RE:RE:RE:RE:RE:RE:Expectations too high. Reset your animus. There's something called Intention to treat, look it up. You don't get to pick out those patient who die/dropout early. They all go into the mix when calculating ORR. So taking your example, assume all 25 had at least one dose of drug, all would be counted as evaluable and you'd have a response rate of 5/25 or 20%. You don't get to pick and choose which patients to count and which to exclude from your analysis. You set a very minimum threshold for patients to be evaluable, usually recieved at least one dose of drug, and you count everybody. All the other drugs you've been looking at work to these standards, they've overcome the problems you outline to get the ORR the report, THTX has to do the same.
BTW I think your 50% dropout/die rate is way to high. I read a study a while back that put phase 1 enrolled patient not making it to 3 months at about 20%, even these get counted if they get the first dose.
THTX do need to show responders, even early on, even with all the difficulties.
Another thing I picked up was the fact that once doctors see the first hints of efficacy in a drug they'll start to put slightly healthier patients on that drug. You have to imagine at a cancer centre there might be 3 or 4 experimental drugs a patient could try. The doctor is going to want to give them some chance. I expect in the dose escalation the sickest of the sick were enrolled mostly. I think with the hints at efficacy they might get strong patient. I thought the 8 previous treatments pointed to pretty far gone individuals, I'm hoping that number comes down in 1b as they get to intervene a little earlier with patients.
Wino115 wrote: It is, no doubt, but to be realistic let's recall that the requirements to enroll are still that you have to be heavily pre-treated and refractory to all standard treatment options. It's still just not a very easy population to deal with in any kind of trial.
For instance, let's just assume they see a good response rate of 40% but also have the same death or curtail rate as the 1a trial. If they've enrolled 25 people in the last 5 months, then a good response rate would be 10 people responding. If the dropout rate due to death/life issues/etc.. is roughly 50% (which seems to be a conservative number based on 1a), then maybe you had 5 get to the point where 3-4-5 cycles showed 30% tumor reduction. What if each was a different cancer? Then still no protocal extension.
I just think that we're seeing, once again, the difficulty in testing your therapy on the kind of population necessary to prove your science up. It's the right population to show amazing things, but it's got a much higher difficulty-rating based on patient baselines. It sucks, but we'll just have to be patient ....
Bucknelly21 wrote: The main thing as rusty said, 5 months in and you dont have efficacy? That seems to be a little concerning to me