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

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.