Hereditary angioedema (HAE) is a rare, debilitating condition marked by unpredictable and potentially life-threatening swelling episodes. While long-term prophylactic therapies have significantly improved disease management, selecting the optimal treatment remains complex—particularly in the absence of direct head-to-head clinical trials.
To help address this gap and support more informed clinical decision-making, the Value and Evidence team at EVERSANA (Sarah Walsh, Anja Haltner, Meaghan Bartlett and Imtiaz Samjoo), in collaboration with CSL Behring (John Sears, Yinglei Li, Maebh Kelly, Simona Gavata-Steiger, Chiara Nenci, Iris Jacobs, Ingo Pragst, and Neelanjana Ray), conducted a matching-adjusted indirect comparison (MAIC) of two advanced prophylactic options: garadacimab and lanadelumab.
By adjusting for differences in baseline characteristics across the phase II (NCT03712228), phase III VANGUARD (NCT04656418), and phase III HELP (NCT02586805) trials, this analysis enabled a more balanced comparison of efficacy and health-related quality-of-life outcomes. The findings showed that garadacimab was associated with a significantly lower number of moderate and/or severe HAE attacks compared to lanadelumab administered biweekly. Fewer patients treated with garadacimab experienced moderate or severe attacks, and patient-reported outcomes—measured by AE-QoL scores—also favored garadacimab, suggesting a potential reduction in overall disease burden.
This work highlights the critical role of advanced indirect comparison methodologies in rare disease research, where traditional head-to-head randomized trials may not be feasible. As the HAE treatment landscape continues to evolve, such evidence can play a pivotal role in guiding personalized care decisions.
For a deeper dive into the analysis, access the full article in the Journal of Comparative Effectiveness Research here.
Author
Purva Barot is a Manager in the Evidence Synthesis group at EVERSANA, with deep expertise in systematic and targeted literature reviews, medical writing, and feasibility assessments for indirect treatment comparisons. She has contributed to…