Department of Physiology and Pharmacology, Karolinska Institutet, Stockholm, Sweden.
Dr Margarete Fischer-Bosch Institute of Clinical Pharmacology, Stuttgart, Germany.
Handb Exp Pharmacol. 2023;280:237-260. doi: 10.1007/164_2022_596.
Over the last decade, next-generation sequencing (NGS) methods have become increasingly used in various areas of human genomics. In routine clinical care, their use is already implemented in oncology to profile the mutational landscape of a tumor, as well as in rare disease diagnostics. However, its utilization in pharmacogenomics is largely lacking behind. Recent population-scale genome data has revealed that human pharmacogenes carry a plethora of rare genetic variations that are not interrogated by conventional array-based profiling methods and it is estimated that these variants could explain around 30% of the genetically encoded functional pharmacogenetic variability.To interpret the impact of such variants on drug response a multitude of computational tools have been developed, but, while there have been major advancements, it remains to be shown whether their accuracy is sufficient to improve personalized pharmacogenetic recommendations in robust trials. In addition, conventional short-read sequencing methods face difficulties in the interrogation of complex pharmacogenes and high NGS test costs require stringent evaluations of cost-effectiveness to decide about reimbursement by national healthcare programs. Here, we illustrate current challenges and discuss future directions toward the clinical implementation of NGS to inform genotype-guided decision-making.
在过去的十年中,下一代测序(NGS)方法已在人类基因组学的各个领域得到越来越多的应用。在常规临床护理中,其已在肿瘤学中用于分析肿瘤的突变景观以及在罕见病诊断中得到应用。然而,其在药物基因组学中的应用却大大落后。最近的全基因组规模数据表明,人类药物基因组携带大量常规基于阵列的分析方法无法检测到的罕见遗传变异,据估计,这些变异可能解释约 30%的遗传编码功能药物遗传学变异性。为了解释这些变异对药物反应的影响,已经开发了多种计算工具,但尽管已经取得了重大进展,但仍需证明其准确性是否足以在稳健的试验中改进个性化药物基因组学建议。此外,传统的短读测序方法在复杂药物基因的检测方面存在困难,而高通量测序测试的高成本需要严格评估成本效益,以决定国家医疗保健计划是否报销。在这里,我们举例说明了当前的挑战,并讨论了朝着以基因型指导决策为目标的 NGS 临床实施的未来方向。