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DATE AND TIME

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

DESCRIPTION/SUMMARY

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).

ABOUT THE PUBLIC WORKSHOP

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.

REGISTRATION

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

ONLINE ATTENDANCE (WEBCAST)

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

Meeting Recording

• Considerations for Progressive Multifocal Leukoencephalopathy (PML) Clinical Trial Designs

EVENT POINT OF 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

Event Materials

• Considerations for PML Clinical Trial Designs FDA Workshop Agenda (PDF - 143 KB)
• PML Workshop Affiliations Disclosures and Bios (PDF - 240 KB)
• Birnkrant Sheikh Intro FDA PML Workshop V7 (PDF - 350 KB)
• Major JCV PML FDA Symp Sept-21-2021 (PDF - 868 KB)
• Clifford PML Therapy for PML CLIFFORD Sept2021 Final V2 (PDF - 2.69 MB)
• Smith PML Clinical Outcomes slides 93 (PDF - 403 KB)
• Sheikh Considerations for PML Clinical Trial Endpoints V5 (PDF - 276 KB)
• Baldassari PML Disability Outcomes draft 9-3-21 (PDF - 546 KB)
• Wattjes Imaging PML FDA Symposium V3 (PDF - 2.34 MB)
• CorteseNoratoCinque JCV Biomarker Workshop slides (PDF - 624 KB)
• Tobin PML Webinar Statement slides (PDF - 97 KB)
• PML Webinar Final Transcript (PDF - 1.28 MB)
• Ohayon Patient Focused Drug Development for PML PMLFDAworkshop V4 (PDF - 192 KB)
• Isabella Patient Perspective Pml slides V3 (PDF - 1.3 MB)
• Lee PML Control Group Considerations 9-3-2021 (PDF - 453 KB)
• Cortese Cheung PML clinical trial designs FDA workshop (PDF - 624 KB)
• Sheikh Closing Remarks (PDF - 173 KB)

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