RE: RE: Professor Platt29 Nov 2018 09:02
UK charity finds disruptive new way to tackle rare diseases funding
Drugs that are already licensed are an even cheaper source of potential therapies for rare diseases. Yet, because many of these drugs are already, or will be imminently, off patent, there is little financial incentive to retrial them for other disorders.
Fran Platt, professor of biochemistry and pharmacology at the University of Oxford, explains: “A repurposed drug could be given orphan designation, but if it is freely available, it would be difficult to enforce pricing, meaning there is a risk drug companies won’t recoup their costs.”
Findacure, a UK-based charity founded to drive research and development for rare disease treatment, wants to bypass pharma altogether.
Chief executive Dr Rick Thompson says orphan designation incentive, though good, often leads to highly priced drugs. “There is a worry we get to a stage where there are drugs developed, but no one can afford to access them,” he says.
Therefore, Findacure is proposing a radical new funding strategy: a social impact bond. The charity wants to issue a bond funded by private investors to pay for ten clinical trials for repurposed drugs, specifically targeting diseases that have a high management cost for the NHS.
In theory, successful trials would lead to off-label prescription of drugs for rare diseases within the NHS, which would then, from the saving it makes via reduced demand for surgery, care, appointments and so on, pay a proportion of money back into the bond to reimburse investors and fund new trials. The NHS has yet to agree to trial the model, however.
“I think it is a really unique and disruptive idea that proposes a different way to tackle a difficult problem,” says Dr Thompson. “It just needs more time to soak into the consciousness to the NHS.”
Findacure is currently working with US charity Cures Within Reach to help develop the idea further.
Sign In
Follow the stocks
