Budget Impact Analysis of Birch Triterpenes for Patients With Epidermolysis Bullosa From a United States Perspective

OBJECTIVES: Epidermolysis bullosa (EB) is a group of severe genetic conditions leading to increased skin fragility and chronic wounds. Birch bark extract gel (known as birch triterpenes) received FDA approval in December 2023 for treating patients with dystrophic and junctional EB (DEB+JEB). Routine care for EB comprises a range of dressings and complex wound management strategies. A budget impact analysis (BIA) was developed from a US payer perspective to estimate total drug/healthcare expenditures and incremental budget impact of introducing birch triterpenes as an add-on to routine care.

METHODS: An Excel-based BIA with a time horizon of 1-5 years was developed. Model inputs were sourced from literature and clinical trial data, with costs adjusted to 2024 US dollars. The modelling approach, inputs and assumptions were validated by a clinician and two ex-payers. The base case considered a mix of payers (commercial, Medicare and Medicaid) and covered medical costs of adding birch triterpenes to routine care in DEB+JEB patients over 3 years (drug acquisition/administration, home health, reimbursed bandages, and other healthcare resource use). Scenario analyses estimated potential medical cost offsets due to reduced wound burden with treatment, and inclusion of indirect costs (productivity and out-of-pocket costs).

RESULTS: In a plan of 1 million lives, there are an estimated 0.4 patients with JEB and 2.8 patients with DEB. In the base case, 1.36 patients are estimated to receive birch triterpenes alongside routine care. Adding birch triterpenes to routine care increased the 3-year total plan costs by an estimated $1,758,806, with a cost per member per month of $0.05. Total 3-year costs before and after the introduction of birch triterpenes are estimated at $286,221 and $2,044,061, respectively.

CONCLUSIONS: EB is an ultra-rare and debilitating disease. As such, introduction of birch triterpenes as a new treatment alongside routine care has a manageable budget impact in a 3-year plan.