It is one of most expensive medicines in the world and goes to heart of the debate about the cost of health care in a civilised society.

But one of the few people in Scotland to be treated with eculizumab says the medication has saved her life and hopes others will be given the same access to the drug she has had.

Joyce Juszczak only received treatment for a life-threatening blood condition after she suffered a potentially fatal blood clot.

After the Herald highlighted her plight, the 67-year-old, appeared at the Scottish Parliament with her daughters to press for access to the drug for paroxysmal nocturnal haemoglobinuria (PNH). She had earlier failed in a series of appeals over six months, including a review of her case after an intervention from then Health Secretary Nicola Sturgeon.

She has now had the eculizumab drug for three years, even though it remains not recommended for use within the NHS Scotland and she says it has worked wonders.

She says: "I am doing fine now. I get the drug every second week and it has certainly worked for me, without a doubt. I wouldn't be here at all if I hadn't got it.

"I still get very tired at times, and if I do too much I have to lie down, but that seems to be part of it all so I can cope with that one."

The family had been angry that it took a life-threatening incident to get the health board to change its mind over the life-saving treatment for PNH, a potentially deadly condition which causes red blood cells to explode and leaves victims at risk of kidney failure and potentially fatal blood clots.

Patient groups, used Mrs Juszczak's case to highlight how Scotland is failing more than 350,000 people affected by a rare disease.

Her first refusal came just weeks after another patient William Devine died shortly after receiving his rejection letter.

Her case sparked a successful campaign for greater access to drugs for rare diseases in Scotland.

And now despite remaining on the 'not recommended' list for the NHS as far as the value for money assessor Scottish Medicines Consortium is concerned it is available through a £40 million New Medicines Fund launched in October.

In September the drug, which is marketed under the name Soliris, was recommended by NICE, the National Institute for Health and Care Excellence for use in the NHS in England and Wales. It was a u-turn on previous guidance.

NICE estimates that Soliris will cost the NHS south of the border up to £58 million in the first year, rising to £82million after five years, with an average patient cost of £330,000 per year.

A source for the PNH Alliance which looks after the interests of patients said: "The New Medicines Fund is essentially funding negative decisions of the SMC and where companies don't submit. It is a mess!"

Lesley Loeliger of PNH Scotland said the new the new drug fund should "in theory" ease the path for approvals of the drug.

The old IPTR (Individual Patient Treatment Request) system which Ms Juszczak had to use remains in place until the new PACS (Peer Approved Clinical System) method comes into force. There are no details of when that will happen, says Ms Loeliger

"There are patients still awaiting approval for eculizumab. They have not been turned down but the system takes a while so I don't know what the outcome will be. There have been a few approvals, though, over the last year so we are hopeful," she said.

The Scottish Government said it hoped to reduce reliance on individual requests for drugs through its changes in the approach to approving medicines.

It said it has raised the issue of a new submission for the eculizumab drug to SMC under the new process with Alexion, the pharmaceutical company which manufactures this drug and "would encourage patient groups to do the same".

A Scottish Government spokeswoman said it was reforming the Individual Patient Treatment Request system and have put in place interim arrangements moving to introduce the Peer Approved Clinical System (PACS) which would improve access.

"These interim arrangements have proved successful with hundreds more patients accessing new treatments across Scotland, and for this drug (eculizumab) the most recent data shows that all requests have been approved since changes to the system were made."