Schuhmacher Alexander, Hinder Markus, Brief Elazar, Gassmann Oliver, Hartl Dominik
Technische Hochschule Ingolstadt, Germany; University of St Gallen, Switzerland.
Novartis, Basel, Switzerland; University of Zürich, Switzerland.
Drug Discov Today. 2025 Feb;30(2):104291. doi: 10.1016/j.drudis.2025.104291. Epub 2025 Jan 11.
Previous analyses provide an industry benchmark of ∼10% for the success rate in clinical development. However, prior analyses were limited by a narrow timeframe, a diverse research focus, biases in phase-to-phase transition methodology or a focus on specific use cases. We calculated unbiased input:output ratios (Phase I to FDA new drug approval) to analyze the likelihood of first approval using data from clinicaltrials.gov, encompassing a total of 2092 active ingredients, 19 927 clinical trials conducted by 18 leading pharmaceutical companies (2006-2022) and 274 new drug approvals. Our study reveals an average likelihood of first approval rate of 14.3% across leading research-based pharmaceutical companies, broadly ranging from 8% to 23%.
以往的分析给出了临床开发成功率约10%的行业基准。然而，之前的分析受到时间框架狭窄、研究重点多样、各阶段过渡方法存在偏差或专注于特定用例的限制。我们计算了无偏差的投入产出比（从一期试验到美国食品药品监督管理局新药批准），以使用来自clinicaltrials.gov的数据分析首次获批的可能性，该数据涵盖了总共2092种活性成分、18家领先制药公司（2006 - 2022年）开展的19927项临床试验以及274项新药批准。我们的研究表明，领先的研发型制药公司首次获批率的平均可能性为14.3%，大致在8%至23%之间。
