Key takeaways - mRNA discussion, 2-pager Here: https://lnkd.in/eqwGWB44
Regulatory streamlining aligning with technological advances jibes well with new FDA thinking and structures around the concept of a “platform approval process”. The FDA Omnibus Reform Act of 2022 (“FDORA”) and the Prepare for and Respond to Existing Viruses, Emerging New Threats, and Pandemics Act (the “PREVENT Pandemics Act”) allow FDA leeway to explore and designate platform technologies. Dr Marks announced FDA is about to name the projects to be included in the new “start pilot” - “It’s basically looking to try to apply the principles of operation warp speed to our rare disease therapeutic development. The goal is to look at various metrics while we give programs the same kind of interactive meeting, instead of [or in addition to] having formal meetings, there’ll be informal interactions on an ad hoc basis, obviously with the appropriate formal minutes, etc, but, but bottom line is, the idea is not to have development slowed down by the need to wait for meetings to take place, and to try to also cut down some of the back and forth that sometimes happens, especially in the CMC space, where we go multiple rounds to get to resolution. So hopefully the metrics that we’ll get back on this will say, hey, it’s a success, and we’ll be able to hire up enough people to be able to put this routinely in place for rare disease product development. Having been up close and personal with Operation warp speed for the vaccines, I think it made a huge difference. It was actually more rewarding on the regulatory side, and I think it was less frustrating on the industry side. So I think it could be a real win-win.. [For example] If we have this question about how many conformance lots do we need to produce before we can go live, instead of having a formal meeting, there’ll be an informal discussion. Sometimes it could be a reply in writing. Sometimes it’ll be an informal teleconference. Sometimes maybe a more formal meeting might be set up a week later, but it’s not going to be, oh, send in a briefing book, and we’ll have a meeting 75 days from now, so that that’s, that’s the that’s the real difference here. The idea is to try to cut this kind of wasted space in development down, which, in aggregate, I think probably it’s got to add anywhere upwards of 25% to development time for some of the small programs that we’ve seen. We did a little net present value calculation about how much industry could pay if we had to put this into practice, and it turns out that it’s like it’s a pittance, what it will cost in terms of the additional headcount compared to the potential upside. Thanks Fouad Atouf Peter Marks Kenneth Chien Constanze Blume BioNTech SE FDA Moderna US Pharmacopeia Karolinska Institutet Pharmaceutical Technology BioPharm International