Hundreds of individualized treatments for rare diseases could become available over the next decade — but only if government regulators handle the new therapeutics properly.
At least that’s how Robert Califf, former commissioner of the Food and Drug Administration, sees it, he said at STAT’s Breakthrough Summit East on Thursday. New bespoke gene therapies, which may only treat a small number of patients, could hold great promise in the aggregate, he suggested, if the agency can properly assess them.
“But if we don’t get this right, we could end up in a dead end that sets things back by a number of years,” he said.
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