OBJECTIVES: Acute hepatic porphyria (AHP) refers to a family of rare, metabolic diseases that includes four types, acute intermittent porphyria (AIP) being the most common. An economic analysis of EXPLORE, the largest prospective, multinational, study in AHP patients experiencing recurrent attacks, estimated an average total of annual healthcare costs between $400,000 to $650,000 per patient (using US cost data). This study aimed to estimate healthcare resource utilization and total healthcare costs of AIP patients in the United States, using a nationally representative health care database.

METHODS: This retrospective analysis utilized the IBM® MarketScan® Commercial Claims and Medicare Supplemental Databases. Patients with at least one claim for AIP (ICD-10 diagnosis code E80.21) and at least one claim for hemin use (HCPCS code J1640) between October 1, 2015 – June 30, 2018 were selected for analyses. The subset of AIP patients with continuous enrollment for at least one year following their first observed AIP diagnosis (index date) were identified for the assessment of baseline characteristics and post-diagnosis (post-index) outcomes. Means were reported as per patient per year (PPPY).

RESULTS: A total of 19 unique patients with AIP receiving hemin were identified, 89.5% of whom were female, with a mean (SD) age of 41.1 (13.9) years. Mean observation time of identified diagnosed patients was 1.8 years. Patients had a mean (SD) of 3.0 (2.0) attacks PPPY. The majority had ≥1 hospitalization (84.2%) and emergency department (ED) visit (57.9%), with a mean (SD) of 1.9 (1.9) admissions and 3.8 (6.0) ED visits PPPY, respectively. Mean (SD) total healthcare costs were $405,035 ($422,226) PPPY.

CONCLUSIONS: Results from this national representative healthcare claims database demonstrated a high rate of healthcare utilization associated with a high average annual expenditure per AIP patient receiving hemin in the United States.