Posted by Michael Wonder on 22 May 2019
ICER releases draft evidence report on treatments for Duchenne muscular dystrophy
22 May 2019 - Public comment period now open until June 18, 2019; requests to make oral comment during public meeting also being accepted.
ICER today released a draft evidence report assessing the clinical effectiveness and value of two gene therapies to treat Duchenne muscular dystrophy (DMD) -- eteplirsen (Exondys 51, Sarepta) and golodirsen (Sarepta) -- as well as deflazacort (Emflaza, PTC Therapeutics), a corticosteroid.
This draft report will be open to public comment until 5pm ET on 18 June 2019. Based on stakeholder feedback, ICER may revise key assumptions and findings for its Evidence Report, which will be published on 11 July 2019. The Evidence Report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER's three independent evidence appraisal committees, on 25 July 2019.
