EMA Opens a Voluntary NAMs Data Pilot Across Four Safety Assessment Areas

A Route for Sharing Data Outside a Marketing Application

The European Medicines Agency (EMA) announced its voluntary data submission (VDS) pilot on September 1, 2026. Companies, method developers, academic laboratories and other organizations can submit and discuss nonclinical NAMs data independently of a marketing authorization application.

EMA describes a gap between the growing use of NAMs in research and early development and the limited amount of data shared with regulators. Through the pilot, developers can present existing work to nonclinical experts across the European medicines regulatory network, while those experts gain experience assessing NAMs data.

Participants receive individual, nonbinding and non-decisional feedback. The pilot is intended to improve scientific dialogue, encourage more consistent assessment across Europe and test a framework for continuing data exchange.

The information document revisits the earlier safe-harbour approach, which permitted novel-method data to accompany established evidence in a marketing application. Uptake was very limited. NAMs data have often reached regulators only in isolated interactions, with too little context for systematic learning. The new pilot allows these discussions to take place separately from a marketing authorization application.

Four Areas Define the Scope

The first area is risk assessment for biologics with no pharmacologically relevant animal species, or with animal responses that only partly reflect human biology. A species may lack target binding, functional activity or the relevant expression pattern; alternatively, it may show target engagement without reproducing downstream human effects.

For these biologics, a submission should explain the mechanism of action, target expression and function, downstream signaling, relevant clinical experience and the safety question being addressed. NAMs may help fill gaps or interpret limitations in animal evidence. With sufficient justification, the resulting evidence could inform first-in-human dosing, clinical trial initiation or decisions about particular non-human primate studies.

The second area is developmental and reproductive toxicity. Both evidence of a hazard and evidence supporting an absence of risk can contribute to a weight-of-evidence assessment. Their implications differ: a well-characterized risk may support waiving subsequent studies when suitable mitigation is in place, while an argument for absence of risk requires a sufficiently complete account of target biology, intrinsic pharmacological risk, the patient population and remaining uncertainties.

NAMs data may also accompany preliminary embryo–fetal development studies to clarify their context of use and address evidence gaps. The information document discusses potential relevance across the medicine lifecycle, including pregnancy, labeling and marketing authorization.

The third area is safety pharmacology. NAMs may contribute to screening and prioritization, targeted investigation of a defined concern, or mechanistic interpretation of an observed finding. Cardiovascular, central nervous system and respiratory risks are among the proposed applications, with data considered in an integrated assessment.

The fourth area is hepatotoxicity and drug-induced liver injury, covering both small molecules and biological products. Mechanistic, functional and exposure–response data may address mitochondrial dysfunction, inhibition of bile acid transport or immune-mediated injury. Potential applications include starting and maximum doses, dose adjustment, clinical monitoring and risk information in product labeling.

Across all four areas, applicants need a clearly defined context of use. The pilot considers standalone methods, batteries of methods and weight-of-evidence approaches that combine NAMs with animal findings, clinical data or established drug-class knowledge. Applicants should explain how the model and its performance relate to the decision they intend to inform.

From an Expression of Interest to a Scientific Package

Organizations first submit an application expressing interest. EMA checks suitability and prioritizes applications before inviting selected participants to provide a full briefing package. Some proposals may be directed to an existing procedure if that offers a more appropriate route.

The briefing package should describe the problem, relevant regulatory area, method, endpoint, biological mechanism and current or anticipated context of use. It should include internal validation or characterization, reference compounds, robustness, biological relevance and measures taken to ensure reliable data. Comparisons with animal evidence or external validation can be included where applicable.

EMA suggests a level of detail comparable to the nonclinical overview in Module 2.4 of the electronic common technical document. It requests underlying reports, datasets or model outputs where they are essential to interpreting the findings.

Where full product disclosure is not possible, the package should still provide relevant information about modality, pharmacological class, target, mechanism, indication and target novelty. Full packages are submitted through EudraLink. Commercially confidential information is to be protected during assessment and when pilot outcomes are published.

Feedback and the Pilot Timetable

An operational expert group drawn from the European regulatory network will provide individual feedback on the readiness of the method, adequacy of the data and possible next steps. Follow-up may involve the Innovation Task Force, Scientific Advice or Qualification Advice.

The VDS pilot does not replace existing regulatory procedures and does not itself make an approval decision. EMA expects submissions through the third quarter of 2027 and plans a lessons-learned report after the pilot, covering scientific, regulatory and operational experience.