ICER Posts Draft Scoping Document for the Assessment of Treatments for Duchenne Muscular Dystrophy

— Document open to public comment until February 1, 2019 —

BOSTON, January 11, 2019 - 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 treatments for Duchenne muscular dystrophy (DMD).

ICER's assessment will focus on eteplirsen (Exondys 51™, Sarepta Therapeutics), golodirsen (Sarepta Therapeutics), and deflazacort (Emflaza®, PTC Therapeutics). Eteplirsen was approved by the FDA in September of 2016, deflazacort was approved in February of 2017, and golodirsen has an anticipated FDA approval decision expected in the middle of 2019.

ICER's report on DMD therapies will be the subject of a July 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 publiccomments@icer-review.org and must be received by 5 PM ET on February 1, 2019. 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 February 11, 2019.

About ICER

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.

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