QURE/BMRN/etc—In hemophilia patients, factor level seems like an endpoint for a phase-2 trial or a phase-3 whose goal is conditional rather than full approval (e.g. in the EU).
Major bleeds seems like the proper phase-3 primary endpoint. Employing a run-in period to establish each patient’s baseline for a calculation of the change in major-bleed frequency (as QURE is doing) is more rigorous than simply comparing the bleed numbers in the treatment arm to those in the control arm (which is what it sounds like BMRN is doing).
In your example, are the two phase-3 trials intended for the same regulatory jurisdictions?
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