Tackling rare diseases: Clinical trials on chips

Area of Science:

• Biomedical Engineering
• Rare Disease Research
• Drug Development

Background:

• Clinical trials for rare diseases are challenging.
• Tissue chip technology is currently used for in vitro safety and efficacy assessments.
• Expanding tissue chip use to therapeutic development is critical for rare disease treatments.

Purpose of the Study:

• To explore the feasibility of using tissue chips for rare disease therapeutic development.
• To identify best approaches for integrating tissue chips into the drug development pipeline.
• To advance the field of organs-on-chips (CToC) and build confidence in their application.

Main Methods:

• Convened a working group of stakeholders and experts.
• Addressed key questions regarding disease settings, test agents, study design, data collection, benefit/risk, and stakeholder engagement.
• Explored current and future use cases and prerequisites for progress.

Main Results:

• Identified critical considerations for using tissue chips in rare disease therapy development.
• Highlighted the need for standardized approaches and robust data collection.
• Emphasized the importance of stakeholder engagement for successful implementation.

Conclusions:

• Tissue chips offer a promising avenue for accelerating rare disease drug development.
• Further research and collaboration are needed to optimize their use in the development phase.
• Successful integration requires addressing challenges in study design, data interpretation, and regulatory pathways.