NEWS | ICER Releases Draft Evidence Report on Treatment for Paroxysmal Nocturnal Hemoglobinuria

The Institute for Clinical and Economic Review (ICER) released on December 5, 2023, a Draft Evidence Report assessing the comparative clinical effectiveness and value of iptacopan (Novartis) and danicopan (Alexion Pharmaceuticals) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 

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.

Register for ICER’s Early Insights Webinar

On December 20th, 2023, as part of ICER’s Early Insights Webinar Series, ICER’s Vice President of Research Foluso Agboola, MBBS, MPH will present the initial findings of this draft report.  This webinar is exclusively available to all users of the ICER Analytics platform; registration for the webinar is now open.

Submit a Public Comment

The Draft Evidence Report and Draft Voting Questions are now open to public comment.  All stakeholders are invited to submit formal comments by email to, which must be received by 5 PM ET on January 9, 2024.

Register for the Virtual Public Meeting

The Evidence Report will be the subject of a virtual public meeting of CTAF on February 16, 2024.  During the meeting, the independent council will vote on key questions raised in the report.  Registration for the virtual public meeting is now open.

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.

Download the report
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