Global Regulatory Challenges Of Rare Diseases
By Erin Harris, Editor-In-Chief, Cell & Gene
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The International Society for Cell & Gene Therapy (ISCT) 2024 Annual Meeting took place in Vancouver, Canada from May 29 to June 1, 2024. This was the 30th annual meeting and the largest global cell and gene therapy translation event of the year with more than 2,500 attendees. The event featured five plenaries and 20 concurrent sessions, covering cutting-edge research and clinical trials in cell and gene therapy. Leading up to the event, Dr. Bambi Grilley, Professor of Pediatrics and the Director of Clinical Research and Early Product Development for the Center of Cell and Gene Therapy (CAGT) at Baylor College of Medicine and Chief Regulatory Officer at ISCT was my guest on Cell & Gene: The Podcast (check out the full episode here). During our chat, Dr. Grilley shared that she would participate in the Global Regulators Summit, which brings together leaders from multiple different countries to talk about different issues facing the cell and gene therapy space. The Global Regulators Summit had its inaugural meeting last year, and participants covered the proliferation of unlicensed stem cell products. This year’s Summit would focus on rare diseases.
Global Regulators Summit 2024
I caught up with Dr. Grilley after ISCT 2024 and the Global Regulators Summit to hear how things went. Dr. Grilley describes the closed, invitation-only, one-day Summit as a crucial part of ISCT's efforts to foster global collaboration in addressing regulatory challenges in cell and gene therapies. The focus was on addressing and understanding global regulatory challenges in the development, manufacture, and access of products to treat rare diseases. Discussions included current global policies and strategies related to rare disease treatments. It brought together representatives from international regulatory organizations across North America, Europe, Asia, Australia, South and Central America, and Africa. “Seventeen regulators attended the Summit, including Fyodor Urnov, Ph.D., Professor of Molecular Therapeutics in the Department of Molecular and Cell Biology at the University of California, Berkeley,” she says. Indeed, Urnov is a prominent figure in the field of genome editing, and according to Dr. Grilley, he kicked off the Summit with his immense knowledge, enthusiasm, and advocacy for children with rare diseases.
“To me, the point of the Summit is to have dialogue that allows people in the field to view other ways of looking at business, because that’s the way regulation works,” she explains. “We talk about harmonization. But regulations looks a certain way in the United States, which is different from the EU. And there are approvals differences even within the EU; for example, there are differences among what is approved in Italy versus Spain versus France. Consider Asia; there’s Korea, South Korea, Japan, Taiwan, Singapore. We are far from being able to say the whole world regulates drugs the same way. But we do have opportunity for the sharing of ideas and best practices.”
Active Dialogue about Platforms
As of August 2024, there are more than 30 approved cell and gene therapy products listed by the FDA. The FDA’s positive and cautious approach to approvals shows the agency is actively supporting the development and approval of CGT platforms while maintaining stringent safety and efficacy standards. Dr. Grilley shared with me that platforms were a topic of discussion at the Summit. “If we are looking at using basically the same type of device, and we're looking at the same types of reagents, couldn’t we regulate that as a group, or what the FDA and other agencies call a platform?” she posits. “And if we could regulate as a platform, then we would be able to move more rapidly to state statistical significance. Instead of having 10 children per year born with a disorder, we’d have all the children who have similar disorders in the same pool. The issue of rare diseases is part of what makes this so challenging, because the standard study enrolls thousands of patients to determine whether it works. If your population is 2,000 or 20,000 children who are going to die by the age of 2, it is very, very difficult to enroll, study, and conduct evaluations for all of them especially worldwide. Our conversation centered around how we can we move to platforms.”
Much of the details from Global Regulators Summit remain confidential, but participants discussed regulatory decisions as compared to reimbursement and how that impacts patient access. “At the heart of all of our conversations is patient access,” says Dr. Grilley. “Honestly, we must do better. We must figure out new ways to move things forward for the patient.”