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From isolated hypotheses to connected practical studies: Statisticians' role in a seamless targeted therapy development

机译:从孤立的假设到相关的实践研究:统计学家在无缝针对性疗法发展中的作用

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摘要

Target-based therapeutic development and personalized cancer medicine is, and will remain, a hot topic in oncology. This reflects advances in understanding the triangular relationship among patient well being, disease prognosis and treatment impact. Although rapid advances in technology have facilitated a wide range of molecular biomarker discoveries, targeted drug development still suffers from a high failure rate. There is a clear gap between the blooming discoveries in laboratory science and the tremendous unmet need in clinical practice for patient care [2]. Scientists from many disciplines contribute their expertise and creativity into drug development; however, often in an isolated fashion. The need for greater collaboration has been widely recognized. An urgent call for an innovative integration is needed, an integration that goes beyond merely collaboration among multidisciplinary scientists, toward a platform that allows interactive and iterative collaboration from early laboratory discovery to confirmation of clinical benefit in randomized controlled studies, through a sophisticated infrastructure to achieve the prospectively defined strategic goal(s).
机译:基于目标的治疗开发和个性化癌症医学一直是并将继续成为肿瘤学的热门话题。这反映出在理解患者健康,疾病预后和治疗影响之间的三角关系方面的进展。尽管技术的飞速发展促进了广泛的分子生物标志物发现,但是靶向药物的开发仍然遭受高失败率的困扰。在实验室科学的蓬勃发展的发现与临床实践中对患者护理的巨大未满足需求之间存在明显的差距[2]。来自许多学科的科学家将他们的专业知识和创造力投入到药物开发中。但是,通常以孤立的方式。人们已经广泛认识到需要加强合作。迫切需要创新的整合,这种整合不仅仅需要多学科科学家之间的协作,还需要一个平台,该平台可以通过复杂的基础架构,从早期的实验室发现到随机对照研究的临床获益确认,进行互动和迭代式协作。预期定义的战略目标。

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