Replies to post #86859 on Biotech Values

[rkrw]

I don't know. You tell me.

You're wearing me out here. I'll be glad to discuss again in 6 months or after the Omapro pdufa.

[AlpineBV_Miller]

Please give me an example of a cancer trial where there was an open label study in an indication where there was an approved drug?

Accelerated approvals from single-arm Phase II trials are relatively common, especially in blood cancers. That's technically open-label, though probably not what you are looking for.

The FDA has granted SPAs to open label trials. The two GVAX trials were open label, for example. None come immediately to mind that actually worked.

[biomaven0]

an example of a cancer trial where there was an open label study in an indication where there was an approved drug?

Well Gleevec itself was approved based entirely on open-label trials. The approval in newly diagnosed chronic phase Philadelphia Chromosome-positive patients was based on an open-label randomized trial against interferon-alpha plus Ara-C (cytarabine) which was an approved drug combination for CML. That trial allowed cross-overs if there was progression, and the majority of the ifn/Ara-C patients eventually crossed-over. The Gleevec approvals for late phase, chronic phase and accelerated phase (and GIST I think) were all based on open-label single arm trials.

It is conceivable that the FDA would require a randomized open-label trial, but if they did it would certainly be stopped early, just as the original Gleevec trial was. I think there would be general consensus among CML doctors that a randomized trial would be stupid - you can basically tell based on PCR just how patients are doing.

Peter