ICER Utilizes Real World Evidence to Update Assessment of HAE Attack Prophylactics Takhzyro, Haegarda, Cinryze

BOSTON, MA, United States – The Institute for Clinical and Economic Review (ICER) will be utilizing real-world evidence (RWE) to update its previous assessment of long-term prophylactics for hereditary angioedema (HAE) attacks, Takeda’s Takhzyro (lanadelumab) and C1 esterase inhibitors CSL Behring’s Haegarda and Takeda’s Cinryze.

ICER’s updated assessment will leverage observational RWE analyzed by Aetion, who will generate RWE from Optum’s de-identified Clinformatics Data Mart, Commercial and Medicare Advantage claims database. Clinical experts and the manufacturers behind these therapies were given several weeks to respond to ICER’s request for data and comment on its new approach, which was considered in the agency’s Revised Protocol and Modeling Analysis Plan.

The joint effort is part of the two organizations’ ongoing collaboration, as well as ICER’s push to expand use of RWE as part of its value assessments. Additionally, Aetion has analyzed RWE to support ICER’s early assessments of sickle cell disease treatments.

John Campbell, PhD, MS, ICER’s Senior Vice President of Health Economics, explained that in ICER’s previous evaluation of HAE therapies, economic models 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.

“By defining the baseline demographics and utilization characteristics of patients who start HAE prophylaxis, evaluating HAE attack rates before initiation of prophylaxis, and updating our health cost assumptions accordingly, ICER aims to further clarify the overall value of these HAE therapies in real-world settings,” said Campbell.

Regarding the utilization of RWE, Carolyn Magill, CEO of Aetion, said, “Pilots like this will help inform efforts of HTA organizations around the world in their quest to evaluate how and when to base their decisions on RWE, while advancing how we as a health care industry—and a society—determine value.”