Pilot Rationale
Rationales for Each of the Four STEP4NAMs Pilot Studies.
New Approach Methodologies (NAMs) have the potential to substantially reduce reliance on animal testing while improving the clinical relevance, efficiency, and ethical robustness of pre-clinical research in the biopharmaceutical and medical technology sectors. The STEP4NAMs project addresses these barriers through the coordinated development and validation of four complementary pilot studies, each focusing on a distinct class of NAMs: digital twins, organ-on-chip systems, advanced bench-top simulation models, and patient-derived organoids. Together, these pilots are designed to generate practical validation evidence, transferable methodologies, and best-practice guidance. In this deliverable, the scientific, technical, and strategic justification for each pilot study is demonstrated. For each pilot, this document outlines the purpose and scope, methodological approach, relevance to regulatory and industrial validation needs, and expected contribution to the overall objectives of STEP4NAMs. The rationales demonstrate how the pilots collectively support SMEs, researchers, regulators, and other stakeholders in building confidence in NAMs and accelerating their uptake across the North-West Europe (NWE) region.