The aim of this project, which was commissioned by NICE through the NIHR HTA Programme as part of the NICE’s Single Technology Appraisal process, was to review the submission of the pharmaceutical company Shire Plc on the evidence for the clinical and cost-effectiveness of lanadelumab for preventing recurrent attacks of hereditary angioedema (HAE).

The clinical effectiveness evidence submitted by the company consists of a phase three, double-blind randomised controlled trial (the HELP-03 trial) that compared subcutaneous lanadelumab with placebo for long-term prophylactic treatment of acute attacks in patients with Type I or Type II HAE, and the open-label extension study (HELP-04). Comparator evidence was limited to the CHANGE crossover study, which tested intravenous C1-INH against placebo.

The Aberdeen HTA Group acted as the Evidence Review Group (ERG) for the project and provided an independent critique of the clinical and cost-effectiveness evidence submitted by the company. Based on the findings of the company submission and of the ERG’s critique, NICE have released appropriate clinical guidance.

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Miriam Brazzelli;