One of the biggest challenges in drug discovery is knowing which preclinical findings are most likely to translate into meaningful outcomes for patients.
Despite the vast amount of data generated during early research, uncertainty remains around target selection, compound prioritisation and predicting treatment response. Human-derived models, including organoids, organ-on-chip systems and advanced imaging technologies, are increasingly being used to address these challenges by providing a more physiologically relevant view of human biology.
Drawing on insights from leading researchers, this report examines where these approaches are helping to improve confidence in drug discovery decisions, and where limitations and practical challenges remain.
Drug discovery still struggles to predict what will translate to patients. Large volumes of preclinical data are generated, but deciding which findings to trust remains a major challenge. Organoids, organ-on-chip systems and advanced imaging are beginning to change how these decisions are made.
This report examines how these models are used to test drug activity in human tissue, analyse resistance and understand differences between patients. Evidence from patient-derived organoids is compared directly with clinical outcomes, while organ-chip systems recreate tissue interfaces, fluid flow and mechanical forces that influence drug response. Imaging approaches track how the same organoids change over time, revealing resistant cell populations that would be missed with end-point assays.
Across early discovery, these approaches are being applied to improve compound selection, refine targets and support patient stratification where conventional models fall short.
Inside the report:
- Organoid screening to identify drug response, resistance and patient variability
- Recreating tissue interfaces, flow and mechanical forces in organ-on-chip systems
- Testing how organoid drug response relates to clinical and patient outcomes
- Tracking treatment response and resistant cell populations over time using imaging
- Applying human-derived models across oncology, neurology and early drug discovery
- Addressing challenges in reproducibility, throughput and integration into discovery workflows.
The report includes contributions from Professor Donald E Ingber, Dr Carla Verissimo, Dr Jeanine Roodhart, Professor Hans Clevers, Dr Mengyang Liu, Professor Rick Livesey and Professor Alysson Muotri.
Taken together, these perspectives show where human-derived models can improve confidence in early drug discovery decisions and where limitations remain.
This report is sponsored by:
Access the full report >>
Download Now
No comments yet