The following is a summary of “Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa,” published in the January 2024 issue of Dermatology by Raymakers, et al.
The approval of the first topical gene therapy, beremagene geperpavec (B-VEC), by the US Food and Drug Administration (FDA) in May 2023 presented a significant advancement in treating both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB), particularly for patients with limited therapeutic options. For a study, researchers sought to estimate US spending on B-VEC therapy for treating autosomal recessive and autosomal dominant DEB.
Using data from the National Epidermolysis Bullosa Registry, the economic evaluation estimated the current population of US patients with autosomal dominant and autosomal recessive DEB. The study aimed to estimate US spending on B-VEC therapy from an all-payer perspective during 1- and 3-year periods following FDA approval. The study estimated overall spending on B-VEC in the first year and over 3 years post-FDA approval. Various sensitivity analyses with different assumptions about the eligible patient population and the cost of therapy were conducted. Additionally, lifetime total costs of treatment per patient were estimated.
In the first year after FDA approval, an estimated 894 US patients with DEB were eligible for treatment with B-VEC. The total expenditure for B-VEC therapy was estimated at $268 million (range, $179 million-$357 million) during the first year and $805 million (range, $537 million-$1.1 billion) over 3 years. The estimated lifetime total costs per patient were $15 million (range, $10 million-$20 million) for autosomal recessive DEB and $17 million (range, $11 million-$22 million) for autosomal dominant DEB.
The economic evaluation’s findings suggested that the FDA’s broad indication for the use of B-VEC in treating both autosomal recessive and autosomal dominant DEB will have significant implications for payers.
Reference: jamanetwork.com/journals/jamadermatology/article-abstract/2814373