THE wait has been “cruel”, Marion Ferguson says. Her son, 15-year-old Thomas, suffers from the “Celtic” form of cystic fibrosis, or type G551D. Since early last year, when Kalydeco started being used in the United States with spectacular results, families across the world whose lives had been affected by the disease had known there was hope.
For the first time, a drug seemed to merit the tag “miracle cure”, effectively halting the onset of the illness.
However, it was not until 4 December last year that the Scottish Medicines Consortium (SMC) – the body which decides which drugs can be prescribed in Scotland – met to decide on whether to support its introduction.
And families have been made to wait until tomorrow to find out whether it is going to be made available on the NHS.
Ferguson, from Hamilton, who has set up a support group for families in her situation, says: “It seems so unfair and cruel to keep them in the dark for five weeks. Why should we have to wait for five weeks for a decision?”
The agony was only intensified when the SMC’s equivalent body in England announced at the turn of the year that it would be pressing ahead with the new drug.
“Now I have people in the parent groups in England who say their lives have already been transformed,” said Ferguson. “They have the drug in their homes. It is wrong there is this inequality going on.”
Thomas, she says, has fought hard to retain good lung capacity. “But the fact that this drug is there and we can’t get access to it is very cruel. We are talking about mostly young people here, because they [CF sufferers] don’t live long.
“This drug targets the ‘Celtic’ gene, and in Scotland we have got the largest percentage in the whole of the UK. Yet we are the last to get it. It doesn’t seem right.”
The wait has been particularly hard on Jonny and Julie Gordon, Accident and Emergency consultants from Glasgow, whose eight-year-old daughter Kate suffers from the same form of CF.
Speaking last week, the pair had just returned from Yorkhill Hospital having left their daughter there, along with two other children who also suffer from the same form of CF. Kate contracted pneumonia last week due to her condition, and is now receiving intravenous antibiotics.
The clock is ticking on her condition, says Jonny. “Her lung capacity is not going to get any better than 90 per cent. We’ve been told it’s now down to 60 per cent because of the acute lung infection.” His fear is that, without a speedy remedy, Kate could permanently lose a large part of her lung function.
Like Marion Ferguson, he wants answers on why a decision has taken so long. “If the answer is ‘yes’, we have wasted five weeks of time,” he says. But mentally he is preparing for a ‘no’, even though such a decision would be baffling.
“It can’t be due to the clinical effectiveness issue, because it is one of the most clinically effective drugs there is,” he says. “It just seems to be one of those no-lose situations. It is a huge cost, but it has been approved in America and they won’t pay for it unless it is going to be cost-beneficial to them.”
If it is a “no”, the couple will move south, taking Kate and her twin Megan. “You can’t accept there is a treatment and say, no, we’ll hold off. How long will it take to get this drug approved [in Scotland]? I don’t know whether it is going to be five months or five years.”
It is just the latest dilemma to hit the SMC, which is charged with appraising all newly licensed medicines and advising NHS boards on their clinical and cost-effectiveness.
Late last year, the body rejected the life-extending ovarian cancer drug Avastin for routine use, on the grounds it was not value for money.
Also last year, it reversed a previous rejection of the prostate cancer drug abiraterone – said to have helped keep Lockerbie bomber Abdelbaset al-Megrahi alive after he returned to Libya.
Previously, it had declared the £3,000-a-month cost did not justify the health benefits.
Ministers say the SMC process is world-leading. The agency was unavailable for comment on the Kalydeco decision, but health secretary Alex Neil has asked for a review of the SMC’s current systems to ensure “consistency of approach” and to see whether “further improvements” can be made.
Campaigners want him, if necessary, to go against precedent and overrule any decision to block a drug which in America, Germany, Holland, France and England is now showing spectacular results.
Ferguson says: “In the event of a ‘no’ decision on Monday, which is very probable, I am calling, on behalf of the cystic fibrosis G551D patients in Scotland, for Alex Neil to do something extraordinary and provide parity with England in Scotland.
“It is simply not good enough to allow the lives of children and young people in Scotland to deteriorate whilst the lives of their fellow sufferers in England are being transformed by receiving this remarkable new medicine.”