Case Study: A pharmaceutical company achieves expedited FDA label expansion for a cancer drug using real-world evidence with IQVIA

Expedited Label Expansion for a Cancer Drug Using Real-World Evidence (RWE)

A pharmaceutical company needed to expand the label of a cancer drug to a new, small patient population but could not run a traditional randomized clinical trial due to low patient numbers. The customer engaged IQVIA’s Regulatory Science and Strategy team to pursue an alternative evidence approach using Real-World Evidence (RWE) to support a regulatory submission.

IQVIA identified robust RWE endpoints for tumor response and safety, built a comprehensive regulatory strategy with protocol synopses and FDA briefing packages, and led FDA engagement and follow-up. As a result, IQVIA’s approach secured FDA approval for the label expansion in approximately one year and delivered substantial cost savings compared with a traditional RCT.