Hereditary Angioedema (HAE) is a rare genetic disorder that causes painful attacks of swelling in the face, hands, feet, and stomach, as well as potentially life-threatening swelling of the throat. Most HAE is caused by a deficiency (Type 1 HAE) or dysfunction (Type 2 HAE) of a protein called C1 esterase inhibitor. Attacks can last for up to four days, and can be spontaneous or triggered by stress, medical procedures, and certain medications like oral
contraceptives or ACE inhibitors. Attacks can occur rarely or as often as once every few days. Because of their severity and unpredictability, attacks can significantly reduce a patient’s quality of life.ICER assessed the comparative clinical effectiveness and value of therapies for hereditary angioedema, a rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa.
Interventions of Interest:
- lanadelumab (Takhzyro™,Shire)
- C1 esterase inhibitors (Haegarda®, CSL Behring; Cinryze®, Shire)
*Ruconest (Pharming) was removed from the ICER review in September 2018 because the treatment is no longer under consideration for FDA approval for long-term prophylaxis.
Below you will find the final documents from the assessments review process: