Posted by Michael Wonder on 03 Apr 2019
ICER issues final report on Spinraza and Zolgensma, provides policy recommendations related to pricing and coverage of treatments for spinal muscular atrophy
3 April 2019 - Independent appraisal committee notes family testimony in votes confirming broad benefits of both treatments; however, committee votes unanimously that price for Spinraza is too high to align fairly with these benefits, and urges fair pricing for emerging gene therapy Zolgensma to support sustainable access to innovation.
The ICER today released a final evidence report and report-at-a-glance assessing the comparative 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 half of 2019.
ICER's assessment was reviewed at the March 2019 public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER's three independent evidence appraisal committees.
