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Message: Patient Years & MACE rate Question

My 2 cents:

1.  Based on my spreadsheet sensitivity analysis, the MACE rate for the placebo is the most important figure.  The trial seems to have been designed at 7 for the placebo and 5 for RVX208.  But based on the various scribblings of Tundup and others, the 7 seems too low.  I read/heard somewhere that some of these patients were getting repeated MACE events.  If we raise the MACE rate to 8, then the RRR just explodes higher.

2.  This is where the # of patient years comes in.  The trial was for 1.5 years and 2400 patients to get an 80% confidence level.  Clearly, the more the patient years, the bigger the confidence level.  And if we are indeed above 4,000 patient years, then the confidence level becomes that much statistically significant and the RRR independently goes up (more patient years mean the MACE from placebo adds up even more).

3. Yes, dropouts are the biggest unknown.  This could be where the Tundup figure of 2,400 patient years and my estimate which is more than 1,000 above, comes in.  Very sick patient group.

4. For my purposes, I took a straight line approach to enrollment.  Between 8/16/16 and 3/17/17, the increase in enrollment was 200 over 213 days, so I took it as 28.2 patients a month added.

5. We do know 250 as of April 18.  I am just using a sensitivity of 255, 260, 265 and 270.  The results change a little.   

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