How to assess new drugs for neuropathies: advances in trial design and methodology.
OBJECTIVE
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.


RESULTS
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.


CONCLUSIONS
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.
van Doorn
Pieter A
PA
Department of Neurology, Erasmus MC Rotterdam, The Netherlands. p.a.vandoorn@erasmusmc.nl
Merkies
Ingemar S J
IS