Development of prediction models for final approval probability in early phase clinical trials for new drug development

2020 Fiscal Year Final Research Report

• Project/Area Number: 18K09939
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 58010:Medical management and medical sociology-related
• Research Institution: The University of Tokyo
• Principal Investigator: Nojima Masanori 東京大学, 医科学研究所, 准教授 (00457699)
• Project Period (FY): 2018-04-01 – 2021-03-31
• Keywords: 新薬開発 / 臨床試験 / 臨床試験デザイン / 新規医薬品開発

Outline of Final Research Achievements

The cost of developing a new drug is enormous, but the probability of approval after the first phase I trial estimated to be just over 10%, and is even more severe in oncology. In this study, we explored the risk factors associated with discontinuation of new drug development and modeled the probability of drug approval by using publicly available information from clinicaltrials.gov and the U.S. Food and Drug Administration (FDA). The database covers development period, whether phase II and phase III trials were conducted, and other information. We found that new drugs in oncology have been in development for more than 10 years and more than half of drugs have not yet been approved even after reaching Phase III trials. We will explore the factors associated with the risk of discontinuation in such late phases.

Free Research Field

臨床試験

Academic Significance and Societal Importance of the Research Achievements

現在本邦においても研究者主導治験・臨床試験が盛んに行われるようになり、新薬開発の機運は高まっているが、開発失敗のリスクというのは研究者にとっても、パートナーとなる製薬企業にとっても重大な問題である。特に、膨大な投資を行った上での後期相での開発中止はより大きな問題と考えられ、その頻度を明らかにし関連する因子を探っていくというのが本研究の目的である。現在、研究用のデータベースが完成間近であり、記述統計的な結果の提示にとどまってはいるが、開発が長期化している実態や第III相での開発中止例が想定以上に多いことが明らかとなるなどの成果が得られている。