Winners and losers in the drugs lottery

Hayleigh Reynolds, eight, is to receive a 100,000-a-year treatment for her rare degenerative condition after a U-turn by her health board.

At the same time, The Scotsman has learned that more than 1,000 multiple sclerosis (MS) patients are set to be denied a powerful new treatment as it is deemed not cost-effective by medicines watchdogs.

These contrasting situations highlight the difficult decisions being made by officials with limited NHS resources and for the patients whose lives are governed by them.

The Scottish Medicines Consortium (SMC) was set up in 2001 with the aim of ending postcode prescribing and making access to drugs fairer.

But doctors must face the daily heartache of having to tell patients they cannot have a certain drug because it is not approved for funding. In some cases, this leads to people selling their homes to fund their treatment privately.

Yesterday the Reynolds family received the good news that NHS Greater Glasgow and Clyde would fund the only treatment for the rare genetic disorder Hurler Scheie syndrome.

The drug laronidase is not approved by the SMC, but the family challenged the health board and have won their case.

While they were celebrating, patients whose MS keeps coming and going - known as relapsing remitting MS - are to be denied access to the drug Tysabri when the SMC announces its decision on Monday.

Tysabri - the first new treatment for the disease in the past ten years - is seen as one of the last hopes for patients with relapsing remitting MS who fail to respond to other therapies.

Patients will now face the prospect of either funding the treatment themselves or hoping their doctor will prescribe the drug anyway. The treatment costs 14,690 a year per patient, compared with about 8,000 for other current MS treatments.

Some 10,400 people in Scotland have MS, and more than 1,000 of these could benefit from Tysabri. There are more people with MS in Scotland per head of the population than anywhere else in the world.

At least two English patients are expected to start on Tysabri, paid for by the NHS down south, and more than 20 patients in Ireland are already receiving it.

Mark Hazelwood, director of the MS Society Scotland, said: "If what we are hearing is correct and the SMC does reject Tysabri, people with severe relapsing remitting MS will be denied a vital treatment option.

"In theory, Tysabri can still be prescribed if a health trust agrees to it, but in practice we know few neurologists and GPs will override SMC advice.

"A negative decision would be bitterly disappointing."

Across the UK, patients have fought long-running battles with health authorities to get access to drugs. The breast cancer therapy Herceptin has been at the centre of the most high-profile campaign and is now widely available.

Professor Jim Cassidy, from the Beatson Oncology Centre in Glasgow, said:

"Our cancer patients know about these drugs because they have read about them, seen them on the internet. They come to us and say, 'can we have this drug' and we have to say no, because it is not approved for funding on the NHS."

He said if patients asked if a treatment was available if they paid privately, he had to tell them that it was. "Then you get people selling their houses and borrowing money to pay for treatment," he said.

Sheila McLean, professor of medical ethics at Glasgow University, said rationing of treatments had always gone on in the health service, but bodies such the SMC and its English equivalent, the National Institute for Clinical Excellence, were now helping to make the process more fair and transparent.

Family celebrate change of heart over costly drug

WINNER

THE Reynolds family described it as the best news they could have hoped for.

Eight-year-old Hayleigh suffers from Hurler-Scheie syndrome - a degenerative disorder caused by an enzyme deficiency which causes organ damage and dwarfism, writes Lyndsay Moss.

Now, thanks to a change of heart by Greater Glasgow and Clyde Health board, she will receive the costly enzyme replacement therapy laronidase.

Hayleigh's father, Robert, 43, said: "This will change Hayleigh's life.

"Any parent would fight for treatment for their child if they knew it was out there." Hayleigh's mother, Heather, 38, said their daughter may even be able to start the treatment before Christmas.

With a price tag of 100,000 a year and no approval from the Scottish Medicines Consortium, the health board had been reluctant to pay for the drug.

But following a legal challenge by the family, who live in the Barmulloch area of Glasgow, that decision was changed.

A health board spokeswoman said: "Following a detailed review, it has been recommended that laronidase should be made available in this case."

Christine Lavery, from the MPS Society, which campaigns for patients with such diseases, said she was "ecstatic" for Hayleigh's family.

But she said they should not have had to wait three years for treatment.

'What price can you put on quality of life?'

LOSER

FIONA Burns leads a full and active life, despite having been diagnosed with MS three years ago, on her 27th birthday.

She currently controls her condition with drugs, but she lives with the possibility that these may one day stop being effective. If that happens, she will have few other options.

So the decision to deny Tysabri on the NHS in Scotland causes the 30-year-old particular concern.

Ms Burns said she would be willing to pay for Tysabri herself when the time came and if the NHS would not fund it.

An account manager in Edinburgh, Ms Burns started off taking the drug Copaxone to control her condition, but this was ineffective.

She now takes beta interferon drugs, but she knows there is a chance these could one day stop working as well.

"Since I began taking them, I have got my life back and I hope that they will continue to work," she said.

"But I know that if it stops [working] there is not much else out there that will work.

"That is why the SMC's decision not to approve Tysabri is so disappointing.

"I would ask the SMC - what price can you put on quality of life? What price can you put on someone's dignity?

"Tysabri would be an amazing Christmas present for people with MS."