Developing an integrated evidence generation strategy for a rare disease requires careful planning, cross-disciplinary collaboration, and adherence to regulatory guidelines. The strategy should be holistic, incorporating real-world evidence (RWE), clinical trials, and patient-centric data to address the unique challenges associated with rare diseases and to support regulatory approvals, market access, and ongoing patient care. … Continue reading Integrated Evidence Generation Strategy for Rare Disease: A Comprehensive Approach Across Clinical Phases