Short abstract:

Drug repurposing is a promising strategy to secure new therapeutic options for rare diseases. This paper identifies main barriers for drug repurposing in Europe and discusses ways to making the current system more economically sustainable.

Long abstract:

Drug repurposing refers to the investigation of existing pharmaceuticals for new therapeutic purposes. While drug repurposing is acknowledged as a strategy for therapeutic innovation, particularly in rare and neglected diseases, the practical application of this strategy raises a number of challenges. In this paper, we a) describe the current drug repurposing ecosystem in Europe, b) identify main systemic barriers in moving repurposed drugs from the clinic to the market in a sustainable fashion, and c) discuss how the approval, launch and reimbursement of repurposed pharmaceuticals might be better supported. Building on analysis of policy reports and stakeholder interviews, we identify barriers relating to the cost of late-stage clinical development, liability at market authorisation, lacking knowledge about regulatory processes among potential sponsors, and issues of reimbursement. Further, there is no proven business model to ensure that innovation centred on drug repurposing will be economically viable or commercially attractive without ongoing support from research funders and philanthropy. Drawing on work in innovation studies and the idea of a sociotechnical niche, we propose that a more sustainable alternative could be to create a circular drug repurposing economy with profits reinvested in R&D. Further, we propose that intermediary institutions (or market translators) can play an important role by mimicking the activities of large, integrated pharmaceutical companies in project finance, clinical trials design, portfolio management, and regulatory affairs. Such intermediary institutions could take different organisational forms (public, private, hybrid) and create new models for commercial or non-profit drug development.