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Real-world evidence was feasible for estimating effectiveness of chemotherapy in breast cancer

Real-world evidence was feasible for estimating effectiveness of chemotherapy in breast cancer
Real-world evidence was feasible for estimating effectiveness of chemotherapy in breast cancer
Objective: Evidence-based guidelines recommend adjuvant chemotherapy in early stage breast cancer whenever treatment benefit is considered sufficient to outweigh the associated risks. However, many groups of patients were either excluded from or underrepresented in the clinical trials that form the evidence base for this recommendation. This study aims to determine whether using administrative healthcare data – Real World Data (RWD) - and econometric methods for causal analysis to provide ‘Real World Evidence’ (RWE) are feasible methods for addressing this gap.
Methods: Cases of primary breast cancer in women from 2001 to 2015 were extracted from the Scottish cancer registry (SMR06) and linked to other routine health records (inpatient and outpatient visits). Four methods were used to estimate the effect of adjuvant chemotherapy on disease-specific and overall mortality: (1) regression with adjustment for covariates (2) propensity score matching (3) instrumental variables analysis and (4) regression discontinuity design. Hazard ratios for breast cancer mortality and all-cause mortality were compared to those from a meta-analysis of randomised trials.
Results: 39,805 cases included in the analyses. Regression adjustment, propensity score matching and instrumental variables were feasible while regression discontinuity was not. Effectiveness estimates were similar between RWE and randomised trials for breast cancer mortality but not for all-cause mortality.
Conclusions: RWE methods are a feasible means to generate estimates of effectiveness of adjuvant chemotherapy in early stage breast cancer. However, such estimates must be interpreted in the context of the available randomised evidence and the potential biases of the observational methods.
Adjuvant chemotherapy, Breast cancer, Feasibility, Instrumental variables, Meta-analysis, Propensity score, Real world evidence, Regression discontinuity
0895-4356
125-132
Gray, Ewan
640f76cd-997b-4a8d-8690-081ffdcd7d01
Marti, Joachim
55e9017a-66c0-4f11-9d38-97cbccac95f4
Brewster, David H.
0ee02f74-62c5-480c-9956-2cf86b4abeb2
Wyatt, Jeremy C.
8361be5a-fca9-4acf-b3d2-7ce04126f468
Piaget-Rossel, Romain
b07442d0-f2da-41aa-9dbd-afcafe8b6d27
Hall, Peter S.
d45f9300-0dd4-4ad7-bd9d-f23873f80616
Gray, Ewan
640f76cd-997b-4a8d-8690-081ffdcd7d01
Marti, Joachim
55e9017a-66c0-4f11-9d38-97cbccac95f4
Brewster, David H.
0ee02f74-62c5-480c-9956-2cf86b4abeb2
Wyatt, Jeremy C.
8361be5a-fca9-4acf-b3d2-7ce04126f468
Piaget-Rossel, Romain
b07442d0-f2da-41aa-9dbd-afcafe8b6d27
Hall, Peter S.
d45f9300-0dd4-4ad7-bd9d-f23873f80616

Gray, Ewan, Marti, Joachim, Brewster, David H., Wyatt, Jeremy C., Piaget-Rossel, Romain and Hall, Peter S. (2019) Real-world evidence was feasible for estimating effectiveness of chemotherapy in breast cancer. Journal of Clinical Epidemiology, 109, 125-132. (doi:10.1016/j.jclinepi.2019.01.006).

Record type: Article

Abstract

Objective: Evidence-based guidelines recommend adjuvant chemotherapy in early stage breast cancer whenever treatment benefit is considered sufficient to outweigh the associated risks. However, many groups of patients were either excluded from or underrepresented in the clinical trials that form the evidence base for this recommendation. This study aims to determine whether using administrative healthcare data – Real World Data (RWD) - and econometric methods for causal analysis to provide ‘Real World Evidence’ (RWE) are feasible methods for addressing this gap.
Methods: Cases of primary breast cancer in women from 2001 to 2015 were extracted from the Scottish cancer registry (SMR06) and linked to other routine health records (inpatient and outpatient visits). Four methods were used to estimate the effect of adjuvant chemotherapy on disease-specific and overall mortality: (1) regression with adjustment for covariates (2) propensity score matching (3) instrumental variables analysis and (4) regression discontinuity design. Hazard ratios for breast cancer mortality and all-cause mortality were compared to those from a meta-analysis of randomised trials.
Results: 39,805 cases included in the analyses. Regression adjustment, propensity score matching and instrumental variables were feasible while regression discontinuity was not. Effectiveness estimates were similar between RWE and randomised trials for breast cancer mortality but not for all-cause mortality.
Conclusions: RWE methods are a feasible means to generate estimates of effectiveness of adjuvant chemotherapy in early stage breast cancer. However, such estimates must be interpreted in the context of the available randomised evidence and the potential biases of the observational methods.

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SATURNE RWE JCE revision 1 clean - Author's Original
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More information

Accepted/In Press date: 16 January 2019
e-pub ahead of print date: 31 January 2019
Published date: 1 May 2019
Keywords: Adjuvant chemotherapy, Breast cancer, Feasibility, Instrumental variables, Meta-analysis, Propensity score, Real world evidence, Regression discontinuity

Identifiers

Local EPrints ID: 430584
URI: http://eprints.soton.ac.uk/id/eprint/430584
ISSN: 0895-4356
PURE UUID: 830161b0-51d0-4203-bc6c-70413ccb0a21
ORCID for Jeremy C. Wyatt: ORCID iD orcid.org/0000-0001-7008-1473

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Date deposited: 03 May 2019 16:30
Last modified: 26 Nov 2021 03:05

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Contributors

Author: Ewan Gray
Author: Joachim Marti
Author: David H. Brewster
Author: Jeremy C. Wyatt ORCID iD
Author: Romain Piaget-Rossel
Author: Peter S. Hall

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