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.

The cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks.

Interventions of Interest:

  • lanadelumab (Takhzyro™,Shire)
  • C1 esterase inhibitors (Haegarda®, CSL Behring; Cinryze®, Shire)

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*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.

Final Documents

Below you will find the final documents from the assessments review process:

“Treatment for HAE attacks is very expensive, as are the prophylactic therapies used to prevent such attacks, and so the overall cost of prophylaxis includes the savings incurred from less on-demand treatment. The economic models produced for this report were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy. Similarly, relatively small changes in the price of prophylactic therapy greatly improved its cost-effectiveness. Insurers and manufacturers should work together to achieve appropriate access to these important therapies at a price that patients and society can afford.”