Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts.
Harvard Medical School, Boston, Massachusetts.
JAMA Dermatol. 2024 Mar 1;160(3):297-302. doi: 10.1001/jamadermatol.2023.5857.
New gene therapies can offer substantial benefits to patients, particularly those with rare diseases who have few therapeutic options. In May 2023, the US Food and Drug Administration (FDA) approved the first topical gene therapy, beremagene geperpavec (B-VEC), for treating both autosomal recessive and autosomal dominant dystrophic epidermolysis bullosa (DEB). However, FDA approval was based on limited data in patients with autosomal dominant disease, even though they comprise approximately 50% of all DEB cases.
To estimate projected spending in the US on B-VEC therapy for treating autosomal recessive and autosomal dominant DEB.
DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation used data from the National Epidermolysis Bullosa Registry to estimate the current population of US patients with autosomal dominant and autosomal recessive DEB, with the aim of estimating US spending on B-VEC therapy from an all-payers perspective during 1- and 3-year periods after FDA approval. A base-case cost of $300 000 per patient per year was assumed based on a report from the manufacturer (Krystal Biotech).
Treatment with B-VEC.
Estimated overall spending on B-VEC in the first year and over a 3-year period after FDA approval. Several prespecified sensitivity analyses with different assumptions about the eligible patient population and the cost of therapy were performed, and lifetime total costs of treatment per patient were estimated.
The estimated number of US patients with DEB who were eligible for treatment with B-VEC in the first year after FDA approval was 894. The estimated total expenditure for B-VEC therapy was $268 million (range, $179 million-$357 million). Over a 3-year period, estimated spending was $805 million (range, $537 million-$1.1 billion). Estimated lifetime total costs per patient were $15 million (range, $10 million-$20 million) per patient with autosomal recessive DEB and $17 million (range, $11 million-$22 million) for patients with autosomal dominant DEB.
Results of this economic evaluation suggest 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.
新的基因疗法可为患者带来实质性的益处，特别是对于那些治疗选择有限的罕见病患者。2023 年 5 月，美国食品和药物管理局（FDA）批准了第一种局部基因疗法——beremagene geperpavec（B-VEC），用于治疗常染色体隐性和常染色体显性营养不良性大疱性表皮松解症（DEB）。然而，FDA 的批准是基于在常染色体显性疾病患者中有限的数据，尽管他们约占所有 DEB 病例的 50%。
估计美国使用 B-VEC 治疗常染色体隐性和常染色体显性 DEB 的预计支出。
设计、设置和参与者：这项经济评估使用了国家大疱性表皮松解症登记处的数据，以估计美国常染色体显性和常染色体隐性 DEB 患者的当前人群，旨在从所有支付者的角度估计 FDA 批准后 1 年和 3 年内 B-VEC 治疗的美国支出。根据制造商（Krystal Biotech）的报告，假设每位患者每年的治疗费用为 30 万美元，这是一个基本的成本。
B-VEC 治疗。
估计 FDA 批准后第一年和三年内 B-VEC 的总支出。进行了几项关于合格患者人群和治疗费用的不同假设的预设敏感性分析，并估计了每位患者的终身治疗总成本。
估计在 FDA 批准后的第一年，有 894 名美国 DEB 患者有资格接受 B-VEC 治疗。预计 B-VEC 治疗的总支出为 2.68 亿美元（范围为 1.79 亿至 3.57 亿美元）。在 3 年内，预计支出为 8.05 亿美元（范围为 5.37 亿至 11 亿美元）。每位患者的终身总成本估计为 1500 万美元（范围为 1000 万美元至 2000 万美元），用于治疗常染色体隐性 DEB 患者，每位患者的终身总成本估计为 1700 万美元（范围为 1100 万美元至 2200 万美元），用于治疗常染色体显性 DEB 患者。
这项经济评估的结果表明，FDA 广泛批准 B-VEC 用于治疗常染色体隐性和常染色体显性 DEB，这将对支付者产生重大影响。
