Dec 07, 2025
Organoid Models for Preclinical Drug Screening: A Systematic Review Protocol
- asmae.toulout 1,
- MALAK MENTAGUI1,
- SOUKAINA HAJJI2
- 1UNIVERSITY EUROMED OF FES;
- 2UNIVERSITY EUROMED OF FES
Protocol Citation: asmae.toulout , MALAK MENTAGUI, SOUKAINA HAJJI 2025. Organoid Models for Preclinical Drug Screening: A Systematic Review Protocol . protocols.io https://dx.doi.org/10.17504/protocols.io.rm7vz9zq5gx1/v1
License: This is an open access protocol distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited
Protocol status: Working
We use this protocol and it's working
Created: November 27, 2025
Last Modified: December 07, 2025
Protocol Integer ID: 233650
Keywords: organoid models for preclinical drug screening, preclinical drug screening, organoid model, organoid type, organoid, assays with conventional preclinical model, toxicity testing, based assay, conventional preclinical model, animal model, applications in efficacy
Abstract
To describe organoid types, experimental methods, and applications in efficacy and toxicity testing. To compare organoid-based assays with conventional preclinical models (2D cultures, animal models). To identify methodological challenges, reproducibility issues, and potential improvements for translational use.
Guidelines
- All records will be imported into Rayyan.ai for blinded screening.
- Step 1: Title and abstract screening by two independent reviewers.
- Step 2: Full-text review to confirm eligibility.
- Discrepancies will be resolved by discussion or a third reviewer.
- Selection will be summarized using a PRISMA 2020 flow diagram.
Quality Assessment / Risk of Bias
- In vitro studies: Checklist adapted from ToxRTool or OECD standards.
- In vivo studies: SYRCLE’s Risk of Bias tool.
Each study will be rated as low, unclear, or high risk of bias.
Data Synthesis
- Narrative synthesis: Results will be summarized descriptively and organized by organoid type, drug category, and outcome.
- Quantitative synthesis: A meta-analysis will not be conducted due to expected heterogeneity in study designs and outcomes.
- Tables and figures will present study characteristics, outcomes, and gaps.
Materials
A structured Excel sheet will be used to extract the following data:
- Author, year, country
- Study design (in vitro / in vivo)
- Type and source of organoid (tissue, species)
- Purpose of study (efficacy, toxicity, etc.)
- Methods of drug screening
- Comparators used
- Main results and limitations
Troubleshooting
Objectives
Primary Objective: To systematically review and synthesize current evidence on the use of organoid models for preclinical drug screening between 2015 and 2025.
Secondary Objectives:
To describe organoid types, experimental methods, and applications in efficacy and toxicity testing.
To compare organoid-based assays with conventional preclinical models (2D cultures, animal models).
To identify methodological challenges, reproducibility issues, and potential improvements for translational use.
Research Question (PICO)
Research Question: What are the applications and challenges of organoid models in preclinical drug screening for evaluating drug efficacy and toxicity in vitro and in vivo?
Eligibility Criteria
Inclusion Criteria
Population: Studies using human- or animal-derived organoid models applied in preclinical drug screening or testing.
Intervention: Any drug exposure, compound testing, or therapeutic evaluation (efficacy, toxicity, pharmacokinetics, pharmacodynamics).
Comparator: Studies that include 2D cultures, animal models, patient-derived xenografts (PDX), or no comparator if the design is justified.
Outcomes: Must report quantitative or qualitative outcomes related to drug efficacy, toxicity, pharmacodynamics, or predictive performance.
Study Design: Experimental or preclinical studies conducted in vitro or in vivo.
Exclusion Criteria
Population: Studies not involving organoid models (e.g., 2D cultures, spheroids, organ-on-chip, or animal-only experiments).
Intervention: Studies not related to drug testing, e.g., focusing only on disease modeling, stem cell differentiation, or genetics.
Study Design: Reviews, commentaries, case reports, conference abstracts, or theoretical studies without experimental data.
Document Type: Full text unavailable or data insufficient for extraction.
Information Sources
Electronic databases:
- PubMed / MEDLINE
- Scopus
- Embase
- Web of Science
- Cochrane Library
Grey literature sources:
- bioRxiv, medRxiv, ProQuest Theses, Google Scholar, and conference proceedings.
Reference lists of all included studies will be screened manually to identify additional relevant publications.
Search Strategy
("organoid"[Title/Abstract] OR "organoids"[Title/Abstract] OR "3D organoid"[Title/Abstract] OR "mini-organ"[Title/Abstract]) AND ("drug screening"[Title/Abstract] OR "drug discovery"[Title/Abstract] OR "preclinical screening"[Title/Abstract] OR "preclinical testing"[Title/Abstract]) AND ("2015/01/01"[Date - Publication] : "2025/12/31"[Date - Publication]) NOT (review[Publication Type] OR "systematic review"[Title]) Equivalent search strings will be adapted for Embase, Scopus, and Web of Science.
Study Screening and Selection
All records will be imported into Rayyan.ai for blinded screening.
Step 1: Title and abstract screening by two independent reviewers.
Step 2: Full-text review to confirm eligibility.
Discrepancies will be resolved by discussion or a third reviewer.
Selection will be summarized using a PRISMA 2020 flow diagram.
Data Extraction
A structured Excel sheet will be used to extract the following data:
- Author, year, country
- Study design (in vitro / in vivo)
- Type and source of organoid (tissue, species)
- Purpose of study (efficacy, toxicity, etc.)
- Methods of drug screening
- Comparators used
- Main results and limitations
Two reviewers will extract data independently to ensure accuracy.
Quality Assessment / Risk of Bias
In vitro studies: Checklist adapted from ToxRTool or OECD standards.
In vivo studies: SYRCLE’s Risk of Bias tool. Each study will be rated as low, unclear, or high risk of bias.
Data Synthesis
Narrative synthesis: Results will be summarized descriptively and organized by organoid type, drug category, and outcome.
Quantitative synthesis: A meta-analysis will not be conducted due to expected heterogeneity in study designs and outcomes.
Tables and figures will present study characteristics, outcomes, and gaps.
Timeline
Registration
This protocol will be registered on OSF Registries, as the topic is primarily preclinical and may not qualify for PROSPERO registration. Any future amendments will be documented and justified according to PRISMA 2020 guidelines.
Acknowledgements
Registration
This protocol will be registered on OSF Registries, as the topic is primarily preclinical and may not qualify for PROSPERO registration.
Any future amendments will be documented and justified according to PRISMA 2020 guidelines.