Real-world evidence: Driving a new drug-development paradigm in oncology

Introduction Evidence generation in oncology is at a key inflection point. Given the rapid pace of scientific innovation, historical approaches to drug development are increasingly burdensome, with randomized controlled trials remaining incredibly costly and time intensive to conduct, and having a low certainty of success. Biopharmaceutical companies will collectively invest USD 50 billion to support oncology research and development in 2018, with a ~3 percent probability of success for any individual product from preclinical through registration phases.1 Despite this investment, many completed trials fail to answer critical questions for payors and healthcare

引言 肿瘤学中的证据生成正处于一个关键的转折点。鉴于科学创新的快速步伐，药物研发的传统方法日益繁重，随机对照试验的开展成本仍然极高且耗时，并且成功的确定性较低。2018年，生物制药公司将总计投资500亿美元用于支持肿瘤学的研发，从临床前到注册阶段，任何单个产品的成功概率约为3%。尽管有此投资，许多已完成的试验未能为支付方和医疗保健机构解答关键问题。