Tuesday, May 23, 2017 11:57:22 AM
CBER Director Focuses on Flexibility to Advance Regenerative Medicines
"The great part of this is this provision (21ST CENTURY CURES ACT)did not lower the safety and effectiveness standard … although we have tremendous flexibility, which we intend to use," Marks said.
"We don't stand on ceremony—forget about the old two randomized controlled trials—single arm, compelling studies are okay in this setting," Marks said, adding that trials of 10 or 20 patients for gene therapy trials for rare diseases may be "compelling enough" for approval, and that the agency could look to creative endpoints for such products.
http://www.raps.org/Regulatory-Focus/News/2017/05/08/27503/CBER-Director-Focuses-on-Flexibility-to-Advance-Regenerative-Medicines/
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