NONE of us should have to face the experience that Lesley Stephen went through. Diagnosed with incurable breast cancer, she faced a battle not only to stay alive but also to obtain a drug which could extend her life by months, if not years. Her world collapsed, though, when the NHS told her that she would not be getting the drug - as it wasn't approved in Scotland.

Devastated, she spent thousands of pounds of her life-savings to access the drug. “It is hard enough physically and emotionally to deal with the cancer," she says, "but knowing there is a drug that could be a real wonder drug for you but you can’t get hold of it unless you have the money is just awful.”

Stephen is just one of many acutely ill patients in Scotland fighting to get life-extending drugs on the NHS despite reforms to the system for approving new medicines, according to damning new claims by campaigners.

One breast cancer charity warned there was “frustration and anger” over not being able to access new life-extending drugs for the disease, with some patients even having to seek private care abroad or paying out of their own pocket rather than accept nothing can be done.

Hundreds of cystic fibrosis patients north of the border have also been unable to obtain a new treatment which can increase lung function and reduce hospital admissions after it was rejected for use by the NHS.

Charities have also called for further transparency over how a £90m Scottish government fund is used to pay for end of life treatments.

A series of submissions made to an independent review of the way drugs are assessed for use by the health service have also raised concerns over patients with rare and terminal conditions being denied the latest treatments on the NHS.

The Scottish Medicines Consortium (SMC) is responsible for assessing which drugs should be available on the NHS in Scotland, with a group of experts which weigh up factors such as how well each new medicine works and how cost-effective it is.

Former NHS Fife medical director Dr Brian Montgomery is currently leading a review of reforms made to the SMC process two years ago, which were aimed at improving patient access to medicines for rare and end-of-life conditions.

The report, which is due to be sent to ministers soon, is also looking more generally at how the system for getting patients access to new drugs is working.

However submissions sent in by charities, which have been obtained by the Sunday Herald, have raised concerns that more needs to be done.

Lawrence Cowan, policy and campaigns manager at Breast Cancer Now, said: “At the moment in the NHS there is a drive to be ‘person-centred’ in the way patients are cared for. But we also need a medicines approval system which is person-centred.

“Patients want the service and the medicines approval system to fight tooth and nail (for them), whereas at the moment they are having to fight personally to get access to these drugs.”

Some ways in which the system could be improved which Breast Cancer Now has suggested, include allowing patients and clinicians more opportunity to take part in question and answer sessions at meetings where it is decided which drugs will be made available on the NHS. Currently, their views can be submitted, but are only read out.

The charity also called for more flexibility in the system, to allow “proactive” negotiation between the SMC and manufacturers over issues such as cost-effectiveness.

“Everyone realises that cost is an issue, but let’s try and resolve that issue,” Cowan said. “That is where the reforms need to go: trying to get everyone round the table to get a deal that works for the NHS and for manufacturers - but most of all for patients, as they are the ones in the middle of all this.”

The NHS, of course, does not have unlimited funds. Gregor McNie, Cancer Research UK's senior public affairs manager in Scotland, pointed out there had been improvements to the system and it should not be one which “says yes to everything”.

But he added: “It is about making sure we are far more informed about making those ‘no’ decisions more confidently when we do.”

One of the improvements Cancer Research UK has called for is the introduction of a “pause” in the negotiation process, to allow the SMC and industry to have conversations at an early stage about any problems which might prevent a drug from being approved. It does, however, note that this could lengthen the time taken to assess drugs.

McNie said the increasing development of personalised medicines – where drugs are targeted at individuals depending on their genetic makeup – was one of the biggest challenges facing the medicines approval system, which has to assess how best limited NHS funds can be spent to benefit most patients.

He said: “Previously you might have had a lung cancer drug, for example, which is regarded as not being very good, as it will only work for some patients and not others.

“We now have a far better understanding of genes and will know the drug shouldn’t be given to the majority of patients, but there is a small sub-group it works for.

“That means patient populations are getting far smaller – there are three lung cancer drugs in the pipeline that have Scottish patient populations for each of them of something like 50, 20, and 10. That is all positive as we are giving drugs to the right patients.

"But there is a challenge about the size of Scotland and whether there is a more UK-wide or more European-wide ways of assessing these personalised medicines needs to be debated.”

