The Value of Integrating Real-world and Trial Data to Increase Patient Benefit

Description: The session will address how real-world, published clinical data and sponsor-generated trial data can be integrated to accelerate drug development and increase patient benefit. The focus will be on two areas; generation and collection of relevant data to allow women of childbearing and breastfeeding age (WoCBA) and their health care professionals to make informed treatment decisions and use of external control arms (ECAs).

Progress in generating data in the use of medicines in WoCBA is often hindered by complex challenges. These challenges are discussed based on a real-life case study, shedding light on the remaining uncertainty by the evidence generated to support efficacy and safety for the WoCBA population and its impact on regulatory decision-making. The role of a composite of data from non-clinical to registries in delivering relevant data post-approval, the perceived limitations of this data in improving label information and the time-consuming nature of these processes are addressed. A special focus will be on the patient/physician perspective, particularly how existing wording is perceived, and the clarity of the message given.

ECAs can accelerate drug development but face regulatory challenges, particularly when using real-world data, due to concerns about data quality and population matching. Approaches to address these concerns will be discussed. Case studies will be presented. The concept of an ECA Steering Group with multidisciplinary expertise will be discussed.