Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity

J. Connor Wells; Adam Fundytus; Shubham Sharma; Wilma M. Hopman; Joseph C. Del Paggio; Bishal Gyawali; Deborah Mukherji; Nazik Hammad; C. S. Pramesh; Ajay Aggarwal; Richard Sullivan; Christopher M. Booth

doi:10.3390/curroncol29040207

Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity

J. Connor Wells, Adam Fundytus, Shubham Sharma, Wilma M. Hopman, Joseph C. Del Paggio, Bishal Gyawali, Deborah Mukherji, Nazik Hammad, C. S. Pramesh, Ajay Aggarwal, Richard Sullivan, Christopher M. Booth^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

3 Citations (Scopus)

Abstract

Background: In this study, we compared and contrasted design characteristics, results, and publications of randomized controlled trials (RCTs) in gastrointestinal (GI), lung, and breast cancer. Methods: A PUBMED search identified phase III RCTs of anticancer therapy in GI, lung, and breast cancer published globally during the period 2014–2017. Descriptive statistics, chi-square tests, and the Kruskal–Wallis test were used to compare RCT design, results, and output across the cancer sites. Results: A total of 352 RCTs were conducted on GI (36%), lung (29%), and breast (35%) cancer. Surrogate endpoints were used in 55% of trials; this was most common in breast trials (72%) compared to GI (47%) and lung trials (43%, p < 0.001). Breast trials more often met their primary endpoint (54%) than GI (41%) and lung trials (41%) (p = 0.024). When graded with the ESMO-MCBS, lung cancer trials (50%, 15/30) were more likely to meet the threshold for substantial benefit. GI trials were published in journals with a substantially lower impact factor (IF; median IF 13) than lung (median IF 21) and breast cancer trials (median IF 21) (p = 0.038). Conclusions: Important differences in RCT design and output exist between the three major cancer sites. Use of surrogate endpoints and the magnitude of benefit associated with new treatments vary substantially across cancer sites.

Original language	English
Pages (from-to)	2530-2538
Number of pages	9
Journal	Current Oncology
Volume	29
Issue number	4
DOIs	https://doi.org/10.3390/curroncol29040207
Publication status	Published - Apr 2022

Keywords

breast
cancer
design
gastrointestinal
lung
outcomes
randomized controlled trial

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.3390/curroncol29040207

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Connor Wells, J., Fundytus, A., Sharma, S., Hopman, W. M., Del Paggio, J. C., Gyawali, B., Mukherji, D., Hammad, N., Pramesh, C. S., Aggarwal, A., Sullivan, R., & Booth, C. M. (2022). Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity. Current Oncology, 29(4), 2530-2538. https://doi.org/10.3390/curroncol29040207

@article{b99f439300e447c2a25a003e28687ad8,

title = "Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity",

abstract = "Background: In this study, we compared and contrasted design characteristics, results, and publications of randomized controlled trials (RCTs) in gastrointestinal (GI), lung, and breast cancer. Methods: A PUBMED search identified phase III RCTs of anticancer therapy in GI, lung, and breast cancer published globally during the period 2014–2017. Descriptive statistics, chi-square tests, and the Kruskal–Wallis test were used to compare RCT design, results, and output across the cancer sites. Results: A total of 352 RCTs were conducted on GI (36%), lung (29%), and breast (35%) cancer. Surrogate endpoints were used in 55% of trials; this was most common in breast trials (72%) compared to GI (47%) and lung trials (43%, p < 0.001). Breast trials more often met their primary endpoint (54%) than GI (41%) and lung trials (41%) (p = 0.024). When graded with the ESMO-MCBS, lung cancer trials (50%, 15/30) were more likely to meet the threshold for substantial benefit. GI trials were published in journals with a substantially lower impact factor (IF; median IF 13) than lung (median IF 21) and breast cancer trials (median IF 21) (p = 0.038). Conclusions: Important differences in RCT design and output exist between the three major cancer sites. Use of surrogate endpoints and the magnitude of benefit associated with new treatments vary substantially across cancer sites.",

keywords = "breast, cancer, design, gastrointestinal, lung, outcomes, randomized controlled trial",

author = "{Connor Wells}, J. and Adam Fundytus and Shubham Sharma and Hopman, {Wilma M.} and {Del Paggio}, {Joseph C.} and Bishal Gyawali and Deborah Mukherji and Nazik Hammad and Pramesh, {C. S.} and Ajay Aggarwal and Richard Sullivan and Booth, {Christopher M.}",

note = "Funding Information: Our findings related to the growing role of industry in funding cancer clinical trials are consistent with our prior work. We have previously described that the proportion of breast, colorectal, and non-small cell lung cancer systemic therapy trials funded by industry increased from 4% in 1975–1984 to 78% in 2005–2009 [8]. Historically, a larger proportion of RCTs were funded by government grants [7]. The reasons for this substantial shift in RCT funding are multifactorial and likely reflect a decreased availability of government grants, high costs of cancer medicines incentivizing industry, and the increased complexity and expense associated with drug development and conducting contemporary RCTs [32,33]. Publisher Copyright: {\textcopyright} 2022 by the authors. Licensee MDPI, Basel, Switzerland.",

year = "2022",

month = apr,

doi = "10.3390/curroncol29040207",

language = "English",

volume = "29",

pages = "2530--2538",

journal = "Current Oncology",

issn = "1718-7729",

publisher = "Multidisciplinary Digital Publishing Institute (MDPI)",

number = "4",

}

Connor Wells, J, Fundytus, A, Sharma, S, Hopman, WM, Del Paggio, JC, Gyawali, B, Mukherji, D, Hammad, N, Pramesh, CS, Aggarwal, A , Sullivan, R & Booth, CM 2022, 'Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity', Current Oncology, vol. 29, no. 4, pp. 2530-2538. https://doi.org/10.3390/curroncol29040207

