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Assessing the robustness of clinical trials regarding novel therapies in inflammatory bowel disease
Jieqi Zheng1, Pinwei Huang1,2, Li Li1
1Department of Gastroenterology, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, 510080, P.R. China.
Background:
Increasing randomized clinical trials evaluating novel therapies for inflammatory bowel disease necessitated the scrutiny of statistical robustness. This study aimed to quantify their fragility and identify factors associated with robustness.
Methods:
This cross-sectional analysis included randomized clinical trials studying biologics, small-molecule inhibitors, fecal microbiota transplantation (FMT), and stem cell therapy (SCT), and then the calculated fragility index (FI) and continuous fragility index (CFI) for binary and continuous outcomes, respectively. Factors affecting robustness were analysed through correlation analysis and multiple linear regression.
Results:
Among 129 trials from 53 studies, the median FI and CFI were 6 and 14.8, respectively. The FI varied significantly by the treatment type, trial phase, outcome type, and P values. The FI was positively correlated with the sample size (ρ = 0.734, P < 0.001), discontinuations (ρ = 0.479, P < 0.001), publication year (ρ = 0.253, P = 0.017), impact factor (ρ = 0.368, P < 0.001), and events percentage (ρ = 0.299, P = 0.005). The CFI was influenced by the outcome type and was strongly correlated with the sample size. After adjustment for other characteristics, biologics/small-molecule drugs displayed enhanced robustness relative to FMT/SCT (correlation coefficient B with the natural logarithm of FI: B = 0.283, P = 0.011). The primary or co-primary outcome exhibited greater robustness than did the other outcomes (FI: B = 0.288, P = 0.013; CFI: B = 0.459, P = 0.024). The sample size was positively correlated with both the FI (B = 0.001, P = 0.021) and the CFI (B = 0.001, P = 0.019), whereas discontinuation did not significantly affect the robustness.
Conclusion:
Randomized clinical trials of novel inflammatory bowel disease therapies exhibit varying robustness and are influenced by multiple study characteristics. Trials of biologics or small molecules, those with positive primary outcomes, and larger studies demonstrated greater robustness, supporting that robustness should be considered in future research when interpreting the efficacy.
Insights
Statistical robustness in inflammatory bowel disease (IBD) trials varies. Larger trials, biologics/small molecules, and positive primary outcomes show greater robustness, informing future research interpretations.
Area of Science:
- Gastroenterology and Hepatology
- Clinical Trial Methodology
- Biostatistics
Background:
- Randomized clinical trials (RCTs) for novel inflammatory bowel disease (IBD) therapies require statistical robustness assessment.
- Quantifying the fragility of IBD clinical trial data and identifying influencing factors is crucial for reliable interpretation.
Purpose of the Study:
- To evaluate the statistical robustness of RCTs for novel IBD therapies.
- To identify specific study characteristics associated with enhanced robustness.
Main Methods:
- Cross-sectional analysis of RCTs involving biologics, small-molecule inhibitors, fecal microbiota transplantation (FMT), and stem cell therapy (SCT).
- Calculation of fragility index (FI) for binary outcomes and continuous fragility index (CFI) for continuous outcomes.
- Correlation analysis and multiple linear regression to identify factors affecting robustness.
Main Results:
- Median FI was 6 and median CFI was 14.8 across 129 trials.
- Robustness (FI/CFI) was positively correlated with sample size, discontinuations, publication year, impact factor, and event percentage.
- Biologics/small molecules and trials with primary outcomes showed significantly greater robustness compared to FMT/SCT and other outcomes.
Conclusions:
- IBD RCTs demonstrate variable statistical robustness influenced by factors like treatment type, outcome definition, and study size.
- Trials utilizing biologics or small molecules, reporting positive primary outcomes, and involving larger sample sizes exhibit superior robustness.
- Consideration of statistical robustness is essential for accurate interpretation of efficacy in future IBD research.
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