Among the first developments following the summer break, the European Medicines Agency (EMA) has launched a new pilot project on the voluntary submission of data (the Voluntary Data Submission, VDS, pilot) generated using New Approach Methodologies (NAMs). These include in vitro, in silico and in chemico methods, as well as refined ex vivo and in vivo approaches designed to progressively reduce reliance on traditional animal testing.
According to EMA, the call for NAM data submissions is expected to remain open until the third quarter of 2027, with the final outcomes of the pilot scheduled for publication by the end of next year.
A pilot to support data sharing
The “safe harbour” mechanism for voluntary data sharing was originally introduced in 2012 through guidance on the regulatory acceptability of testing approaches based on the 3Rs principles (replacement, reduction and refinement). However, industry uptake has so far been limited. Companies were given the opportunity to voluntarily submit data from NAM studies alongside data generated through conventional approaches as part of marketing authorisation applications, with the aim of helping regulatory authorities gain practical experience with these emerging methods.
The recently published EMA document highlights that experience with NAMs remains very limited among European regulators. Fundamental questions regarding the feasibility, incentives, data management requirements and regulatory added value of these novel preclinical assessment methodologies also remain largely unexplored.
The new VDS pilot significantly redesigns the approach to voluntary NAM data sharing by decoupling it from marketing authorisation procedures. Through this initiative, EMA aims to gain practical experience, identify scientific and operational challenges, and assess the feasibility of developing a more structured VDS framework in the future. Among the Agency’s key objectives are improving understanding and confidence in the evaluation of NAM data and providing a platform for discussion of NAM-related proposals, including their Contexts of Use (CoU) and Regulatory Applicability Areas (RAA).
EMA intends to provide participating organisations with non-binding scientific feedback on the regulatory relevance and fitness for purpose of the submitted methods, together with recommendations on possible next steps for further discussion through mechanisms such as the Innovation Task Force (ITF), Scientific Advice (SA) or Qualification Advice (QA) procedures.
The initiative is also expected to contribute to a harmonised interpretation of NAM data and Weight of Evidence (WoE) approaches across the EU regulatory network.
How to participate in the pilot
The pilot project is open to pharmaceutical companies, contract research organisations (CROs), method developers, non-governmental organisations, academic laboratories and consortia. Interested parties may submit an expression of interest to EMA using the dedicated application form available on the Agency’s website.
Four priority Regulatory Applicability Areas (RAAs) have been identified for participation in the pilot:
- Risk assessment for biological medicinal products lacking a pharmacologically relevant animal species;
- Developmental and reproductive toxicity (ICH S5(R3));
- Safety pharmacology (ICH S7A);
- Hepatotoxicity and drug-induced liver injury (DILI).
Following its internal assessment, EMA may redirect applicants to other, more appropriate procedures, such as ITF, SA or QA. Selected participants will be invited to submit a more comprehensive briefing package and will be provided with an EMA account and access to EudraLink.
Data will be shared through EudraLink and securely stored within EMA’s SharePoint system, accessible only to the Operational Expert Group (OEG) and relevant Agency staff. The briefing package should be aligned, in terms of format and level of detail, with Module 2.4 of the electronic Common Technical Document (eCTD). It should include a description of the RAA, the endpoints of interest, internal validation criteria and the intended regulatory application of the proposed methodology.
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