[Federal Register Volume 83, Number 156 (Monday, August 13, 2018)]
[Notices]
[Pages 40055-40057]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-17273]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2777]


Expansion Cohorts: Use in First-In-Human Clinical Trials To 
Expedite Development of Oncology Drugs and Biologics; Draft Guidance 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the availability of a draft guidance for industry entitled ``Expansion 
Cohorts: Use in First-In-Human Clinical Trials to Expedite Development 
of Oncology Drugs and Biologics.'' The purpose of this draft guidance 
is to provide advice to sponsors regarding the design and conduct of 
first-in-human (FIH) clinical trials intended to efficiently expedite 
the clinical development of cancer drugs, including biological 
products, through multiple expansion cohort study designs.

DATES: Submit either electronic or written comments on the draft 
guidance by October 12, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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

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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-2777 for ``Expansion Cohorts: Use in First-In-Human Clinical 
Trials to Expedite Development of Oncology Drugs and Biologics.'' 
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 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: Lee Pai-Scherf, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 2314, Silver Spring, MD 20993-0002, 301-
796-3400; 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 ``Expansion Cohorts: Use in First-In-Human Clinical Trials to 
Expedite Development of Oncology Drugs and Biologics.'' The purpose of 
this draft guidance is to provide advice to sponsors regarding the 
design and conduct of FIH clinical trials intended to efficiently 
expedite the clinical development of cancer drugs, including biological 
products, through multiple expansion cohort study designs. These are 
study designs that employ multiple, concurrently accruing, patient 
cohorts, where individual cohorts assess different aspects of the 
safety, pharmacokinetics, and anti-tumor activity of the drug. This 
guidance provides FDA's current thinking regarding: (1) Characteristics 
of drug products best suited for consideration for development under a 
multiple expansion cohort study; (2) information to include in 
investigational new drug application submissions to justify the design 
of individual expansion cohorts; (3) when to interact with FDA on 
planning and conduct of multiple expansion cohort studies; and (4) 
safeguards to protect patients enrolled in FIH expansion cohort 
studies.
    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 Expansion 
Cohorts: Use in First-In-Human Clinical Trials to Expedite Development 
of Oncology Drugs and Biologics. 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. 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 the draft guidance have been approved 
under 21 CFR part 312 at OMB control number 0910-0014; the guidance for 
industry entitled ``Formal Meetings Between the FDA and Sponsors or 
Applicants of PDUFA Products'' under OMB control number 0910-0429 is 
available at https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM590547.pdf; the 
guidance for clinical trial sponsors entitled ``Establishment and 
Operation of Clinical Trial Data Monitoring'' under OMB control number 
0910-0581 is available at https://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm127073.pdf; the guidance for 
industry entitled ``Oversight of Clinical Investigations--A Risk-Based 
Approach to Monitoring''

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under OMB control number 0910-0733 is available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm269919.pdf; 21 CFR parts 50 and 56 under OMB control number 0910-
0755; the guidance for industry entitled ``Expedited Programs for 
Serious Conditions--Drugs and Biologics'' under OMB control number 
0910-0765 is available at https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf; and 
the ICH guidance for industry entitled ``E6(R2) Good Clinical Practice: 
Integrated Addendum to ICH E6(R1)'' under OMB control number 0910-0843 
is available at https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM464506.pdf.

III. Electronic Access

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

    Dated: August 8, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-17273 Filed 8-10-18; 8:45 am]
 BILLING CODE 4164-01-P


