A pharma company sought to expand its cancer drug label to a rare patient subgroup, but traditional RCTs were infeasible due to small populations. IQVIA’s Regulatory Science and Strategy team designed a real-world evidence (RWE) approach, identifying tumor response and safety endpoints to build a robust regulatory case. They developed detailed protocol synopses, briefing packages, and engaged FDA directly, addressing feedback quickly. Within about one year, FDA approved the label expansion, cutting both time and cost compared to RCTs. IQVIA’s RWE strategy provided a viable, accelerated path to approval.

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