A new model of orphan drug development: AI, drug repurposing and patient groups

Jin Ding (University of Sheffield, UK)

Panel: Imagined and abandoned futures

Abstract: Drug repurposing is a strategy of finding new uses for approved or investigational drugs. It has become an alternative but efficient way of developing orphan drugs. The emerging AI technology, such as big data and machine learning, is changing the traditional way of drug repurposing, mainly through serendipity. The AI-powdered drug repurposing leads to the increase in the chance of success in clinical trials and the reduction of the development time and cost. Rare disease patient groups and small AI companies have started to create a new model of drug development through the use of AI-powered drug repurposing to accelerate orphan drug development. The majority of orphan drug research focused on economic and regulatory fields, there was very limited research studying the role of technology and patient groups. To address the research gap, this paper will take a case study of one of the pioneering companies in this field and its collaborations with patient organizations. By using ideas from science and technology studies (STS), this paper will investigate the impact of niche technology and emerging patient-centred drug development models on the rare disease landscape and reflect on the establishment of an orphan drug innovation ecosystem.