Traditionally, Real-World Evidence (RWE) has had its place in satisfying post-launch regulatory requirements principally related to drug safety. RWE has since been expanding well beyond its beginnings in pharmacovigilance and has found widespread acceptance for a range of use cases with different healthcare stakeholders, including regulators, Health Technology Assessment (HTA) bodies, payers and Healthcare Professionals (HCPs). Initial scepticism and uncertainty about the robustness of real-world data, and the analytical methodologies used to derive RWE, have given way to the appreciation by those stakeholders of the value RWE brings in addressing their needs. This expansion has gone hand in hand with the digitisation of healthcare combined with innovation in technology and analytics.
In this white paper, we will review the landscape of use cases for RWE among different healthcare stakeholders with a focus on Europe, while highlighting variations and limitations in RWE acceptance. We will further look at trends that are shaping this dynamic picture and how they might play out over the medium term. Finally, we will draw out implications for pharmaceutical companies of how to embrace this new world.
Although not a focus for this white paper pharmaceutical companies’ internal use of RWE is increasing. They are using it to inform strategic and operational decisions, for example example target product profile (TPP) development, clinical trial design, patient recruitment, financial forecasting or commercial resource allocation.
The march of RWE has been relentless. Today, we find a wide range of use cases for RWE along the entire product lifecycle, spanning both the pre- and postlaunch phase. Applications of RWE have also moved downstream from their original regulatory focus, to address the specific needs of different healthcare stakeholders, including payers and HCPs.
The sheer scale and pace of medical innovation is creating increasing complexity and uncertainty for healthcare stakeholders and is a major driver behind the expanding use of RWE. For example, regulators and payers struggle with understanding clinical value supported by limited evidence packages associated with an accelerated or early approval, where outcomes may have been extrapolated, e.g., overall survival (OS) from progression-free survival (PFS) for oncology products, or where comparators are missing, e.g., in single arm trials. At the same time, HCPs are overwhelmed by the proliferation of treatment options and the fragmentation of patient populations into smaller and smaller sub-segments, which making it increasingly challenging for HCPs to accurately identify and characterise patients and then match them with the optimal treatment option. Against this backdrop, RWE has an invaluable role to play in eliminating uncertainty and guiding healthcare stakeholders’ decisions across the board.