Posted by Michael Wonder on 21 Dec 2018
Institute for Clinical and Economic Review releases draft evidence report on treatments for spinal muscular atrophy
20 December 2018 - Public comment period now open until 31 January 2019; requests to make oral comment during public meeting also being accepted.
The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the clinical effectiveness and value of nusinersen (Spinraza, Biogen) and onasemnogene abeparvovec (Zolgensma, Novartis/AveXis) for the treatment of spinal muscular atrophy (SMA). Spinraza was approved in 2016 for treatment of SMA in both children and adults. Zolgensma is a gene therapy that has been studied in infants with Type I SMA, and an FDA decision is expected in the first quarter of 2019.
This draft report will be open to public comment until 5pm ET on January 31, 2019. Based on stakeholder feedback, ICER may revise key assumptions and findings for its Evidence Report, which will be published on February 21, 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 March 7, 2019.
