes).
The purpose of this guidance is to DEPARTMENT OF HEALTH AND
‘‘confidential.’’ Any information marked
facilitate drug development in pediatric HUMAN SERVICES
as ‘‘confidential’’ will not be disclosed
except in accordance with 21 CFR 10.20 rare diseases, with a focus on Gaucher
Food and Drug Administration
and other applicable disclosure law. For disease. In this guidance, Gaucher
more information about FDA’s posting disease is provided as a disease model. [Docket No. FDA–2013–N–1161]
of comments to public dockets, see 80 However, the principles underlying this
FR 56469, September 18, 2015, or access proposal may be extended to other areas Agency Information Collection
the information at: https://www.gpo.gov/ of drug development in rare diseases. Activities; Submission for Office of
fdsys/pkg/FR-2015-09-18/pdf/2015- The guidance was originally a document Management and Budget Review;
23389.pdf. developed as a strategic collaboration Comment Request; Food Safety
Docket: For access to the docket to between FDA and the European Survey
read background documents or the Medicines Agency to enhance the AGENCY: Food and Drug Administration,
electronic and written/paper comments efficiency of drug development in HHS.
received, go to https:// Gaucher disease, which was released in
ACTION: Notice.
www.regulations.gov and insert the 2014 for public comment. The draft
docket number, found in brackets in the guidance is an updated version of the SUMMARY: The Food and Drug
heading of this document, into the document and has no fundamental Administration (FDA, Agency, or we) is
‘‘Search’’ box and follow the prompts changes to the original intent and announcing that a proposed collection
and/or go to the Dockets Management content. of information has been submitted to the
Staff, 5630 Fishers Lane, Rm. 1061, This draft guidance is being issued Office of Management and Budget
Rockville, MD 20852. consistent with FDA’s good guidance (OMB) for review and clearance under
You may submit comments on any practices regulation (21 CFR 10.115). the Paperwork Reduction Act of 1995.
guidance at any time (see 21 CFR The draft guidance, when finalized, will DATES: Fax written comments on the
10.115(g)(5)). represent the current thinking of FDA collection of information by January 8,
Submit written requests for single on ‘‘Pediatric Rare Diseases—A 2018.
copies of the draft guidance to the Collaborative Approach for Drug
ADDRESSES: To ensure that comments on
Division of Drug Information, Center for Development Using Gaucher Disease as
a Model.’’ It does not establish any the information collection are received,
Drug Evaluation and Research, Food OMB recommends that written
and Drug Administration, 10001 New rights for any person and is not binding
on FDA or the public. You can use an comments be faxed to the Office of
Hampshire Ave., Hillandale Building, Information and Regulatory Affairs,
4th Floor, Silver Spring, MD 20993– alternative approach if it satisfies the
requirements of the applicable statutes OMB, Attn: FDA Desk Officer, Fax: 202–
0002. Send one self-addressed adhesive 395–7285, or emailed to oira_
label to assist that office in processing and regulations. This guidance is not
subject to Executive Order 12866. submission@omb.eop.gov. All
your requests. See the SUPPLEMENTARY comments should be identified with the
INFORMATION section for electronic II. The Paperwork Reduction Act of OMB control number 0910–0345. Also
access to the draft guidance document. 1995 include the FDA docket number found
FOR FURTHER INFORMATION CONTACT: This draft guidance refers to in brackets in the heading of this
Hong Vu, Center for Drug Evaluation previously approved collections of document.
and Research, Food and Drug information found in FDA regulations. FOR FURTHER INFORMATION CONTACT: Ila
Administration, 10903 New Hampshire These collections of information are S. Mizrachi, Office of Operations, Food
Ave., Bldg. 22, Rm. 5345, Silver Spring, subject to review by the Office of and Drug Administration, Three White
MD 20993–0002, 301–796–7401. Management and Budget (OMB) under Flint North, 10A–12M, 11601
SUPPLEMENTARY INFORMATION: the Paperwork Reduction Act of 1995 Landsdown St., North Bethesda, MD
I. Background (44 U.S.C. 3501–3520). The collections 20852, 301–796–7726, PRAStaff@
of information in 21 CFR parts 312 and fda.hhs.gov.
FDA is announcing the availability of 314 have been approved under OMB
a draft guidance for industry entitled SUPPLEMENTARY INFORMATION: In
control numbers 0910–0014 and 0910–
‘‘Pediatric Rare Diseases—A compliance with 44 U.S.C. 3507, FDA
0001, respectively. The collections of
Collaborative Approach for Drug has submitted the following proposed
information in 21 CFR 201.57 for the
Development Using Gaucher Disease as collection of information to OMB for
content and format of prescription drug
a Model.’’ The emergence of review and clearance.
sradovich on DSK3GMQ082PROD with NOTICES
labeling was approved under OMB
concomitant trials for multiple control number 0910–0572. Food Safety Survey
investigational drug products for the
treatment of rare disease can pose III. Electronic Access OMB Control Number 0910–0345—
significant challenges to effective drug Persons with access to the internet Extension
development, given the limited number may obtain the draft guidance at either Under section 1003(b)(2) of the
of patients worldwide with these https://www.fda.gov/Drugs/Guidance Federal Food, Drug, and Cosmetic Act
diagnoses. This guidance discusses, ComplianceRegulatoryInformation/ (21 U.S.C. 393(b)(2)), we are authorized
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Document Created: 2017-12-07 00:34:50
Document Modified: 2017-12-07 00:34:50
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of availability. | |
| 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. | |
| 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. | |
| FR Citation | 82 FR 57759 |
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