RE:IMO MCNA will be approved by the FDa... I will reply to your comments. I'm not perfect and of course, cannot predict the future. I fully expected a 'yes' vote on the grounds that there are limited options for treating bladder cancers and endorsing the approval of this therapy would encourage the industry to continue pouring money into research and further clinical trials that would allow them to improve and optimize their therapies. As a human being, obviously I'm hurt by the outcome, while I didn't expect it to be easy for the team at TST, I was taken aback by the callous nature of the voters. The second thing is that I feel they didn't answer the question appropriately. Sure, it can be argued that if a certain clause was added to the end of the question, it would have changed the outcome, but I wouldn't even go that far. It is very clear the question stated does the drug "[...] an overall risk/benefit profile [...]" for treating BC, etc; and it unequivacally does. For the little risk you are putting patients at, this drug presenting itself as a second line therapy is an almost no-brainer. It appeared to me that they were answering some other question and the reluctance in their answer definitely tells me that they just weren't sure. Also lastly, I did send an application to TST to join their regulatory team because what better way to help the company by actually lending my talents to assist in their submission.
1) The adcom makeup and vote
Again, I placed my faith in the indication they were seeking which absolutely does make a difference in the outcome. If they were going for a different label that would have made them a first line therapy, with the single Phase 3 study and their clinical trial demographics and results, there would have been no way in hell. Perhaps it is a little naive of me, but I expected the non-oncologists to be swayed by the plights of some of the cancer patients who were pleading with them to just have the option available to them. Now, I was at work yesterday, but I kept it on in the background, not sure if I missed a portion of it but I didn't ever hear them recommend the possibility of another clinical trial (which is what I would have expected as a worst-case scenario).
2) Stats Irrelevant
Agreed with all your points.
3) Safety
Again, agreed. I was expecting this to carry it over the "yes" line in the end.
4) Efficacy
I must have missed Bartlett encouraging off-label use because if that's the case, it's embarrassing. Everyone in pharma knows very well the consequences of promoting off-label use. Sure doctors can do it, but it defeats the whole purpose of the FDA and the regulatory process imo. That would have actually made me somewhat angry to hear.
I did catch "MCNA getting underserved credit". In a sense, that can sort of be a positive because it means they're getting their biostatiticians to develop arguments against such a notion for the actual BLA.
5) Unmet need
Every single bladder oncologist that spoke made a clear point that the options and treatments are incredibly limited. Some even expressed remorse at the lack of innovation in this area.
6) Label
Don't forget that TST has also committed to post-market studies. While its initial approval may be conditional, it is the start of a platform that they can build upon in the future.
7) TST leadership
No opinion, I will just agree to say that the management did seem unprepared, and anyone in regulatory will know what kind of a grilling to expect.
In all, I am sure they're currently corresponding with their FDA Reviewer contact and drawing up a plan to best mitigate this. It makes me question certain things from my own experiences however. Usually there's an End of Phase/End of Phase 3 meeting with the FDA, and again, a pre-submission meeting before you even file for the BLA. Typically, the data for these studies was already well-known before they even submitted and if there were any significant issues, it would have been raised before filing and a second phase 3 clinical trial would have been held instead of wasting their time with the BLA.
Alas, as I said. my shares and my money in the company are on ice until February, and I trust them to pull it off. The indication they're seeking, for the data that they possess is definitely not classified as a task of "unheard of" proportions.I did my research, and from this superficial point of view, nothing stood out to me as a massive red flag.