TY - JOUR

T1 - Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers

T2 - An Overview of Global Research Activity

AU - Connor Wells, J.

AU - Fundytus, Adam

AU - Sharma, Shubham

AU - Hopman, Wilma M.

AU - Del Paggio, Joseph C.

AU - Gyawali, Bishal

AU - Mukherji, Deborah

AU - Hammad, Nazik

AU - Pramesh, C. S.

AU - Aggarwal, Ajay

AU - Sullivan, Richard

AU - Booth, Christopher M.

N1 - Funding Information: Our findings related to the growing role of industry in funding cancer clinical trials are consistent with our prior work. We have previously described that the proportion of breast, colorectal, and non-small cell lung cancer systemic therapy trials funded by industry increased from 4% in 1975–1984 to 78% in 2005–2009 [8]. Historically, a larger proportion of RCTs were funded by government grants [7]. The reasons for this substantial shift in RCT funding are multifactorial and likely reflect a decreased availability of government grants, high costs of cancer medicines incentivizing industry, and the increased complexity and expense associated with drug development and conducting contemporary RCTs [32,33]. Publisher Copyright: © 2022 by the authors. Licensee MDPI, Basel, Switzerland.

PY - 2022/4

Y1 - 2022/4

N2 - Background: In this study, we compared and contrasted design characteristics, results, and publications of randomized controlled trials (RCTs) in gastrointestinal (GI), lung, and breast cancer. Methods: A PUBMED search identified phase III RCTs of anticancer therapy in GI, lung, and breast cancer published globally during the period 2014–2017. Descriptive statistics, chi-square tests, and the Kruskal–Wallis test were used to compare RCT design, results, and output across the cancer sites. Results: A total of 352 RCTs were conducted on GI (36%), lung (29%), and breast (35%) cancer. Surrogate endpoints were used in 55% of trials; this was most common in breast trials (72%) compared to GI (47%) and lung trials (43%, p < 0.001). Breast trials more often met their primary endpoint (54%) than GI (41%) and lung trials (41%) (p = 0.024). When graded with the ESMO-MCBS, lung cancer trials (50%, 15/30) were more likely to meet the threshold for substantial benefit. GI trials were published in journals with a substantially lower impact factor (IF; median IF 13) than lung (median IF 21) and breast cancer trials (median IF 21) (p = 0.038). Conclusions: Important differences in RCT design and output exist between the three major cancer sites. Use of surrogate endpoints and the magnitude of benefit associated with new treatments vary substantially across cancer sites.

AB - Background: In this study, we compared and contrasted design characteristics, results, and publications of randomized controlled trials (RCTs) in gastrointestinal (GI), lung, and breast cancer. Methods: A PUBMED search identified phase III RCTs of anticancer therapy in GI, lung, and breast cancer published globally during the period 2014–2017. Descriptive statistics, chi-square tests, and the Kruskal–Wallis test were used to compare RCT design, results, and output across the cancer sites. Results: A total of 352 RCTs were conducted on GI (36%), lung (29%), and breast (35%) cancer. Surrogate endpoints were used in 55% of trials; this was most common in breast trials (72%) compared to GI (47%) and lung trials (43%, p < 0.001). Breast trials more often met their primary endpoint (54%) than GI (41%) and lung trials (41%) (p = 0.024). When graded with the ESMO-MCBS, lung cancer trials (50%, 15/30) were more likely to meet the threshold for substantial benefit. GI trials were published in journals with a substantially lower impact factor (IF; median IF 13) than lung (median IF 21) and breast cancer trials (median IF 21) (p = 0.038). Conclusions: Important differences in RCT design and output exist between the three major cancer sites. Use of surrogate endpoints and the magnitude of benefit associated with new treatments vary substantially across cancer sites.

KW - breast

KW - cancer

KW - design

KW - gastrointestinal

KW - lung

KW - outcomes

KW - randomized controlled trial

UR - http://www.scopus.com/inward/record.url?scp=85128698422&partnerID=8YFLogxK

U2 - 10.3390/curroncol29040207

DO - 10.3390/curroncol29040207

M3 - Article

C2 - 35448181

AN - SCOPUS:85128698422

SN - 1718-7729

VL - 29

SP - 2530

EP - 2538

JO - Current Oncology

JF - Current Oncology

IS - 4

ER -

Randomized Controlled Trials in Lung, Gastrointestinal, and Breast Cancers: An Overview of Global Research Activity

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Keywords

UN SDGs

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