Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

162
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
162
Comparing the Survival Analysis of Two or More Groups01:20

Comparing the Survival Analysis of Two or More Groups

261
Survival analysis is a cornerstone of medical research, used to evaluate the time until an event of interest occurs, such as death, disease recurrence, or recovery. Unlike standard statistical methods, survival analysis is particularly adept at handling censored data—instances where the event has not occurred for some participants by the end of the study or remains unobserved. To address these unique challenges, specialized techniques like the Kaplan-Meier estimator, log-rank test, and...
261
Bioequivalence: Overview01:16

Bioequivalence: Overview

1.2K
Pharmaceutical equivalents, by definition, are drug products with the same active ingredient in the same quantities, encapsulated in identical dosage forms, and intended for the same administration routes. These pharmaceutical equivalents are deemed bioequivalent if the bioavailability of the active entity in the drug preparations is similar. Moreover, pharmaceutical equivalents demonstrating bioequivalence are also regarded as therapeutically equivalent. This means that when used as directed,...
1.2K
Kaplan-Meier Approach01:24

Kaplan-Meier Approach

235
The Kaplan-Meier estimator is a non-parametric method used to estimate the survival function from time-to-event data. In medical research, it is frequently employed to measure the proportion of patients surviving for a certain period after treatment. This estimator is fundamental in analyzing time-to-event data, making it indispensable in clinical trials, epidemiological studies, and reliability engineering. By estimating survival probabilities, researchers can evaluate treatment effectiveness,...
235
Strategies for Assessing and Addressing Confounding01:25

Strategies for Assessing and Addressing Confounding

146
Confounding is a critical issue in epidemiological studies, often leading to misleading conclusions about associations between exposures and outcomes. It occurs when the relationship between the exposure and the outcome is mixed with the effects of other factors that influence the outcome. Given that, addressing confounding is of high importance for drawing accurate inferences in research.
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
146
Study Designs in Epidemiology01:20

Study Designs in Epidemiology

367
Epidemiological study designs are fundamental tools for investigating the distribution, determinants, and control of health conditions in populations. They help researchers understand the relationships between exposures and outcomes, and they broadly fall into two categories: "observational" and "experimental" studies.
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...
367

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Cancer risk with methotrexate-TNF antagonist combination therapy compared to TNF-antagonist monotherapy in IBD.

Inflammatory bowel diseases·2026
Same author

Toward Artificial Intelligence-driven Clinical Decision Support Tools in Rheumatology.

Rheumatic diseases clinics of North America·2026
Same author

Statistics and AI - A Fireside Conversation.

Harvard data science review·2026
Same author

Predicting the timing of first sustained cognitive worsening in Alzheimer's disease using real-world clinical data and machine learning.

medRxiv : the preprint server for health sciences·2026
Same author

Nonparametric estimation of the total treatment effect with multiple outcomes in the presence of terminal events.

Biometrics·2026
Same author

Stratification of Alzheimer's disease patients using knowledge-guided unsupervised latent factor clustering with electronic health record data.

Communications medicine·2026

Related Experiment Video

Updated: Aug 29, 2025

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index
06:55

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index

Published on: January 8, 2020

14.6K

Identifying surrogate markers in real-world comparative effectiveness research.

Larry Han1, Xuan Wang1, Tianxi Cai1

  • 1Department of Biostatistics, Harvard T.H. Chan School of Public Health, Boston, Massachusetts.

Statistics in Medicine
|September 5, 2022
PubMed
Summary

New statistical methods using real-world data (RWD) can now evaluate how well short-term surrogates predict long-term treatment effects, aiding healthcare policy decisions. This research introduces novel estimators for identifying and validating surrogate markers in complex RWD settings.

Keywords:
comparative effectiveness researchdouble robustnessinflammatory bowel diseaseproportion of treatment effect explainedsemi-nonparametric estimationsurrogate marker

More Related Videos

In Vitro Methods for Comparing Target Binding and CDC Induction Between Therapeutic Antibodies: Applications in Biosimilarity Analysis
07:25

In Vitro Methods for Comparing Target Binding and CDC Induction Between Therapeutic Antibodies: Applications in Biosimilarity Analysis

Published on: May 4, 2017

17.8K
Heterogeneity Mapping of Protein Expression in Tumors using Quantitative Immunofluorescence
07:54

Heterogeneity Mapping of Protein Expression in Tumors using Quantitative Immunofluorescence

Published on: October 25, 2011

18.7K

Related Experiment Videos

Last Updated: Aug 29, 2025

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index
06:55

Inverse Probability of Treatment Weighting Propensity Score using the Military Health System Data Repository and National Death Index

Published on: January 8, 2020

14.6K
In Vitro Methods for Comparing Target Binding and CDC Induction Between Therapeutic Antibodies: Applications in Biosimilarity Analysis
07:25

In Vitro Methods for Comparing Target Binding and CDC Induction Between Therapeutic Antibodies: Applications in Biosimilarity Analysis

Published on: May 4, 2017

17.8K
Heterogeneity Mapping of Protein Expression in Tumors using Quantitative Immunofluorescence
07:54

Heterogeneity Mapping of Protein Expression in Tumors using Quantitative Immunofluorescence

Published on: October 25, 2011

18.7K

Area of Science:

  • Biostatistics
  • Epidemiology
  • Health Services Research

Background:

  • Comparative Effectiveness Research (CER) increasingly uses real-world data (RWD).
  • Short-term surrogates are crucial for inferring long-term treatment effects in RWD.
  • Existing methods for surrogate evaluation are limited to randomized clinical trials (RCTs).

Purpose of the Study:

  • To develop and validate statistical methods for evaluating surrogate marker performance in RWD.
  • To estimate the Proportion of Treatment Effect (PTE) explained by surrogates in RWD.
  • To address the knowledge gap in surrogate marker validation using non-trial data.

Main Methods:

  • Proposed novel inverse probability weighted (IPW) and doubly robust (DR) estimators.
  • Developed estimators for optimal surrogate transformation and PTE.
  • Evaluated estimator consistency and asymptotic normality, including DR robustness.

Main Results:

  • The proposed IPW and DR estimators are consistent and asymptotically normal.
  • The DR estimator demonstrates robustness, requiring only one model (propensity score or outcome regression) to be correctly specified.
  • Simulations and RWD applications successfully identified and validated surrogate markers for inflammatory bowel disease (IBD).

Conclusions:

  • The developed statistical methods provide a robust framework for surrogate marker evaluation in RWD.
  • These methods enhance the utility of RWD for inferring treatment effects on long-term outcomes.
  • The approach has practical implications for evaluating treatments and informing health policy using real-world evidence.