Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status.

Abstract

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. 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. 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. 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.