Drug repurposing offers a potentially rich source of safe and effective therapies for both common and rare diseases, but its challenges are often overlooked, or not fully understood, when planning development programmes. This can result in failure to reach the patients for whom the therapies are being developed. In 2021 LifeArc held a workshop to explore the opportunities and challenges of repurposing. Visit LifeArc for a publication of the findings from this workshop.

It became clear that there is an opportunity to improve awareness of the challenges that can be faced in repurposing projects, and ways to overcome them, in order to increase the likelihood of potentially life-changing medicines reaching patients. In this toolkit we aim to provide a guide for researchers, charities and others embarking on repurposing projects. We highlight common activities, issues and considerations at each development stage and signpost to other available resources. The content is mostly styled as questions – what should I be considering at each development stage? It is intended to stimulate thinking and not as a step-by-step guide, which reflects the variable nature of repurposing.

The stage at which a repurposing project enters the development path varies from project to project, but in every case it makes sense to begin with construction of a Target Product Profile (TPP). The TPP sets out the characteristics of the planned treatment, to drive a focused development plan.

The toolkit has been co-developed by Life Arc and the Medical Research Council (MRC) with input from many organisations and individuals with expertise and experience of drug repurposing. Both LifeArc and the MRC support repurposing through several funding schemes and activities. Visit Funding for further information. The MRC Regulatory Support Centre provides guidance on the legal and ethical requirements for research involving human participants, their tissues or data.

We welcome feedback on the toolkit - it is intended to be a living resource, so please get in touch with any comments.