The emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment and their high prices introduce critical access and affordability concerns. The aim of this article is to analyze the challenges of traditional value assessment and price and reimbursement methods in the evaluation of ATMPs and to characterize the current and prospective financing solutions that may ensure patient access and affordability for these health technologies. Standard Health Technology Assessment (HTA) is not designed for ATMPs, and may delay access to these health technologies, thus a broader concept of value is required. As a consequence, value-based pricing methodologies have been gaining terrain to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure the balance between encouragements to innovation and maximization of value for money for payers, through the attribution of a fair price to new health technologies. Early scientific advice by regulatory and HTA bodies to developers is key, as it will contribute to diminish the perspective gap between developers, regulators and payers. The high efficacy/high price dynamic of many advanced therapies will demand novel financing models, both in the EU and US. Managed entry agreements (MEA), with financing being conditional to the submission of additional evidence, associated with methods of leased payments, may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access.

先进治疗药物产品 (ATMP) 的出现是一种颠覆性的健康技术，在价值评估方面产生了重大挑战，其高昂的价格引发了关键的获取和可负担性问题。本文的目的是分析传统价值评估、价格和报销方法在评估 ATMP 时面临的挑战，并描述当前和未来的融资解决方案，以确保患者获得这些医疗技术的可及性和可负担性。标准卫生技术评估 (HTA) 不是为 ATMP 设计的，可能会延迟获得这些卫生技术，因此需要更广泛的价值概念。因此，基于价值的定价方法已经获得了应对ATMP特定挑战的领域。定价和报销框架应通过为新的卫生技术提供公平的价格，确保在鼓励创新和最大限度地提高支付者的性价比之间取得平衡。监管机构和 HTA 机构对开发商的早期科学建议是关键，因为它将有助于缩小开发商、监管机构和付款人之间的观点差距。许多先进疗法的高效/高价格动态将需要新的融资模式，无论是在欧盟还是美国。管理准入协议 (MEA) 的融资以提交与租赁付款方式相关的额外证据为条件，可以提供有效的策略来解决与 ATMP 相关的证据差距所造成的不确定性，确保负担得起和持续的访问。