Document ID: FDA-2021-N-0512-0001
Agency: fda
Document Type: Notice
Title: Considerations for Progressive Multifocal Leukoencephalopathy Clinical Trial Designs; Public Workshop; Request for Comments
Posted Date: 2021-06-24T04:00Z

[Federal Register Volume 86, Number 119 (Thursday, June 24, 2021)]
[Notices]
[Pages 33312-33313]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[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 https://www.regulations.gov 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:
     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-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 https://www.regulations.gov or at the Dockets 
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
     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.govinfo.gov/content/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

[[Page 33313]]

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, 
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:
     Unmet need for PML therapeutics.
     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 
ONDPublicMTGSupport@fda.hhs.gov 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: https://collaboration.fda.gov/fdaworkshop092121.
    If you have never attended a Connect Pro event before, test your 
connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get a quick overview of the Connect Pro program, 
visit https://www.adobe.com/go/connectpro_overview. 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 https://www.regulations.gov. It may be viewed at the Dockets Management Staff 
(see ADDRESSES). A link to the transcript will also be available on the 
internet at https://www.fda.gov/drugs/news-events-human-drugs/considerations-progressive-multifocal-leukoencephalopathy-clinical-trial-designs-09212021-09212021.

    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