EMA Qualification

In 2023 PRIME-CKD project leader Hiddo Lambers Heerspink and Lesley Inker (Tufts Medical Center, Boston), co-authored an important paper in Nature Medicine on the use of GFR slope as a surrogate endpoint for kidney failure. The authors developed and validated a kidney hierarchical composite end point (HCE) that combines clinical kidney outcomes with longitudinal GFR changes (GFR slope).

Recently, this proposed novel method was submitted to and qualified by the EMA to be used as a validated surrogate endpoint for CKD progression in randomized controlled clinical trials. This qualification can support market authorization and extension of indication approvals when the use of clinical endpoints is not feasible. This may serve as a case study for the research efforts conducted in Work Package 4 of PRIME-CKD as, amongst others, we aim to characterize the evidence demonstrating correlation between surrogate biomarkers and clinical outcomes. Elucidating the evidentiary basis on which eGFR slope has been approved as surrogate endpoint provides learnings for future surrogate biomarker qualifications.

*The European Medicines Agency (EMA) publishes opinions on the qualification of innovative development methods for novel methodologies that have been shown to be promising in the context of research and development into pharmaceuticals.