Related Experiment Video
Updated: Oct 22, 2025
An Intestine/Liver Microphysiological System for Drug Pharmacokinetic and Toxicological Assessment
Published on: December 3, 2020
Applicability of organ-on-chip systems in toxicology and pharmacology
Marlon R Schneider1, Michael Oelgeschlaeger1, Tanja Burgdorf1
1German Centre for the Protection of Laboratory Animals (Bf3R), German Federal Institute for Risk Assessment (BfR), Berlin, Germany.
Abstract:
Organ-on-chip (OoC) systems are microfabricated cell culture devices designed to model functional units of human organs by harboring an in vitro generated organ surrogate. In the present study, we reviewed issues and opportunities related to the application of OoC in the safety and efficacy assessment of chemicals and pharmaceuticals, as well as the steps needed to achieve this goal. The relative complexity of OoC over simple in vitro assays provides advantages and disadvantages in the context of compound testing. The broader biological domain of OoC potentially enhances their predictive value, whereas their complexity present issues with throughput, standardization and transferability. Using OoCs for regulatory purposes requires detailed and standardized protocols, providing reproducible results in an interlaboratory setting. The extent to which interlaboratory standardization of OoC is feasible and necessary for regulatory application is a matter of debate. The focus of applying OoCs in safety assessment is currently directed to characterization (the biology represented in the test) and qualification (the performance of the test). To this aim, OoCs are evaluated on a limited scale, especially in the pharmaceutical industry, with restricted sets of reference substances. Given the low throughput of OoC, it is questionable whether formal validation, in which many reference substances are extensively tested in different laboratories, is feasible for OoCs. Rather, initiatives such as open technology platforms, and collaboration between OoC developers and risk assessors may prove an expedient strategy to build confidence in OoCs for application in safety and efficacy assessment.
Insights
Organ-on-chip (OoC) systems offer enhanced predictive value for chemical and pharmaceutical safety testing. However, challenges in standardization and throughput must be addressed for regulatory acceptance.
Area of Science:
- Biotechnology and Biomedical Engineering
- Toxicology and Pharmacology
- In Vitro Toxicology Models
Background:
- Organ-on-chip (OoC) systems are advanced in vitro models using microfabrication to mimic human organ function.
- These systems offer a broader biological context compared to traditional cell culture assays.
- Their application in chemical and pharmaceutical safety assessment presents both opportunities and challenges.
Purpose of the Study:
- To review the issues and opportunities associated with applying OoC systems in safety and efficacy assessments.
- To identify necessary steps for the regulatory acceptance of OoC technology.
- To discuss the feasibility of standardization and validation for OoC use in risk assessment.
Main Methods:
- Literature review and analysis of current issues and opportunities in Organ-on-chip (OoC) applications.
- Evaluation of OoC complexity, predictive value, throughput, standardization, and transferability.
- Discussion on characterization, qualification, and interlaboratory standardization requirements for regulatory use.
Main Results:
- OoC systems provide enhanced biological relevance but face challenges in throughput, standardization, and transferability.
- Regulatory use necessitates detailed, standardized protocols for reproducible interlaboratory results.
- Current evaluation focuses on characterization and qualification, with limited validation due to low throughput.
Conclusions:
- Achieving regulatory acceptance for OoC requires addressing standardization and throughput limitations.
- Collaborative strategies, such as open technology platforms, are essential for building confidence in OoC for safety and efficacy assessment.
- The feasibility and necessity of interlaboratory standardization for OoC regulatory application remain subjects of debate.
