Identification of Real-World Data to Study HAE Unmet Need: A Targeted Literature Review
Author(s)
Temitope Adebayo-Ojo, PhD1, Mateo Delclaux Rodriguez-Rey, BSc1, Meena Venkatachalam, BSc, MSc1, Madeleine Thursfield, MA2, Charlotte Heeks, BSc, MSc3.
1Lumanity, London, United Kingdom, 2Otsuka Europe, London, United Kingdom, 3Otsuka Europe, Windsor, United Kingdom.
1Lumanity, London, United Kingdom, 2Otsuka Europe, London, United Kingdom, 3Otsuka Europe, Windsor, United Kingdom.
OBJECTIVES: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable and debilitating swelling attacks, adversely affecting patients' quality-of-life (QoL). There is no disease-specific code to diagnose HAE, making identification of patients in secondary real-world data (RWD) challenging. This study aimed to conduct a targeted literature review to identify secondary RWD sources for future research on HAE attacks, healthcare resource utilization, treatment patterns, QoL, and adverse events in Europe.
METHODS: A search strategy was developed in PubMed for the period 2015 - 2025 to identify RWE literature on all HAE types, based on disease and data source specific terms, existing HAE treatments, and outcomes of interest. Studies referencing only US databases were excluded. Additionally, desk-based research was conducted to identify conference publications, disease-specific registries, HAE centers, claims data, and national health databases that may not have been published.
RESULTS: We reviewed a total of 98 sources of information: 72 selected from 156 screened articles and 26 identified through additional desk research. Of these 98, 20 referenced single-site studies, 22 multi-site studies, 17 disease-specific registries referenced across 46 publications, 5 administrative/claims data sources, and 5 sources categorized as other data types. Among the 17 registries, 5 were prioritized for an in-depth assessment of the data due to expected suitability for secondary RWE research in HAE based on their coverage and data elements. The assessment of data focused on the availability, structure of variables, and HAE patient counts.
CONCLUSIONS: Results indicate that RWE research in HAE is challenging and primarily uses hospital or disease-specific registry data, with limited use of large administrative databases. Identifying appropriate secondary RWD will be crucial for advancing HAE research and patient care. This review identified 5 disease specific registries as potentially valuable sources for HAE research.
METHODS: A search strategy was developed in PubMed for the period 2015 - 2025 to identify RWE literature on all HAE types, based on disease and data source specific terms, existing HAE treatments, and outcomes of interest. Studies referencing only US databases were excluded. Additionally, desk-based research was conducted to identify conference publications, disease-specific registries, HAE centers, claims data, and national health databases that may not have been published.
RESULTS: We reviewed a total of 98 sources of information: 72 selected from 156 screened articles and 26 identified through additional desk research. Of these 98, 20 referenced single-site studies, 22 multi-site studies, 17 disease-specific registries referenced across 46 publications, 5 administrative/claims data sources, and 5 sources categorized as other data types. Among the 17 registries, 5 were prioritized for an in-depth assessment of the data due to expected suitability for secondary RWE research in HAE based on their coverage and data elements. The assessment of data focused on the availability, structure of variables, and HAE patient counts.
CONCLUSIONS: Results indicate that RWE research in HAE is challenging and primarily uses hospital or disease-specific registry data, with limited use of large administrative databases. Identifying appropriate secondary RWD will be crucial for advancing HAE research and patient care. This review identified 5 disease specific registries as potentially valuable sources for HAE research.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
RWD99
Topic
Epidemiology & Public Health, Real World Data & Information Systems
Topic Subcategory
Distributed Data & Research Networks
Disease
Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases