Joyce Juszczak, 65, from Gourock, Inverclyde, was initially refused the “miracle drug” eculizumab for a rare and deadly condition that destroys red blood cells and leaves victims at risk of kidney failure and potentially fatal blood clots.
Despite Mrs Juszczak being severely weakened by the illness, her daughters Beverley Hardie and Marlyn Milne will provide the physical support she needs to allow her to go to the Scottish Parliament to hear Greenock and Inverclyde MSP Duncan McNeil question the Health Secretary about her case.
Campaigners hope her plight will help put pressure on the Scottish Government to end a north-south divide in treatment for rare diseases in Scotland.
Eculizumab would be available to Mrs Juszczak, who suffers from paroxysmal nocturnal haemoglobinuria (PNH), if she was living south of the Border.
Mrs Juszczak’s failed appeal to an NHS Greater Glasgow and Clyde (NHSGGC) panel to prescribe the drug, despite support from her medical consultants, was made just a matter of weeks after another patient, William Devine, died within days of receiving a similar rejection letter.
It comes after the family made a desperate plea through Mr McNeil to get Ms Sturgeon to personally intervene. Ms Sturgeon said she had asked health directorate officials to contact NHSGGC to investigate.
Consultants have now confirmed to the family that Mrs Juszczak’s symptoms have become more advanced, as she is bleeding more regularly, is left severely fatigued and has difficulty swallowing.
Mrs Juszczak, who has had to undergo eight blood transfusions in the past year and described herself as a “walking timebomb”, said: “I am hoping Ms Sturgeon will have a rethink. We hoped she would intervene, but she passed the buck and I was quite angry about that. I didn’t think I could go to Edinburgh in case I just didn’t feel too good, but my daughters managed to take some time off to help me.
“I am just very, very fatigued and up during the night in a lot of pain down my left side and down my back. Even when I am having a cup of tea, I have to stop to get my breath. It is quite frightening.”
Mrs Hardie added: “I just hope that Nicola Sturgeon will end the postcode lottery for healthcare and urge NHSGGC to allow mum access to this vital drug before it’s too late.
“I want to discuss the inequity of care provision across Scotland and the UK, and why they allow the Scottish Medicines Consortium to give advice to NHSGGC about rare diseases when it does not have the knowledge and expert advice required when dealing with such cases.”
The Scottish Medicines Consortium, which decides which newly licensed drugs represent good value, ruled against eculizumab in November last year, saying it has yet to be convinced of the cost-effectiveness of the £250,000-a-year drug. It is prescribed in England and Wales.
Mr McNeil said: “Mrs Juszczak and her family remind us all that this is about more than health outcomes and value for money, this is about real people and the quality of their lives.
“They are only asking for what anyone else in their position would want, and it seems only bureaucracy is preventing my constituent from receiving treatment that would improve and prolong her life.”
Dr Henry Hambley, the consultant haematologist who is continuing to fight Mrs Juszczak’s case in retirement, has written to Andrew Robertson, chairman of NHSGGC, expressing his “disappointment” at the health board’s decision.
Ms Sturgeon said: “I cannot comment on individual cases, but if Mrs Juszczak is at the parliament I will be more than happy to meet with her.”
She earlier told the family in response to Mr McNeil’s letter: “I am sorry to learn of the concerns raised on behalf of Mrs Juszczak. Please be assured that I appreciate the anxiety and frustration caused.”
She added: “Decisions regarding whether or not to fund ‘not recommended’ drugs on a ‘case by case’ basis for individual patients ... are a matter for each NHS Board. I hope therefore that you appreciate that it would be inappropriate for Scottish ministers to comment on individual cases.
“It is open to patients who have been refused access to eculizumab to pursue this directly with their NHS board.”
PNH affects one in a million, causes red blood cells to explode, leaves patients too exhausted for simple tasks and at risk of kidney failure and potentially fatal blood clots.
Experts say eculizumab is a potentially life-saving drug, obviating the need for regular blood transfusions in two-thirds of patients and virtually removing the risk of blood clots, the main killer.