Jeremy Schafer，药学博士，工商管理硕士，高级副总裁，价值的精确性

来自贸易组织PhRMA的一份2018年报告指出，目前有近300种细胞和基因疗法正在研发中。癌症是最大的靶点，有111种药物正在研制中，但其他各种疾病也有靶点，包括血友病、神经系统疾病、自身免疫性疾病，甚至阿尔茨海默氏症。虽然这些疗法中有相当一部分可能在开发阶段的某个时候失败，但基因/细胞疗法革命似乎正在兴起正在迅速增长。这对付款人和我们目前的医疗体系来说可能是一个问题，我们的医疗体系受到短期回报和即时成本规避的激励，而不是改善患者健康的长期利益。基因疗法正在彻底改变医疗保健，但我们能否及时改变支付模式以跟上时代的步伐？

一些最新的基于基因或细胞的治疗方案强调了这个难题。两个被认可的CAR-T代理的费用都超过35万美元。Luxturna是一种治疗罕见失明的代理，治疗双眼的费用超过80万美元。从血友病到遗传性肌肉疾病等罕见疾病的未来治疗理论预计将超过100万美元或更多。然而，这些以基因和细胞为基础的治疗方法也可能帮助付费者避免未来数百万美元的潜在成本，因为患者可能不再需要慢性治疗和病情恶化带来的健康资源。不幸的是，目前的付款人和医疗保健系统侧重于短期回报。一般的健康计划可能只有几年的成员，因此，如果最初的健康计划支付昂贵的治疗费用，却看到竞争对手后来实现的收益，那么终身储蓄的价值就失去了。

Clearly new payment models will be needed. In 2018, our organization conducted market research amongst 20 health plans and 5 integrated delivery networks on perceptions of gene therapy and payment models. The information was presented at the Academy of Managed Care Pharmacy 2018 spring meeting. Respondents were asked about their interest in alternative payment models and the operational capacity to implement them. Outcomes contracting, where less, or nothing, would be paid if the drug didn’t work, was most popular with 56% being very or extremely interested and 88% confident they could operationalize the agreement. Distribution through a specialty pharmacy in order to eliminate provider/hospital upcharge was second with 44% very or extremely interested and 92% able to implement. Annuity payments, where payments would be remitted over time versus all at once, garnered interest from 16%, with 72% believing that such an agreement could be made operational. Respondents proposed a number of other potential payment mechanisms including payment over time that would follow the patient (in case he or she switched health plans), placing such patients in high-risk pools, paying for gene therapy like a plan would pay for end stage renal disease, and even requiring a treated patient to stay with the health plan. The number of proposals was encouraging in that it indicates the industry is thinking, but the lack of consistency shows there is still a long way to go.

The healthcare industry needs to begin experimenting with longer-term payment models and building incentives for improved long-term health. The market forces will require health plans to continue to focus on short-term financial performance, but a few goals that focus on improvement in patient long-term health and reduced future expenses can be piloted. Manufacturers should spend time developing different payment models for gene therapy with a focus on what is operationally possible for their customers.

基因治疗领域的创新令人震惊，给患者带来了新的希望。业界现在需要确保我们在支付这些治疗费用的方式上有所创新。

工具书类

弗拉玛。药品开发for cell therapy and gene therapy. 2018. Accessed January 18, 2019.

福布斯。Novartis' $4 million gene therapy—real price or a negotiation ploy?Nov 13, 2018. Accessed January 18, 2019.

今天的新闻。#AAN2018-试验显示，使用AVXS-101治疗的1型SMA婴儿取得了新的里程碑. April 24, 2018. Accessed January 18, 2019.

从欧洲到美国：基因治疗的挑战和潜力。AMCP管理护理和专业药房年会；2018年4月23-26日；马萨诸塞州波士顿。