Objective:

To evaluate the efficiency of home optical coherence tomography (OCT) combined with pharmacokinetic/pharmacodynamic (PK/PD) modeling in reducing sample size requirements for early ophthalmology clinical trials, highlighting the potential for significant improvements in trial efficiency.

Key Findings:

• Home OCT with PK/PD modeling requires only 33 to 35 patients per arm, compared to 41 to 54 patients for traditional monitoring, indicating a significant reduction in trial burden.
• This represents a 20% to 40% reduction in sample size while maintaining statistical power, which could lead to faster trial completion.
• The model was validated using profile likelihood analysis and prediction-corrected visual predictive checks, ensuring reliability of the findings.

Interpretation:

Integrating home OCT data with advanced modeling can streamline clinical trials in ophthalmology, improving endpoint precision and reducing recruitment needs, though challenges in implementation should be considered.

Limitations:

• The model was based on a small observational dataset, which may limit the robustness of the findings.
• Generalizability may be limited due to the uniform demographic of the sample, necessitating further research across diverse populations.

Conclusion:

Prospective validation of this approach in interventional trials is recommended to assess its practical impact on trial endpoints and modeling performance, with specific attention to real-world applicability.