Autologous skin cell suspension plus phototherapy in stable vitiligo: findings from a US economic model

A cell harvesting device for preparing non-cultured autologous skin cell suspension (ASCS) at the point-of-care is FDA-approved for repigmentation of stable depigmented vitiligo lesions in patients 18 years and older. The pivotal RSVP trial showed ≥80% repigmentation at Week-24 in 36% of lesions treated with laser ablation, ASCS, and narrowband ultraviolet B phototherapy compared to 0% with phototherapy alone (p = 0.012). The objective of this analysis was to evaluate the potential economic impact of laser ablation plus ASCS with phototherapy versus phototherapy alone for repigmentation of stable vitiligo lesions from a US payer perspective. A 5-year decision-tree model was developed reflecting clinical pathways of adults with stable vitiligo lesions who had an inadequate response to prior topicals and phototherapy. Patients entering the model were treated with ASCS plus phototherapy or phototherapy alone and assessed for treatment response at Weeks-24 and 52 based on the RSVP trial's effectiveness endpoints. Durable response for Year-2 onwards was proxied by melanocyte-keratinocyte transplantation data. Model outcomes included per-patient total and incremental healthcare costs, treatment costs and total costs, cost per-patient per-month (PPPM), and cost per-patient per-year (PPPY). One-way sensitivity analyses assessed model result robustness. The cumulative total per-patient cost for ASCS plus phototherapy increased from $28,177 to $92,779 between Year-1 and Year-5. Phototherapy alone increased from $21,146 to $101,518 over the same period. Compared to phototherapy alone, ASCS plus phototherapy incurred $7,030 more total per-patient cumulative costs in Year-1 and $8,738 less by Year-5 (-$146 PMPM; -$1,748 PPPY). Breakeven occurred between Years 2-3. Results were most sensitive to changes in ASCS response at Weeks-24 and 52 and healthcare costs. Among adults with stable vitiligo with prior inadequate response to topicals or phototherapy, ASCS treatment may lead to lower all-cause direct medical costs over 5 years compared to phototherapy alone.

一款可在床旁制备非培养自体皮肤细胞悬液（ASCS）的细胞采集设备，已获美国食品药品监督管理局（Food and Drug Administration, FDA）批准，用于18岁及以上患者稳定型色素脱失性白癜风皮损的复色治疗。关键RSVP临床试验结果显示，接受激光消融、ASCS与窄谱紫外线B光疗联合治疗的皮损中，36%在第24周时达到≥80%的复色率，而仅接受光疗的皮损复色率为0%（p=0.012）。本分析旨在从美国支付方视角，评估激光消融联合ASCS联合光疗对比单纯光疗用于稳定型白癜风皮损复色治疗的潜在经济学影响。我们构建了一款5年期决策树模型，用以刻画曾对既往外用药物及光疗应答不足的稳定型白癜风成年患者的临床诊疗路径。入组模型的患者被分配至ASCS联合光疗组与单纯光疗组，并依据RSVP临床试验的有效性终点，在第24周与第52周评估治疗应答情况。第2年及以后的持久应答以黑素细胞-角质形成细胞移植数据作为替代指标。模型结局指标包括每位患者的总医疗成本与增量医疗成本、治疗成本与总成本、每位患者每月成本（PPPM）以及每位患者每年成本（PPPY）。单因素敏感性分析用于评估模型结果的稳健性。ASCS联合光疗组的每位患者累计总成本从第1年的28177美元升至第5年的92779美元；单纯光疗组同期则从21146美元升至101518美元。与单纯光疗组相比，ASCS联合光疗组在第1年的每位患者累计总成本高出7030美元，但到第5年时则低出8738美元（每月人均成本较对照组减少146美元，每年人均成本较对照组减少1748美元）。两组的收支平衡点出现在第2年至第3年之间。研究结果对第24周与第52周的ASCS应答率以及医疗成本的变化最为敏感。对于曾对外用药物或光疗应答不足的稳定型白癜风成年患者而言，相较于单纯光疗，ASCS治疗在5年随访期内可降低全因直接医疗成本。