The evolution of the healthcare ecosystem over the past decade has resulted in the increasing availability of big data – i.e., real-world data (RWD) for patients. As a corollary, real-world evidence (RWE) derived from the analysis of RWD is becoming a critical component throughout the product development lifecycle, especially in support of health technology assessment (HTA) and reimbursement discussions. The potential to evaluate the relative effectiveness of therapies in actual use as well as augment clinical trial data where there are few patients or short time horizons makes RWE a compelling solution for evidence generation. However, there is still some uncertainty around the HTA agency requirements, types of RWD, and the analytic approaches to synthesizing such data. The symposium will explore the use of RWE to enable patient access, drawing contrasts across borders to highlight potential strategies for respective HTA agencies.

Specifically,

• Matthew Gordon will provide an overview of RWE and growth of application across the product development lifecycle in his opening comments
• Jackie Vanderpuye-Orgle will present considerations for analyzing RWD, current evidence gaps in HTA, and some analytic approaches for addressing these gaps.
• Emtiyaz Chowdhury will present NICE’s recent real-world evidence framework and provide a case study of how RWE has been used in a recent NICE HTA submission for an anti-CMV drug in post-transplant patients.
• Gianluca Biao will discuss using RWE to augment clinical trial data and potential use cases.
• Nick Latimer will present a case study on estimating cost-effectiveness using cancer registry data.