Department of Nuclear Medicine, Fiona Stanley Fremantle Hospitals Group, The University of Western Australia, Murdoch, Australia.
Cancer Biother Radiopharm. 2021 Feb;36(1):1-9. doi: 10.1089/cbr.2020.4129. Epub 2020 Sep 15.
Pharmaceutical industry clinical trials are ethically problematic: human research subjects are being used as a means to the end of demonstrating statistically significant efficacy of novel anticancer agents to achieve regulatory registration and marketing approval. Randomized controlled trial design is inequitable since control arm patients are denied access to the postulated best treatment. Most pharma studies do not provide clinically meaningful benefit of increased overall survival and enhanced quality of life (QOL) to cohorts and are not reliably generalizable to real-world patients. Precision oncology now enables prospective identification of patients expressing a specific cancer biomarker to determine their particular eligibility for evaluation of efficiency of molecular-targeted treatments. A patient-centered approach, collecting prospective real-world data in large populations, could provide real-world evidence of cost-effective, sustained clinical benefits of survival and QOL, while preserving the ethical beneficent compact between patient and doctor.
人类研究对象被用作手段，以证明新型抗癌药物在统计学上的疗效，从而获得监管注册和市场批准。随机对照试验设计是不公平的，因为对照臂患者被剥夺了接受假设的最佳治疗的机会。大多数制药研究并没有为队列提供增加总体生存和提高生活质量（QOL）的临床意义上的益处，并且不能可靠地推广到实际患者。精准肿瘤学现在能够前瞻性地识别表达特定癌症生物标志物的患者，以确定他们是否有资格接受分子靶向治疗的效果评估。以患者为中心的方法，在大量人群中收集前瞻性的真实世界数据，可以提供具有成本效益、持续生存和 QOL 临床获益的真实世界证据，同时保护患者和医生之间的伦理有益契约。
