PURPOSE: To provide a high-level overview of real-world evidence guidance from regulators and HTA agencies, exploring indicators of their influence and opportunities for greater alignment.

DESCRIPTION: In recent years, regulators and HTA bodies have signalled their intent to make greater and better use of routinely collected data and real-world evidence (RWE) to support their decision making processes.

This movement has resulted in the publication of several pieces of guidance from regulators including multiple FDA guidance on assessing electronic health records and medical claims data, and registries for decision making and multiple EMA guidance on data quality and methodological standards in pharmacoepidemiology. Meanwhile, HTA agencies have also produced frameworks including NICE’s RWE framework, CADTH’s framework for reporting standards for RWE studies, and the REALISE framework which supports the use of real-world data and RWE to support drug reimbursement decision making in Asia.

However, to what extent can we currently see the influence of these publications – in terms of their application in evidence submissions, and the expansion of different RWE use cases to support decision making? Are they sufficiently aligned to support joint scientific advice both laterally (regulator to regulator and HTA to HTA) and vertically (from regulator to reimbursement)?

The panel will start with an overview of published and drafted frameworks to guide the use of real-world data and RWE for regulatory and reimbursement decisions, with similarities and differences highlighted. Following this, regulatory and HTA representatives will present exemplary case studies showing their guidance in action. Next the perspective of an industry representative applying a guidance in their evidence submission will be provided. The session will end with interactive polls and a discussion of the main challenges impacting the application of these guidance, key areas missing, and the potential for greater alignment between different HTA bodies, and with regulators.