HEALTH managers have told a retired barmaid they will not reconsider their refusal to give her potentially life-saving NHS treatment for a rare blood dis-order despite admitting the medication would be "clinically effective".

Joyce Juszczak, 65, from Gourock, was initially refused the 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.

Her unsuccessful appeal to an NHS Greater Glasgow and Clyde (NHSGGC) panel was made just a matter of weeks after another patient, William Devine, died within days of receiving a similar rejection letter.

Her case prompted patient groups to call for the Scottish Government to end what they say is a postcode lottery in health provision, as the drug would be available to Mrs Juszczak, who suffers from paroxysmal nocturnal haemoglobinuria (PNH), if she was living south of the Border.

However, the PNH Alliance pressure group, which wrote to Health Secretary Nicola Sturgeon to ask her to intervene, told The Herald she has declined a meeting.

Dr Henry Hambley, former consultant haematologist at Inverclyde Royal Hospital, who tried in vain on his last day before retirement to get Mrs Juszczak the drug for the stem-cell disorder, has continued to fight for the pensioner in his retirement.

However, Andrew Robertson, chairman of NHS Greater Glasgow and Clyde, has told Mr Hambley that it will not revisit her case although he acknowledged “the variance in approach across the home countries and the uncertainty this may create for patients with the condition”.

The Scottish Medicines Consortium (SMC), which decides which newly-licensed drugs represent good value, ruled against eculizumab in November last year, saying it is yet to be convinced of the cost-effectiveness of the £250,000-a-year drug.

And Mr Robertson told Mr Hambley: “It is clear that eculizumab is clinically effective as defined through its licence approval by the medicine regulatory authorities. However, it has not been established that this medicine is cost-effective as defined by the SMC.”

He said he was advised the assessment of the medicine was “unusual” because the manufacturer did not supply any health economic analysis.

“An independent economic analysis was, therefore, commissioned which was unable to demonstrate cost-effectiveness,” said Mr Robertson.

“I recognise that your patient has been assessed as a candidate for eculizumab by national specialists from England and by a consultant haematologist based in Lanarkshire NHS Board.

“It is regrettable that expectations were raised and she was told she fulfils the criteria for acceptance for treatment without reference to the qualifications which apply within NHS Scotland, the host authority.”

He said a devolved health service “inevitably leads to differing priorities”.

“Clearly if your patient’s clinical circumstances change, or there is a significant change in national guidance, it may be possible to re-examine the case. However, in the absence of such change, I regret that it will not be justifiable to re-examine the case.”

Patient groups say Scotland is failing more than 350,000 people who will be affected by a rare disease. They have demanded an advisory group to deal with specialist medication, similar to that in England and Wales, is established in Scotland.

Dr Hambley, who revealed another elderly patient had been refused eculizumab for PNH and died of an unrelated lung cancer, had told the health board to “think outside the box” in relation to Mrs Juszczak’s case.

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