Evaluating the Economic Burden: Lifetime Direct and Indirect Costs of Managing Alpha-1 Antitrypsin Deficiency for Patients With the PiZZ Genotype Receiving Augmentation Therapy in the United States

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Abstract RATIONALE: Alpha-1 antitrypsin deficiency (AATD) is a rare genetic condition characterized by accumulation of the protein alpha-1 antitrypsin (AAT) in the liver. Low levels of this protein in circulation predispose individuals to lung and liver disease. Patients with AATD-associated lung disease typically have early onset emphysema or bronchiectasis and are often managed with medications for chronic obstructive pulmonary disease and/or augmentation therapy to replace the deficient protein. This study aimed to estimate the economic burden affecting patients, society, and the healthcare system by assessing lifetime direct and indirect costs of AATD patients (PiZZ genotype) receiving augmentation therapy in the United States. METHODS: A literature review was performed, and a cohort-level Markov model was constructed to simulate healthcare resource utilization from diagnosis (age 55) until death in the United States (U.S.). Three health states were modeled: 1) alive on augmentation therapy, 2) post-lung transplant, and 3) death. Utilization and costs were calculated for hospitalizations, outpatient visits, augmentation therapy (Prolastin-C 1000 mg / 20 ml vials; highest U.S. market share), other medications, oxygen, lab tests, and scans. The annual probability of lung transplant was considered, and the costs of lung transplant/post-transplant management were included. Prolastin-C's wholesale-acquisition cost (Red Book) was used, and we ran the model for 5 Prolastin-C vials per week (Base Case) and 6 vials to account for differences in body weight. Other direct costs were sourced from the literature and Medicare fee schedule, while indirect costs were estimated based on productivity losses and early retirement (8.9 years earlier). All costs were adjusted to reflect the commercial payer perspective in 2024 values and adjusted for time value using a 3% discount rate. A scenario analysis of higher mortality was conducted where the Mortality Relative Risk (MRR) was set to 3.2 versus 1.91 in the Base Case. RESULTS: For the Base Case (5 Prolastin-C vials/week), lifetime direct costs were $4,004,870 and indirect costs were $1,296,517, and lifetime overall (direct and indirect) costs were $5,301,387 (undiscounted). In the Base Case, the annual direct cost for patients in the alive on augmentation therapy health state was $203,839. Additional scenarios using a higher mortality relative risk, 6 vials/week, and earlier/older age of diagnosis along with associated costs, can be found in Table 1. CONCLUSIONS: AATD (PiZZ) imposes a significant economic burden, including both direct and indirect costs.

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