CRED Understanding Clinical Development 2024

14/10/2024

Real-World-Data – Regulatory Decision Making

Post-marketing studies

- COVID-19 Vaccine

Label expansions

IBRANCE® (Palbociclib)- breast cancer in men (US)

evidence from Electronic Health Records/ Health Insurance Claims Data

New filings - Zolgensma ® - external control arm for Phase 3 open label study in spinal muscular atrophy

Translarna (ataluren) – RWE in EMA re-examination procedure

The Organisation for Professionals in Regulatory Affairs

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Is RWD replacing Phase III ?

• RWD complements but does not substitute Phase III

RCTs remain gold standard

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• Many MAAs will contain some RWD – potential for extended indications

Challenges with RWD

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• Unobserved confounders, variability of clinical setting, less monitoring of adverse events, risk of incomplete data

• Helpful to better understand rare diseases

• Useful in post-approval surveillance and safety monitoring

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7093234/

The Organisation for Professionals in Regulatory Affairs

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