I'm the first to admit I'm out of my depth with statistics, But I'm curious to know where the length of time starts to become more important than the number of participants [12] in the first study.
For example what would be more significant, 24 patients for 12 months or 12 patients for 24 ? or 30 months ?
People often say "oh there are only 12 patients", but the mortality rate in ALS/MND trials has historically been high, and on this occasion has been reasonably low with patients with advanced disease.
Some patients are now 40 months since diagnosis, I imagine the expected mortality rate past 40 months would be very high. Possibly past 50% or more.
Any insights would be appreciated.
Cheers
Ann: Positive Interim Study Results from OLE Study in ALS, page-10
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