Notice2021-13371
Considerations for Progressive Multifocal Leukoencephalopathy Clinical Trial Designs; Public Workshop; Request for Comments
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Published
June 24, 2021
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the following public workshop entitled "Considerations for Progressive Multifocal Leukoencephalopathy Clinical Trial Designs." The purpose of the public workshop is to discuss the challenges and clinical trial design considerations for developing therapeutic products for the treatment of progressive multifocal leukoencephalopathy (PML).
Full Text
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<title>Federal Register, Volume 86 Issue 119 (Thursday, June 24, 2021)</title>
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[Federal Register Volume 86, Number 119 (Thursday, June 24, 2021)]
[Notices]
[Pages 33312-33313]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2021-13371]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2021-N-0512]
Considerations for Progressive Multifocal Leukoencephalopathy
Clinical Trial Designs; Public Workshop; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the following public workshop entitled ``Considerations for Progressive
Multifocal Leukoencephalopathy Clinical Trial Designs.'' The purpose of
the public workshop is to discuss the challenges and clinical trial
design considerations for developing therapeutic products for the
treatment of progressive multifocal leukoencephalopathy (PML).
DATES: The public workshop will be held virtually on September 21,
2021, from 10 a.m. to 4:15 p.m., Eastern Time. Submit either electronic
or written comments on this public workshop by November 1, 2021. See
the SUPPLEMENTARY INFORMATION section for registration date and
information.
ADDRESSES: The public workshop will be held in virtual format only.
You may submit comments as follows. Please note that late, untimely
filed comments will not be considered. Electronic comments must be
submitted on or before November 1, 2021. The <a href="https://www.regulations.gov">https://www.regulations.gov</a> electronic filing system will accept comments until
11:59 p.m. Eastern Time at the end of November 1, 2021. Comments
received by mail/hand delivery/courier (for written/paper submissions)
will be considered timely if they are postmarked or the delivery
service acceptance receipt is on or before that date.
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-2021-N-0512 for ``Considerations for Progressive Multifocal
Leukoencephalopathy Clinical Trial Designs.'' Received comments, those
filed in a timely manner (see ADDRESSES), will be placed in the docket
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
[[Page 33313]]
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.
FOR FURTHER INFORMATION CONTACT: Lori Benner and/or Antoinette
Ziolkowski, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 6221, Silver
Spring, MD 20993-0002, 301-796-1300.
SUPPLEMENTARY INFORMATION:
I. Background
Progressive Multifocal Leukoencephalopathy (PML) is a rare, often
fatal viral disease of the central nervous system that affects patients
with immunosuppressive conditions and those treated with
immunomodulatory agents. No products are approved for the treatment of
PML and no therapeutic development pathway is established for PML. FDA
seeks to discuss scientific and regulatory challenges associated with
designing clinical trials evaluating PML treatments, and to develop PML
clinical trial designs that are feasible, adequate to establish
substantial evidence of effectiveness, adequate to characterize the
safety profile of investigational treatments, and acceptable to PML
patients, clinicians, regulators, and industry.
The Agency encourages healthcare providers, employees of other U.S.
Government agencies, academic experts, industry experts, patients and
patient advocates, and other stakeholders to attend this public
workshop.
II. Topics for Discussion at the Public Workshop
Discussions are planned around the following topics areas:
<bullet> Unmet need for PML therapeutics.
<bullet> Key trial design considerations, including feasibility,
trial populations, selection of control groups, endpoints, adaptive
designs, and master protocols.
III. Participating in the Public Workshop
Registration: Persons interested in attending this public workshop
must register online by September 20, 2021, midnight Eastern Time using
the weblink for this workshop noted in the Transcripts section below.
Please provide complete contact information for each attendee,
including name, title, affiliation, address, email, and telephone.
Requests for Oral Presentations: During online registration, you
may indicate if you wish to present during the virtual public comment
session and which topic(s) you wish to address. We will do our best to
accommodate requests to make public comments. Individuals and
organizations with common interests are urged to consolidate or
coordinate their presentations, and request time for a joint
presentation, or submit requests for designated representatives to
participate in the focused sessions. We will determine the amount of
time allotted to each presenter and the approximate time each oral
presentation is to begin, and will select and notify participants by
September 10, 2021. All requests to make oral presentations must be
received by September 3, 2021. If selected for presentation, any
presentation materials must be emailed to the
<a href="/cdn-cgi/l/email-protection#145b5a50446176787d77594053476164647b6660547270753a7c7c673a737b62"><span class="__cf_email__" data-cfemail="93dcddd7c3e6f1fffaf0dec7d4c0e6e3e3fce1e7d3f5f7f2bdfbfbe0bdf4fce5">[email protected]</span></a> no later than September 16, 2021. No
commercial or promotional material will be permitted to be presented or
distributed at the public workshop.
Streaming webcast of the public workshop: This public workshop will
be webcast at the following site: <a href="https://collaboration.fda.gov/fdaworkshop092121">https://collaboration.fda.gov/fdaworkshop092121</a>.
If you have never attended a Connect Pro event before, test your
connection at <a href="https://collaboration.fda.gov/common/help/en/support/meeting_test.htm">https://collaboration.fda.gov/common/help/en/support/meeting_test.htm</a>. To get a quick overview of the Connect Pro program,
visit <a href="https://www.adobe.com/go/connectpro_overview">https://www.adobe.com/go/connectpro_overview</a>. FDA has verified
the website addresses in this document, as of the date this document
publishes in the Federal Register, but websites are subject to change
over time.
Transcripts: Please be advised that as soon as a transcript of the
public workshop is available, it will be accessible at <a href="https://www.regulations.gov">https://www.regulations.gov</a>. It may be viewed at the Dockets Management Staff
(see ADDRESSES). A link to the transcript will also be available on the
internet at <a href="https://www.fda.gov/drugs/news-events-human-drugs/considerations-progressive-multifocal-leukoencephalopathy-clinical-trial-designs-09212021-09212021">https://www.fda.gov/drugs/news-events-human-drugs/considerations-progressive-multifocal-leukoencephalopathy-clinical-trial-designs-09212021-09212021</a>.
Dated: June 15, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-13371 Filed 6-23-21; 8:45 am]
BILLING CODE 4164-01-P
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