Related Experiment Video
Updated: Dec 22, 2025
07:37
An Adoptive Transfer Model of Rheumatoid Arthritis in Mice
Published on: June 6, 2025
868
Improving rheumatoid arthritis comparative effectiveness research through causal inference principles: systematic
Sizheng Steven Zhao1,2, Houchen Lyu2,3, Daniel H Solomon2,4
1Musculoskeletal Biology, Institute of Lifecourse and Medical Sciences, University of Liverpool, Liverpool, UK s.zhao8@liverpool.ac.uk.
Annals of the Rheumatic Diseases
|May 9, 2020
Summary
Most observational comparative effectiveness research (CER) studies in rheumatoid arthritis (RA) have design limitations. Target trial emulation offers a structured framework to improve CER study design and reduce bias in rheumatoid arthritis research.
Area of Science:
- Comparative Effectiveness Research (CER)
- Rheumatoid Arthritis (RA) Treatment
Background:
- Observational studies are crucial for comparative effectiveness research (CER) in rheumatoid arthritis (RA).
- However, these studies often suffer from design limitations that can introduce bias.
- Target trial emulation provides a structured framework to address these limitations.
Purpose of the Study:
- To systematically review CER studies in RA.
- To identify design limitations in RA CER studies.
- To demonstrate how target trial emulation can mitigate these limitations and reduce bias.
Main Methods:
- Systematic review of head-to-head CER studies of biologic disease-modifying anti-rheumatic drugs (DMARDs) in RA.
- Evaluation of study designs based on seven components of the target trial emulation framework.
- Assessment of hypothetical trials to identify design limitations and potential biases.
Main Results:
- 31 CER studies were reviewed, with 29 (94%) exhibiting at least one design limitation.
- Common limitations included: using post-baseline information for eligibility criteria (61%), unclear causal contrasts (81%), and inadequate handling of confounding by indication (42%).
Conclusions:
- Design limitations are prevalent in observational CER studies for RA.
- Target trial emulation is a valuable structured approach for designing observational CER studies.
- Implementing target trial emulation can help avoid potential sources of bias in RA research.
Keywords:
causal inferencecomparative effectivenessobservational studyrheumatoid arthritistarget trial emulationMore Related Videos
Related Concept Videos
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
326
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,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
326
01:11Genome-wide Association Studies-GWAS
15.1K
Genome-wide association studies or GWAS are used to identify whether common SNPs are associated with certain diseases. Suppose specific SNPs are more frequently observed in individuals with a particular disease than those without the disease. In that case, those SNPs are said to be associated with the disease. Chi-square analysis is performed to check the probability of the allele likely to be associated with the disease.
GWAS does not require the identification of the target gene involved in...
GWAS does not require the identification of the target gene involved in...
15.1K
01:16Clinical Trials
10.1K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
10.1K
01:20Study Designs in Epidemiology
770
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...
Observational studies are those where the researcher does not intervene but rather observes natural variations. They include cross-sectional, cohort, and...
770
01:12Hazard Ratio
472
The hazard ratio (HR) is a widely used measure in clinical trials to compare the risk of events, such as death or disease recurrence, between two groups over time. It reflects the ratio of hazard rates—the instantaneous risk of the event occurring—between a treatment group and a control group. This measure provides valuable insights into the relative effectiveness of a treatment by assessing how the risk of an event differs between the two groups.
For example, in a clinical trial...
For example, in a clinical trial...
472
01:16Crossover Experiments
4.4K
Crossover experiments, also called the repeated-measurements design, is a study design in which all experimental units are exposed to all treatments in different periods. Crossover experiments are generally used in psychology, the pharmaceutical industry, agriculture, and medicine.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
4.4K