Mixed ICER findings on treatments for hereditary angioedema

US cost-effectiveness watchdog the Institute for Clinical and Economic Review (ICER) has released an Evidence Report assessing the comparative clinical effectiveness and value of therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks.

HAE is an ultra-rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. ICER’s report reviews three therapies for the prevention of HAE attacks: lanadelumab (Takhzyro, from Shire [LSE: SHP]), and two C1 inhibitors (Haegarda, CSL Behring GmbH; and Cinryze, Shire). The ICER’s earlier draft report also included an additional C1 inhibitor, Ruconest (Pharming [Euronext: PHARM]), which has since been removed from the assessment because the treatment is no longer under consideration for Food and Drug Administration approval for long-term prophylaxis.

“While long-term prophylaxis with all three therapies resulted in fewer acute attacks and improved quality of life for people living with hereditary angioedema, these improvements come at a substantial financial cost,” said Dr David Rind, chief medical officer at ICER. “At their current prices, these treatments do not meet traditional cost-effectiveness thresholds. However, decision makers will need to be aware that even minor adjustments in our key assumptions could result in substantially different cost-effectiveness results, particularly for the two therapies that are administered subcutaneously, lanadelumab and Haegarda. During our public meeting, we will discuss these therapies and the limitations inherent to this particular analysis,” he added.