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Given the long follow-up periods that are often required for treatment or intervention studies, the potential to use surrogate markers to decrease the required follow-up time is a very attractive goal. However, previous studies have shown…

Methodology · Statistics 2016-08-12 Layla Parast , Tianxi Cai , Lu Tian

The development of statistical methods to evaluate surrogate markers is an active area of research. In many clinical settings, the surrogate marker is not simply a single measurement but is instead a longitudinal trajectory of measurements…

Methodology · Statistics 2024-02-28 Denis Agniel , Layla Parast

Estimating the long-term effects of treatments is of interest in many fields. A common challenge in estimating such treatment effects is that long-term outcomes are unobserved in the time frame needed to make policy decisions. One approach…

Methodology · Statistics 2024-08-23 Susan Athey , Raj Chetty , Guido Imbens , Hyunseung Kang

In many real-world causal inference applications, the primary outcomes (labels) are often partially missing, especially if they are expensive or difficult to collect. If the missingness depends on covariates (i.e., missingness is not…

Machine Learning · Statistics 2024-05-24 Zhenghao Zeng , David Arbour , Avi Feller , Raghavendra Addanki , Ryan Rossi , Ritwik Sinha , Edward H. Kennedy

Surrogate markers are often employed in clinical trials to replace primary outcomes that may be difficult, expensive, or time-consuming to measure directly. These markers can accelerate the evaluation of new treatments, provided they…

Methodology · Statistics 2026-03-17 Pietro Carlotti , Layla Parast

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect,…

Methodology · Statistics 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast

The identification of surrogate markers is motivated by their potential to make decisions sooner about a treatment effect. However, few methods have been developed to actually use a surrogate marker to test for a treatment effect in a…

Methodology · Statistics 2024-09-17 Layla Parast , Jay Bartroff

A surrogate marker is a biomarker or other physical measurement used to replace a primary outcome in clinical trials to evaluate a treatment effect when the primary outcome of interest is costly, invasive, or takes a long time to observe.…

Methodology · Statistics 2026-04-24 Emily Hsiao , Layla Parast

When evaluating the effectiveness of a treatment, policy, or intervention, the desired measure of effectiveness may be expensive to collect, not routinely available, or may take a long time to occur. In these cases, it is sometimes possible…

Methodology · Statistics 2022-11-10 Denis Agniel , Layla Parast , Boris Hejblum

Surrogate markers are often used in clinical trials to evaluate treatment effects when primary outcomes are costly, invasive, or take a long time to observe. However, reliance on surrogates can lead to the surrogate paradox, where a…

Methodology · Statistics 2025-06-17 Emily Hsiao , Lu Tian , Layla Parast

Surrogate markers are most commonly studied within the context of randomized clinical trials. However, the need for alternative outcomes extends beyond these settings and may be more pronounced in real-world public health and social science…

Methodology · Statistics 2025-04-23 Rebecca Knowlton , Layla Parast

Motivated by increasing pressure for decision makers to shorten the time required to evaluate the efficacy of a treatment such that treatments deemed safe and effective can be made publicly available, there has been substantial recent…

Methodology · Statistics 2022-09-20 Xuan Wang , Layla Parast , Lu Tian , Tianxi Cai

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data…

Statistics Theory · Mathematics 2026-05-15 Ziyang Liu , Niwen Zhou , Peng Wu , Xu Guo

There are many scenarios where short- and long-term causal effects of an intervention are different. For example, low-quality ads may increase short-term ad clicks but decrease the long-term revenue via reduced clicks. This work, therefore,…

Applications · Statistics 2020-12-23 Lu Cheng , Ruocheng Guo , Huan Liu

A common practice in clinical trials is to evaluate a treatment effect on an intermediate endpoint when the true outcome of interest would be difficult or costly to measure. We consider how to validate intermediate endpoints in a…

Methodology · Statistics 2022-11-30 Emily K. Roberts , Michael R. Elliott , Jeremy M. G. Taylor

Policy makers typically face the problem of wanting to estimate the long-term effects of novel treatments, while only having historical data of older treatment options. We assume access to a long-term dataset where only past treatments were…

Econometrics · Economics 2022-02-11 Keith Battocchi , Eleanor Dillon , Maggie Hei , Greg Lewis , Miruna Oprescu , Vasilis Syrgkanis

In many experimental and observational studies, the outcome of interest is often difficult or expensive to observe, reducing effective sample sizes for estimating average treatment effects (ATEs) even when identifiable. We study how…

Machine Learning · Statistics 2024-10-11 Nathan Kallus , Xiaojie Mao

A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential…

Methodology · Statistics 2022-02-04 Emily Roberts , Michael Elliott , Jeremy M. G. Taylor

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

Methodology · Statistics 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity…

Methodology · Statistics 2022-09-20 Layla Parast , Tianxi Cai , Lu Tian
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