@Phaedrus, HC not letting me post wit the quote function...

@Phaedrus, HC not letting me post wit the quote function tonight...

Quote: "I think you and I can agree that, provided MSB has controlled and other study data which the FDA can accept as showing significant reduction in mortality, the absence of any RCTs should not be a barrier to marketing approval, and the LANTIDRA approval based on open-label trials appears to be consistent with that conclusion."

Yes, you and I and everyone else can agree on that... but it's important to understand that the level of statistical significance shown by Lantidra is not easy to achieve. Per the discussion with @otherperspective above, the FDA has the discretion of making approvals based on single arm studies/RWE in orphan indications when statistical significance is under p=.01. If you're going to bypass RCT's you need some powerful numbers. As he and I discussed up above FDA probably applies a correction factor in small studies making it harder still to get under .01 and achieve extraordinary significance. But Lantidra got there... with room to spare. Lantidra should be looked at more as a transplant than a therapeutic. Transplants are generally not regulated by the FDA beyond giving an exemption. It's medically evident that you can't live without functioning.... kidneys...liver...lungs...heart. So, transplanting those organs does not require phase 3 clinical trials. A physician can simply order it be done, the mechanism of action is self-evident, and any degree of survival, certainly 70% survival, in a condition with 100% mortality sends the p-value down close to zero, too low to be calculated. Lantidra is a transplant of islet cells without which one cannot live short of insulin injections. The only reason the FDA is involved is because more than "minimal manipulation" is required to isolate and prepare the islet cells for injection. Rem-L is actually much safer than Lantidra, but... GvHD is a more complex illness, multiple organs may be involved, multiple other immuno-suppressives/modulators are implicated, treatment of the illness is evolving, mortality is not 100% leading the FDA to debate historical control mortality rates proposed by the company, and rem-L doesn't graft it acts as a therapeutic. The company didn't achieve p under .01 on its mortality reduction even with the advantage of non-randomized controls, and with the Yates correction may not even be under .05, see analysis above... Technically, that's why the FDA asked for more randomized, controlled data, imho.

Look, I think the product most likely works, that's why I'm invested. But I think management horribly botched the approval process going back years and misled investors in the process. Successful products require great science AND great management. You asked, what is the message of the FDA? To my mind it's this: The onus of proof of efficacy rests with the sponsor, aka MANAGEMENT.