European Medicines Agency (EMA), Amsterdam, The Netherlands.
Medical University of Vienna, Vienna, Austria.
Clin Pharmacol Ther. 2021 May;109(5):1212-1218. doi: 10.1002/cpt.2083. Epub 2020 Nov 12.
Compared with drugs from the blockbuster era, recently authorized drugs and those expected in the future present a heterogenous mix of chemicals, biologicals, and cell and gene therapies, a sizable fraction being for rare diseases, and even individualized treatments or individualized combinations. The shift in the nature of products entails secular trends for the definitions of "drugs" and "target population" and for clinical use and evidence generation. We discuss that the lessons learned from evidence generation for 20th century medicines may have limited relevance for 21st century medicines. We explain why the future is not about randomized controlled trials (RCTs) vs. real-world evidence (RWE) but RCTs and RWE-not just for the assessment of safety but also of effectiveness. Finally, we highlight that, in the era of precision medicine, we may not be able to reliably describe some small treatment effects-either by way of RCTs or RWE.
与重磅炸弹药物相比，最近批准的药物和未来预期的药物呈现出化学物质、生物制品、细胞和基因治疗的混合体，相当一部分是针对罕见疾病的，甚至是个体化治疗或个体化组合。产品性质的转变需要对“药物”和“目标人群”的定义以及临床使用和证据生成进行长期的趋势分析。我们讨论了从 20 世纪药物的证据生成中获得的经验教训可能对 21 世纪药物的相关性有限。我们解释了为什么未来不是随机对照试验（RCT）与真实世界证据（RWE）的对抗，而是 RCT 和 RWE——不仅用于安全性评估，也用于有效性评估。最后，我们强调，在精准医学时代，我们可能无法可靠地描述某些较小的治疗效果——无论是通过 RCT 还是 RWE。
