Navigate the FDA regulatory landscape for New Approach Methodologies. Understand qualification pathways, ISTAND, and how to get your NAMs data accepted for submissions.
Organ-on-chip data has been accepted in FDA submissions. The first OoC platform (Emulate liver-chip) received ISTAND designation in 2024. For your submission:
Clearly articulate what question your OoC data will answer and what regulatory decision it will support.
Submit a Type B meeting request to discuss your NAMs strategy with the relevant review division.
Include validation data, performance characteristics, and reference to ISTAND-qualified platforms if applicable.
Include OoC data alongside (or instead of) traditional data, with clear rationale for interpretation.
Organoid data is increasingly accepted, especially for oncology (PDO drug response) and disease modeling. There is no formal qualification pathway yet, but strong precedent exists.
Provide genotypic/phenotypic validation, passage stability, and comparison to clinical outcomes if available.
Discuss with review division how organoid data will be evaluated in your specific context.
Reference published studies and prior FDA acceptances for similar organoid applications.
If seeking qualification for broader use, consider ISTAND consultation for organoid platform.
Computational models (PBPK, QSP) have the longest regulatory acceptance history among NAMs. The DDT program provides formal qualification pathways.
PBPK for PK questions, QSP for efficacy/safety, AI/ML for predictions - each has different acceptance levels.
FDA has issued guidance on PBPK (2018), model-informed drug development, and computational modeling credibility.
PBPK for DDI, pediatric extrapolation, and renal/hepatic impairment are routinely accepted.
Submit through Drug Development Tool program for formal qualification of novel computational approaches.
Traditional in vitro assays and newer NAMs can be included in submissions. The key is demonstrating relevance and validation for your specific context.
What specific question does your NAMs data answer? Safety, efficacy, mechanism, patient selection?
Demonstrate assay performance, reproducibility, and relevance to human biology or clinical outcomes.
For unprecedented applications, request FDA feedback through appropriate meeting type.
ISTAND is FDA's program to qualify NAMs platforms for a defined Context of Use. Once qualified, sponsors can reference the platform without needing to re-validate.
Request a meeting to discuss qualification strategy and get FDA feedback on your approach.
Include validation studies, performance characteristics, and proposed Context of Use.
FDA evaluates evidence and may request additional data or clarification.
If qualified, FDA publishes letter of support; platform can be referenced in submissions.
Under FDA Modernization Act 2.0, animal studies are no longer mandated. To replace a specific study, you'll need to demonstrate your NAMs provides equivalent or superior information.
Which specific animal study? What question does it answer? What endpoint does it measure?
Design a NAMs approach that answers the same regulatory question with comparable or better data.
Demonstrate concordance with historical animal data or clinical outcomes where available.
Present your replacement strategy to FDA and get agreement before proceeding.
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FDA Modernization Act 3.0 Educational Resource
© 2025 Patient Analog. All rights reserved.
Educational content created by J Radler for the biotech and scientific community. Last updated: February 4, 2026.
Free to share for educational purposes with attribution.