FDA panels: real‑world data and evidence can substitute where trials are infeasible — examples include alpelisib and Zolgensma FDA panelists reviewed standards for RWD/RWE (fit‑for‑purpose data, prespecified protocols, traceability) and cited cases where registries or expanded‑access data supported regulatory decisions — for example, alpelisib (PIK3CA‑related overgrowth) and Zolgensma (SMA) — while noting challenges around index dates, missing data and device identification.

FDA experts reveal how real-world data can reshape regulatory decisions, highlighting groundbreaking approvals like alpelisib and Zolgensma.

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