effectiveness demo'd in 102 patient treatment group without control group. will this "pivotal phase II" trial be enough? or will FDA ask for Phase III? i have my doubts, but still seems like ratio of upside to downside risk is skewed to the upside... JMHO
<<zebraboy snip>>The studies they've done so far are not good enough to prove to the FDA that GDC-0449 should be first line therapy for BCC and when they submit for approval, they will submit for GDC-0449 to be used only in BCC patients that have failed first line.<<snip>>
Zebraboy may write what some don't want to read, but it is still spot on.
Neither DNA/Roche or Passeri has ever made a comment about the broader BCC market. They know that the data generated to date is not about to sway CMS into approving added cost when the surgical SOC for "normal" BCC is so robust.
On the positive side, any validation of the platform is a very good thing.
I am not quite sure myself but I think it relates that not all BCCs respond to 0449 (less than 10%) and the respond may be only partial and it may reappear again even it has vanished. But operation takes always the nodulus away. So a long observation time may be nesessary to see real healing of cancer.
oh yah and the study you posted could potentially address the question of whether GDC-0449 is better than surgery as first-line but to be honest the way they've filled out the form i don't really understand what they intend to do (like what is happening to cohort 1 versus cohort 2?).
i don't know, i think if they wanted to challenge the current standard, it's up to them to prove that GDC-0449 is significantly better than the standard in a direct head-to-head study. Efficacy of standard care is quite high (between 70-90%), so it would be difficult to show that GDC-0449 is better in such a study (in my opinion). The studies they've done so far are not good enough to prove to the FDA that GDC-0449 should be first line therapy for BCC and when they submit for approval, they will submit for GDC-0449 to be used only in BCC patients that have failed first line.
Roughly, that means, if there are ~900,000 new cases of BCC each year and standard care shows efficacy in 70-90% patients, that's 90,000 to 270,000 patients that could get treated with GDC-0449. If Curis is only to receive a ~10% royalty from Roche/Genentech it's as if Curis is treating (and making money off of) 9,000 - 27,000 patients per year.
this condition of participation will probably be a sticking point for some potential participants:
Willingness to delay excision of the target tumor site until the time mandated in the protocol, unless evidence of disease progression or lack of drug tolerability
Look at this:
This is the study to demonstrate that with this drug 0449 we do not need anything else (?) and so it could be the new standard, perhaps, if proven and is safe. It is still the only study to challenge the present standard. I think they could need more. 0449 is compeating with surgery but it could also help surgery by srinking the cancer for surgery. So there are a lot of possibilities for 0449 even in this diagnosis.
They normally dont do control groups with terminal cancer patients, it's unethical. Even in PhI they routinely use cancer patients instead of healthy volunteers. My biggest concern going into this week was the few deaths that occurred during the trial. It's now clear that none of the deaths are attributable to GDC-0449. Genentechs track record with the FDA is pretty good, I think there's a good chance that roche will file and get approval for this small population of patients. The EU, which is much more conservative, is probably requiring a further testing thus the new trials in europe. We should see 44 in the next week or two as this is all digested by the investing community. GLTAL