The European Medicines Agency (EMA) has launched a public consultation on a concept paper that aims to develop a reflection paper on non-clinical requirements for medicinal products targeting severely debilitating or life-threatening diseases. The initiative seeks to establish a harmonised regulatory framework allowing tailored non-clinical testing programmes, potentially accelerating patient access to therapies for conditions with high unmet medical need, while maintaining safety standards.
The concept paper, published on 30 July 2026 and adopted by the Committee for Medicinal Products for Human Use (CHMP) on 13 July 2026, addresses the current lack of EMA guidance outside oncology. In oncology, the ICH S9 guideline already facilitates faster clinical development, but for other serious diseases, deviations from core non-clinical guidelines (ICH M3(R2) and ICH S6(R1)) are handled case-by-case, leading to inconsistency. The reflection paper would outline general principles for when flexibility is appropriate, drawing on lessons from the COVID-19 pandemic and existing scientific advice.
The document emphasises the 3Rs (replacement, reduction, refinement of animal use) and novel approach methods (NAMs) to complement weight-of-evidence assessments. It will provide examples of streamlined non-clinical packages, circumstances where standard safety testing remains warranted, timing considerations for studies, and modality-specific guidance for small molecules versus biologics. The reflection paper will exclude products already covered by ICH S9 (advanced cancer treatments).
Stakeholders are invited to submit comments via the EUSurvey form by 30 September 2026. The initiative is expected to benefit patients with life-threatening conditions by reducing development timelines, while pharmaceutical companies may gain regulatory clarity, potentially lowering costs and uncertainty. However, patient safety advocates may raise concerns about the adequacy of abbreviated testing, and animal welfare groups could welcome the emphasis on 3Rs. The reflection paper, once finalised, will guide future marketing authorisation applications and scientific advice procedures, impacting regulatory decision-making across the EU.