The Rare Disease Drug Repurposing Social Impact Bond

(RDDR SIB)

Drug Repurposing for Rare Diseases

When searching for new treatments for a disease the normal approach is to invent an entirely new drug; however, this is a very expensive and time consuming process. Drug repurposing is another approach to drug discovery. Drug repurposing is like recycling.  It takes existing generic drugs – those that are approved for human use and no longer protected by patents – and identifies new illnesses that they could treat. This is potentially cheaper and faster than traditional drug discovery. We believe this is a great approach for rare diseases where there is a huge need for new treatments, but little funding available for conventional drug discovery.

You can see some examples of drug repurposing on our case studies page. We are also looking for new repurposing ideas. If you would like to find out more about our Rare Repurposing Open Call, please visit this page.

To help support and promote drug repurposing research, Findacure is developing a new mechanism to fund clinical trials in drug repurposing. This is our social impact bond.

What is a Social Impact Bond?

A social impact bond (SIB) is a financial tool. It allows investors to put their money into projects that are designed to benefit society, which will be delivered by third sector organisations, like charities. These organisations use the investors’ money to set up and run their project, and receive payments from the government based upon the project’s success. They use these a portion of these success payments to repay investors. This is why social impact bonds are known as payment by results models.

What have Social Impact Bonds got to do with Findacure?

Findacure has found an innovative way to use this financial tool to fund drug repurposing research for rare diseases. This is the rare disease drug repurposing social impact bond – RDDR SIB for short.

The RDDR SIB aims to help the large number of rare disease patients with no treatment and little hope of ever receiving a treatment. By finding existing generic drugs with the potential to treat a rare disease, and running clinical trials to test their effect on patients, Findacure aims to repurpose drugs to treat rare diseases. When prescribed to patients via the NHS, this will improve their health while reducing the NHS’ expenditure: a win-win situation.

The SIB works in the following way:

By delivering low cost medicines to some of the most neglected patients in the healthcare system, the social impact bond has the potential to transform drug development for rare disease patients.

Proving the Concept

At the end of 2015 Findacure was awarded a grant from the Big Lottery Fund to run a study to prove that a social impact bond can be used to fund drug repurposing clinical trials for rare diseases. We selected three specific rare diseases to investigate: congenital hyperinsulinism, Wolfram syndrome, and Friedreich’s ataxia. Each of these has candidate drugs suitable for repurposing. We ran patient focus groups and built health economic models to assess the current impact of each disease on the NHS.

Patient focus groups – These groups brought together patients and parents affected by our rare diseases and asked them to share their experience of treatment, the cost of living with a rare disease, their thoughts on clinical trials, and their hopes for future treatment. Our focus groups helped to establish the need for the SIB. You can find the reports about each focus group on our resources page.

Health economic models – In order to prove that the RDDR SIB can work we had to do two things. First, demonstrate that each of our rare diseases has a high cost of care to the NHS. Our cost of illness models do this by estimating the cost of caring for the UK patient population for each disease for a year, within the NHS. Second, demonstrate that a repurposed drug could save the NHS money by improving their heath. To do this we built budget impact models which assess the effect of prescribing a low cost generic repurposed drug to the NHS budget.

Results

Completion of the proof of concept study has demonstrated a high burden of care to the NHS for all three rare diseases: each year congenital hyperinsulinism, Wolfram syndrome, and Friedreich’s ataxia, were found to cost the NHS approximately £4.5 million, £1 million, and £7.5 million respectively. Furthermore, conservative estimates of the effect of newly repurposed treatments on patient health all yielded a significant saving to the NHS budget across a five year period. Combined, the projects could save the NHS £2.3million over five years – more than sufficient to cover the cost of the three phase two clinical trials required.

Moving the RDDR SIB Forward

Findacure is now developing a full business plan for the social impact project based on the proof of concept study. We are working with our collaborators, the NHS, and potential investors to find a way to make it a reality. We are also working on publications which summarise the results of our modelling, which we hope will be available by mid 2017.

On 28th February 2017 we launched an open call for drug repurposing projects from rare disease researchers, clinicians, patient groups and companies. Find out more here.

If you are interested in finding out more about our RDDR SIB, or think you have a drug repurposing project that could suit it, please contact our Head of Research at [email protected].