Access to gene therapies and other innovative therapies for rare diseases: Ethical, IP and regulatory issues

This research project at the intersection of bioethics, law, medicine, regulatory science, and health economics will look at four main challenges to the successful development and market launch of innovative medicines for rare diseases, identified during ITINERARE Phase I: intellectual property, ethics in clinical trial design, regulatory approval, and pricing. In line with ITINERARE research priorities, the main focus of the project will be on gene therapy medicinal products (GTMPs) for rare genetic disorders. In a second step, we will employ the methodology devised for our analysis of GTMPs to develop research-based guidance on artificial intelligence (AI)-enabled medicines.

Our findings are expected to support Swiss researchers and their industry partners to successfully bring GTMPs to the Swiss and global market, and to support lawmakers and regulators to devise approaches to foster, in an ethically responsible way, the development and accessibility of gene therapies for rare diseases in Switzerland. In addition, our project is intended to support Swiss researchers prepare for the ethical, intellectual property, and regulatory challenges posed by the advent of AI-based pharmaceutical research and development, and to support lawmakers, regulators, academia, and industry in devising research-based strategies for the digital transformation of pharmaceutical research and development.