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Short Time to Market and Forward Planning Will Enable Cell Therapies to Deliver R&D Pipeline Value
Human Gene Therapy ( IF 3.9 ) Pub Date : 2021-05-17 , DOI: 10.1089/hum.2020.212
Delfi Krishna 1 , Laure Rittié 1 , Hoang Tran 1 , Xuan Zheng 1 , Chia-En Chen-Rogers 1 , Amanda McGillivray 1 , Timothy Clay 1 , Amol Ketkar 1 , Joseph Tarnowski 1
Affiliation  

There is considerable industry excitement about the curative potential of cell and gene therapies, but significant challenges remain in designing cost-effective treatments that are accessible globally. We have taken a modeling-based approach to define the cost and value drivers for cell therapy assets during pharmaceutical drug development. We have created a model development program for a lentiviral modified ex vivo autologous T cell therapy for Oncology indications. Using internal and external benchmarks, we have estimated the total out-of-pocket cost of development for an Oncology cell therapy asset from target identification to filing of marketing application to be $500–600 million. Our model indicates that both clinical and Chemistry Manufacturing and Controls (CMC) cost of development for cell therapies are higher due to unique considerations of ex vivo autologous cell therapies. We have computed a threshold revenue-generating patient number for our model asset that enables selection of assets that can address high unmet medical need and generate pipeline value. Using statistical approaches, we identified that short time to market (<5 years) and reduced commercial cost of goods (<$65,000 per dose) will be essential in developing competitive assets and we propose solutions to reduce both. We emphasize that teams must proactively plan alternate development scenarios with clear articulation of path to value generation and greater patient access. We recommend using a modeling-based approach to enable data driven go/no-go decisions during multigenerational cell therapy development.

中文翻译:

较短的上市时间和前瞻性规划将使细胞疗法能够提供研发管道价值

业界对细胞和基因疗法的治疗潜力感到相当兴奋,但在设计可在全球范围内获得的具有成本效益的治疗方法方面仍然存在重大挑战。我们采用基于模型的方法来定义药物开发过程中细胞治疗资产的成本和价值驱动因素。我们已经为慢病毒修饰的离体创建了模型开发程序自体 T 细胞疗法用于肿瘤学适应症。使用内部和外部基准,我们估计肿瘤细胞治疗资产从目标识别到提交营销申请的总自付费用开发成本为 500-6 亿美元。我们的模型表明,由于离体的独特考虑,细胞疗法的临床和化学制造和控制 (CMC) 开发成本更高自体细胞疗法。我们已经为我们的模型资产计算了一个创收患者数量的阈值,从而可以选择能够满足高未满足医疗需求并产生管道价值的资产。使用统计方法,我们发现缩短上市时间(<5 年)和降低商品商业成本(每剂<65,000 美元)对于开发具有竞争力的资产至关重要,我们提出了减少这两者的解决方案。我们强调,团队必须主动规划替代开发方案,明确表达价值创造的途径和更多的患者访问。我们建议在多代细胞疗法开发过程中使用基于建模的方法来实现数据驱动的去/不去决策。
更新日期:2021-05-18
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