Replies to post #45080 on Biotech Values

[lumpy9200]

Yes, there is a suggestion of efficacy with Provenge, but given that the MOA does not stand up under close scrutiny, and given the small numbers of patients enrolled in the trials, any respectable scientist would be cautious about saying the treatment works.

jellybean, the panel voted 13-4. I believe the panel knew whether they were voting for approval or approvable.

Much of the conversation since March 29th has centered on Drs. Hussain and Scher, two of the no votes. The 13 yes votes came from professionals most would consider respectable.

Best,
Geoff

[drbio45]

Jelly

You are getting to excited over the dndn business.

I was more agitated about geoff Alan's self imposed injunction, and I didn't even own Insmed at the time. I just felt bad for the kids and the people with MMD

Alan is a POS in capital letters.

Have you ever looked at dorb Dor Biopharma. Go to dor board I would like your input

[AlpineBV_Miller]

DNDN - Any real statistician will tell you a trial shooting a p=0.01 with 125 people is more clinically persuasive than a trial shooting p=0.01 with 1250 patients.

Docs and amateur biostaticians hear "small trials pose the following problems..." and zone out the entire rest of the biostats lecture. With time and distance away from the lesson, this turns into "small trials are bad." Investors, who have an even shorter attention span than pre-med students, are even worse.

They miss the important part about describing strategies to ensure the hazards that come with small trials (mainly the magnification of small imbalances) do not harm the overall survival benefit.

When I see a 125-patient trial a company claims as a demonstration of a survival benefit, I do not automatically think, "Oh, that's too small." I think, "what have they done to correct for the probable imbalances inherent in small trials?"

Quibble if you want about the extraordnary steps Dendreon went through to prove the imbalances that were found did not explain the survival advantage they claim exists. But don't say "it was a small trial" and expect someone who knows better to listen.

Subset analysis of the control arm isn't bad science, its bad statistics when looked at in isolation. The fact guys who didn't crossover did worse than guys who did is not conclusive supportive evidence for efficacy -- but it doesn't hurt the case.

As Dr. Mule pointed out, we have no idea how any of these works. If we wait until we do, we won't approve one of these drugs any time in the next decade or so. If 9902b shoots p<0.05, would you withhold approval because the MOA isn't precisely known? If the answer is no, then the MOA argument is moot.