Anavex Life Sciences Corp (AVXL)

[nidan7500]

read this re peer reviewed criteria being met, pending admin details...otherwise , looks good to go.

The New Big Is Small: Leveraging Knowledge from Small Trials for Rare Disease Drug Development - Blarcamesine for Rett Syndrome
Ene I. Ette, Emmanuel O. Fadiran, Christopher Missling, Edward Hammond
First published: 10 July 2023 https://doi.org/10.1111/bcp.15843
This article has been accepted for publication and undergone full peer review but has not been through the copyediting, typesetting, pagination and proofreading process, which may lead to differences between this version and the Version of Record. Please cite this article as doi: 10.1111/bcp.15843.
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https://bpspubs.onlinelibrary.wiley.com/doi/10.1111/bcp.15843

Abstract
Big data in drug development may not satisfactorily address the demands of precision medicine in a rare disease population, making the use of smaller clinical trials necessary. Consequently, the use of innovative design and analysis of these clinical trials using model-informed approaches have become indispensable. This requires informative exposure-outcome analysis, together with formal statistical analysis, which should include the strength of evidence for a study outcome. We demonstrate how knowledge can be gained, with supporting strength of evidence, from a small (data) clinical trial with a low dose of blarcamesine in the treatment of Rett syndrome (RTT). Based on a small data paradigm, pharmacometrics item response theory modeling and Bayes factor analysis were used to show that blarcamesine is efficacious in RTT.