From real‐world patient data to individualized treatment effects using machine learning: current and future methods to address underlying challenges

I Bica, AM Alaa, C Lambert… - Clinical Pharmacology …, 2021 - Wiley Online Library
Clinical decision making needs to be supported by evidence that treatments are beneficial to
individual patients. Although randomized control trials (RCTs) are the gold standard for …

Model-informed precision dosing: background, requirements, validation, implementation, and forward trajectory of individualizing drug therapy

AS Darwich, TM Polasek, JK Aronson… - Annual review of …, 2021 - annualreviews.org
Model-informed precision dosing (MIPD) has become synonymous with modern approaches
for individualizing drug therapy, in which the characteristics of each patient are considered …

Leveraging external data in the design and analysis of clinical trials in neuro-oncology

R Rahman, S Ventz, J McDunn, B Louv… - The Lancet …, 2021 - thelancet.com
Integration of external control data, with patient-level information, in clinical trials has the
potential to accelerate the development of new treatments in neuro-oncology by …

[HTML][HTML] Real-world data analyses unveiled the immune-related adverse effects of immune checkpoint inhibitors across cancer types

F Wang, S Yang, N Palmer, K Fox, IS Kohane… - NPJ precision …, 2021 - nature.com
Immune checkpoint inhibitors have demonstrated significant survival benefits in treating
many types of cancers. However, their immune-related adverse events (irAEs) have not …

[HTML][HTML] Replication of randomized, controlled trials using real-world data: what could go wrong?

D Thompson - Value in health, 2021 - Elsevier
With the growing interest in using real-world evidence (RWE) for regulatory purposes,
researchers and policy makers are considering how best to assess the credibility of RWE …

Historical controls in randomized clinical trials: opportunities and challenges

KT Hall, L Vase, DK Tobias, HT Dashti… - Clinical …, 2021 - Wiley Online Library
Randomized control trials (RCTs) with placebo are the gold standard for determining
efficacy of novel pharmaceutical treatments. Since their inception, over 75 years ago …

Evolving use of real-world evidence in the regulatory process: a focus on immuno-oncology treatment and outcomes

JC O'Donnell, TK Le, R Dobrin, M Higashi… - Future …, 2021 - Taylor & Francis
In recent years, regulatory bodies have increasingly recognized the utility of real-world
evidence (RWE) for supplementing and supporting clinical trial data in new drug …

Efficient and robust semi-supervised estimation of ATE with partially annotated treatment and response

J Hou, R Mukherjee, T Cai - arXiv preprint arXiv:2110.12336, 2021 - arxiv.org
A notable challenge of leveraging Electronic Health Records (EHR) for treatment effect
assessment is the lack of precise information on important clinical variables, including the …

Heterogeneous Treatment Effect Estimation using machine learning for Healthcare application: tutorial and benchmark

Y Ling, P Upadhyaya, L Chen, X Jiang… - arXiv preprint arXiv …, 2021 - arxiv.org
Developing new drugs for target diseases is a time-consuming and expensive task, drug
repurposing has become a popular topic in the drug development field. As much health …

[HTML][HTML] 基于文献计量与知识图谱分析的真实世界研究发展现状及趋势

承鹭郭, 文惠, 林何, 玲李, 鑫孙 - Sheng Wu Yi Xue Gong Cheng …, 2021 - ncbi.nlm.nih.gov
文章通过回顾性可视化分析方法, 探讨有关真实世界研究中文文献的热点与趋势。
本文检索了知网、 维普、 万方及中国生物医学文献数据库, 文献收录时间从数据库起始截至 …