Patients suffering from rare conditions to get vital treatment after ‘wonder-drug’ fund approved

Cystic Fibrosis drug  Kalydeco will be available under the fund
Cystic Fibrosis drug Kalydeco will be available under the fund
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THE Scottish Government has announced it has found extra funds to pay for the cost of medicines of rare conditions, in a bid to dampen a fierce row over the lack of access to a “wonder drug” which helps sufferers of a rare form of cystic fibrosis.

• Drug treatement to be allocated on case-by-case basis

• Cystic fibrosis drug Kalydeco to be included in scheme

In a timed announcement this afternoon, Health Secretary Alex Neil revealed he would put £21m extra cash towards the new Fund which will pay for so-called “orphan drugs” which affect fewer than 1 in 2,000 people.

It came as the Scottish Medicines Consortium announced that a new drug for a special “Celtic” form of cystic fibrosis known as Kalydeco would not be sanctioned for use across the NHS in Scotland, despite the fact it has been approved in England.

A Scottish Government spokeswoman said that Kalydeco would be one of the drugs that could be applied for under the new Fund. It costs £182,000 per patient per year to fund.

Parents and campaigners had lobbied intensively for the drug to be used, pointing to its proven ability to arrest the debilitating symptoms of the G551D strain of the disease, which affects as many as 80 people across the country.

Proportionally, double the number of cystic fibrosis patients suffer from the strain compared to those in England, where health chiefs backed the drug’s use at the turn of the year.

Parents said they were unconvinced by the announcement from the Scottish Government which states that the new fund will only pay for drugs on a case-by-case basis.

The announcement also made no specific mention of Kalydeco. But a Scottish Government spokeswoman said that Kalydeco would be among the drugs which will be considered by the new £21m Fund.

Deserving patients

But one parent, Moira Anderson, said: “Parents are now faced with having to say that my child is more deserving than so and so. How can we accept that?”

Another parent, Jonny Gordon, whose eight year old daughter Kate is recovering from pneumonia caused by the disease, said: “We read this announcement with cautious optimism. What Alex Neil has done today is side step the question by finding this money. There’s nothing to say that this money will be spent and nothing to say that we will get access to the drug. It will still be on a case-by-case basis.

“It is a step closer to what we want but we are still being treated differently to people in England and Northern Ireland,” he added.

Only those medicines not recommended for routine use by the Scottish Medicines Consortium will be covered by the fund and will be available from March this year.

Health Secretary Alex Neil said: “It is only right that Scottish patients with rare conditions have access to innovative medicines which are clinically justified and that they are not disadvantaged due to the very high cost of these treatments.”

He added: “We recognise that the Scottish Medicines Consortium is globally respected and has the fastest and most efficient medicine review process anywhere in the UK, but that for some individual patients with rare conditions there is a need for further support.”

Requests to the fund will be assessed on a case by case basis, based on the clinical circumstances of individual patients.

Ed Owen, the Cystic Fibrosis Trust’s chief executive said: “We warmly welcome the decision by the Scottish Executive to respond to today’s SMC’s decision by making additional funding available to enable Kalydeco to be made available to those that need it.”

He added: “We now appeal to Vertex, the manufacturers of Kalydeco and the NHS inScotland to work together to reach a fair and affordable solution as soon as possible. The CF Trust has campaigned for all people with the G551D mutation of cystic fibrosis who are eligible to receive Kalydeco should be able to receive it as soon as possible.”

“The Trust will be looking for further clarification about the process going forward, including how soon a consultant can make an individual treatment request so that patients have access to Kalydeco as soon as possible after 1 March 2013,” he went on.