RE:Long, very long CanadaPiet you have to remember what the current standard of care benchmarks are for these refractory and advanced cancer patients. At this point they have tried and exhausted all the current standards used in the hospital. In most of those third and fourth line treatments we have seen that response rates in the 30% level are considered both good and commercial. As for efficacy or progression free survival, it depends on the actual cancer type, but it is anywhere from 6 months of prolonging life to 12 months. In a few of these types, even 4 months would be additive. That is all you need to show in order to be considered as the 4th or 5th line for refractory and become a standard of care. In all likelihood if you hit those benchmarks, you would see oncologists use it earlier.
Many of the blockbuster ADCs hit those 30 to 40% response rates and 6 to 12 months of progression free survival. They also spend a lot of time subsequent in the P2, P3 and later to test different cohort groups to boost those ORR and PFS rates based on baseline criteria. You may recall a post I put up a few weeks back about the newest Trodelvy data and how for HR+ breast, the interim study group only showed a 1.6 month OSS (Tropics2 study). They then added more patients and had much better results than the initial trial cohort and it was because of a particular "screening" they had done (HER2 low and someth8ng called IHC). They haven't released the data but say it met all primary and secondary endpoints, so it showed it expanded the survival rate and months more then the interim readout. The point being, as QWERTY points out, you want to know how your drug responds in all sorts of conditions for the powerful reason that it will help you determine what kinds of patients in what kind of cancer situations your drug has the most chance of success in working and for them to see the full benefits of the therapy and work as it should. Will some doctors try to use it without "hitting" all the characteristics the label is optimized for --of course, because there's so few therapies for these patients. But the patient and doctor know that the fewer of the "optimal" signals are there, the less it may work. But as JFM and others have been saying, cancers and tumors are not homogeneous and each case is almost always unique in some way. No drug label or screening will take into account all the possible permutations. It's all about probabilities and increasing chances. Don't expect perfection, just expect a commercial opportunity to arise if you extend the current SOC slightly.
canadapiet wrote: @SPCEO: my deepest condolences for the loss!
I'm becoming more convinced of the minimal results for now! It takes way to long to recruit patients and the ones we have doesn't seem to benefit that much. They aren't directly targeting those with high sortilin expression, it is all comers, so the trials are more of "a lucky match"....!
Maybe in the long run we will succeed when we can detect the "better patients" and when we can give it "earlier".
The only "thing" that can save us in the near time is some kind of "POC". But how when you can't select the "targeted peatients???