With both the EMA and FDA committed to using real-world evidence (RWE) to support medical-product regulatory decision making and increasing interest by both in patient-centered endpoints, there is need to understand if and how this work can be done across countries. Several current initiatives will be discussed that can provide insights to inform future work:

The DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative aims to coordinate the gathering of evidence on the use, safety, and effectiveness of medicines across the EU. It can provide insights on how to handle the capture of patient-reported data for symptom and health-related quality of life improvements across borders.

Beginning with the landmark Patient Reported Outcomes Measurement (PROMs) programme in the English NHS, other health care funders are also embedding generic (e.g., EQ-5D) and condition-specific patient reported outcomes in routine outcomes measurement (ROM). We review key learnings from implementation of large-scale PRO data collection in the real world, including the OECD’s Patient-Reported Indicators Surveys (PaRIS) initiative, and consider scalability across the EU.

Core outcomes sets (COS) have been posited to help overcome difficulties when comparing trial results due to differing endpoints across trials. More recently, patient-centered core impact sets (PC-CIS) were proposed to overcome lack of patient centricity across a wide range of activities such as outcome and endpoint selection, protocol development, PRO measure development and selection; regulatory decision making; etc. Might PC-CIS and subsequent COS be developed internationally to improve both patient centricity and standardization across studies?

Speakers will discuss advances and lessons learned for capturing patient-centric data and whether doing so across borders is realistic to aid in effectiveness evaluation of new medicines.