The "plan," as of now, is still BETonMACE ending at 250 MACE events and they "anticipate" reaching 250 MACE events by end of 2018. However, the "plan" may change. SSRA and recommendations by CSC/DSMB may results in increasing the number of patients and target MACE events beyond the current 2400 patients and 250 MACE events. Even though they "anticipate" reaching 250 MACE events by end of 2018 there is no guarantee that they will reach 250 MACE events by end of 2018. Top-line data will only be released when they hit their current MACE event target of 250 MACE events. No 250 events in 2018, no top-line data. If the MACE event target is increased, then top-line data would be delayed until the new target it reached. The only caveat is if the non-USA portion is allowed to read out first, but this is only conjecture at this time.

Here's an example of "top-line data" from the SUSTAIN trial and ASSURE trial. Top-line simply indicates whether or not the trial achieved its primary endpoint(s) with statistical significance and is typically released in advance of the full trial results. Often in cardiovascular outcomes trials, the full trial results will be presented at a major cardio meeting and published simultaneously in a major peer-reviewed journal such as NEJM.

As discussed previously, there are many potential scenarios with how changes to enrollment numbers and target MACE events may affect the trial timeline. Furthermore, we do not know for sure how the logistics of the USA vs. non-USA portion of the trial may affect things. See scenarios 1-4 in that post I linked to for just a few examples. Right now we have no idea if the non-USA portion would be permitted to read out prior to the USA portion. If the non-USA portion is only ~200 patients (last enrollment count was 2200 of 2400 target), it would be difficult for a 200 patient sub-trial to be able to stand on its own without the legs of the other 2200 patients. If total patient number is increased to a much higher number, thus allowing for a larger number of USA patients, then the idea of a stand-alone USA sub-trial becomes more viable.

Lastly, the SSRA decision may have little to do with the magnitude of the RRR and more to do with that statistics. I caution reading too much into the SSRA decision and the RRR.

BearDownAZ