Boston – July 31, 2018 – The Institute for Clinical and Economic Review (ICER) will develop a report assessing the comparative clinical effectiveness and value of AVXS-101 (Novartis) and nusinersen (Spinraza®, Biogen) for treatment of spinal muscular atrophy (SMA). ICER’s report is set to be reviewed during 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.
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 many analysts expect FDA approval of this treatment to occur in the first quarter of 2019. The formal scope of ICER’s review – including the drugs reviewed, special subpopulations of interest, and basic approach to economic modeling – will be finalized with the benefit of input from all stakeholders, including clinical experts and patients.
An “Open Input” period begins today, and comments on the topic will be accepted from all interested stakeholders until August 17, 2018 at 5 PM ET. The Open Input period is intended to allow stakeholders to share key information relevant to the development of the evidence report. During this time, ICER will also contact key patient groups and clinical experts to gain further insights on the patient perspective and clinical context of these treatments.
For more information about the Open Input period, visit ICER’s website. ICER’s Manufacturer Engagement Guide, Patient Participation Guide, and Patient Guide to Open Input provide additional information for manufacturers and patient groups, including an explanation of what types of information may be most informative.
There are no page limits to Open Input submissions, and input received will be incorporated throughout report development. All input can be emailed to firstname.lastname@example.org and must be received by 5 PM ET on August 17, 2018 in order to be considered.
A Draft Scoping Document, which will provide more detail on ICER’s proposed scope for the review, will be available on or about August 22, 2018 and will be open to public comment until September 7, 2018.
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.