VRTX – The fact that the interim PROVE-3 numbers for patients who had relapses in the first line are immensely better than the numbers for patients who had non-responses or breakthroughs has the effect of fragmenting the second-line setting and making it harder to handicap what the FDA will request from VRTX.
I had not noticed the start contrast between the 3 arms until I read it closer. I was just so taken with the 73% result and 52% overall.
Given that the largest population with this unmet need is being presented with an extraordinary new option. Half the time and perhaps triple to quadruple the SVR rate, that is going to be awfully hard not to move forward because there are some inter-subgroup differences. Particularly when the SVR results for each subgroup is likely to be 30% or higher.
A cost benefit/analysis strongly and overwhelmingly dictates that the drug, that we know works, and that we have a large safety database on, there are no alternative treatments, and that there is a dire unmet need (albeit it is not pancreatic cancer, but we are talking 10,000 some deaths + how many thousands, to ten thousands of liver transplants each year which is no picnic, and not cheap for medicare) to approve the drug, and further evaluate the drug and the subgroup specifics in the phase 3 trial.
Unlike Provenge, where the cost benefit/analysis was quite compelling given the dire unmet need and horrible death and dearth of alternative treatment, in this case, we know for a practical certainty that the drug works, that it is reasonably safe, and we know that now without any real and reasonable doubt.
As such, that is the rational analysis in this instance. It would not even be controversial, it would not be a negative precedent for the FDA, and it would be a situation where the FDA could boast about moving safe drugs to tackle dire unmet needs to market quickly. I just cannot see this holding accelerated approval back, particularly if all 3 arms turn out SVR 30% or better.
This, even without much political pressure. With political pressure, it becomes even more likely not to make a difference.
But it is the FDA, so you are correct. There may be some serious discussion of this as to why the large distinction between groups. I am sure VRTX is anticipating this and has prepared their responses accordingly. Perhaps we will find out on Wednesday at VRTX's next presentation. Should be interesting.
Tinker
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