How to assess new drugs for neuropathies: advances in trial design and methodolo

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Curr Opin Neurol. 2008 Oct;21(5):519-26.

How to assess new drugs for neuropathies: advances in trial design

and methodology.

van Doorn PA, Merkies IS.

Department of Neurology, Erasmus MC Rotterdam, The Netherlands.

PURPOSE OF REVIEW: New randomized controlled trials are very much

needed to improve the outcome in patients with a variety of

peripheral neuropathies. A relatively low incidence of immune-

mediated neuropathies such as Guillain-Barré syndrome, chronic

inflammatory demyelinating polyradiculoneuropathy and multifocal

motor neuropathy, and slow progression in diabetic or hereditary

neuropathies may hinder a rapid inclusion and may lead to undesirable

extended trial duration.

RECENT FINDINGS: Some recent randomized controlled trials use modern

trial methodology. Identification of prognostic factors,

stratification for important variables at randomization, and the

selection of appropriate outcome measurements using a modern

clinimetric approach may contribute to a more proper randomized

controlled trial design for trials that can be conducted within a

limited time frame.

SUMMARY: Modern trial methodology and the appropriate use of outcome

measurements may improve the quality and reduce the numbers of

patients needed in randomized controlled trials in patients with

peripheral neuropathies.