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

被引：0

作者：

Rahman, Rifaquat ^{[1
]}

Ventz, Steffen ^{[2
]}

McDunn, Jon ^{[4
]}

Louv, Bill ^{[4
]}

Reyes-Rivera, Irmarie ^{[5
]}

Polley, Mei-Yin C. ^{[6
]}

Merchant, Fahar ^{[7
]}

Abrey, Lauren E. ^{[8
]}

Allen, Joshua E. ^{[9
]}

Aguilar, Laura K. ^{[10
]}

Aguilar-Cordova, Estuardo ^{[10
]}

Arons, David ^{[11
]}

Tanner, Kirk ^{[11
]}

Bagley, Stephen ^{[12
]}

Khasraw, Mustafa ^{[13
]}

Cloughesy, Timothy ^{[14
]}

Wen, Patrick Y. ^{[3
]}

Alexander, Brian M. ^{[1
,15
]}

Trippa, Lorenzo ^{[2
]}

Roche, F. Hoffmann-La

Aguilar-Cordova, E.

Tanner, K.

机构：

[1] Harvard Med Sch, Dept Radiat Oncol, Dana Farber Brigham & Womens Canc Ctr, Boston, MA 02115 USA

[2] Harvard TH Chan Sch Publ Hlth, Dept Data Sci, Boston, MA USA

[3] Harvard TH Chan Sch Publ Hlth, Dana Farber Canc Inst, Ctr Neurooncol, Boston, MA USA

[4] Project Data Sphere, Morrisville, NC USA

[5] F Hoffmann La Roche, Basel, Switzerland

[6] Univ Chicago, Dept Publ Hlth Sci, Chicago, IL 60637 USA

[7] MedicennaTherapeut, Toronto, ON, Canada

[8] Novartis, Basel, Switzerland

[9] Chimerix, Durham, NC USA

[10] Candel Therapeut, Needham, MA USA

[11] Natl BrainTumor Soc, Newton, MA USA

[12] Univ Penn, Perelman Sch Med, Div Hematol & Oncol, Philadelphia, PA 19104 USA

[13] Duke Univ, Med Ctr, Preston Robert Tisch Brain Tumor Ctr, Dept Neurosurg, Durham, NC USA

[14] Univ Calif Los Angeles, Dept Neurol, David Geffen Sch Med, Los Angeles, CA USA

[15] Fdn Med, Cambridge, MA USA

来源：

LANCET ONCOLOGY | 2021年 / 22卷 / 10期

基金：

美国国家卫生研究院;

关键词：

HISTORICAL CONTROL DATA; REAL-WORLD EVIDENCE; SINGLE-ARM; ADJUVANT PACLITAXEL; ADJUSTMENT; SURVIVAL; RANDOMIZATION; TEMOZOLOMIDE; TRASTUZUMAB; BEVACIZUMAB;

D O I：

暂无

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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 contextualising single-arm studies and improving decision making (eg, early stopping decisions). Based on a series of presentations at the 2020 Clinical Trials Think Tank hosted by the Society of Neuro-Oncology, we provide an overview on the use of external control data representative of the standard of care in the design and analysis of clinical trials. High-quality patient-level records, rigorous methods, and validation analyses are necessary to effectively leverage external data. We review study designs, statistical methods, risks, and potential distortions in using external data from completed trials and real-world data, as well as data sources, data sharing models, ongoing work, and applications in glioblastoma.

引用

页码：E456 / E465

页数：10

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