Real-world data (RWD) has been utilized in pharmacoepidemiologic studies for decades to enhance understanding of the benefits and risks of drugs following regulatory approval. Historically, these Real-world evidence (RWE) studies primarily focused on post-marketing safety surveillance. However, more recently, data from drug utilization and real-world effectiveness studies have been increasingly used to support label expansions for already approved drugs. Beyond label expansions, RWE is also gaining acceptance for inclusion in new drug applications, either as supportive evidence or as primary clinical evidence, particularly for drugs with orphan designation or in disease areas with significant unmet needs.

This webinar will highlight a case study featuring the use of RWE from a U.S.-approved product to secure the approval of a new drug in the European Union (EU). The marketing authorization application (MAA) incorporated data from one pivotal clinical trial, post-marketing data from the U.S. market since FDA approval four years earlier, and a real-world effectiveness and cardiovascular safety study. This RWE study was specifically conducted to address concerns raised by the Committee for Medicinal Products for Human Use (CHMP) during the initial MAA review four years prior. The successful use of RWE in this instance addressed a critical data gap, ultimately leading to the drug's approval in the EU.

The Webinar will:

• Discuss recent guidance documents regarding the use of RWE in regulatory applications in both the U.S. and EU.
• Review common biases, pitfalls, and concerns related to RWD quality from various stakeholder perspectives.
• Explore study design considerations when using RWE for regulatory submissions.
• Consider the importance of timely engagement of internal and external experts and stakeholders to enable successful outcomes.