The rare disease drug repurposing social impact bond

Drug repurposing can only move ahead if clinical trials can be funded.

Our rare disease drug repurposing social impact bond proposes an innovative funding source for such research.

Why do we need a new funding source for research?

Although generic drug repurposing has so much potential for rare diseases, it tends to be avoided by the pharmaceutical industry. This is because intellectual property, which protects their research from being copied by competitors, cannot easily be secured for existing generic drugs: after a company has spent millions on research, patients could be prescribed a non-branded version of the same drug from any pharmacy.

The research is instead left to rare disease researchers, specialists, and charities. However, with small patient populations and such high competition for funding, these groups can struggle to secure the budget needed to test repurposing projects and run full clinical trials.

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. These projects are delivered by third sector organisations like charities. They use the investors’ money to set up and run their project and receive payments from the government based upon the project’s success – the more successful, the more money they save the government, and the more money they are paid. These payments allow investors to be repaid. This is why social impact bonds are known as payment by results models.

How can a social impact bond fund repurposing?

BM Manifesto 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.

But how would it work?

Our SIB is based on the premise that successfully repurposing low cost generic drugs for rare disease patients will save the NHS money.

Rare diseases left untreated cost the NHS lots in surgery, care, appointments, hospital, stays, and much more – a cost that can be significantly reduced by a cheap, repurposed treatment. The NHS can then use a portion of these savings to reimburse BM Manifesto for the cost of the clinical trial. We will use this reimbursement to pay back private investors, whose money was required to initially run the trial. Surplus reimbursement will be invested by us in more clinical trials: the project is self-sustaining and non-profit.

The SIB would work in the following way:

By finding existing low cost generic drugs with the potential to treat a rare disease, and running clinical trials to test their effect on patients, BM Manifesto 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.

What has BM Manifesto done?

Since the inception of the RDDR SIB project in 2015, BM Manifesto has:

  • Secured a grant from the Big Lottery Fund Commissioning Better Outcomes Fund to deliver a proof of concept study into the SIB, with the support of Simon Stevens, Chief Executive of NHS England.
  • Identified three viable repurposing projects in Wolfram syndrome, Friedreich’s ataxia, and congenital hyperinsulinism for the proof of concept study.
  • Delivered a proof of concept study, showing that all three untreated rare diseases have a high financial burden on the NHS (roughly £13 million per year in total). Repurposing a generic drug to treat each of these conditions would save the NHS roughly £2.3 million over the five years after the drug had been paid for. This would be sufficient to cover the cost of the three phase II clinical trials required.
  • Held discussions with NHS England’s highly specialised services team about the RDDR SIB – while there was strong initial interest they decided that it did not fit with their strategy in mid-2017.
  • Two of the three repurposing projects we identified have been funded independently since the completion of our study, and are moving into clinical trials.
  • Held a global rare disease drug repurposing open call to identify new repurposing ideas and highlight their prevalence. We received 38 different proposals from around the world; however with the NHS deciding not to take the RDDR SIB forward we were unable to fund any using our model.

Where we are now

We are exceptionally proud of the work we have done in developing the rare disease drug repurposing social impact bond model. This innovate concept has the potential to provide much needed funding for rare disease research, to save money for global health systems, and deliver much needed new treatments to some of the world’s most underserved patients. While we have not yet been able to convince the NHS to commission the programme, we will continue to search for routes to move it forward with our partners around the globe, and to champion both drug repurposing and innovative financial models as crucial in delivering new treatments to the world’s rare disease population.

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 get in touch.