China’s Center for Drug Evaluation has opened a voluntary NAMs pilot with separate routes for drug-specific regulatory evidence and method validation. Each application must define one context of use and provide evidence suited to the proposed decision.
China’s Center for Drug Evaluation (CDE) has issued the PIONEER pilot plan to support the development, validation and use of new approach methodologies (NAMs) in nonclinical drug evaluation. The plan covers human-relevant in vitro systems, computational approaches, ex vivo methods and other methods that do not rely on conventional animal testing. Its focus is how evidence from a specified method can inform a specified drug-development or regulatory question.
The pilot does not cover NAMs used only for early lead-compound screening. Participation is voluntary, and products included in the pilot still follow the existing drug registration process. Applicants who do not join may continue to submit NAMs data through the usual application route.
The first route concerns a particular drug. A registration applicant that plans to use, or has already used, a NAM in nonclinical evaluation may seek to submit its results as supporting evidence. The method must have a clear context of use, feasible operation, and initial evidence of robustness and reproducibility. The existing validation package should give preliminary support for the method’s reliability, accuracy and relevance to the proposed decision.
The second route is for developers seeking to validate a NAM for a defined use. Eligible applicants include drug developers, universities, research institutes and technical service organizations. The proposal must describe a practicable validation plan and the evidence already available. Joint applications are encouraged, but the applicant remains responsible for carrying out the agreed validation work.
CDE defines context of use as both the way a method is used and the purpose it serves in drug evaluation. Each pilot application covers one context of use. A developer can later submit another application if accumulating evidence supports a broader or different use. The plan does not prescribe one validation package for all NAMs; the evidence required depends on the proposed use and the weight the data would carry in a registration decision.
Applicants submit an application form and an implementation framework through a Type I regulatory communication request. CDE expects a quality system matched to the intended use, with data that are accurate, complete and traceable; it also asks applicants to refer to relevant Good Laboratory Practice principles. For accepted projects, the implementation framework becomes a working record that is updated as studies and regulatory discussions proceed.
The recruitment window lasts five years from the plan’s launch. Proposed projects are assessed by a CDE working group and the relevant therapeutic-area team. Those selected for inclusion are posted for five working days before their inclusion is confirmed. Drug-specific projects receive early and continuing dialogue; method-validation projects may involve joint or third-party validation where needed. Completion of an agreed validation program can support use of that method for the same context of use in subsequent drug-development projects.
A new FDA draft describes how in vitro potency and safety data, anchored to clinical evidence, could support variants of individualized genome-editing therapeutics within a defined product boundary.
Australia’s TGA already considers non-animal evidence in therapeutic-goods assessment. Its October 2026 statement ties acceptance of a NAM to its intended use, human biological relevance, technical performance and reliability, with validation evidence scaled to the consequences of a wrong conclusion.
