BRIEF—ICER to assess Duchenne muscular dystrophy therapies

The USA’s Institute for Clinical and Economic Review (ICER) has released a Draft Evidence Report assessing the clinical effectiveness and value of therapies to treat Duchenne muscular dystrophy (DMD).

These are two gene therapies from US RNA-based drug developer Sarepta Therapeutics - Exondys 51 (eteplirsen) and golodirsen - as well as PTC Therapeutics’ corticosteroid drug Emflaza (deflazacort).

This draft report will be open to public comment until 5pm ET on June 18, 2019. Based on stakeholder feedback, the ICER may revise key assumptions and findings for its Evidence Report, which will be published on July 11, 2019. The Evidence Report will be subject to deliberation during a public meeting of the New England Comparative Effectiveness Public Advisory Council, one of the ICER’s three independent evidence appraisal committees, on July 25, 2019.