Other concerns raised include the need for more transparency over how the Scottish Government’s £90million New Medicines Fund, which pays for treatments for patients with rare or end-of-life conditions, is being spent.

A submission to the Montgomery review by charity Prostate Cancer UK said: “There is no information currently publicly available that indicates how this money is being used or what the money is being spent on.

"There would be value in Scottish Government sharing how the New Medicines Fund has been used so far and the extent to which it has improved access to new medicines.”

The Cystic Fibrosis Trust pointed to the example of a new drug called Orkambi, which is targeted at the most common genetic mutation which causes cystic fibrosis and can help improve lung function and reduce infections.

It said around 300 people in Scotland could benefit from the drug – but it was rejected by the SMC earlier this year.

With debate over the longer-term impact of the treatment, the charity said one solution could be to make the treatment available for two years and measure its effectiveness - similar to an approach which has been taken in Germany and France.

A spokesman for the SMC said it was committed to continually reviewing changes to increase transparency, give patients and their representatives a greater role and increase access to new medicines, but added negotiation on price was not part of the assessment process.

He said: “We recognise decisions not to recommend medicines will be disappointing for patients and carers. However, the SMC’s job is to decide whether a medicine is clinically effective and offers value for money to NHS and the people of Scotland. NHS funds are not unlimited.

"When making decisions the committee has to consider not only the patients that will be treated with that medicine, but every patient NHS Scotland treats. If SMC were to accept for routine use a medicine whose benefits are not clear, it might mean that patients with other conditions would lose out.”

A Scottish Government spokeswoman said there had been significant reforms and investment to improve access to new medicines in recent years.

She added: “As a result Scotland is now one of the top nations in the world for accessing new medicines for cancer. We do think that more can be done to build on the progress made to date and that is why the Cabinet Secretary has asked Dr Montgomery to conduct an independent review on the access to new medicines.

“This review is making progress and has already heard the views of a range of stakeholders - a report is due to the Cabinet Secretary later in the summer.

“The overarching aim of the review is to ensure there are robust mechanisms in Scotland to provide safe and timely access to clinically effective medicines at a fair price.”

'I feel like I am hanging off the edge of a cliff by my fingertips'

When mother-of-four Lesley Stephen was diagnosed with incurable breast cancer in 2014, she was given some hope after learning a new life-extending drug was available which could potentially help her.

But the 50-year-old, who lives in Edinburgh, faced a battle to access the treatment, called Kadcyla, as it was rejected for NHS use in Scotland by the Scottish Medicines Consortium (SMC).

Stephen said: “Maybe I was in denial, but I wasn’t too worried when it was rejected (by the SMC). I thought somehow there will be a way of getting hold of it as it was available in England through their NHS Cancer Drug Fund.

“There are not that many women in Scotland who need the drug, so it is not like we are talking mega amounts of money for the NHS.”

Stephen initially had conventional chemotherapy treatment but in 2015 it was discovered the cancer had spread to her brain. An application to her health board to consider her for Kadcyla through a system known as an individual patient treatment request was rejected.

In the end, she used money left to her by her mother, who had recently died of cancer, and spent £13,000 on three cycles of the drug, with the hope the health board would change their decision if it was shown the treatment was successful.

She said: “It is awful. I didn’t cause this cancer myself, I have three children and a stepson that need me. I had heard stories of some women being on this drug for four years and going back to work.

“It is hard enough physically and emotionally to deal with the cancer, but knowing there is a drug that could be a real wonder drug for you but you can’t get hold of it unless you have the money is just awful.”

The drug did stabilise her condition, but she did not respond as well as hoped and she could not afford to continue to pay for it herself.

She has now managed to get on a clinical trial for a brand new treatment which is in the early stages of being developed which she says has been a “miracle drug” and allowed her a good quality of life.

Stephen said she wanted to see more consistency in the way the system for accessing new drugs is applied in Scotland, as there have been examples of other NHS boards funding Kadcyla for patients.

She said: “If I hadn’t had the chance to access Kadcyla, I would have always wondered could it have been the one that gave me an extra few years of life.

“You have to be quite proactive as a patient and a lot of patients just won’t even know it is out there and available.

“I sometimes say I feel like I am hanging off the edge of a cliff by my fingertips - for women like me with this disease, we don’t have lots of time.

“There needs to be real urgency about reviews and making decisions.”