How Early and Ongoing Engagement Boosted Vijoice’s FDA Journey: A Case Study in Building Regulatory Confidence in RWE
Facing the challenge of demonstrating treatment benefits for the ultra-rare and complex disease PIK3CA-related overgrowth spectrum (PROS), Novartis took an innovative approach with the evidence generation for Vijoice (alpelisib). With few patients worldwide and no approved therapies, clinical trials weren’t feasible—so they designed a retrospective, single-arm study using real-world data from patients in a Compassionate Use Program across multiple countries. From the start, Novartis engaged openly with the FDA, discussing whether this novel approach could support approval and aligning on study design, endpoints, and analysis plans.
They focused on objective measures like tumor reduction assessed through independent radiology review to minimize investigator bias, while adhering to strict data management and regulatory standards. By submitting patient-level and standardized datasets, Novartis enabled the FDA to replicate original findings and conduct additional analyses, evenutally culminating in accelerated approval. This case study illustrates how early engagement and open communication, along with thoughtful study design and rigorous data handling, can transform real-world evidence into a powerful regulatory tool .
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