Challenges in the translation and commercialization of cell therapies.
Challenges in the translation and commercialization of cell therapies.
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DOI：
10.1186/s12896-015-0190-4
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发表时间：
2015-08-07
影响因子：
3.5
通讯作者：
Levine AD
中科院分区：
文献类型：
--
作者：
Dodson BP;Levine AD
细胞疗法是一种新兴的医疗保健形式，为改善医学实践提供了巨大的潜力，并为目前治疗选择有限或没有治疗选择的患者提供了益处。理想情况下，这些创新疗法可以补充现有的小分子、生物和设备方法，形成所谓的医学第四支柱，并允许临床医生为每位患者确定最佳治疗方法。尽管有这种潜力，但细胞疗法比小分子或生物干预要复杂得多。这种复杂性给开发细胞疗法的科学家和公司以及寻求监督这一不断增长的医学领域的监管机构带来了挑战。在这个项目中，我们回顾性地研究了七种细胞疗法的发展，包括三种自体干预和四种同种异体干预，目的是确定阻碍新细胞疗法推向市场的共同挑战。我们通过对细胞治疗各个方面的专家进行一系列定性访谈来补充这一分析。通过我们的分析，包括对从公司文件、报纸、期刊、分析师报告和类似来源收集的现有文献的回顾，以及对定性访谈的分析，我们确定了细胞治疗公司在上市前和上市后阶段必须解决的几个常见挑战。关键的上市前挑战包括确定和维持稳定的资金，以帮助公司度过漫长的发展时间表和不确定的监管程序。当然，这些挑战并不是细胞疗法所独有的，但与小分子或生物方法相比，基于细胞的干预措施的新奇使这些努力变得复杂。细胞疗法的非典型性也导致了上市后的困难，包括在报销过程中遇到的挑战，以及说服供应商改变他们的治疗方法。此外，扩大生产、分配细胞疗法和管理生产成本是从上市前开始并持续到上市后阶段的挑战。我们的分析强调了阻碍细胞疗法发展的几个相互关联的挑战。确定应对这些挑战的策略可能会加速开发并增加新型细胞疗法的影响。
Cell therapies are an emerging form of healthcare that offer significant potential to improve the practice of medicine and provide benefits to patients who currently have limited or no treatment options. Ideally, these innovative therapies can complement existing small molecule, biologic and device approaches, forming a so-called fourth pillar of medicine and allowing clinicians to identify the best treatment approach for each patient. Despite this potential, cell therapies are substantially more complex than small molecule or biologic interventions. This complexity poses challenges for scientists and firms developing cell therapies and regulators seeking to oversee this growing area of medicine. In this project, we retrospectively examined the development of seven cell therapies – including three autologous interventions and four allogeneic interventions – with the aim of identifying common challenges hindering attempts to bring new cell therapies to market. We complemented this analysis with a series of qualitative interviews with experts in various aspects of cell therapy. Through our analysis, which included review of extant literature collected from company documents, newspapers, journals, analyst reports and similar sources, and analysis of the qualitative interviews, we identified several common challenges that cell therapy firms must address in both the pre- and post-market stages. Key pre-market challenges included identifying and maintaining stable funding to see firms through lengthy developmental timelines and uncertain regulatory processes. These challenges are not unique to cell therapies, of course, but the novelty of cell-based interventions complicates these efforts compared to small molecule or biologic approaches. The atypical nature of cell therapies also led to post-market difficulties, including challenges navigating the reimbursement process and convincing providers to change their treatment approaches. In addition, scaling up production, distributing cell therapies and managing the costs of production were challenges that started pre-market and continued into the post-market phase. Our analysis highlights several interrelated challenges hindering the development of cell therapies. Identifying strategies to address these challenges may accelerate the development and increase the impact of novel cell therapies.