Publications
Papers that shaped the field, plus notable new work.
- Review
New approach methodologies in neurotherapeutics development
Tamiz AP, et al. (NIH NINDS, Instem, JHU, FDA). Neurotherapeutics. Sep 17, 2026
Government, academic and industry view of NAMs for CNS drugs, including blood-brain barrier and neurotoxicity models.
- Perspective
Exponential toxicology
Hartung T (Johns Hopkins CAAT). ALTEX. Q3 2026
Argues chemical exposure grows faster than animal testing can scale, so testing must move to high-throughput, computational and AI methods.
- Organoids & 3D modelsReview
Human organoids: Fit for drug discovery?
Wittich A, et al. (Fraunhofer ITMP). Stem Cell Reports. May 7, 2026
PSC-derived organoid screening: readouts, ML methods, and open problems in assay robustness, throughput and standardization.
- LandmarkReview
Reimagining human-centric drug development with new approach methodologies
Wu X, et al. (Stanford, NIH). Science. Apr 16, 2026
Synthesizes biological and computational NAMs and the regulatory reforms positioning them as a complement to or replacement for animal testing.
- LandmarkReview
New approach methodologies for drug discovery
Liu W, et al. (Stanford, NCATS). Cell. Apr 2, 2026
Review of stem cell, organoid and in silico NAMs across discovery, plus the biological, technical and regulatory bottlenecks to adoption.
- Organ-on-chip & MPSReview
Microphysiological systems to accelerate and de-risk AAV gene therapy development
Tornabene P, et al. Advanced Drug Delivery Reviews. Mar 5, 2026
Proposes organoids and organ-chips as a human-relevant filter for AAV tropism, liver toxicity and immune response.
- Method
New approach methodologies (NAMs) for improved developmental and reproductive toxicity (DART) assessment with focus on endocrine disruption: a PARC project
Ardenkjaer-Skinnerup J, et al. (PARC). Frontiers in Toxicology. Feb 3, 2026
EU PARC work on human-relevant NAMs for developmental and reproductive toxicity and endocrine disruption.
- Organ-on-chip & MPSReview
Challenges and opportunities for human Organ Chips in FDA assessments and pharma pipelines
Ingber DE. Cell Stem Cell. Jan 20, 2026
Organ Chips are widely explored in pharma labs but not yet built into development pipelines; reviews what must change to get there.
- LandmarkRegulatory
A CDER perspective: Landscape of New Approach Methodologies (NAMs) submitted in drug development programs
Dao T, Sadrieh N (FDA CDER). Regulatory Toxicology and Pharmacology. Dec 6, 2025
15 years of submission data: 93% of NAM submissions were in silico (49%) or in vitro (44%). 3D and organ-chip data were less common than standard in vitro models.
- Review
Integrating New Approach Methodologies (NAMs) into Preclinical Regulatory Evaluation of Oncology Drugs
Mirlohi MS, et al. Biomimetics. Nov 24, 2025
Reviews organoid, organ-chip and AI-based NAMs for oncology and their regulatory context.
- Regulatory
FDA/CDER/OND Experience With New Approach Methodologies (NAMs)
Yao J, et al. (FDA CDER). International Journal of Toxicology. Nov 13, 2025
CDER reviewers describe how NAMs became accepted alternatives and what they look for in NAM submissions.
- Organ-on-chip & MPSMethod
Challenges and solutions in measuring commonly used biomarkers for drug-induced liver injury in a liver-on-a-chip platform
Shi Q, et al. (FDA NCTR, CDER, Emulate). Toxicology Letters. Oct 1, 2025
FDA scientists found standard ALT activity assays can fail in liver-chip perfusate and urea and albumin assays need care. Assay choice can explain conflicting DILI results.
- LandmarkReview
Application of new approach methodologies for nonclinical safety assessment of drug candidates
Beilmann M, et al. (EFPIA/PDEG). Nature Reviews Drug Discovery. May 2, 2025
Cross-pharma review of cases where animal models fell short and four drug candidate categories where human in vitro approaches apply today.
- LandmarkOrgan-on-chip & MPSIndustry adoption
The Current Status and Use of Microphysiological Systems by the Pharmaceutical Industry: The IQ MPS Affiliate Survey and Commentary
Baker TK, et al. (IQ MPS Affiliate). Drug Metabolism and Disposition. Feb 14, 2024
Surveys of 20+ pharma companies (2019 and 2021) on MPS resourcing, organs of interest, contexts of use and barriers to using MPS data in submissions.
- LandmarkIndustry adoption
Industry Adoption of Organoids and Organs-on-Chip Technology: Toward a Paradox of Choice
Homan KA (Genentech). Advanced Biology. Mar 2, 2023
Genentech view that the supply of human model systems has outrun pharma's ability to choose among them; argues for shared multi-omic data across models.
- LandmarkOrgan-on-chip & MPSValidation
Performance assessment and economic analysis of a human Liver-Chip for predictive toxicology
Ewart L, et al. (Emulate). Communications Medicine. Dec 6, 2022
870 Liver-Chips tested on a blinded set of 27 drugs from IQ consortium benchmarks: 87% sensitivity, 100% specificity. Company-authored; includes an economic model of R&D productivity.
Bibliographic metadata from PubMed.