Effectiveness, safety and costs of orphan drugs: an evidence-based review

Effectiveness, safety and costs of orphan drugs: an evidence-based review Evidence based practice . Igho J Onakpoya, Elizabeth A Spencer, Matthew J Thompson, Carl J Heneghan; BMJ Open 2015;5:e007199 doi:10.1136/bmjopen-2014-007199

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The available evidence suggests that there is inconsistency in the quality of evidence of approved orphan drugs, and there is no clear mechanism for determining their prices. In some cases, far cheaper generic agents appear to be available. A more robust, transparent and standard mechanism for determining annual costs is imperative

Targeting mitochondrial metal dyshomeostasis for the treatment of neurodegeneration

Jeffrey R Liddell, Neurodegenerative Disease Management, Posted online on August 21, 2015. (doi:10.2217/nmt.15.19)


Several trials indicate that redistributing iron may be an effective treatment for FRDA, but precise dosage control is required for optimal results.