Lanadelumab in Hereditary Angioedema: Real-World Outcomes and Implications for Access Practices in Europe

Background/Objectives: Lanadelumab is approved in the EU for long-term HAE prevention in patients aged ≥2 years. While trials show high efficacy, real-world data on reimbursement and outcomes are limited. This study presents real-world clinical results in Poland and compares reimbursement criteria across European countries, assessing how effectiveness influences access restrictions. Methods: This retrospective analysis examined patients in the Polish drug program for lanadelumab. It collected demographics, disease features, attack frequency, and rescue medication use before and after at least six months of treatment. Additionally, a review of European reimbursement policies was conducted using health technology assessments, policy documents, and literature. Results: The data of 72 patients with HAE with C1 inhibitor deficiency were analyzed. The median follow-up was 20.0 months (IQR 15.0-25.0). The median baseline attack frequency was 15 over 6 months. After 6 months of lanadelumab, attacks dropped to 0 (IQR 0.0-0.0; p < 0.001), with 77.8% achieving >90% reduction. Most remained attack-free beyond 6 months; on-demand medication use decreased from 16 to 0 doses (p < 0.001). Outcomes persisted beyond 6 months. No demographic or baseline variables predicted response. No discontinuations due to adverse events. Reimbursement criteria across Europe vary, from broad access to restrictions based on attack frequency or treatment response, with differences in care settings. Conclusions: Data from Poland confirm lanadelumab nearly eliminates severe HAE attacks in practice, regardless of attack frequency. Some European reimbursement models may exclude patients who could benefit. Using real-world effectiveness evidence in policies could improve access and outcomes for HAE patients.