Replies to post #109580 on Biotech Values

[jq1234]

You are off on this. RR in randomized portion isn't important anymore, PFS is the key in randomized portion.

Mutiple cohort RDT is quite expensive. Development stage biotechs don't run multiple Cohort RDT on their own, usually supported by large partners. The most successful drug out of RDT is Nexavar. It was Bayer's team idea to run RDT trial, and Bayer got the credit for it. Not every drug candidate is good for RDT trial. XL184 was a good candidate. Multi kinase inhibitors have been good candidate for RDT. It isn't to impress investors, rather to find appropriate indications to move forward. Multi kinase inhibitors do have more difficulty in identifying appropriate indications from typical phase I oncology studies. XL184 was good example in this regard as well, got two rather niche indications MTC, and GBM.

[AlpineBV_Miller]

I agree, but at least they are randomized. Mark Ratain, the arguable father of this trial design (or at least its biggest promoter) argues the enrichment is a byproduct but the important thing is tohave some randomization to help design the Phase III trials.

If you ever get a chance to chat with him about this design, it's worth the conversation. He's a great guy and one of the very few practicing clinicians (he's a hematologist) I've met who really understands statistics.