ICER Releases Draft Evidence Report on Therapies for Sickle Cell Disease

— ICER’s ongoing assessment emphasizes that policymakers will need to consider, in addition to traditional measures of clinical effectiveness and cost-effectiveness, that new interventions for sickle cell disease will differentially benefit an historically disadvantaged and underserved community —

— Public comment period now open until February 20, 2020; Requests to make oral comment during public meeting also being accepted —

BOSTON, January 23, 2020 – The Institute for Clinical and Economic Review (ICER) today released a Draft Evidence Report assessing the comparative clinical effectiveness and value of treatments for sickle cell disease, including crizanlizumab (Adakveo®, Novartis), voxelotor (Oxbryta™, Global Blood Therapeutics), and L-glutamine (Endari®, Emmaus Life Sciences). This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions.

ICER’s draft report notes that sickle cell disease has been an historically underserved area for research, with no new treatments for decades; as such, patients and clinicians have struggled with a system plagued by racism and underfunding. While assessing the value of these treatments, policymakers will need to consider the substantial lifelong burden associated with sickle cell disease, as well as the potential for these treatments to reduce the disparity in life expectancy between black and white Americans.

This draft report will be open to public comment until 5pm ET on February 20, 2020. Based on stakeholder feedback, ICER may revise key assumptions and findings for its Evidence Report, which will be published on March 12, 2020. 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 26, 2020.

ICER is committed to engaging with all stakeholders in a thorough and transparent manner. During this review, ICER has spoken with patients, clinical experts, insurers, and manufacturers. Public comments were also accepted on a Draft Scoping Document. The current draft report incorporates input received from patients, clinicians, and other stakeholders during each of these opportunities for engagement.

Submit a Public Comment

The Draft Evidence Report and Draft Voting Questions are now open to public comment until 5 PM ET on February 20, 2020. All stakeholders are invited to submit formal comments by email to publiccomments@icer.org. Guidelines for submitting public comments, including formatting specifications, are available on ICER’s website. ICER’s Manufacturer Engagement Guide and Patient Participation Guide provide additional detail on what types of information may be most informative to the report.

ICER will review all comments and incorporate any necessary changes in the Evidence Report and Revised Voting Questions that will be posted on or about March 12, 2020. All comments and ICER’s response to comments will be posted publicly along with the Evidence Report.

Register for the Public Meeting

The Evidence Report will be the subject of a public meeting of the New England CEPAC on March 26, 2020 in Boston, MA. During the meeting, the independent council will vote on key questions raised in the report. Registration for the public meeting and live webcast is now open.

Register to Make an Oral Comment

During the public meeting, there will be a limited amount of time available for interested stakeholders to make an oral comment on the report. Requests to submit oral comments must be emailed to publiccomments@icer.org by 5 PM ET on February 20, 2020. Individuals who wish to deliver oral comments must separately register to attend the meeting. For more information about registering for oral comment, please visit our website.

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.