Bullement, A. orcid.org/0000-0001-7091-0972 and Kearns, B. orcid.org/0000-0001-7730-668X (2022) Incorporating external trial data to improve survival extrapolations: a pilot study of the COU-AA-301 trial. Health Services and Outcomes Research Methodology, 22. pp. 317-331. ISSN 1387-3741
Abstract
Survival extrapolation plays a key role within cost effectiveness analysis and is often subject to substantial uncertainty. Use of external data to improve extrapolations has been identified as a key research priority. We present findings from a pilot study using data from the COU-AA-301 trial of abiraterone acetate for metastatic castration-resistant prostate cancer, to explore how external trial data may be incorporated into survival extrapolations. External trial data were identified via a targeted search of technology assessment reports. Four methods using external data were compared to simple parametric models (SPMs): informal reference to external data to select appropriate SPMs, piecewise models with, and without, hazard ratio adjustment, and Bayesian models fitted with a prior on the shape parameter(s). Survival and hazard plots were compared, and summary metrics (point estimate accuracy and restricted mean survival time) were calculated. Without consideration of external data, several SPMs may have been selected as the ‘best-fitting’ model. The range of survival probability estimates was generally reduced when external data were included in model estimation, and external hazard plots aided model selection. Different methods yielded varied results, even with the same data source, highlighting potential issues when integrating external trial data within model estimation. By using external trial data, the most (in)appropriate models may be more easily identified. However, benefits of using external data are contingent upon their applicability to the research question, and the choice of method can have a large impact on extrapolations.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © The Author(s) 2022. Open Access: This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. |
Keywords: | Survival analysis; external data; extrapolation; cost-effectiveness analysis |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) > ScHARR - Sheffield Centre for Health and Related Research |
Funding Information: | Funder Grant number NATIONAL INSTITUTE FOR HEALTH RESEARCH DRF-2016-09-119 |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 15 Nov 2021 14:46 |
Last Modified: | 03 Feb 2023 12:43 |
Status: | Published |
Publisher: | Springer |
Refereed: | Yes |
Identification Number: | 10.1007/s10742-021-00264-6 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:180227 |
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