For patients with HAE 1/2, prophylaxis with Cinryze, Haegarda, and Takhzyro showed significant clinical benefits by reducing the number and severity of HAE attacks, without significant adverse events when compared with no long-term prophylaxis. However, the data were not sufficient to distinguish any drug as clinically superior to the other. In the absence of long-term safety data for lanadelumab-flyo, which targets a different pathway than the C1-INH products, we are less certain about its overall net health benefit. In the base case and at current pricing, all 3 drugs were judged to represent low or intermediate long-term value for money. However, these findings were highly influenced by the baseline attack rate, with improved cost-effectiveness for a population with a higher mean attack rate per month and by the cost of treating patients with ondemand therapy, with higher on-demand treatment costs leading to improved cost-effectiveness for prophylaxis. Further efforts are needed to help align the price of these treatments with their demonstrated benefit in order to ensure sustainable access to high-value care for all patients.
