Abstract
In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker and genetically defined patient subgroups. This has produced a shift toward development of biomarker-targeted agents during a time when funding for cancer research has been limited; as a result, the need for improved operational efficiency in studying many agent-and-target combinations in parallel has emerged. Platform trials, basket trials, and umbrella trials are new approaches to clinical research driven by this need for enhanced efficiency in the modern era of increasingly specific cancer subpopulations and decreased resources to study treatments for individual cancer subtypes in a traditional way. In this review, we provide an overview of these new types of clinical trial designs, including discussions ofmotivation for their use, recommended terminology, examples, and challenges encountered in their application.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 34-43 |
| Number of pages | 10 |
| Journal | Annals of Oncology |
| Volume | 28 |
| Issue number | 1 |
| DOIs | |
| State | Published - Jan 1 2017 |
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Keywords
- Basket trialbiomarker-based trialmaster protocolplatform trialprecision medicineumbrella trial
ASJC Scopus subject areas
- Hematology
- Oncology
Cite this
Statistical controversies in clinical research : Basket trials, umbrella trials, and other master protocols: A review and examples. / Renfro, L. A.; Sargent, D. J.
In: Annals of Oncology, Vol. 28, No. 1, 01.01.2017, p. 34-43.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Statistical controversies in clinical research
T2 - Basket trials, umbrella trials, and other master protocols: A review and examples
AU - Renfro, L. A.
AU - Sargent, D. J.
PY - 2017/1/1
Y1 - 2017/1/1
N2 - In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker and genetically defined patient subgroups. This has produced a shift toward development of biomarker-targeted agents during a time when funding for cancer research has been limited; as a result, the need for improved operational efficiency in studying many agent-and-target combinations in parallel has emerged. Platform trials, basket trials, and umbrella trials are new approaches to clinical research driven by this need for enhanced efficiency in the modern era of increasingly specific cancer subpopulations and decreased resources to study treatments for individual cancer subtypes in a traditional way. In this review, we provide an overview of these new types of clinical trial designs, including discussions ofmotivation for their use, recommended terminology, examples, and challenges encountered in their application.
AB - In recent years, cancers once viewed as relatively homogeneous in terms of organ location and treatment strategy are now better understood to be increasingly heterogeneous across biomarker and genetically defined patient subgroups. This has produced a shift toward development of biomarker-targeted agents during a time when funding for cancer research has been limited; as a result, the need for improved operational efficiency in studying many agent-and-target combinations in parallel has emerged. Platform trials, basket trials, and umbrella trials are new approaches to clinical research driven by this need for enhanced efficiency in the modern era of increasingly specific cancer subpopulations and decreased resources to study treatments for individual cancer subtypes in a traditional way. In this review, we provide an overview of these new types of clinical trial designs, including discussions ofmotivation for their use, recommended terminology, examples, and challenges encountered in their application.
KW - Basket trialbiomarker-based trialmaster protocolplatform trialprecision medicineumbrella trial
UR - http://www.scopus.com/inward/record.url?scp=85015747268&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85015747268&partnerID=8YFLogxK
U2 - 10.1093/annonc/mdw413
DO - 10.1093/annonc/mdw413
M3 - Article
C2 - 28177494
AN - SCOPUS:85015747268
VL - 28
SP - 34
EP - 43
JO - Annals of Oncology
JF - Annals of Oncology
SN - 0923-7534
IS - 1
ER -