A PATIENT refused a life- transforming drug by Scotland's largest health board despite a plea from a top clinician has died within days of receiving the rejection letter.
William Devine’s plight was featured in The Herald last month, when we reported how he was told he could not have a treatment for the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH), at the same time as other patients were receiving the drug eculizumab.
His consultant Professor Peter Hillmen said, in an email to Mr Devine’s son, that if he had been prescribed eculizumab even a few weeks before his death, the 68-year-old would probably still be alive today.
The body which approves new treatments for use by NHS Scotland, the Scottish Medicines Consortium, rejected eculizumab last year – leaving health boards to make up their own minds whether to treat individual cases of PNH, a disorder which causes red blood cells to explode.
NHS Greater Glasgow and Clyde refused Mr Devine’s application for the drug last November. An appeal hearing was held on Wednesday, February 23.
In a letter supporting his case, Mr Hillmen told the board Mr Devine had stage four kidney failure and it was critical he started treatment sooner rather than later.
At the hearing Mr Devine, a retired draughtsman from Newton Mearns, asked the panel how long he would have to live if they did not allow him to have the drug. As it turned out, he had seven weeks.
Mr Devine was taken into hospital last month, but he died within hours from pulmonary oedema – fluid on the lungs.
In the email to Mr Devine’s son, Doug, Mr Hillmen says: “I would say that his PNH definitely contributed to his death (if it was due to pulmonary oedema) and that eculizumab if started some time before (even a few weeks) would have reduced the risk of this happening and would probably have stopped it all together.”
Doug Devine, who fought on his father’s behalf, said it would stay with him forever that he had been unable to secure the treatment doctors felt his father deserved. He added: “I am just completely devastated that this has happened and it could so easily have been avoided.”
He said living with PNH, which causes extreme exhaustion, and knowing there was a potentially effective treatment that other patients were receiving made the last year of his father’s life much harder.
He said: “They need to review the process because I would hate for anyone to go through what he went through and what I went through and to get this end result. It is heart-breaking.”
Mr Hillmen, consultant haematologist and expert on PNH who leads the only Scottish clinic for patients with the condition, said he and his staff were extremely upset by Mr Devine’s death and it re-emphasised the need to reconsider the way Scotland deals with treatments for rare diseases.
In England there is a special body which considers treatments for rare conditions, which can carry a hefty price tag. It has backed the use of eculizumab, which costs around £250,000-a-year, in patients who are severely ill with PNH as long as they meet certain criteria.
According to Mr Hillmen, the only health authority in the UK that has refused to fund the treatment for patients who are that ill is NHS GGC. It is understood the board has turned down another sufferer as well as Mr Devine.
Lesley Loeliger, a PNH sufferer living in Glasgow whose life was transformed after the health board agreed to let her have the drug, is setting up a support group for patients to campaign for equal access to treatment.
She said last month she felt “heartbroken” after meeting Mr Devine and listening to the details of his situation. After hearing about his death, she told The Herald: “I am devastated that we did not meet soon enough to try and get the medication for Mr Devine and to raise awareness in time.
“The work of PNH Scotland is to try to highlight the issue of funding and to stop anyone else suffering the way he did.”
Dr Brian Cowan, medical director for NHS GGC, said: “Our thoughts and sympathies are with Mr Devine’s family at this very difficult and distressing time.
“With regards to the prescribing of eculizumab, NHS GGC has clearly stated this treatment is not recommended by SMC and therefore each case has to be carefully considered on an individual basis.
“In reaching these decisions, we follow guidelines set out by the Scottish Government, we consider the SMC advice and we examine the individual patient circumstances.
“There were very sound clinical reasons why this patient was not prescribed the drug.”
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