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  4. Considerations for Progressive Multifocal Leukoencephalopathy Clinical Trial Designs - 09/21/2021 - 09/21/2021
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Event Title
Considerations for Progressive Multifocal Leukoencephalopathy Clinical Trial Designs
September 21, 2021

September 21, 2021
10:00 AM - 4:15 PM ET


The public workshop will be held virtually on September 21, 2021 from 10 a.m. to 4:15 p.m.


This public workshop is currently being scheduled and planned as a virtual meeting. The Food and Drug Administration is announcing the following public workshop titled "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).


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 health care providers, employees of other U.S. Government agencies, academic experts, industry experts, patients and patient advocates, and other stakeholders to attend this 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.


This meeting will be held in virtual format only. Registration is required for online attendance and will be available until midnight on September 20th, 2021.

To register for this workshop, visit https://www.eventbrite.com/e/consideration-for-progressive-multifocal-leukoencephalopathy-clinical-trial-tickets-151666908807


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. 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 10th, 2021. All requests to make oral presentations must be received by September 3rd, 2021. If selected for presentation, any presentation materials must be emailed to ONDPublicMTGSupport@fda.hhs.gov no later than September 16th, 2021. No commercial or promotional material will be permitted to be presented or distributed at the public workshop.


A full agenda will be available before the workshop. All other meeting materials, including speaker slides and recorded webcast, will be also available here before and after the workshop. Please visit this page for any updates and posting of materials before and after the meeting.


This meeting will be available for online viewing, on the day of the meeting via the following link: https://fda.zoomgov.com/j/1604331150?pwd=dUp3aDBRcW5LVXhBSm5Ebndoa2podz09 | Passcode: n?kV4G


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

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