Certain types of gene therapies could qualify for a faster US Food and Drug Administration approval process, Stat News reports.
The agency first plans to focus on hemophilia treatments for this faster approval process, Bloomberg adds. In this case, rather than basing approval decisions on whether therapies lead to fewer bleeding episodes experienced by patients, it would be based on a surrogate endpoint — whether the treatment increases the amount of clotting factors patients produce, it reports.
According to Bloomberg, FDA Commissioner Scott Gottlieb noted during a speech to the annual board meeting of the Alliance for Regenerative Medicine that while this is a faster path to approval, it doesn't come with the same certainty that the end goal of lower bleeding rates would be achieved. That, he added, would have to be ascertained after approval.
"These products are initially being aimed at devastating diseases, many of which are fatal and lack available therapy," Gottlieb said, according to Stat News. "In these settings, we've traditionally been willing to accept more uncertainty to facilitate timely access to promising therapies."
Stat News adds that the FDA plans to soon issue a policy framework to help companies determine how to assess therapies for different conditions.