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http://hdl.handle.net/1942/37192
Title: | Surrogacy Beyond Prognosis: The Importance of “Trial-Level” Surrogacy | Authors: | BUYSE, Marc Saad, Everardo D. BURZYKOWSKI, Tomasz Regan, Meredith M. Sweeney, Christopher S. |
Issue Date: | 2022 | Publisher: | OXFORD UNIV PRESS | Source: | The Oncologist, 2022, Vol. 17, N°.4 | Status: | Early view | Abstract: | Many candidate surrogate endpoints are currently assessed using a 2-level statistical approach, which consists in checking whether (1) the potential surrogate is associated with the final endpoint in individual patients and (2) the effect of treatment on the surrogate can be used to reliably predict the effect of treatment on the final endpoint. In some situations, condition (1) is fulfilled but condition (2) is not. We use concepts of causal inference to explain this apparently paradoxical situation, illustrating this review with 2 contrasting examples in operable breast cancer: the example of pathological complete response (pCR) and that of disease-free survival (DFS). In a previous meta-analysis, pCR has been shown to be a strong and independent prognostic factor for event-free survival (EFS) and overall survival (OS) after neoadjuvant treatment of operable breast cancer. Yet, in randomized trials, the effects of experimental treatments on pCR have not translated into predictable effects on EFS or OS, making pCR an "individual-level" surrogate, but not a "trial-level" surrogate. In contrast, DFS has been shown to be an acceptable surrogate for OS at both the individual and trial levels in early, HER2-positive breast cancer. The distinction between the prognostic and predictive roles of a tentative surrogate, not always made in the literature, avoids unnecessary confusion and allows better understanding of what it takes to validate a surrogate endpoint that is truly able to replace a final endpoint. | Notes: | Buyse, M (corresponding author), Int Drug Dev Inst, Ave Prov 30, B-1340 Louvain La Neuve, Belgium. marc.buyse@iddi.com |
Document URI: | http://hdl.handle.net/1942/37192 | ISSN: | 1083-7159 | e-ISSN: | 1549-490X | DOI: | 10.1093/oncolo/oyac006 | ISI #: | WOS:000769640000001 | Rights: | This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited. | Category: | A1 | Type: | Journal Contribution | Validations: | ecoom 2023 |
Appears in Collections: | Research publications |
Files in This Item:
File | Description | Size | Format | |
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oyac006.pdf | Published version | 1.57 MB | Adobe PDF | View/Open |
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