Sherri Coleman recently spent a week paralysed, imprisoned in her upstairs bedroom, because her doctors say they can’t give her the drug she needs.
‘If it hadn’t been for Violet, my 11-year-old daughter, and mum, Rose, bringing me drinks and food, I would have starved to death,’ she says in measured tones that belie the desperation of her condition.
Sherri, 41, was born with a rare form of muscular dystrophy, inherited from her father, that worsens with age, causing her body to become paralysed at random — the attacks may last anything from a few minutes to a number of days.
The recent episode involved her arms, legs, neck and even her tongue — meaning she could barely eat or swallow.
In the past, such episodes were contained with the drug dichlorphenamide.
Until 2012 it was branded as Daranide and was readily available on the NHS, costing £400 for a year’s supply. However, the equivalent drug costs £35,000 for a year’s supply and Sherri has been told it is too expensive — so she can’t have it.
Daranide was originally approved to treat the eye disease glaucoma. It fell out of use because it didn’t work well, but it was found to help in muscle disorders, including some types of muscular dystrophy. Doctors could prescribe it ‘off label’ — that is, for conditions the drug was not originally licensed for.
It was a lifeline for Sherri and her father, Paul Toms. Yet, because the cost of production was higher than the profit from global sales — around 200 other people took it worldwide — it was withdrawn in 2008 and stocks finally ran out in 2012.
The U.S. government asked the drug’s manufacturer, Taro Pharmaceuticals, to undertake the trials and testing needed so the drug could be licensed to treat periodic paralysis.
It was relaunched 18 months later, rebranded as Keveyis, with new packaging — and a new price, almost 90 times higher, of £35,000 a year.
Instead of Keveyis, Sherri was offered a cheaper alternative called acetazolamide or Diamox which, although it helps, is much less effective.
The former factory worker, who lives in Huntingdon, Cambs, with daughter Violet, can barely walk — and is largely housebound.
She is matter of fact as she describes her worsening condition: ‘I can only walk a few steps, and I risk falling over because my balance is so bad,’ she says.
‘Daranide made a massive difference and since it’s been taken away I’ve had many more attacks. Every time I have a bad one it does a bit more damage and I never get as much strength back as I had before.’
Sherri did not want to be photographed for Good Health because she says her face and body are covered with disfiguring brown blotches, which doctors have told her are a side-effect of Diamox. Keveyis is a so-called orphan drug, meaning the number of patients who can benefit from it is too small to make it profitable from sales. In these cases, a manufacturer is asked by a government to set up a production line.
The trade-off is the company then has a monopoly, and there are no rules about what it charges for the treatment.
In the absence of new blockbuster profitable drugs such as the impotence treatment Viagra, orphan drugs are increasingly important to drug companies.
A report from the London-based data agency Evaluate, published in June, found orphan drugs for diagnosing, preventing or treating rare diseases will account for a third of all drug sales by 2022. And while the situation with Keveyis — where a cheap, pre-existing drug is relaunched as a pricier product — is not common, orphan drugs are often prohibitively expensive.
Last February, for example, it was announced that the watchdog NICE was restricting access to the drug Strensiq, so only some patients with the rare genetic disease hypophosphatasia, which causes malformed breakable bones, would get access to a vital enzyme replacement therapy priced at £367,000 a year.
Other conditions where effective orphan drug treatment is often withheld, include a variety of less common cancers, narcolepsy, the blood clotting disorder haemophilia, Crohn’s disease and childhood arthritis.
Another problem relating to drug companies’ capture of a limited market is where they simply overcharge the NHS because they have a monopoly. Recently, Canadian drug company Concordia International was accused by the Competitions and Markets Authority (CMA) of overcharging the NHS by more than £100m for a thyroid drug — liothyronine — which went from £4.46 a pack in 2007 to £258.19 by July.
Until this year, Concordia was the sole supplier of the drug — the only effective option for around 15 per cent of people with a thyroid hormone deficiency.
Concordia could be fined if it is found guilty of overcharging, but it is not the only company to find itself in the frame.
Last year, the CMA fined drugs giant Pfizer £90 million for overcharging by 2,600 per cent for an anti-epilepsy drug. There are seven other ongoing CMA investigations into overcharging. Whatever is driving the rising prices, the personal cost to patients is enormous. Sherri, who has suffered with the condition all her life, struggled at school.
Unsurprisingly, she left without taking A-levels and got a job in a factory, sitting at a bench soldering components for computers, where she worked for the next 13 years before she had to quit.
The guilt of having passed the condition to his daughter weighs heavily on Paul, 63, a former London taxi driver, who is also suffering from lack of access to Keveyis and has to use a wheelchair.
He has campaigned tirelessly for Sherri to get the drug. ‘I’ve had my life, but she’s the mother of a young child. She needs to be there for Violet,’ says Paul. ‘I’m determined not to let Sherri get to the state I’m in.’
The Muscular Dystrophy UK has also lobbied health funding bodies and politicians on behalf of Sherri and Paul.
‘This ongoing battle needs to be resolved,’ said its spokesman. ‘The situation is particularly agonising for Paul and Sherri, and the other patients who could benefit from this life-changing drug.’
The bitter irony of the Keveyis story is that the price the NHS charged was agreed by the Department of Health earlier this year, ‘recognising the costs involved in developing a licensed product for a small group of patients’.
Good Health has learned that at £4,100 for 100 tablets, the price being charged in the UK by Sun Pharmaceuticals, the European distributor, is significantly cheaper than everywhere else in Europe where it costs €5,700 (£5,100) per 100 tablets — and yet patients there are already receiving it.
NHS England says it is down to Paul and Sherri’s doctors to request funding for their patients’ treatment.
However, a spokesman for the National Hospital for Neurology and Neurosurgery in London, where they are both treated, insisted the drug has not yet been approved by NHS England.
A spokesman for Sun Pharma, which distributes Keveyis in Europe on behalf of Taro, told Good Health: ‘The price agreed for the UK is very competitive compared with other countries in Europe. It has been negotiated and approved by your Department of Health.
‘It is up to the NHS to give us the prescriptions to process. Doctors in France are getting it through the equivalent of special funding requests, and it gets to the patient within two days.’