The health economics of cell and gene therapies.

Q2 Medicine Handbook of clinical neurology Pub Date : 2024-01-01 DOI:10.1016/B978-0-323-90120-8.00004-6

Laura Beswick

引用次数: 0

Abstract

The health economics of cell and gene therapies is complex; due to resource-intensive manufacturing, high prices are required for commercial viability that are challenging for healthcare systems to accommodate. Despite high prices, cell and gene therapies can provide value when they deliver substantial clinical benefits and displace long-term healthcare costs compared with existing treatment options. In this chapter, the cost utility approach of economic evaluation is discussed, focusing on the considerations that occur more commonly in cell and gene therapies compared to conventional medicines, how these considerations create challenges in interpreting the evidence and coming to conclusions on value, and what tools exist for understanding the level of decision uncertainty. A summary of the economic evaluation of onasemnogene abeparvovec for spinal muscular atrophy is provided as a real-world example that features the considerations discussed.

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细胞和基因疗法的卫生经济学。

细胞和基因疗法的卫生经济学非常复杂；由于制造过程需要大量资源，因此需要高昂的价格才能实现商业可行性，这对医疗保健系统来说具有挑战性。尽管价格高昂，但与现有治疗方案相比，细胞和基因疗法如果能带来显著的临床疗效，并替代长期医疗成本，就能产生价值。本章将讨论经济评估中的成本效用法，重点是细胞和基因疗法与传统药物相比更常见的考虑因素，这些考虑因素如何给解释证据和得出价值结论带来挑战，以及有哪些工具可用于了解决策的不确定性程度。本文提供了对onasemnogene abeparvovec治疗脊髓性肌萎缩症的经济评估摘要，作为一个现实世界的例子，其中包含了所讨论的各种考虑因素。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Handbook of clinical neurology Medicine-Neurology (clinical)

CiteScore

4.10

自引率

0.00%

发文量

302

期刊介绍： The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.

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