– Document open to public comment until September 12, 2018 –
BOSTON, August 22, 2018 – The Institute for Clinical and Economic Review (ICER) has posted a Draft Scoping Document outlining a planned review of the comparative clinical effectiveness and value of AVXS-101 (AveXis) and nusinersen (Spinraza®, Biogen) for the treatment of spinal muscular atrophy (SMA). The scoping document will be open to public comment until 5pm ET on September 12, 2018.
Nusinersen was approved in 2016 for treatment of SMA in both children and adults. AVXS-101 is a gene therapy currently being studied for use in infants with SMA, and an FDA decision is expected in the first quarter of 2019. ICER’s report on these therapies will be the subject of a March 2019 meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.
All interested stakeholders are encouraged to submit comments and suggested refinements to the scope to ensure all perspectives are adequately considered. Comments can be submitted by email to email@example.com and must be received by 5 PM ET on September 12th. All comments submitted must meet ICER’s formatting specifications.
ICER’s Patient Participation Guide and Manufacturer Engagement Guide provide additional guidance for submitting public comments, including suggestions for what types of information may be most useful.
In addition to comments on the scope, ICER also welcomes submissions from stakeholders on examples of low-value care practices within this clinical area. These submissions will inform a report section focused on strategies to reduce waste and preserve resources for high-value, potentially higher cost treatments. More information is available in the scoping document.
Following the public comment period, a revised scoping document will be posted on or about September 19th.
NOTE: An earlier version of this release stated the public comment deadline as September 7th. This release has been updated to reflect the correct deadline of September 12th.
The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.
ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.