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Gottlieb Outlines the FDA’s Future Approach to Precision Medicine in Davos
February 5, 2018

The FDA plans to base future approvals of precision medicines largely on clinical trials that examine long-term durability and safety issues, Commissioner Scott Gottlieb said during a panel discussion at the World Economic Forum in Davos, Switzerland. Gottlieb said the key regulatory issues will be less about determining efficacy, especially with products demonstrating a strong proof-of-principle and early observations of effectiveness in small trials — and instead more focused on product issues related to potential off-target effects and their implications. The agency will also seek accelerated approvals for precision medicines designed to optimize benefits for particular groups of patients, especially through genomic or molecular profiling. “I think we’re really at an inflection point right now, where we’re … defining the modern rules for how these technologies are going to be regulated,” he said. “We’re going to be looking at accelerated approval endpoints for earlier approval on questions of efficacy, with more vigorous long-term follow up in some of these constructs where we have authority to do that under accelerated approval.”