Quote: "This is likely music to the ears of someone like Emil Kakkis, M.D., Ph.D., CEO of Ultragenyx, who’s long been advocating for expanding the use of accelerated approval for rare disease patients. Core to his argument, described in a previous interview with Fierce Pharma, is that if the FDA leaned into surrogate endpoints for rare disease patients, then the development process would hasten and there’d be a more viable commercial landscape."
Another quote: The FDA doesn’t “have to get to 100% certainty between an accelerated approval endpoint and a clinical endpoint," Marks added.
“If that were the case, there wouldn't be a difference, then we wouldn't have accelerated approval,” he said. The agency can mitigate uncertainty by “using science to the fullest extent," according to Marks—be it available animal data or strong human biomarkers related to, for example, the expression of a particular protein."
"Though we're still in early days here and I think we'll see some refinements, and I think we don't want to be overly dogmatic with this, I think it's overall an advance," said Marks.
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