15 August 2019 - Public meeting reveals tensions inherent around high-priced treatments that lack adequate evidence of efficacy.

The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and value of the corticosteroid deflazacort (Emflaza, PTC Therapeutics), and two exon-skipping therapies — eteplirsen (Exondys 51, Sarepta Therapeutics) and golodirsen (Sarepta Therapeutics). ICER’s report on these three treatments for Duchenne muscular dystrophy (DMD) was reviewed at the July 2019 public meeting of the New England Comparative Effectiveness Public Advisory Council (NE CEPAC), one of ICER’s three independent evidence appraisal committees.

During the meeting, a majority of CEPAC members voted that the evidence suggests that deflazacort achieves a net health benefit over prednisone, a more affordable corticosteroid. However, the panel did not find sufficient evidence to show a net health benefit of either eteplirsen or golodirsen over supportive care alone. During their deliberation, panel members also weighed the therapies’ other benefits and contextual considerations, highlighting that all three therapies target a condition of high severity and a high lifetime burden of illness, and that the use of deflazacort may reduce caregiver burden.

Read ICER Announcement