Hernandez-Villafuerte, K., Fischer, A. and Latimer, N.R. orcid.org/0000-0001-5304-5585 (2018) Challenges and methodologies in using progression free survival as a surrogate for overall survival In oncology. International Journal of Technology Assessment in Health Care, 34 (3). pp. 300-316. ISSN 0266-4623
Abstract
Objectives: A primary outcome in oncology trials is overall survival (OS). However, to estimate OS accurately requires a sufficient number of patients to have died, which may take a long time. If an alternative end point is sufficiently highly correlated with OS, it can be used as a surrogate. Progression-free survival (PFS) is the surrogate most often used in oncology, but does not always satisfy the correlation conditions for surrogacy. We analyze the methodologies used when extrapolating from PFS to OS.
Methods: Davis et al. previously reviewed the use of surrogate end points in oncology, using papers published between 2001 and 2011. We extend this, reviewing papers published between 2012 and 2016. We also examine the reporting of statistical methods to assess the strength of surrogacy.
Results: The findings from 2012 to 2016 do not differ substantially from those of 2001 to 2011: the same factors are shown to affect the relationship between PFS and OS. The proportion of papers reporting individual patient data (IPD), strongly recommended for full assessment of surrogacy, remains low: 33 percent. A wide range of methods has been used to determine the appropriateness of surrogates. While usually adhering to reporting standards, the standard of scholarship appears sometimes to be questionable and the reporting of results often haphazard.
Conclusions: Standards of analysis and reporting PFS to OS surrogate studies should be improved by increasing the rigor of statistical reporting and by agreeing to a minimum set of reporting guidelines. Moreover, the use of IPD to assess surrogacy should increase.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2018 Cambridge University Press. This is an author produced version of a paper subsequently published in International Journal of Technology Assessment in Health Care. Article available under the terms of the CC-BY-NC-ND license (https://creativecommons.org/licenses/by-nc-nd/4.0/). |
Keywords: | Cancer; surrogate endpoints; overall survival (OS); progression-free survival (PFS); post-progression survival (PPS) |
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 |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 18 May 2018 10:55 |
Last Modified: | 19 May 2020 10:52 |
Published Version: | https://doi.org/10.1017/S0266462318000338 |
Status: | Published |
Publisher: | Cambridge University Press (CUP) |
Refereed: | Yes |
Identification Number: | 10.1017/S0266462318000338 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:130810 |
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