The National Center for Advancing Translational Sciences' virtual 2021 conference on gene-targeted therapies (GTTs) encouraged multidisciplinary dialogue on a wide range of GTT topic areas. Each of three parallel working groups included social scientists and clinical scientists, and the three major sessions included a presentation on economic issues related to their focus area. These experts also coordinated their efforts across the three groups. The economics-related presentations covered three areas with some overlap: (1) value assessment, uncertainty, and dynamic efficiency; (2) affordability, pricing, and financing; and (3) evidence generation, coverage, and access. This article provides a synopsis of three presentations, some of their key recommendations, and an update on related developments in the past year. The key high-level findings are that GTTs present unique data and policy challenges, and that existing regulatory, health technology assessment, as well as payment and financing systems will need to adapt. But these adjustments can build on our existing foundation of regulatory and incentive systems for innovation, and much can be done to accelerate progress in GTTs. Given the substantial unmet medical need that exists for these oft-neglected patients suffering from rare diseases, it would be a tragedy to not leverage these exciting scientific advances in GTTs.

中文翻译：

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基因靶向疗法的经济问题回顾：价值、可负担性和可及性

国家转化科学促进中心 2021 年虚拟基因靶向治疗 (GTT) 会议鼓励就广泛的 GTT 主题领域进行多学科对话。三个平行工作组中的每一个都包括社会科学家和临床科学家，三个主要会议包括与其重点领域相关的经济问题的介绍。这些专家还协调了三个小组的工作。与经济学相关的演讲涵盖三个领域，但有一些重叠：(1) 价值评估、不确定性和动态效率；(2) 负担能力、定价和融资；(3) 证据生成、覆盖和访问。本文提供了三个演讲的概要、他们的一些主要建议，以及过去一年相关发展的最新情况。关键的高层调查结果是，GTT 提出了独特的数据和政策挑战，现有的监管、卫生技术评估以及支付和融资系统需要进行调整。但这些调整可以建立在我们现有的创新监管和激励体系基础上，并且可以做很多事情来加速 GTT 的进展。鉴于这些经常被忽视的罕见疾病患者存在大量未满足的医疗需求，如果不利用这些令人兴奋的 GTT 科学进步，将是一场悲剧。可以做很多事情来加快全球贸易条约的进展。鉴于这些经常被忽视的罕见疾病患者存在大量未满足的医疗需求，如果不利用这些令人兴奋的 GTT 科学进步，将是一场悲剧。可以做很多事情来加快全球贸易条约的进展。鉴于这些经常被忽视的罕见疾病患者存在大量未满足的医疗需求，如果不利用这些令人兴奋的 GTT 科学进步，将是一场悲剧。