An Assault On Vertex, Enthusiasm Building Around Xoma [View article]
I strongly agree. the data in problematic and same goes for the company conference call. Although CF is a rare disease, the primary end point are known from previous studies and do not include dividing the data to "better" periods. lets look at VRTX data - The interim gave 4% absolute improvement from baseline, in all doses - this is the most relevant figure because it reflects the patient improvement. The final results were 3.3% in the higher dose! assuming :12 patients in the 600mg interim group, and assuming they had a greater than 4% improvement.(dose related) - you will get that the additional 8 patients(20 in the final results) had 0-2% improvement! Same thing with the placebo group - interim -4.6% - 11 patients final - -3.3% 21 patients - the "new" 10 patients had -1.8%. Choosing to trust those numbers, with that kind of variability, is risky. just a reminder - the saline trial gave 3-4% absolute improvement.
An Assault On Vertex, Enthusiasm Building Around Xoma [View article]
the data in problematic and same goes for the company conference call. Although CF is a rare disease, the primary end point are known from previous studies and do not include dividing the data to "better" periods.
lets look at VRTX data -
The interim gave 4% absolute improvement from baseline, in all doses - this is the most relevant figure because it reflects the patient improvement.
The final results were 3.3% in the higher dose!
assuming :12 patients in the 600mg interim group, and assuming they had a greater than 4% improvement.(dose related) - you will get that the additional 8 patients(20 in the final results) had 0-2% improvement!
Same thing with the placebo group - interim -4.6% - 11 patients
final - -3.3% 21 patients - the "new" 10 patients had -1.8%.
Choosing to trust those numbers, with that kind of variability, is risky.
just a reminder - the saline trial gave 3-4% absolute improvement.