ArticlePloS one2019
A standardised framework to identify optimal animal models for efficacy assessment in drug development.
Article in PloS one, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 30 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
30 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Neuroimaging findings in preclinical amyotrophic lateral sclerosis models-How well do they mimic the clinical phenotype? A systematic review.Frontiers in veterinary science · 2023Pooled it
- Current status of neuroprotective and neuroregenerative strategies in multiple sclerosis: A systematic review.Multiple sclerosis (Houndmills, Basingstoke, England) · 2022Pooled it
- Engineering a chitosan-based nanoemulsion for non-invasive CNS-targeting approach: dual neuroprotection against epileptic and depressive disorders.Naunyn-Schmiedeberg's archives of pharmacology · 2026Article
- Pharmacological Switching in Minor Phytocannabinoids: A Mechanistic Framework for Compound-Indication Matching and Precision Neurotherapeutic Development.Pharmaceuticals (Basel, Switzerland) · 2026Review
- Translational assessment instruments for preclinical to first-in-human decision-making: a scoping review.Journal of translational medicine · 2026Article
- A standardized imaging and analysis workflow for quantitative evaluation of cutaneous neurofibromas in Nf1-KO mice.PloS one · 2026Article
- A scoping review of ethical decisions and decision tools for experimental animal protocols.BMC medical ethics · 2025Article
- Idiopathic Intracranial Hypertension Animal Models and Venous Sinus Stenting: Status of Disease and Device-Focused Evidence.Brain sciences · 2025Review
- Biosimilars Targeting Pathogens: A Comprehensive Review of Their Role in Bacterial, Fungal, Parasitic, and Viral Infections.Pharmaceutics · 2025Review
- Discordance between preclinical and clinical testing of Na V 1.7-selective inhibitors for pain.Pain · 2025Review
- A Machine Learning Pipeline to Screen Large In Vivo Molecular Data to Curate Disease Signatures of High Translational Potential.Methods in molecular biology (Clifton, N.J.) · 2025Article
- Validity of mental and physical stress models.Neuroscience and biobehavioral reviews · 2024Review
- Animal models of Takotsubo syndrome: bridging the gap to the human condition.Frontiers in cardiovascular medicine · 2024Review
- On the value of diverse organisms in auditory research: From fish to flies to humans.Hearing research · 2023Review
- Predictive validity in drug discovery: what it is, why it matters and how to improve it.Nature reviews. Drug discovery · 2022Review
- Sex Differences in Intestinal P-Glycoprotein Expression in Wistar versus Sprague Dawley Rats.Pharmaceutics · 2022Article
- Comparative transcriptomic analysis reveals translationally relevant processes in mouse models of malaria.eLife · 2022Article
- Dogs as a Natural Animal Model of Epilepsy.Frontiers in veterinary science · 2022Review
- A Structured Approach to Optimizing Animal Model Selection for Human Translation: The Animal Model Quality Assessment.ILAR journal · 2021Article
- Do Lipid-based Nanoparticles Hold Promise for Advancing the Clinical Translation of Anticancer Alkaloids?Cancers · 2021Review
Corrections and comments
- Erratum issued
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionPoor translation of efficacy data derived from animal models can lead to clinical trials unlikely to benefit patients-or even put them at risk-and is a potential contributor to costly and unnecessary attrition in drug development.
objectivesTo develop a tool to assess, validate and compare the clinical translatability of animal models used for the preliminary assessment of efficacy. DESIGN AND
resultsWe performed a scoping review to identify the key aspects used to validate animal models. Eight domains (Epidemiology, Symptomatology and Natural History-SNH, Genetic, Biochemistry, Aetiology, Histology, Pharmacology and Endpoints) were identified. We drafted questions to evaluate the different facets of human disease simulation. We designed the Framework to Identify Models of Disease (FIMD) to include standardised instructions, a weighting and scoring system to compare models as well as factors to help interpret model similarity and evidence uncertainty. We also added a reporting quality and risk of bias assessment of drug intervention studies in the Pharmacological Validation domain. A web-based survey was conducted with experts from different stakeholders to gather input on the framework. We conducted a pilot study of the validation in two models for Type 2 Diabetes (T2D)-the ZDF rat and db/db mouse. Finally, we present a full validation and comparison of two animal models for Duchenne Muscular Dystrophy (DMD): the mdx mouse and GRMD dog. We show that there are significant differences between the mdx mouse and the GRMD dog, the latter mimicking the human epidemiological, SNH, and histological aspects to a greater extent than the mouse despite the overall lack of published data.
conclusionsFIMD facilitates drug development by serving as the basis to select the most relevant model that can provide meaningful data and is more likely to generate translatable results to progress drug candidates to the clinic.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.