ICER Publishes Evidence Report on Treatments for Duchenne Muscular Dystrophy

-- 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 --

-- For deflazacort, discounts of at least 73% from its list price would be needed to achieve commonly cited thresholds for cost-effectiveness --

-- As with all treatments for ultra-rare conditions, judgments of overall value require consideration of contextual issues and broader benefits for patients and families; these additional factors will be discussed during a July 25 public meeting --

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ICER Issues Final Report and Policy Recommendations on Esketamine for Treatment-Resistant Depression

-- Independent appraisal committee votes that evidence is adequate to demonstrate clinical benefits of esketamine versus placebo, but concerns remain about the study criteria used to define treatment-resistant depression and the lack of longer-term data on esketamine’s safety and effectiveness --

-- Esketamine’s list price would require a 25-52% discount to reach a fair value-based price benchmark, and the potential short-term budget impact associated with treating the eligible patient population leads ICER to issue an Access and Affordability Alert --

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