Biotech Values

[mouton29]

1. On the first point, consider Mr. X, who enrolled one month ago. He does not know whether he received treatment or placebo. Maybe he suspects it was placebo as he did not any fever. He wants to quit and enter CU, where he has a 100% chance of receiving treatment, not a less than 2/3rds chance of treatment (recall his suspicion he in the placebo arm) to be followed by a crossover, which he views as too little, too late.

2. And if the answer to Mr. X is tough, if you quit the trial you get nothing from us, then the company is discriminating against men who have entered their trial. Maybe that can be done, but it feels "wrong." If the answer to Mr. X is OK, then isn't there a strong incentive to leave the trial? Again, as time passes and men progress and crossover, that incentive strongly diminishes.

3. Let's say that the FDA believes given the totality of the evidence, the "true" p-value is .04. I.e., a 96% chance the results were not due to chance. I think they have a strong interest in confirming this result and reducing the chance of a type I error as much as possible. Why are the considerations materially different than those that obtain where approval is based on a surrogate endpoint followed by a phase IV study? (I understand the REGULATORY framework is different).

4. As to waiting till 2010, that is what they said at the panel presentation, and they also said that the SPA provides for termination upon 350 or 360 deaths (I don't recall which of those is the number, but it one of them).