By Nancy Bradish Myers
The DIA annual meeting session “Regulatory Frameworks: How Can Regulators Facilitate Disruptive Innovation?” earlier this month offered a rare window into companies that are developing cutting edge technologies and the regulators who must prepare for their review. I had the opportunity to serve as moderator of this fascinating panel.
The panel featured:
- Rich Moscicki, FDA, Deputy Center Director for Science Operations, CDER
- Anne-Virginie Eggimann, Vice President, Regulatory Science, bluebird bio
- Tal Zaks, Chief Medical Officer, Moderna Therapeutics
New technologies and approaches to therapeutic products carry great promise. At the same time, they often challenge existing regulatory frameworks and require both companies and regulators to think creatively about how to prepare for getting game-changing technologies from pipeline to patients.
What is disruptive innovation?
Panelists offered several examples of disruptive technologies.
Eggimann, who began her career in cell therapy, described bluebird bio’s vector technology for gene therapy and gene editing that will treat and potentially cure rare diseases in a single dose.
Zaks described Moderna’s mRNA technology platform as functioning very much like an operating system on a computer. It is designed so that it can plug and play interchangeably with different programs. The “program” or “app” is Moderna’s mRNA drug – the unique mRNA sequence that codes for a protein.
How can regulators and companies interact to foster innovation?
It can take years from the advent of a game-changing technology to the point where it bears fruit.
FDA’s Moscicki calculates that it typically takes 20 years for the story that appears on the cover of Time to translate to a therapeutic product. Therefore, FDA reviewers have time to research and learn about the technologies when an IND is submitted, and it is part of their job to do so.
Zaks noted that companies and FDA have a common language around science and data. However, sometimes it is appropriate to involve senior management – how should sponsors approach elevating certain issues?
Moscicki encouraged dialogue with FDA. Some key resources include the Critical Path group and the new Emerging Technologies group within the agency. CDER’s medical policy office also encourages companies to come in on a yearly basis to discuss any gaps or difficulties. In addition, FDA holds internal symposia and public meetings to gain insight into evolving technologies. The agency uses these opportunities to learn, evaluate, and create appropriate policies.
What are other models for collaboration?
bluebird bio’s Eggimann noted the importance of international convergence on science and policy. Collaboration between FDA, EMA and Japan can enhance efficiency and predictability for innovators.
The panelists discussed the pros and cons of other models for collaboration, including a model in breast cancer in which patients, researchers, and regulators were all engaged.