ICER to Assess Value of Novartis Drug for Rare Blood Disorder

The Institute for Clinical and Economic Review (ICER) will assess the comparative clinical effectiveness and economic value of Novartis’s drug iptacopan for treating paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic blood disease.

PNH is caused by a genetic mutation that results in the creation of defective red blood cells susceptible to premature destruction by the complement system, a part of the human immune system. The condition may cause back and abdominal pain, blood clots, headache, weakness, fatigue and shortness of breath and affects one to two persons per million. Most patients are diagnosed in their 30s and have an average survival rate of 10 years.

An independent evidence review panel will deliberate and vote on ICER’s findings at a meeting of the California Technology Assessment Forum in February 2024.

Comments are being accepted until Aug. 14.

Read the draft scoping document here.

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