ALD Connect, Middleton, MA, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
REGENXBIO Inc., Rockville, MD, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Mol Ther. 2018 Dec 5;26(12):2717-2726. doi: 10.1016/j.ymthe.2018.10.017. Epub 2018 Oct 30.
Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to optimize patient access. Maximizing patient access to effective gene therapies is one integral part of the overall mission of the American Society of Gene and Cell Therapy, along with maximizing the quality of therapies and minimizing their costs.
尽管基因疗法的高前期成本导致了人们对充分报销以允许患者获得这些疗法的关注，但除非患者能够获得这些疗法，否则基因疗法的巨大益处将无法实现。利益相关者正在讨论这些问题，并且正在为新批准的基因疗法制定的支付模式为治疗制造商、支付者和政策制定者提供了一个早期迹象，表明需要灵活性，以优化患者的获得途径。最大限度地增加患者获得有效基因疗法的机会是美国基因与细胞治疗学会总体使命的一个组成部分，同时还需要最大限度地提高疗法的质量并降低其成本。
