Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Aug;61(8):1659-1667.
doi: 10.1111/epi.16609. Epub 2020 Jul 13.

Statistical efficiency of patient data in randomized clinical trials of epilepsy treatments

Juan Romero  1 , Daniel M Goldenholz  1
Affiliations

Statistical efficiency of patient data in randomized clinical trials of epilepsy treatments

Juan Romero et al. Epilepsia. 2020 Aug.

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.

Keywords: clinical trials; deep learning; epilepsy; seizures; statistics.

PubMed Disclaimer

Comment in

References

REFERENCES

    1. Schulz KF, Grimes DA. Sample size calculations in randomised trials: mandatory and mystical. Lancet. 2005;365(9467):1348-53.
    1. Perucca E, Wiebe S. Not all that glitters is gold: a guide to the critical interpretation of drug trials in epilepsy. Epilepsia Open. 2016;1(1-2):9-21.
    1. PhRMA. Profile biopharmaceutical research industry. 2015. [cited 2020 July 1]. Available at: http://www.phrma.org/sites/default/files/pdf/2015_phrma_profile.pdf
    1. DiMasi JA, Grabowski HG, Hansen RW. Innovation in the pharmaceutical industry: new estimates of R&D costs. J Health Econ. 2016;47:20-33.
    1. Oliveira A, Romero JM, Goldenholz DM. Comparing the efficacy, exposure, and cost of clinical trial analysis methods. Epilepsia. 2019;60:128-32.