临床试验成功率2011-2020（经典）

2021

05/08

求实医药

A-

A+

成功的首要因素是疾病适应症、靶点、形态和药物创新性。

BIO、QLS Advisors、Biomedtracker发布的最近10年临床研发成功率及其贡献因素的报告。

The overall likelihood of approval，LOA

2011-2020年所有候选药物从I期开始到最后的总体批准可能性（LOA）为7.9%，而2006-2015年这一数据为9.6%；
II期仍然是药物开发的最大障碍，只有28.9%的候选药物进入下一期临床；
在14个主要疾病中，血液疾病从I期开始的LOA最高（23.9%），是成功率最低的泌尿领域疾病（3.6%）的7倍。
免疫肿瘤学疗法在肿瘤药物研发方面取得了罕见的成功，总体LOA为12.4%，而所有肿瘤疗法的LOA为5.3%。
罕见疾病药物非常成功，总体LOA为17.0%。
慢性、高发性疾病药物不太成功，总体LOA为5.9%。
药物的复杂性通常带来更高的LOA，CAR-T和RNA干扰分别达到17.3%和13.5%的最高LOA。
采用患者预选生物标志物进行试验的研发项目具有两倍高的LOA（15.9%），II期临床成功率接近1/2。
成功的首要因素是疾病适应症、靶点、形态和药物创新性。
在研发时间上，从I期临床到批准平均需要10.5年，这包括I期临床2.3 年，II期临床3.6 年, III期临床3.3 年，审批阶段1.3年。高于平均LOA的病种往往开发时间最短。

（Key Takeaways
• The overall likelihood of approval (LOA) from Phase I for all developmental candidates over 2011–2020 was 7.9%.
• Phase II development remains the largest hurdle in drug development, with just 28.9% of candidates
achieving this critical phase transition.
• Of the 14 major disease areas, Hematology therapies had the highest LOA from Phase I (23.9%), representing a seven-fold increase over the least successful group, Urology (3.6%).
• Immuno-oncology therapies provide a rare pocket of success in oncology R&D with an overall LOA of 12.4% vs 5.3% for all oncology approaches.
• Rare disease therapies were notably successful with an overall LOA of 17.0%.
• Chronic, high prevalence disease therapies were less successful with an overall LOA of 5.9%.
• Biological complexity in drug modalities generally leads to higher LOA, with CAR-T and RNA interference
achieving the highest LOAs of 17.3% and 13.5%, respectively.
• Development programs with trials employing patient preselection biomarkers have two-fold higher LOAs
(15.9%), driven by a Phase II success rate of nearly one-in-two.
• The top contributing factors toward phase success are disease indication, target, modality, and drug
novelty.
• On average, it takes 10.5 years for a Phase I asset to progress to regulatory approval. Disease areas with
above-average LOAs tend to have the shortest development timelines.）