Prevalence, Real-World Healthcare Resource Utilization, and Costs of Patients With Paroxysmal Nocturnal Hemoglobinuria in the US: A Retrospective Claims Database Analysis
Author(s)
Tantravahi S1, Latremouille-Viau D2, Desai R3, Lee S4, Paulose J4, Geevarghese A4, Guérin A2, Seshasayee S3, Tabatabaeepour N3, Chanpura M5, Yen G4
1Huntsman Cancer Institute, Salt Lake City, UT, USA, 2Analysis Group, Inc., Montreal, QC, Canada, 3Analysis Group, Inc., Boston, MA, USA, 4Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA, 5Novartis Pharmaceuticals Corporation, Lawrence Township, NJ, USA
Presentation Documents
OBJECTIVES: To estimate the 5-year prevalence of paroxysmal nocturnal hemoglobinuria (PNH) and describe the healthcare resource utilization (HRU) and direct healthcare costs associated with C5 inhibitors (C5i; eculizumab or ravulizumab) among commercially insured patients in the US.
METHODS: The 5-year prevalence of adult (age ≥18 years) patients with PNH (ICD-10-CM code D59.5) in the IQVIA PharMetrics® Plus was estimated (2018-2022). A retrospective cohort study was also conducted in adult patients with PNH treated with C5i and ≥3 months of continuous health plan coverage following the first claim for C5i (index date) (2011-2022). PNH-related (PNH diagnosis or C5i procedure codes) HRU and direct healthcare costs were assessed from the index date until the earliest of treatment discontinuation/end of continuous health plan coverage/end of data.
RESULTS: From 2018-2022, the 5-year prevalence of PNH was 2.4 per 100,000 persons; 30.0% were treated with ≥1 complement inhibitors (eculizumab: 16.4%, ravulizumab: 21.9%, or pegcetacoplan: 2.2%). From 2011-2022, 371 patients treated with C5i (median age: 40 years; female: 55.3%; eculizumab: 53.9%; ravulizumab: 46.1%) were followed for a mean (SD) of 19.3 (16.9) months. The annual incidence rate of blood transfusion and breakthrough hemolysis (BTH) in any setting were 1.2 and 4.5 per person per year (PPPY), respectively. The mean (SD) costs of blood transfusion and BTH were $1,483 ($7,148) and $24,200 ($35,413) per event, respectively. Among patients treated with C5i, the annual total PNH-related costs PPPY were estimated at $660,533 for the first year and $633,984 for subsequent years; of which treatment costs accounted for 94.3%-94.6%.
CONCLUSIONS: Findings of this study indicated the 5-year prevalence of PNH to be 2.4 per 100,000 persons in commercial claims. Despite treatment with C5i associated with high annual PNH-related costs, patients with PNH still exhibited BTH and required blood transfusions, potentially indicating an unmet clinical need for more effective treatments.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 6, S1 (June 2024)
Code
EE452
Topic
Economic Evaluation, Epidemiology & Public Health
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)