A Developers Journey Towards Regulatory Qualification of a Drug Discovery Tool
This talk is scheduled as part of the “In vitro and in silico models for sustainable drug discovery” track.
Pre-clinical safety testing is required prior to progressing a candidate drug into the clinic. With safety-related findings continuing to account for approximately 30% of drug attrition in the clinical phase of drug development¹, there is a pressing need to continue improving pre-clinical testing strategies to protect patient safety and reduce costly drug attrition.
Organ-on-a-chip technology, developed in the early 2000’s, is emerging as a powerful tool for predicting toxicity during pre-clinical testing. For example, it has been demonstrated that a human Liver-Chip had an 87% sensitivity and 100% specificity for detecting drug-induced liver injury in a small molecule drug set with a known clinical outcome².
Recent U.S. regulatory policy updates encourage greater use of innovative approaches, such as organ-on-a-chip, within drug discovery programs. To gain acceptance, these tools must undergo regulatory qualification to demonstrate scientific validity, technical robustness, and reproducibility across laboratories. Accelerated regulatory acceptance of data will be enabled if sponsors use a qualified drug discovery tool within a specific context of use. To facilitate qualification of such tools, the U.S. FDA launched the Innovative Science and Technology Approaches for New Drugs (ISTAND). The program consists of three main phases; (1) Letter of Intent, (2) Qualification Plan and (3) Acceptance as a qualified tool.
This presentation will introduce organ-on-a-chip technology and will detail the study that was undertaken to assess the performance of Liver-Chip in the prediction of small molecule drug-induced liver injury. The presentation will describe how such a tool can be used in project decision-making as well as showcasing the journey of the Liver-Chip through to an accepted qualification plan with the ISTAND program.
References:
1. Sun et al., 2022, Acta Pharm Sin B., 12(7):3049–3062.
2. Ewart et al., 2022, Communications Medicine, 2, 154.
