As fans of all good crime dramas will know, proof is a hard thing to establish. Over the last year, the Findacure team has found itself in the position of having to build its own body of evidence – not to catch a criminal – but to prove our own theory: that a social impact bond can be used to fund generic drug repurposing clinical trials. Success could lead to a strong collaboration with the NHS, and funding for vital clinical trials for rare disease treatments, ultimately changing, or even saving lives.
Never one to duck a challenge, Rick took the lead.
For those who don’t remember, a social impact bond (SIB) is a financial tool designed to reward projects that deliver a benefit to society in a cost effective manner. We will use private investment to fund phase II clinical trials in a range of rare disease drug repurposing projects, targeting diseases that cost the NHS a lot to manage. Successful trials would provide the NHS with more robust evidence to prescribe the generic drug to rare disease patients off-label. This means that the NHS saves money by treating patients in a new and effective way.
In our SIB a proportion of those financial savings made by the NHS will be used to reimburse investors, and to fund more drug repurposing research.
To prove that the rare disease drug repurposing social impact bond is more than just a bright idea, we needed to:
- Show that there were scientifically valid drug repurposing ideas out there.
- Demonstrate a need for new drugs for rare disease patients.
- Show that untreated rare diseases currently cost the NHS a lot of money.
- Show that repurposing a generic drug could save the NHS money by improving patient health.
- Show that the money saved by the NHS would be sufficient to cover the costs of a clinical trial into a repurposed drug for a rare disease.
Naturally, we did it all.
We’ve run studies into three specific rare diseases with strong candidate drugs for repurposing: congenital hyperinsulinism (CHI), Wolfram syndrome and Friedreich’s ataxia. For each we have run a patient focus group to highlight the huge patient need for improved treatments (you’ll be able to read the results of these studies on our website soon). Rick’s trip to Birmingham gave us the chance to run one such focus group with Wolfram syndrome patients.
The core of our work though has been to understand the cost of rare diseases to the NHS – though we’ve also looked into costs outside the NHS. Our financial models have allowed us to build a case for drug repurposing in each of our three target diseases. Overall we have found that congenital hyperinsulinism, Wolfram syndrome, and Friedreich’s ataxia cost the NHS approximately £4.5 million, £1 million, and £7.5 million respectively each year. By repurposing specific generic drugs for each of our target rare diseases, the NHS could help CHI patients keep their pancreas, preventing life-long diabetes, while slowing the progression of both Wolfram syndrome and Friedreich’s ataxia. At the same time, we estimate the NHS could save £2.3million over five years – more than sufficient to cover the cost of the three phase two clinical trials required.
We’ve compiled all of our hard gained evidence into a detailed report which we’re now discussing with collaborators, investors, and the NHS. After months of hard work, we now have the proof – it is now time to test the idea. Keep watching this space for more information on SIB project, and our progress in taking it to the next stage.
If you like to learn more about Findcure’s social impact bond project and cost of illness models you can contact our Head of Research, Rick, by emailing [email protected].