学 术 报 告
2020年6月11日（周四），上午9:00～10:00（北京时间）
ZOOM 会议ID：984 8059 3697 （密码: 490397）
Incorporating historical information and real-world evidence to improve phase I clinical trials
| Dr Ying Yuan Bettyann Asche Murray Distinguished Professor Department of Biostatistics University of Texas MD Anderson Cancer Center |
摘要:
Incorporating historical data or real-world evidence has a great potential to improve the efficiency of phase I clinical trials and to accelerate drug development. For model-based designs, such as the continuous reassessment method (CRM), this can be conveniently carried out by specifying a ``skeleton, i.e., the prior estimate of dose limiting toxicity (DLT) probability at each dose. In contrast, little work has been done to incorporate historical data or real-world evidence into model-assisted designs, such as the Bayesian optimal interval (BOIN) and keyboard designs. This has led to the misconception that model-assisted designs cannot incorporate prior information. In this paper, we propose a unified framework that allows for incorporating historical data or real-world evidence into model-assisted designs. The proposed approach uses the well-established ``skeleton approach, combined with the concept of prior effective sample size, thus it is easy to understand and use. More importantly, our approach maintains the hallmark of model-assisted designs: simplicity---the dose escalation/de-escalation rule can be tabulated prior to the trial conduct. Extensive simulation studies show that the proposed method can effectively incorporate prior information to improve the operating characteristics of model-assisted designs, similarly to model-based designs.
报告人简介：
Dr. Ying Yuan is a Bettyann Asche Murray Distinguished Professor and Deputy Chair in the Department of Biostatistics at the MD Anderson Cancer Center. Dr. Yuan is an internationally renowned researcher in Bayesian adaptive clinical trial design. He has published over 100 methodology papers on innovative Bayesian adaptive designs, including early phase trials, seamless trials, biomarker-guided trials, and basket and platform trials. The designs and software (www.trialdesign.org) developed by Dr. Yuan are widely used in medical research institutes, pharmaceutical companies and the National Cancer Institute. Dr. Yuan was elected as an American Statistical Association (ASA) fellow in 2017. Dr. Yuan is the Chair of Data Safety Monitoring Board (DSMB) at MD Anderson Cancer Center, overseeing over 140 ongoing phase II and III randomized oncology clinical trials. He wrote book “Bayesian Designs for Phase I-II Clinical Trials” published by Chapman & Hall/CRC.