ArticleStatistics in medicine2018
Five criteria for using a surrogate endpoint to predict treatment effect based on data from multiple previous trials.
Article in Statistics in medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 13 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Correlation between pathologic complete response, event-free survival/disease-free survival and overall survival in neoadjuvant and/or adjuvant HR+/HER2-breast cancer.Frontiers in oncology · 2023Pooled it
- Pooled it
- A framework to evaluate surrogate endpoints at the trial level: analysis in colorectal cancer.Journal of the National Cancer Institute · 2026Article
- Albuminuria Changes as a surrogate endpoint inmedRxiv : the preprint server for health sciences · 2026Article
- Impact of LEAP-012 and EMERALD-1 in the management of HCC.JHEP reports : innovation in hepatology · 2026Review
- Improving the efficiency of drug resistant tuberculosis treatment trials: a time-to-event alternative marker for bacteriological response and adaptive minimization for randomization.BMC medical research methodology · 2025Article
- Aesthetics and Patient-Reported Outcomes in Periodontology and Implant Dentistry: Consensus Report.Journal of clinical periodontology · 2025Article
- A New Frequentist Implementation of the Daniels and Hughes Bivariate Meta-Analysis Model for Surrogate Endpoint Evaluation.Biometrical journal. Biometrische Zeitschrift · 2025Article
- Surrogate endpoint metaregression: useful statistics for regulators and trialists.Journal of clinical epidemiology · 2024Article
- Considerations for using potential surrogate endpoints in cancer screening trials.The Lancet. Oncology · 2024Review
- Criteria for assessing evidence for biomarker-targeted therapies in rare cancers-an extrapolation framework.Therapeutic advances in medical oncology · 2024Article
- Simple Methods for Evaluating 4 Types of Biomarkers: Surrogate Endpoint, Prognostic, Predictive, and Cancer Screening.Biomarker insights · 2020Review
- Evaluation of amyloid change as a surrogate for cognitive decline: demonstration in individual-level data from the A4 study of solanezumab.Alzheimer's & dementia (New York, N. Y.)Article
Corrections and comments
- Erratum issued
Authors and funding
1 author.
Funding
Abstract
A surrogate endpoint in a randomized clinical trial is an endpoint that occurs after randomization and before the true, clinically meaningful, endpoint that yields conclusions about the effect of treatment on true endpoint. A surrogate endpoint can accelerate the evaluation of new treatments but at the risk of misleading conclusions. Therefore, criteria are needed for deciding whether to use a surrogate endpoint in a new trial. For the meta-analytic setting of multiple previous trials, each with the same pair of surrogate and true endpoints, this article formulates 5 criteria for using a surrogate endpoint in a new trial to predict the effect of treatment on the true endpoint in the new trial. The first 2 criteria, which are easily computed from a zero-intercept linear random effects model, involve statistical considerations: an acceptable sample size multiplier and an acceptable prediction separation score. The remaining 3 criteria involve clinical and biological considerations: similarity of biological mechanisms of treatments between the new trial and previous trials, similarity of secondary treatments following the surrogate endpoint between the new trial and previous trials, and a negligible risk of harmful side effects arising after the observation of the surrogate endpoint in the new trial. These 5 criteria constitute an appropriately high bar for using a surrogate endpoint to make a definitive treatment recommendation.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.