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Statistical efficiency of patient data in randomized clinical trials of epilepsy treatments
Author(s) -
Romero Juan,
Goldenholz Daniel M.
Publication year - 2020
Publication title -
epilepsia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.687
H-Index - 191
eISSN - 1528-1167
pISSN - 0013-9580
DOI - 10.1111/epi.16609
Subject(s) - clinical trial , epilepsy , statistical significance , statistical power , medicine , randomized controlled trial , baseline (sea) , clinical significance , physical therapy , statistics , psychiatry , mathematics , oceanography , geology
Abstract Objective Previous research suggests that natural fluctuations in seizure rates within individuals have a quantifiable impact on therapeutic clinical trial outcomes. Methods A trial simulator estimated the statistical power of clinical trials with a typical trial design with and without patients included who exhibited a range of means (1‐15 seizures/mo) and standard deviations (1‐15 seizures/mo) in their baseline seizure rates. Trial outcomes were evaluated using 50% responder rates, median percentage change, and time to prerandomization. Results Patients with higher seizure frequencies and lower standard deviations during their baseline contribute more to the statistical power regardless of the method used to evaluate the trial. Power varied from −20% to 30% depending on baseline seizure characteristics. Significance Patient‐specific characteristics can predict the contributions to the statistical power of clinical trials for epilepsy treatments. It may be possible to characterize this contribution with baseline data, leading to more efficient clinical trials.