Retiring Office of Tissues and Advanced Therapies’ Director Wilson Bryan expects the expedited pathway will be used more frequently moving forward, but worries that ambiguous confirmatory trial results many years down the line will still leave efficacy questions unanswered.
Accelerated approval is likely to be used more extensively in the future for gene therapies that treat very rare diseases, but the slowly progressive nature of some conditions means there may be many years of uncertainty about the clinical benefits provided by the one-time treatments, the head of the US FDA’s Office of Tissues and Advanced Therapies said.
OTAT Director Wilson Bryan opined on the future use of accelerated approval for gene therapies, worries about ambiguous confirmatory trial...
The UK drug regulator acknowledges that technology will “move on” and, as such, has designed its decentralized manufacturing regulation to be as “enduring” as possible. Experts from the MHRA explain what products are covered by the new framework and how it has been future-proofed.
A differentiated profit-sharing model to accelerate the use of AI in drug development using clinical and patient data is being proposed in South Korea, but the idea faces multiple practical challenges
Office of Therapeutic Products Director Nicole Verdun and her deputy Rachael Anatol were escorted out of FDA headquarters on June 18. Disagreements over CAR-T regulation and Capricor’s DMD treatment may be to blame.
