ICER publishes evidence report on treatments for Duchenne muscular dystrophy


11 July 2019 - No price can be suggested as a fair value-based price for eteplirsen or golodirsen because no persuasive evidence yet exists to demonstrate the clinical effectiveness of either drug.

The ICER today released an Evidence Report assessing the comparative clinical effectiveness and value of two exon-skipping therapies to treat Duchenne muscular dystrophy (DMD) — eteplirsen (Exondys 51, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics) — as well as deflazacort (Emflaza, PTC Therapeutics), a corticosteroid.

This Evidence Report will be reviewed at an upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), in Cambridge, MA on 25 July 2019.

Read ICER Announcement

Michael Wonder

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Michael Wonder