By: Alind Gupta
In the evolving landscape of clinical research, single-arm trials have become essential tools, particularly when traditional randomized controlled trials (RCTs) are not feasible. However, the challenge arises when attempting to evaluate these trials without a comparison group. This is where external control arms (ECA), based on real-world data (RWD), come into play. But, what if sharing patient-level real-world data isn't an option due to privacy or logistical concerns? Enter the world of synthetic data.
What Is the ECLIPSE Study?
The ECLIPSE study (External Control Arm with Synthetic Real-world Data for Comparative Oncology using Single Trial Arm Evidence), aims to explore whether synthetic real-world data can be a reliable replacement for traditional patient data in external control arm analysis. Specifically, the study focuses on comparing two cancer treatments: nivolumab combined with ipilimumab versus nivolumab alone in patients with metastatic non-small cell lung cancer (mNSCLC).
Study Design: Real-World Meets Synthetic Data
Traditional trials in oncology often involve randomized groups to assess the efficacy of different therapies. However, randomization isn’t always feasible. For example, in the Lung-MAP S1400I study (NCT02785952), only a single arm of patients receiving nivolumab plus ipilimumab is available. The ECLIPSE study aims to compare this treatment arm with an ECA, which includes patients who received nivolumab monotherapy based on RWD from Alberta, Canada, and the Nordic countries (Denmark and Norway).
However, instead of relying solely on original real-world data (which may not be accessible to all researchers), ECLIPSE explores the creation of synthetic real-world data—statistically generated datasets based on real-world data. This synthetic data is expected to preserve the integrity of the original data while addressing privacy and sharing restrictions.
Why Synthetic Data?
With increasing concerns over patient privacy and data security, especially in small sample sizes, researchers are turning to synthetic data. Using advanced generative models, synthetic datasets can mimic the structure and characteristics of real-world data without revealing sensitive information. The ECLIPSE study seeks to validate this synthetic data approach, asking: Can synthetic data provide the same level of insight as real-world data when used in external control arms?
The ECLIPSE Approach
The study will compare the efficacy of the two treatments (nivolumab + ipilimumab vs. nivolumab monotherapy) using both the original and synthetic real-world data. Baseline confounding adjustments will be applied to ensure the comparison is as accurate as possible. If successful, the findings will be a significant step forward in clinical research, particularly for oncology trials where data-sharing limitations often hinder progress.
Why This Matters for Oncology Research
The implications of this study are broad. If synthetic data can effectively replace original data in external control arm analyses, this could streamline the way future trials are conducted. Researchers will no longer be limited by data access restrictions or privacy concerns. They could more freely collaborate and share findings without compromising patient confidentiality.
For patients, this means faster access to new, potentially life-saving therapies. By enabling researchers to use synthetic data, treatments that show promise in single-arm trials can be compared against existing therapies more efficiently, speeding up the approval and adoption of new therapies.
Conclusion: A Future Driven by Synthetic Data
The ECLIPSE study is paving the way for a new era in clinical trials. By validating the use of synthetic data in external control arm analysis, this study could significantly impact oncology research practice. If successful, synthetic real-world data could become a staple in trials where traditional RCTs aren't possible, providing a powerful tool to compare treatment options and improve patient outcomes.
Stay tuned for more updates as the ECLIPSE study progresses and the potential of synthetic data in oncology research unfolds!
External Control Arm with Synthetic Real-world Data for Comparative Oncology using Single Trial Arm Evidence (ECLIPSE): A Case Study using Lung-MAP S1400I
Alind Gupta, Luke Segars, David Singletary, Johan Liseth Hansen, Kirk Geale, Anmol Arora, Manuel Gomes, Sreeram Ramagopalan, Winson Cheung, Paul Arora
medRxiv 2024.09.10.24313417;
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