Johns Hopkins Home Care Group, Baltimore, MD, USA.
Am J Health Syst Pharm. 2021 May 24;78(11):953-961. doi: 10.1093/ajhp/zxab064.
To provide health systems with baseline knowledge on existing and pipeline gene therapy treatments, including considerations that health-system pharmacies and specialty pharmacy programs may reference when evaluating and implementing services around gene therapies.
Advancements in research and biotechnology have recently led to the development and launch of the first commercially available gene therapy treatments in the United States. These treatments have the ability to significantly alter and even effectively cure diseases. Alongside these significant advances and clinical benefits, these therapies present unique challenges due to their cost and complexity. Given the large number of additional gene therapy treatments that are currently in late-stage clinical development, stakeholders across the healthcare industry must increasingly adapt and ready themselves to meet these challenges. The diagnosis and treatment of patients with diseases being targeted by gene therapies largely occurs within health systems, and judging by the gene therapy pipeline, this trend is likely to continue. To prepare for these novel treatments, health systems must understand and consider the methods in which gene therapies are developed, procured, reimbursed, administered, and monitored.
The future of health-system pharmacy practice must include comprehensive gene therapy services and stakeholder engagement strategies to ensure patients have access to these life-changing treatments.
为卫生系统提供现有和即将推出的基因治疗药物方面的基础知识，包括卫生系统药房和专业药房项目在评估和实施基因治疗相关服务时可能参考的因素。
研究和生物技术的最新进展使得美国首批商业上可获得的基因治疗药物得以开发和推出。这些疗法具有显著改变甚至有效治愈疾病的能力。除了这些显著的进步和临床益处之外，由于这些疗法的成本和复杂性，它们带来了独特的挑战。鉴于目前有大量的其他基因治疗药物处于后期临床开发阶段，医疗保健行业的利益相关者必须不断调整和准备好迎接这些挑战。基因治疗药物针对的疾病的诊断和治疗主要发生在卫生系统内，从基因治疗药物的研发情况来看，这种趋势很可能会持续下去。为了准备这些新的治疗方法，卫生系统必须了解并考虑基因疗法的开发、采购、报销、管理和监测方法。
未来的卫生系统药房实践必须包括全面的基因治疗服务和利益相关者参与策略，以确保患者能够获得这些改变生活的治疗方法。
