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AdminApr 12, 20214 min read

ICER to use Aetion observational RWE to update value assessment of treatments for hereditary angioedema

  • New ICER pilot will incorporate more than 24 months of observational real-world demographic, utilization, and cost data to further clarify therapies' value
  • Through continued use of the Aetion Evidence Platform®, ICER advances the use of transparent, replicable real-world evidence in health technology assessment
BOSTON and  NEW YORKApril 12, 2021 /PRNewswire/ -- The Institute for Clinical and Economic Review ( ICER) today announced it is using observational real-world evidence (RWE) to update its  2018 assessment of three therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks: lanadelumab (Takhzyro®, Takeda), and two C1 esterase inhibitors (Haegarda®, CSL Behring; and Cinryze®, Takeda). ICER's updated assessment will leverage observational RWE analyzed by Aetion, as part of the  ongoing collaboration between the two organizations, and consistent with ICER's  commitment to expand use of RWE to complement other sources of information used in its value assessments. Aetion researchers will use Optum's de-identified Clinformatics® Data Mart, Commercial and Medicare Advantage claims database, to generate the RWE. In addition to this specific pilot to use RWE to update ICER assessments completed more than 24 months ago, Aetion has analyzed RWE to support ICER's earlier assessment of treatments for  sickle cell disease. "As ICER  noted throughout our earlier assessment of these HAE therapies, our 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," said  Jon Campbell, PhD, MS, ICER's Senior Vice President of Health Economics. "Three years later, we hope to use observational RWE from the Aetion Evidence Platform® to help close some of these information gaps. 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. We appreciate Aetion's continued partnership as we work together to set new standards for how decision-grade RWE can be appropriately applied alongside high-quality clinical trial data to better inform the consequential decisions that biopharma manufacturers, insurers, and other stakeholders make every single day around drug pricing and patient access." "We commend ICER for continuing to improve the ways in which RWE can be integrated responsibly into US health technology assessment," said  Carolyn Magill, CEO of Aetion. "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." As part of this pilot, ICER has engaged clinical experts and the manufacturers of these HAE therapies, and each manufacturer was provided several weeks to respond to ICER's data request and comment on the proposed approach. ICER, Aetion, and ICER's academic collaborators from the  University of Washington incorporated manufacturer and clinical expert feedback into the revised Research Protocol and Modeling Analysis Plan. As broad stakeholder input was included within the initial assessment of these therapies, ICER will be proceeding with a condensed timeline for incorporating RWE into this re-assessment, and we expect to publish our RWE update on  July 27, 2021. About ICER The Institute for Clinical and Economic Review ( ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER's reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system. ICER's reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum ( CTAF), the Midwest Comparative Effectiveness Public Advisory Council ( Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council ( New England CEPAC). These independent panels review ICER's reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit  ICER's website. About Aetion Aetion is a health care analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value, across the product life cycle. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs health care’s most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation into health care’s modern era. Aetion is based in New York City, and backed by investors including New Enterprise Associates (NEA), Flare Capital Partners, Lakestar, Greenspring Associates, Town Hall Ventures, McKesson Ventures, Sanofi Ventures, EDBI, Amgen Ventures, Johnson & Johnson Innovation—JJDC, Inc., UCB, and Horizon Health Services, Inc. Learn more at and follow us at @aetioninc.