Biotech Values

[jq1234]

Clearly EXEL would have been better simply not trying to obtain an SPA...lessons for future such management

I am not sure that is the right lesson. SPA process had improved EXEL 306 trial design IMO. If EXEL went on with their original composite primary endpoint design, I don't think they are going to get approved even if trial sigficantly exceeds primary endpoint because it will be difficult to interpret the data. The new 306 trial separates pain primary endpoint from bone scan secondary endpoint, which makes data interpretation more clearly. Pain reduction is an approvable endpoint.

I think people who think FDA is unreasonable for not granting EXEL SPA doesn't completely understand clinical development process. For FDA to grant SPA, FDA virtually not only has to endorse trial design but also endorse the data and interpretation of how much bone scan contributes to clinical benefit when they have no data to make such endorsement. Thus FDA's suggestion of making bone scan as exploratory endpoint is completely right on mark. Only with further data, maybe from 306 trial, would we know how much bone scan contributes to clinical benefit.

To me, the lession is not to make big deal out of SPA, rather having FDA's input on trial design. Maybe I am biased because I really thought composite endpoint had no chance to succeed unless you have separate data to make that interpretation possible.