In Vivo Models & Study Design

Key Considerations:

  • Ethical considerations and animal welfare
  • Selection of infection models (e.g., neutropenic thigh, pneumonia, UTI)
  • Use of humanised PK models
  • Translational relevance to human disease
  • Statistical powering of studies


Preclinical efficacy evaluation in clinically relevant animal infection models forms the scientific foundation for demonstrating proof of concept, dose selection and regulatory approval, requiring integration of pharmacokinetic/pharmacodynamic (PK/PD) principles throughout study design. This section addresses ethical animal use frameworks and the ARRIVE 2.0 reporting guidelines that ensure reproducibility and transparency; systematic selection of infection models (neutropenic thigh, pneumonia, UTI) matched to clinical indication and pathogen characteristics; and the strategic use of humanised dosing regimens that translate animal PK parameters to mimic human drug exposure, enabling predictive clinical relevance. Statistical powering, strain selection across geographic and resistance backgrounds, and translational considerations for establishing preliminary breakpoints are integrated throughout. Advanced in vitro approaches including hollow-fibre models, dose-fractionation study design, and mechanism-based PK/PD modelling to predict human efficacy and suppress resistance emergence are examined.


Resources

5 resources

Webinar / Video
GARDP

Animal models to study the activity of antibiotics – REVIVE

Provides an overview of commonly used animal models for studying antibiotic activity, including the rationale for selecting different models and their limitations in specific research contexts.


Webinar / Video
GARDP

Models for antimicrobial R&D: Development and use of in vivo models for infectious disease research – REVIVE

Part 1: Outlines key considerations in designing animal models for antimicrobial research, addressing model selection, clinical relevance, and the predictive value of in vivo results for human efficacy.


Webinar / Video
GARDP

Models for antimicrobial R&D: Advanced and complex in vivo models for infectious disease research – REVIVE

Part 2: Explores the design and execution of advanced animal models in antimicrobial research, focusing on pneumonia, UTI, tissue and biofilm infections, non-traditional drug evaluation, and key insights for generating robust PK/PD data.


Guidance
NC3Rs

ARRIVE Guidelines 2.0

Checklist to improve the reporting of animal research, enhancing reproducibility and transparency.


Online Tool
NC3Rs

NC3Rs Experimental Design Assistant

Help researchers to design robust and reproducible animal experiments.






PACE Delivery Partners

Delivery Partner Network members with expertise relevant to this theme