irector Peter Marks explains the benefits of Accelerated Approval and CBER’s START (Support for clinical Trials Advancing Rare disease Therapeutics) Pilot Program in this DIA 2024 interview with Lori Ellis, Head of Insights, BioSpace. “We want to lean into accelerated approval whenever we can, particularly in the rare disease space. Because if you have patients in need, getting something to them a year or two sooner can make the difference, for instance, in a child with Duchenne muscular dystrophy, from the ability to walk or not walk,” Marks said. “Getting there fast is important. We want to get there with good accuracy because we want the confirmatory trials to confirm, but if we do our scientific work well, that will happen.

“EMA and FDA have agreed to do a pilot jointly reviewing a product at the same time (Collaboration on Gene Therapies Global Pilot [CoGenT]), which is a pretty big deal because the EU uses a different review process: Member States have rapporteurs, whereas our agency staff reviews the products. Once we work that out, we’re going to expand to a larger number of high-income country regulators that will include Canada, the UK, Switzerland, and Japan. A nucleus of high-income countries doing this will hopefully set the way for a much more facile way of getting products across multiple regulatory environments, hopefully with the same application, to reduce regulatory rework. The dream is: If we can then work out reliance procedures, it would be nice to have an organization like WHO register and list that. You could have listed products in the gene therapy space, so that regulators who just don’t have the capacity to review could say, ‘US and EU approve this product; we can now use their approval to key off our approval.’”