The lives of thousands of women at increased risk of breast cancer could be saved each year after the NHS began the process of seeking approval to redeploy a cheap medication already in use.
Anastrozole costs less than 10p per dose and has been shown to halve cases of breast cancer in post-menopausal women, who are at higher risk of the disease.
It is the first drug to be targeted by a little-known UK scheme to research new uses for medications already in use, with the potential to revolutionise NHS care at relatively little cost.
Documents seen by i show that a contract was offered late last year for funding for an application to Britain’s medicines regulator to allow anastrozole, which is currently used to treat breast cancer, to also be prescribed as a preventive therapy for the one in seven women deemed to be at moderate to high risk of developing cancer.
At present, anastrozole can only be used as a treatment for advanced breast cancer, but it is estimated it could save 4,000 lives a year if used to prevent disease. One leading research charity, Breast Cancer Now, told i that the benefits of the drug had long been recognised and it welcomed any steps to improve access to it for those at risk of the disease.
“Research has shown that anastrozole can reduce incidence of breast cancer by nearly half over 10 years in postmenopausal women at increased risk of the disease,” a spokesperson said.
“Any work to extend the licence for anastrozole to include preventative use will pave the way to improving access to it for women who are at increased risk.”
The initiative, overseen by NHS England and NHS Improvement, is the latest example of the “repurposing” of medications, a technique that researches so-called “off-patent” or “generic” drugs to see if they can be redeployed to an alternative use such as treating rare, or indeed common, diseases.
But while there is progress being made on anastrozole, a leading pharmaceutical industry body has told i that too little is being done to exploit a potential treasure trove of similarly inexpensive but effective treatments because companies lack any incentive to conduct the necessary research.
Ministers have previously trumpeted the success of innovative British repurposing research after a publicly funded study during the pandemic found that an inexpensive steroid, dexamethasone, was highly effective in treating hospitalised Covid-19 patients. The medication is estimated to have since saved the lives of more than one million patients worldwide.
But drug producers say they are being “disincentivised” from seeking similar breakthroughs because of a “perverse” system in which Britain offers no protection for research into new uses for generic drugs whose patent has expired but which continue to be licensed for their original purpose, such as dexamethasone.
The British Generic Manufacturers Association (BGMA), which represents the segment of the pharmaceutical industry responsible for providing about 80 per cent of the drugs used in the NHS, said that while the UK was effective at protecting the research needed to produce entirely new but expensive medicines – more often than not developed by multinational pharmaceutical giants – it is failing to prioritise potentially cheaper breakthroughs.
Mark Samuels, chief executive of the BGMA, told i: “The current system disincentivises repurposing like dexamethasone, and that is incredibly unhelpful because those medicines are affordable. We have a UK system that incentivises high-price medicines and disincentivises research into more affordably priced medicines. We are saying to Government, ‘please stop ignoring the sector that provides four out of five NHS medicines’.”
The success of rapid research during the pandemic to identify new treatments for Covid among the world’s existing 20,000 medicines has prompted excitement on both sides of the Atlantic at the possibility that effective treatments for other diseases may be effectively hiding in plain sight.
One of the most established examples of repurposing is the widespread use of low-dose aspirin to lower the risk of heart disease and strokes. About one billion people worldwide are now estimated to take the pill regularly, although medics have warned that it may offer only modest long-term benefits.
Experts aruge there are dozens, possibly hundreds, of other avenues ripe for exploration, including the potential use of some chemotherapy drugs to reverse or alleviate symptoms of dementia.
LifeArc, a UK-based medical research charity, is calling for a particular focus on rare diseases for repurposing research, pointing to the success of using rapamycin, a compound originally identified in a soil sample from Easter Island and used to treat kidney transplant patients, as the first effective treatment for a rare progressive lung disease, LAM, which almost exclusively affects women of childbearing age.
The charity is also supporting a programme to have tamoxifen, currently used to treat breast cancer, to help improve the conditions of children with myotubular myopathy, a rare genetic muscle wasting disease that mostly affects boys and means fewer than one in five with the condition make it to adulthood.
Last year, in response to the advances made during the pandemic, the NHS, supported by central government, established its Medicines Repurposing Programme to support further research. It is understood the scheme, which was set up in March 2021 and allows patient groups, researchers and companies to put forward candidate drugs, will eventually support formal trials on up to five medicines a year.
The process of varying the licence of anastrozole is understood to be the first product selected by the programme after research, first published in 2014, showed it could lead to a 49 per cent reduction in breast cancer cases among post-menopausal women at higher risk of disease due to factors such as family history. Studies have estimated that 3,750 cases of breast cancer would be prevented annually by taking anastrozole over a five to 10-year period.
A document seen by i shows that NHS England and NHS Improvement offered a contract and funding late last year to a company or organisation to prepare a dossier to apply to the Medicines and Healthcare Products Regulatory Agency to vary the drug’s licence for preventive use. Scotland has its own separate drug licensing system, but will be watching closely.
Drug industry figures have applauded the Medicines Repurposing Programme, but they warn it is small scale and claim that the Government is failing to fully exploit an opportunity that exists for far wider scale advances.
The BGMA said that the generics sector, which saves the NHS £15bn a year on its drugs bill, was caught in a Catch 22 situation whereby a company stands to gain no benefit, or even lose out, if it invests the sum required to research a drug for repurposing and obtains regulatory approval. It is argued that the absence of any legal or regulatory protection for that work means that any competitor could step in to produce the repurposed drug and be able to do so at a lower price because it has no research and development costs to recoup.
Mr Samuels said the industry would like to see avenues explored to support a beefed-up system to research on repurposed drugs, possibly by developing a mechanism for companies to recoup research costs or expanding the remit for publicly-funded research. He said: “Coming up with a solution to this would seem to be a very sensible thing to do because we want more new medicines that are affordably priced.”
Repurposing has the benefit of being based on drugs that have already gone through rigorous clinical testing to ensure their safety. Because generic drugs are off-patent, they can be produced at a fraction of their original cost – often at a discount of 80 to 90 per cent.
An industry source told i: “The NHS scheme is great but in terms of volume, it is like bringing a peashooter to a gun fight. We need to be looking at bringing forward dozens of compounds for novel use, not just a handful. If we can get this right, the potential for cheap, innovative medications for the health service is huge.”
Experts point out that the repurposing process is nonetheless still complex, in particular when it comes to arranging trials for uses in rare diseases where the number of patients is necessarily limited.
The Department for Health and Social Care pointed to the success of the UK’s dexamethasone research as the basis for future work on repurposing and said it is spending £5bn on health-related research to boost innovation.
A spokesperson said: “We are committed to ensuring the UK continues to be a world leader in the research, development and deployment of innovative and cost-effective medicines to NHS patients.”
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