Rare Repurposing Open Call

Findacure is a UK rare disease charity that is building the rare disease community to drive research and develop treatments. In collaboration with Cures Within Reach, and supported by Healx, Findacure and these partners are working to promote drug repurposing to the rare disease community.

Rare diseases can be a challenging field for drug development, due to their small fragmented patient populations. However, the power of the patient group is transforming this situation, unifying the patient voice, increasing awareness, and driving research. Drug repurposing is a great avenue for such active patient groups to pursue, as it has the potential to deliver drugs to patients more quickly and affordably than conventional new drug discovery.

To promote repurposing for rare diseases, Findacure, Cures Within Reach, and Healx are launching the Rare Repurposing Open Call, searching for existing drug and other repurposing ideas for rare diseases that have yet to be tested via a robust efficacy-based clinical trial. We are seeking to demonstrate the huge potential of clinic-led, patient group-led, and researcher-driven innovation in drug repurposing, and the need for new funding streams to help these ideas bridge the “translational gap”.

In parallel to the open call, we are working to develop the world’s first rare disease drug repurposing social impact bond (RDDR SIB). This is a unique investment structure that is designed to fund the repurposing of generic drugs to create rare disease treatments – a type of project that almost always lacks standard commercial viability.

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:

• Target a rare disease with no currently 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 ability to document the improvement in patient outcome through reliable and objective measurements
• Have the potential to dramatically reduce healthcare spend on treated patients, be 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.

Submissions are welcome beyond this scope of the SIB model, with lead researchers having the option to open up their projects to other CureAccelerator funders, and/or submit ideas with commercial potential.

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