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“She’s our child, nothing else matters – I’d lose my house before I take her off the treatment,” says Nina White from Kent, mother of seven-year-old Beatrice.

Beatrice has cystic fibrosis, a genetic condition causing progressive worsening of lung function, which is thought to affect more than 10,000 people in the UK. People with cystic fibrosis may spend hours a day having physiotherapy or inhaling medicines, and their life expectancy is currently about 47 years.

Three years ago a new medicine called Orkambi arrived, designed to reverse one of the genetic flaws that causes the disease. So far, studies of this drug have had mixed results but one of the most optimistic found that it .

But the official price of the drug is just over £100,000 a year. NICE, the body that assesses the cost-effectiveness of new medicines for England, decided that the benefits weren’t worth that price tag. Some other countries have concluded the same.

So Beatrice’s family began to pay for the drug privately, using contributions from all their relatives to pay the £8000-a-month costs. “We’re going month-to-month, everyone’s digging in. It’s completely unsustainable,” says White. She says Beatrice’s health has improved markedly.

Generic drug hope

White is not alone in doing this. Rob Long, in Sussex, is using his pension savings to pay for the drug for his nine-year-old son Aidan. Long had seen other children’s health deteriorate and not recover. “Their health can change rapidly.”

The price tag, however, is too much for most of the other estimated 5000 people in the UK who could benefit from this type of medicine. Cystic fibrosis can be caused by many different variants of the same gene, but Orkambi targets the most common one.

But there may be a way to get the medicine much more cheaply. Because Vertex does not have a valid patent on Orkambi in Argentina, a pharmaceutical firm called Gador is legally making a cheaper version of the medicine there, and selling it for about £23,000 a year.

The campaign group Just Treatment is lobbying for the UK to suspend Vertex’s patent through a Crown use licence, which would allow the country to buy the generic drug from Argentina through a rarely-used legal process.

Alternatively, the patent could be circumvented by beginning a massive clinical trial, in which there is no placebo arm and every participant receives the generic medicine.

This may sound almost like cheating, but a similar trial was set up in 2017 for a pill that can help people avoid catching HIV.

Families’ buyers club

However, Vertex says getting around their patent in such ways is not fair. “Companies who claim to be able to produce a product similar to Orkambi have not had to bear the cost of drug discovery and development,” the firm said in a statement. “It has taken twenty years and billions of dollars to fund our research.”

It’s unclear if the UK government would go down either of these routes to supply the generic medicine through the National Health Service. But there is a third option – for families to club together to buy the generic directly from Argentina. A newly-formed Cystic Fibrosis Buyers Club hopes to get a bulk purchasing discount, bringing the annual price per person to £18,000.

But many parents still won’t be able to afford the lower price. “It’s not a fair solution,” says Diarmaid McDonald, of Just Treatment. “It’s far from the way that patients should get access to life-changing treatment. Setting up these buyers’ clubs is a desperate act.”