Notice2022-04398
Master Protocols: Efficient Clinical Trial Design Strategies To Expedite Development of Oncology Drugs and Biologics; Guidance for Industry; Availability
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Published
March 2, 2022
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics." This guidance provides advice to sponsors of drugs and biologics for cancer treatment regarding the design and conduct of clinical trials, other than first- in-human trials, intended to simultaneously evaluate more than one investigational drug and/or more than one cancer type within the same overall trial structure (master protocols) in adult and pediatric cancers. In contrast to traditional trial designs, where a single drug is tested in a single disease population in one clinical trial, master protocols use a single infrastructure, trial design, and protocol to simultaneously evaluate multiple drugs and/or disease populations in multiple substudies, allowing for efficient and accelerated drug development.
Full Text
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<title>Federal Register, Volume 87 Issue 41 (Wednesday, March 2, 2022)</title>
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[Federal Register Volume 87, Number 41 (Wednesday, March 2, 2022)]
[Notices]
[Pages 11722-11724]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2022-04398]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-3292]
Master Protocols: Efficient Clinical Trial Design Strategies To
Expedite Development of Oncology Drugs and Biologics; 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 final guidance for industry entitled ``Master
Protocols: Efficient Clinical Trial Design Strategies to Expedite
Development of Oncology Drugs and Biologics.'' This guidance provides
advice to sponsors of drugs and biologics for cancer treatment
regarding the design and conduct of clinical trials, other than first-
in-human trials, intended to simultaneously evaluate more than one
investigational drug and/or more than one cancer type within the same
overall trial structure (master protocols) in adult and pediatric
cancers. In contrast to traditional trial designs, where a single drug
is tested in a single disease population in one clinical trial, master
protocols use a single infrastructure, trial design, and protocol to
simultaneously evaluate multiple drugs and/or disease populations in
multiple substudies, allowing for efficient and accelerated drug
development.
DATES: The announcement of the guidance is published in the Federal
Register on March 2, 2022.
ADDRESSES: You may submit either electronic or written comments on
Agency guidances at any time as follows:
Electronic Submissions
Submit electronic comments in the following way:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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-3292 for ``Master Protocols: Efficient Clinical Trial Design
Strategies to Expedite Development of Oncology Drugs and Biologics.''
Received comments will be placed in the docket
[[Page 11723]]
and, except for those submitted as ``Confidential Submissions,''
publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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, 240-402-7500.
You may submit comments on any guidance at any time (see 21 CFR
10.115(g)(5)).
Submit written requests for single copies of this 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 to the Office of
Communication, Outreach and Development, Center for Biologics
Evaluation and Research (CBER), 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 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, 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 final guidance for industry
entitled ``Master Protocols: Efficient Clinical Trial Design Strategies
to Expedite Development of Oncology Drugs and Biologics.''
This guidance provides recommendations to sponsors of drugs or
biologics for the treatment of cancer regarding the design and conduct
of clinical trials intended to simultaneously evaluate more than one
investigational drug and/or more than one cancer type within the same
overall trial structure (master protocols) in adult and pediatric
cancers. In general, the recommended phase 2 dose should have been
established for an investigational drug or drugs evaluated in a master
protocol. It also describes aspects of master protocol designs and
trial conduct and related considerations, such as biomarker
codevelopment and statistical analysis considerations, and provides
advice on the information that sponsors should submit to FDA and on how
sponsors can interact with FDA to facilitate efficient review.
This guidance does not address all issues relating to clinical
trial design, statistical analysis, or the biomarker development
process. Those topics are addressed in the International Council for
Harmonisation (ICH) guidances for industry ``E9 Statistical Principles
for Clinical Trials'' and ``E10 Choice of Control Group and Related
Issues in Clinical Trials'' and the guidance for industry and FDA staff
``In Vitro Companion Diagnostic Devices.''
This guidance finalizes the draft guidance of the same name issued
on October 1, 2018 (83 FR 49398). FDA considered comments received on
the draft guidance as the guidance was finalized. Changes from the
draft to the final guidance include adding information about a dose-
finding or safety lead-in component in basket trials when evaluating an
investigational drug combination and comparison between experimental
arms in umbrella trials and acceptable statistical approaches.
Revisions were also made to various sections of the draft guidance to
clarify the information to be submitted to FDA to support amendments to
expand the protocol, the frequency of cumulative safety updates, the
role of ad hoc institutional review board meetings, the role of the
safety assessment committee, and informed consent requirements.
This guidance is being issued consistent with FDA's good guidance
practices regulation (21 CFR 10.115). The guidance represents the
current thinking of FDA on ``Master Protocols: Efficient Clinical Trial
Design Strategies 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.
II. Paperwork Reduction Act of 1995
While this guidance contains no collection of information, it does
refer to previously approved FDA collections of information. Therefore,
clearance by the Office of Management and Budget (OMB) under the
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not
required for this guidance. The previously approved collections of
information are subject to review by OMB under the PRA. The collections
of information in 21 CFR 314.50(d)(5) (clinical data section) and
(d)(6) (statistical section) have been approved under OMB control
number 0910-0001; the collections of information in 21 CFR part 312,
subpart B, for the submission of investigational new drug applications
(INDs), including protocols, protocol amendments, and information
amendments, have been approved under OMB control number 0910-0014.
Sponsors may request comment and advice on an IND as well as request
meetings with FDA under 21 CFR part 312, subpart C (OMB control number
0910-0014).
The collections of information in 21 CFR part 11 have been approved
under OMB control number 0910-0303; the collections of information in
parts 50 and 56 for the protection of human subjects and institutional
review boards have been approved under OMB control numbers 0910-0130;
Responsibilities of sponsors and investigators (21 CFR part 312,
subpart D) are also covered under OMB control number 0910-0014; the
collections of information in 21 CFR part 601 have been approved under
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OMB control number 0910-0338; the collections of information in
Sec. Sec. 201.56 and 201.57 for the content and format requirements
for labeling of human prescription drug and biological products have
been approved under OMB control number 0910-0572.
In addition, the following collections of information in FDA's
guidances have been approved by OMB (<a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>):
<bullet> Collections in FDA's draft guidance for industry entitled
``Formal Meetings Between the FDA and Sponsors and Applicants for PDUFA
Products'' have been approved under OMB control number 0910-0429.
<bullet> Collections in FDA's guidance for industry entitled
``Special Protocol Assessment'' have been approved under OMB control
number 0910-0470.
<bullet> Collections in FDA's guidance for industry entitled
``Establishment and Operation of Clinical Trial Data Monitoring
Committees'' have been approved under OMB control number 0910-0581.
<bullet> Collections in FDA's guidance for industry entitled
``Oversight of Clinical Investigations--A Risk-Based Approach to
Monitoring'' have been approved under OMB control number 0910-0733.
<bullet> Collections in FDA's guidance for industry entitled
``Expedited Programs for Serious Conditions--Drugs and Biologics'' have
been approved under OMB control number 0910-0765.
<bullet> Collections in FDA's guidance for industry entitled
``E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1)''
have been approved under OMB control number 0910-0843.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
<a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Dated: February 24, 2022.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2022-04398 Filed 3-1-22; 8:45 am]
BILLING CODE 4164-01-P
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