From consent to participatory governance: The need for multi-stakeholder alignment in iPSC-based drug repurposing

Induced pluripotent stem cells (iPSCs) are increasingly used as disease models to accelerate drug repurposing, especially for rare diseases. While ethical and regulatory issues in iPSC research have been widely discussed, little is known about how these challenges are addressed in practice. To provide insights into the current practice, challenges, and opportunities in the governance of research on iPSC-based drug repurposing for rare neurological disorders, semi-structured interviews were conducted with various experts from eight countries in the context of the SIMPATHIC project. The results indicate that this research context requires improved information provision for minors and individuals with cognitive impairment and more clarity regarding commercial use and reporting of findings. Uncertainty in governance procedures hinders collaboration, validation, and clinical translation in iPSC-based drug repurposing. Responsible (re)use of iPSCs requires dynamic, transparent, and participatory governance structures based on shared decision-making with all stakeholders involved.