OBJECTIVES:

Regulatory bodies, including the FDA and EMA, endorse real-world evidence (RWE) as a part of patient-focused drug development, encouraging manufacturers to conduct RWE research. Despite the potential for RWE to strengthen randomized clinical trial (RCT) data, there is notable divergence in how HTA and payer organizations incorporate RWE into their decision-making. This research aimed to understand payer perception and evolution of patient-centric approaches in clinical trials and RWE in health technology assessment (HTA) submissions and payer decision-making.

METHODS:

Quantitative research was conducted using a web-enabled, 15-minute questionnaire to identify payer preference and collect insights on the evolving use and acceptance of RWE in drug assessments in the US, UK, Germany, France, and Spain from a total of 20 Payer stakeholders. Survey questions evaluated payer inclination towards patient-centric research, including the type of study and specific outcomes of interest as part of understanding the impact of RWE on HTA and payer submissions.

RESULTS:

Several institutions have published their perspectives on the use of RWE in drug assessments. In Germany, the G-BA compiled a report on the potential use of RWE, including the use of patient registry studies, to evaluate the additional benefit of novel drugs with no direct comparison available. Similarly, NICE in the UK developed an RWE framework to increase the use of RWE to bridge data gaps in knowledge and improve patient access to novel technologies. Despite the efforts to increase the use of RWE in drug assessments, the acceptance of such evidence and its impact on access outcomes remains heterogeneous and in flux across markets.

CONCLUSIONS:

This research found a gradual increase in the recognition of the value of real-world studies among payers over time, suggesting a trend towards greater acceptance of RWE in future drug assessments.