Case Study on the Use of Real-World Evidence in a Feasibility Assessment of a Randomized Controlled Trial Design to Support the Fulfillment of Pediatric Requirements with the FDA and EMA.
Ther Innov Regul SciJun 19, 2026 (epub)
Neal E Storm, Christine Kubik, Ghislaine Priem et al.
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In 30 seconds
This study assessed the feasibility of a randomized controlled trial (RCT) for denosumab in pediatric patients with glucocorticoid-induced osteoporosis (GiOP). Using real-world data from three databases, the research found that only about 1 eligible subject per 1,000,000 people per year could be recruited for the trial, indicating significant challenges in enrollment.
Key findings
- Approximately 1 eligible pediatric subject per 1,000,000 people per year was estimated for the trial.
- Recruitment challenges persisted despite multiple mitigation measures implemented by the study sponsor.
- Real-world epidemiological analyses provided insights into the clinical feasibility of the RCT design.
- The study questioned the feasibility of alternative clinical trial designs for pediatric GiOP.
Why it matters
Understanding the feasibility of clinical trials in rare pediatric conditions is crucial for regulatory compliance and ensuring that new treatments are developed responsibly. This study highlights the importance of real-world evidence in assessing trial viability.