Societal implications of the pharmacoeconomics of α1-antitrypsin deficiency

The use of intermittent augmentation therapy for patients with alpha(1)-antitrypsin deficiency assists in slowing disease progression and may, as a result, reduce the likelihood of hospitalization and significant clinical events. There are significant cost and access issues related to current therapy management, since currently marked augmentation therapy is expensive and there have been periodic shortages. The high cost, access and reimbursement concerns have prompted questions about the potential pharmacoeconomic advantages of future treatments. In this review, we provide an overview of published research findings concerning the effectiveness of current therapy, reimbursement and cost issues and potential future advances in treatment for alpha(1)-antitrypsin deficiency.