Budget Impact Analysis of Gene Therapies for Recessive Dystrophic Epidermolysis Bullosa

Although recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare disease, two recently approved gene therapies for the condition could have large financial implications for US payers. The estimated budget impact of these therapies, beremagene geperpavec (B-VEC; Vyjuvek) and prademagene zamikeracel (PZ-CEL; Zevaskyn), was substantial across a range of uptake and utilization scenarios, highlighting the challenges high-cost gene therapies can create for health care budgets even when the eligible patient population is very small.