The Institute for Clinical and Economic Review (ICER), a non-profit research institute that evaluates potential therapies and medical devices, announced it will turn to observational real-world evidence (RWE) analyzed by Aetion to update its 2018 assessments of three potential angioedema treatments. The treatments are intended for long-term prophylaxis against hereditary angioedema (HAE) attacks: Takeda’s lanadelumab (Takhzyro), and two C1 esterase inhibitors: CSL Behring’s Haegarda, and Takeda’s Cinryze).
The updated assessment is part of an ongoing research partnership between the two organizations, in line with ICER’s stated commitment to expand the use of RWE in its value assessments. According to the companies, Aetion researchers will use Optum’s de-identified Clinformatics Data Mart, Commercial, and Medicare Advantage claims database, to generate the RWE.
ICER senior vice president of health economics Jon Campbell commented that during its earlier assessment of the HAE therapies, the economics were sensitive to small changes in assumptions in a number of areas, such as the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy.
“Three years later, we hope to use observational RWE from the Aetion Evidence Platform to help close some of these information gaps,” Campbell remarked. “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.”
Carolyn Magill, CEO of Aetion, said the collaboration will help further ICER’s commendable mission to continually look for ways that life sciences professionals can make better use of RWE.
“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,” Magill said.
As part of this pilot, ICER has engaged clinical experts and the manufacturers of the HAE therapies. Each of the pharma firms was given a number of weeks to respond to the ICER data request, and to comment on the approach being proposed. ICER, Aetion, and academic collaborators from the University of Washington incorporated manufacturer and clinical expert feedback into the revised Research Protocol and Modeling Analysis Plan.
ICER included broad stakeholder input in the initial assessment of the three potential therapies; the organization plans to go ahead with a condensed timeline for integrating RWE in the re-assessment, with the goal of publishing the RWE update on July 27.