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Mapping strategies towards improved external validity in preclinical translational research
Clarissa F D Carneiro1, Natascha Drude1, Maren Hülsemann1
1QUEST Center for Responsible Research, Berlin Institute of Health at Charité, Berlin, Germany.
Expert Opinion on Drug Discovery
|September 11, 2023
Summary
Improving the robustness of preclinical research is crucial for successful clinical translation. This review covers strategies like systematic heterogenization and multi-center experiments to enhance the external validity of in vitro and in vivo studies.
Area of Science:
- Preclinical research
- Translational science
- Experimental design
Background:
- Clinical trials often fail despite positive preclinical results.
- Limited robustness in preclinical research contributes to these failures.
- Improving external validity of in vitro and in vivo experiments is a key solution.
Purpose of the Study:
- To review strategies for enhancing external validity in preclinical experiments.
- To identify and discuss sources of variability in heterogenized experiments.
- To examine sample size, analysis, and feasibility considerations for these strategies.
Main Methods:
- Literature review of strategies to improve external validity.
- Identification of articles on variability in systematically heterogenized experiments.
- Review of generalizability tests and multi-center/multi-batch experiments.
Main Results:
- Various strategies exist to improve experimental external validity.
- Prevalent sources of variability in heterogenized experiments are identified.
- Considerations for sample size, analysis, and feasibility are discussed.
Conclusions:
- Different strategies offer unique strengths for various research goals.
- Combining approaches can increase the success rate of preclinical research.
- Enhanced external validity in preclinical studies may improve clinical translation outcomes.
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