Linking Specialty Pharmacy Data With Healthcare Claims Data in a Rare Disease: Comprehensively Characterizing Patients With Hereditary Angioedema Treated With Berotralstat
Author(s)
Sean D. MacKnight, MScPH1, Tracy Yee, PhD, MPH2, François Laliberte, MA1, Colleen Spencer, MA1, Sandra Nestler-Parr, MPhil, MSc, PhD2, Patrick Gillard, PharmD, MS2.
1Groupe d'analyse SRI, Montreal, QC, Canada, 2BioCryst Pharmaceuticals, Durham, NC, USA.
1Groupe d'analyse SRI, Montreal, QC, Canada, 2BioCryst Pharmaceuticals, Durham, NC, USA.
OBJECTIVES: To link Optime Care Specialty Pharmacy Data with Komodo Healthcare Map data to characterize patients treated with Orladeyo® (berotralstat) for long-term prophylaxis of hereditary angioedema (HAE) in the United States.
METHODS: Optime Care Specialty Pharmacy data (Dec. 3, 2020 - Jan. 31, 2024) was linked with Komodo Healthcare Map administrative claims data (Oct. 1, 2015 - Jan. 31, 2024) at the patient level using de-identified Datavant patient tokens based on patients’ date of birth, sex, and first and last name, accounting for spelling variations. Optime Care is the sole dispenser of berotralstat in the United States, and the database includes self-assessments of HAE attacks and laboratory results (C1 inhibitor [C1INH] levels, C1INH function, and C4 levels) for HAE type identification. Patients selected into the study population had ≥2 berotralstat dispensings (first=index) recorded in Optime data, had ≥6 months of continuous insurance enrollment pre-index in Komodo data, and were ≥12 years of age at index.
RESULTS: The study population consisted of 523 patients. Mean age was 41 years, 75.0% were female, and 69.4% had commercial insurance at index; 37.5% had C1INH deficiency (HAE type 1 or 2) and 30.8% had HAE with normal C1INH, with the remainder having undeterminable HAE type. Among the subset of patients with self-assessments of HAE attacks (n=435), 23.9% had 0 attacks/month, 24.8% had 1 attack/month, 26.4% had 2-4 attacks/month, and 24.8% had ≥5 attacks/month in the 3 months pre-index. In the 6 months pre-index, 16.3% and 11.1% of patients had an angioedema-related hospitalization and emergency department visit, respectively.
CONCLUSIONS: The innovative linking of Optime Care Specialty Pharmacy and Komodo Healthcare Map data allows for comprehensive characterization of patients with HAE treated with berotralstat. The linked database enables future research in a rare disease to address knowledge gaps and support decision-making by healthcare practitioners and payers.
METHODS: Optime Care Specialty Pharmacy data (Dec. 3, 2020 - Jan. 31, 2024) was linked with Komodo Healthcare Map administrative claims data (Oct. 1, 2015 - Jan. 31, 2024) at the patient level using de-identified Datavant patient tokens based on patients’ date of birth, sex, and first and last name, accounting for spelling variations. Optime Care is the sole dispenser of berotralstat in the United States, and the database includes self-assessments of HAE attacks and laboratory results (C1 inhibitor [C1INH] levels, C1INH function, and C4 levels) for HAE type identification. Patients selected into the study population had ≥2 berotralstat dispensings (first=index) recorded in Optime data, had ≥6 months of continuous insurance enrollment pre-index in Komodo data, and were ≥12 years of age at index.
RESULTS: The study population consisted of 523 patients. Mean age was 41 years, 75.0% were female, and 69.4% had commercial insurance at index; 37.5% had C1INH deficiency (HAE type 1 or 2) and 30.8% had HAE with normal C1INH, with the remainder having undeterminable HAE type. Among the subset of patients with self-assessments of HAE attacks (n=435), 23.9% had 0 attacks/month, 24.8% had 1 attack/month, 26.4% had 2-4 attacks/month, and 24.8% had ≥5 attacks/month in the 3 months pre-index. In the 6 months pre-index, 16.3% and 11.1% of patients had an angioedema-related hospitalization and emergency department visit, respectively.
CONCLUSIONS: The innovative linking of Optime Care Specialty Pharmacy and Komodo Healthcare Map data allows for comprehensive characterization of patients with HAE treated with berotralstat. The linked database enables future research in a rare disease to address knowledge gaps and support decision-making by healthcare practitioners and payers.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
MSR138
Topic
Methodological & Statistical Research
Topic Subcategory
Missing Data
Disease
Rare & Orphan Diseases