The groundwork being done in 2024 is building the foundation for global collaboration in the future.
At the DIA Global 2024 Conference, Dr. Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA), and Lori Ellis, head of insights at BioSpace, discussed the advancements being made to help drive forward drug and medical device development. Not surprisingly, Dr. Marks stressed the need for regular communication between the FDA and the biopharma industry as science and technology continue to evolve. Below summarizes the discussion highlights.
Key Takeaways
Dr. Marks spoke candidly about the progress being made on the FDA and EMA’s joint review process, saying that he sees the collaboration as a blueprint that he hopes will expand to regulators in other countries. The goal, he said, is to have a global registry to assist regulators around the world in their review process.
He further expanded on the need to lean into the accelerated approval process, particularly for rare disease therapies, because the quality of life for these patients dramatically increases when treatments are made available sooner. For gene therapies, he suggested that a guidance would be released on how to do navigate the complexities of accelerated approval submissions.
The second, third and fourth generation of CRISPR genome editors offer a robust platform technology. Predicting that 99% of products using this technology will be similar, Dr. Marks advocated for regulating them in such a way to maximize approvals while minimizing the regulatory burden. For Dr. Marks, the next few months are exciting as the FDA focuses on creating the regulatory framework for this platform.
Dr. Marks stressed the need for regular communication and collaboration between regulators and drug/device sponsors at early stages, particularly for cell and gene therapies, as the FDA is willing to help companies identify the best approach for development of their assets.
Dr. Marks noted that the industry is lagging behind in some areas, such as the ability to manufacture certain drug products.
