Interventions of Interest:

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

In 2018, 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.

In 2021, ICER launched a new initiative to re-assess these treatments using observational real-world evidence to help remove some of the data uncertainty we identified during our 2018 review.

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Final Documents from 2018 Assessment and 2021 RWE Update

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.”

On October 25, 2018, the California Technology Assessment Forum convened to deliberate over the clinical evidence, the other contextual considerations, and the cost-effectiveness modeling: