It's all relative.
That couldn't be truer for Vertex Pharmaceuticals'
Turns out, while they were presented as absolute improvements, those numbers were actually relative to placebo, which inflated the numbers because the placebo group's lung function decreased. The number of patients taking the combination that actually improved 5 or 10 percentage points on an absolute basis was 35% and 19%, respectively.
Vertex blamed the issue on a misinterpretation of the data that it received from a contractor responsible for doing the statistical analysis. That's certainly better than being accused of having employees who faked data, like Sequenom
Let's not forget that this was interim data from a trial with a mere 58 patients, 37 of whom got the drug combination. The data was likely to change anyway, something that investors in Celldex Therapeutics
Vertex's new data certainly doesn't look as good as it did before, but given how much of a game-changer the combination is for the treatment of cystic fibrosis, whether the drug works is a lot more important than how well it works. If the combination improves lung functions, cystic fibrosis patients will take it because there aren't any other options, besides drugs such as Gilead Sciences'
The decrease in placebo group might not be reproducible, but even ignoring that, VX-809 and Kalydeco caused a mean improvement of 4 percentage points in FEV1. If the combination can hit that number in the phase 3 trial, Vertex shouldn't have any problems getting it on the market and quickly reaching blockbuster status.
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