Takeda, CSL hereditary angioedema meds need steep discounts following real-world evidence review, ICER says

With real-world data in hand, the Institute for Clinical and Economic Review (ICER) is retooling its cost-effectiveness models for three rare disease drugs. And the new discounts it has in mind are steep.

The meds in question—Takeda’s Takhzyro and Cinryze, plus CSL Behring’s Haegarda—all carry FDA approvals for long-term prevention of hereditary angioedema (HAE) attacks. Using real-world evidence (RWE) garnered with the help of Aetion and the University of Washington, ICER found patients suffered fewer baseline HAE attacks in the real world than investigators noticed in clinical studies.