EY, 1 More London Place, London, SE1 2AF, UK.
EY, Harcourt Centre, Harcourt Street, Dublin 2, Ireland.
Pharmaceut Med. 2023 Sep;37(5):365-375. doi: 10.1007/s40290-023-00480-0. Epub 2023 Jun 7.
Clinical development paradigms for cell and gene therapies appear to be different to those of more conventional treatments: therefore, it is informative to explore this from the perspective of investments required to bring a new cell and/or gene therapy to the market. While there are a number of studies in the literature analyzing clinical-stage R&D costs for novel therapeutics, these are 'modality-agnostic' and thus do not elucidate costs specifically for the emerging class of cell and gene therapies.
The objective of this study was to understand the research and development (R&D) costs associated with the clinical development of new cell and gene therapy assets METHODS: As part of our analysis of clinical-stage R&D costs for cell and gene therapies, we focused our efforts on cell and gene therapy assets recently approved by the US Food and Drug Administration (FDA) or expected to receive FDA approval by the end of 2024. A total of 25 therapies were identified for the study, 11 of which had sufficient level of detail for our clinical-stage R&D costing study. We calculated the clinical-stage R&D costs to bring a new cell and/or gene therapy to the market following a three-step approach, starting with (1) calculation of the out-of-pocket investment reported in US SEC reports; (2) we adjusted these figures for the risk of failure by applying a clinical trial phase-dependent attrition risk rate; (3) we accounted for the cost of capital of 10.5%.
After accounting for R&D attrition rate (i.e., costs of failed programs) and applying a cost of capital at 10.5%, we estimate that the clinical-stage R&D investment required to bring a new cell and/or gene therapy to market is US$1943 M (95% CI US$1395 M, US$2490 M).
This knowledge can inform financial planning for biopharma companies looking to enter the space and inform policy makers within the context of the commercialization and pricing of such therapies.
细胞和基因治疗的临床开发模式似乎与更传统的治疗方法不同：因此，从将新的细胞和/或基因疗法推向市场所需的投资角度来探索这一点是有意义的。虽然文献中有许多研究分析了新型疗法的临床阶段研发成本，但这些研究是“无模式”的，因此无法阐明专门针对新兴细胞和基因治疗类别的成本。
本研究旨在了解与新的细胞和基因治疗资产的临床开发相关的研发成本。
作为我们对细胞和基因治疗临床阶段研发成本分析的一部分，我们专注于最近获得美国食品和药物管理局 (FDA) 批准或预计在 2024 年底前获得 FDA 批准的细胞和基因治疗资产。共有 25 种疗法被确定用于该研究，其中 11 种疗法有足够的细节供我们进行临床阶段研发成本研究。我们采用三步法计算将新的细胞和/或基因疗法推向市场的临床阶段研发成本，首先（1）计算美国证券交易委员会报告中报告的自付投资；（2）通过应用临床试验阶段相关的淘汰风险率来调整这些数字以考虑失败风险；（3）考虑 10.5%的资本成本。
在考虑研发淘汰率（即失败项目的成本）并应用 10.5%的资本成本后，我们估计将新的细胞和/或基因疗法推向市场所需的临床阶段研发投资为 1.943 亿美元（95%置信区间为 1.395 亿美元至 2.490 亿美元）。
这一知识可以为希望进入该领域的生物制药公司提供财务规划，并在这些疗法的商业化和定价背景下为决策者提供信息。
