Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status
Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status
复制标题
DOI：
10.1007/s43441-019-00100-7
复制
发表时间：
2020-01-06
影响因子：
1.5
通讯作者：
Parker, Jonathon M.
中科院分区：
文献类型：
--
作者：
Muensterman, Elena Tomaselli;Luo, Yijia;Parker, Jonathon M.
由于对治疗危及生命和罕见疾病的药物的需求不断增加，监管机构已经开发了各种加速监管途径。这些项目旨在优先考虑最有希望的候选药物，以治疗缺乏令人满意的治疗方法的疾病。引进的最突出的加速项目是美国的突破性治疗指定（BTD），欧盟的优先药物（PRIME）和日本的Sakigake。本文回顾了这些名称，并着眼于如何在神经科学和肿瘤学的3个司法管辖区授予它们的差异。方法：我们的目标是分析2012年至2019年期间BTD、PRIME和Sakigake的批准，重点是两个治疗领域之间批准的数量差异。对有关BTD、PRIME和Sakigake主题的公共资源进行了搜索。该分析显示，48%的BTD被授予肿瘤学领域，而神经科学领域获得了8%的批准。在PRIME申请中，肿瘤学领域获得了27%，神经科学领域获得了15%。在日本，Sakigake的50%被授予肿瘤学领域，22%被授予神经科学产品。鉴于药物开发的全球性质和这些监管机制的相对相似性，美国授予特殊地位的比例为6:1（肿瘤学：神经科学），欧盟和日本授予特殊地位的比例为2:1，两者之间存在明显差异。这种不成比例的比例可能受到多因素问题的影响；然而，这种差异值得进一步研究。
Background Because of the increasing demand for drugs addressing life-threatening and rare diseases, regulatory agencies have developed a variety of accelerated regulatory pathways. These programs are aimed at prioritizing the most promising drug candidates for diseases lacking satisfactory treatments. The most prominent accelerated programs introduced have been Breakthrough-Therapy Designation (BTD) in the United States, Priority Medicine (PRIME) in the European Union and Sakigake in Japan. This article reviews these designations and looks at differences in how they are granted across the 3 jurisdictions focusing on neuroscience and oncology. Methods Our objective was to analyze BTD, PRIME, and Sakigake approvals between 2012 and 2019 with a focus on numerical disparities of designations granted between the 2 therapeutic areas. A search of public sources pertaining to topics of BTD, PRIME, and Sakigake was undertaken. Results This analysis revealed that 48% of BTD were granted in oncology, while neuroscience received 8% of these designations, for PRIME designations were 27% received by oncology and 15% by neuroscience and in Japan, 50% of Sakigake were granted to oncology and 22% to neuroscience products. Conclusion Given the global nature of drug development and relative similarity of these regulatory mechanisms, there is an apparent disparity between the US granting special status at 6:1 (oncology: neuroscience) and both the EU and Japan granting at 2:1. This disproportionate ratio is likely impacted by multifactorial issues; however, this difference is worth further investigation.