Posted by Michael Wonder on 11 Jan 2019
ICER posts draft scoping document for the assessment of treatments for Duchenne muscular dystrophy
11 January 2019 - Document open to public comment until 1 February 2019.
The ICER has posted a draft scoping document outlining a planned review of the comparative clinical effectiveness and value of treatments for Duchenne muscular dystrophy.
ICER’s assessment will focus on eteplirsen (Exondys 51, Sarepta Therapeutics), golodirsen (Sarepta Therapeutics), and deflazacort (Emflaza, PTC Therapeutics). Eteplirsen was approved by the FDA in September of 2016, deflazacort was approved in February of 2017, and golodirsen has an anticipated FDA approval decision expected in the middle of 2019.
