Introduction

The rare repurposing open call is a collaborative project between Findacure, Cures Within Reach, and Healx. They are searching for existing drug repurposing ideas for rare diseases that have yet to receive a full clinical trial. Their primary aim is to demonstrate the huge potential of clinic-led, patient group-led, and researcher-driven innovation in drug repurposing for rare diseases, and the need for new funding streams to help these ideas bridge the translational gap, including Findacure's Rare Disease Drug Repurposing Social impact Bond (RDDR SIB).

The two key aims of the RDDR SIB are:

• To deliver new treatments to underserved rare disease patients
• To deliver low cost treatments that can reduce the spend of health care providers (crucially the UK National Health Service) on treated rare diseases, while improving patient outcomes.

This Request for Proposals is for Phase II efficacy trials for repurposed generic drugs, devices or nutraceuticals to treat rare genetic conditions. These clinical trials can be introduced into our innovative Social Impact Bond (SIB) funding model (see www.findacure.org.uk/drug-repurposing). As such it is crucial that there is a strong argument that the proposed repurposing project will significantly improve patient outcomes AND generate direct healthcare savings, as well as being scientifically viable.

Ideal projects will have some form of collaborator in the UK (note: the PI is not required to be based in the UK), and multinational clinical trials are welcome.

Ideal projects will fit into the RDDR SIB and:

• Target a rare disease with no effective existing treatment.
• Have a candidate generic drug, device or nutraceutical for repurposing supported by multiple pre-clinical studies.
• Have some in-human case studies.
• Have a potentially disease-modifying effect, which has significant patient benefit.
• Have the potential to dramatically reduce healthcare spend on treated patients, and able to show evidence of the current high healthcare cost for this patient cohort.
• Have connections with a rare disease patient group (an organisation or charity representing the opinions, rights, and treatment needs of a rare disease patient population).
• Have some form of collaborator in the UK.
• Be able to launch a clinical trial in early 2018.
• Have ready access to a patient population.

This call is not just aiming to identify some suitable projects for the Findacure RDDR SIB project. We are also keen to demonstrate to health care providers, research funders, and the rare disease community that there are a significant number of plausible rare disease repurposing projects held by the research and healthcare professions, and that these are struggling to secure conventional funding. We welcome all applications to further this aim, with lead researchers having the option to open up their projects to other CureAccelerator funders, or submit ideas with commercial potential.

To submit your repurposing projects, sign up for CureAcceleratorTM and search for the Rare Repurposing Open Call.

More information at

http://www.findacure.org.uk/open-call/

https://app.cureaccelerator.org/public/view/rfp/72