Project Summary

This research project is in progress. PCORI will post the research findings on this page within 90 days after the results are final.

One of PCORI’s goals is to improve the methods that researchers use for patient-centered outcomes research. PCORI funds methods projects like this one to better understand and advance the use of research methods that improve the strength and quality of comparative effectiveness research.

What is the project about?

Patients often have multiple treatment options for a single health problem. When choosing a treatment, patients and their doctors consider how well each treatment is likely to work based on the patient’s traits, such as age or the number and type of health problems they have. Researchers use statistical methods to predict how well a treatment may work for patients based on their traits. But current methods are limited when multiple treatment options exist.

In this study, the research team is developing methods to identify patients who will benefit most from a treatment when multiple treatment options exist.

How can this project help improve research methods?

Results may help researchers predict which patients benefit most from a treatment when they have multiple treatment options and outcomes to consider.

What is the research team doing?

First, the research team is developing a way to combine findings from published studies on treatment outcomes with individual patient data from electronic health records. The team is looking at ways to analyze the data to reduce biases in individual studies. Next, the team is developing new methods for analyzing data that capture the relationships between patient traits and the way patients respond to treatment. The team is testing the new methods using data from trials of treatments for Crohn’s disease in children within PEDSnet. PEDSnet is a national pediatric learning health system made up of 8 children’s hospital systems in 12 states.

After testing the methods, the research team is creating software that researchers can use to identify patients who benefit most from a treatment when multiple treatment options exist.

Patients and healthcare providers are helping to choose research questions for the study.

Research methods at a glance

Design Elements Description
  • Develop a framework for combining aggregated results from the published literature with individual patient data derived from a research network.
  • Propose and evaluate a powerful two-stage hypothesis testing procedure to identify treatment-covariate interactions.
  • Evaluate the proposed methods in pragmatic trials of treatments for pediatric Crohn’s disease within the PEDSnet consortium.
Approach Multivariate multiple treatment meta-regression analysis

Project Information

Yong Chen, PhD, MA
Jing Huang, PhD
University of Pennsylvania
A Two-Stage Meta-Regression Framework for Precision Medicine Using Data from Clinical Data Research

Key Dates

August 2019
January 2024

Study Registration Information


Health Conditions Health Conditions These are the broad terms we use to categorize our funded research studies; specific diseases or conditions are included within the appropriate larger category. Note: not all of our funded projects focus on a single disease or condition; some touch on multiple diseases or conditions, research methods, or broader health system interventions. Such projects won’t be listed by a primary disease/condition and so won’t appear if you use this filter tool to find them. View Glossary
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: November 10, 2022