Posted by Michael Wonder on 11 Oct 2018
ICER finds treatments prevent hereditary angioedema attacks, but cost-effectiveness uncertain and sensitive to frequency of attacks
11 October 2018 - As with all treatments for ultra-rare conditions, judgments of overall value require consideration of the broader patient benefits and contextual issues that will be discussed at the October 25 public meeting.
The ICER today 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 characterised 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, Shire), and two C1 inhibitors (Haegarda, CSL Behring; and Cinryze, Shire).
ICER’s earlier draft report also included an additional C1 inhibitor, Ruconest (Pharming), which has since been removed from the assessment because the treatment is no longer under consideration for FDA approval for long-term prophylaxis.
