[Federal Register Volume 83, Number 200 (Tuesday, October 16, 2018)]
[Notices]
[Pages 52223-52224]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-22435]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-3268]


Rare Diseases: Early Drug Development and the Role of Pre-
Investigational New Drug Application Meetings; Draft Guidance for 
Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Rare 
Diseases: Early Drug Development and the Role of Pre-Investigational 
New Drug Application Meetings.'' The purpose of this draft guidance is 
to assist sponsors of drug and biological products for the treatment of 
rare diseases in planning and conducting more efficient and productive 
pre-investigational new drug application (pre-IND) meetings. Drug 
development for rare diseases has many challenges related to the nature 
of these diseases. This draft guidance is intended to advance and 
facilitate the development of drugs and biological products for the 
treatment of rare diseases.

DATES: Submit either electronic or written comments on the draft 
guidance by December 17, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-2018-D-3268 for ``Rare Diseases: Early Drug Development and the 
Role of Pre-Investigational New Drug Application Meetings; Draft 
Guidance for Industry.'' 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 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

[[Page 52224]]

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 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, or Office 
of Communication, Outreach, and Development, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, 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: Lucas Kempf, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6460, Silver Spring, MD 20993-0002, 301-
796-1140; or Stephen Ripley, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Rare Diseases: Early Drug Development and the Role of Pre-
Investigational New Drug Application Meetings.'' This guidance is 
intended to assist sponsors of drug and biological products for the 
treatment of rare diseases in planning and conducting more efficient 
and productive pre-IND meetings through a discussion of selected issues 
commonly encountered in the early phases of rare disease drug 
development. Although these issues are encountered in other drug 
development programs, the issues are frequently more difficult to 
address in the context of a rare disease than in the context of a 
common disease, of which there is often greater and more widespread 
medical experience. A rare disease is defined in section 526(a)(2) of 
the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(2)) as a 
disease or condition that affects fewer than 200,000 people in the 
United States or affects more than 200,000 in the United States and for 
which there is no reasonable expectation that the cost of developing 
and making available in the United States a drug for such disease or 
condition will be recovered from sales in the United States of such 
drug. Most rare diseases affect far fewer than 200,000 people.
    Most rare disorders are serious conditions with no approved 
treatments, and rare disease patients often have considerable unmet 
medical needs. Collectively, rare diseases are highly diverse. FDA is 
committed to helping sponsors of drugs for rare diseases have 
successful pre-IND meetings that address the particular challenges 
posed by each drug.
    This guidance addresses the following important topics related to 
pre-IND meetings:
     Regulatory considerations across various FDA disciplines 
including chemistry, manufacturing, and controls; nonclinical; clinical 
pharmacology; and clinical.
     Additional considerations, including expedited programs 
for serious conditions, companion diagnostics, orphan drug incentives, 
pediatric studies, and data standards.
    Early consideration of these issues allows sponsors to efficiently 
and adequately plan for a productive pre-IND meeting with FDA.
    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 ``Rare 
Diseases: Early Drug Development and the Role of Pre-Investigational 
New Drug Application Meetings.'' 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 guidance refers to previously approved collections of 
information that 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 collection of information resulting from the draft 
guidance for industry ``Formal Meetings Between the FDA and Sponsors or 
Applicants of PDUFA Products'' (available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm590547.pdf) has been approved under OMB control number 0910-0429. 
The collection of information resulting from the guidance for industry 
``Expedited Programs for Serious Conditions--Drugs and Biologics'' 
(available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm358301.pdf) has been approved under OMB 
control number 0910-0765.

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, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or https://www.regulations.gov.

    Dated: October 10, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-22435 Filed 10-15-18; 8:45 am]
 BILLING CODE 4164-01-P