The objective of this paper is to identify the extent to which real world data (RWD) is being utilized, or could be utilized, at scale in drug development. Through screening peer-reviewed literature, we have cited specific examples where RWD can be used for biomarker discovery or validation, gaining a new understanding of a disease or disease associations, discovering new markers for patient stratification and targeted therapies, new markers for identifying persons with a disease, and pharmacovigilance. None of the papers meeting our criteria was specifically geared toward novel targets or indications in the biopharmaceutical sector; the majority were focused on the area of public health, often sponsored by universities, insurance providers or in combination with public health bodies such as national insurers. The field is still in an early phase of practical application, and is being harnessed broadly where it serves the most direct need in public health applications in early, rare and novel disease incidents. However, these exemplars provide a valuable contribution to insights on the use of RWD to create novel, faster and less invasive approaches to advance disease understanding and biomarker discovery. We believe that pharma needs to invest in making better use of Electronic Health Records and the need for more precompetitive collaboration to grow the scale of this ‘big denominator’ capability, especially given the needs of precision medicine research.

本文的目的是确定在药物开发中大规模使用或可以利用真实世界数据（RWD）的程度。通过筛选经过同行评审的文献，我们列举了一些具体的例子，其中RWD可用于生物标志物的发现或验证，对疾病或疾病关联的新了解，发现用于患者分层和靶向治疗的新标志物，用于识别患有以下疾病的人的新标志物疾病和药物警戒。符合我们标准的论文均未专门针对生物制药领域的新目标或适应症。多数集中在公共卫生领域，通常由大学，保险提供商赞助或与公共卫生机构（如国家保险公司）联合发起。该领域仍处于实际应用的早期阶段，在早期，罕见和新型疾病事件中，在公共卫生应用中最直接需要的地方得到广泛应用。但是，这些示例为使用RWD创建新颖，更快，侵入性更强的方法以促进疾病理解和生物标记物发现提供了宝贵的见解。我们认为，制药公司需要投资以更好地利用电子病历，并且需要开展更多的竞争前合作以扩大这种“大分母”能力的规模，特别是考虑到精密医学研究的需求。这些示例为使用RWD创建新颖，更快，侵入性更强的方法以促进疾病理解和生物标记发现的见解提供了宝贵的帮助。我们认为，制药公司需要投资以更好地利用电子病历，并且需要开展更多的竞争前合作以扩大这种“大分母”能力的规模，特别是考虑到精密医学研究的需求。这些示例为使用RWD创建新颖，更快，侵入性更强的方法以促进疾病理解和生物标记发现的见解提供了宝贵的帮助。我们认为，制药公司需要投资以更好地利用电子病历，并且需要开展更多的竞争前合作以扩大这种“大分母”能力的规模，特别是考虑到精密医学研究的需求。