Page last updated at 00:02 GMT, Sunday, 2 November 2008

'I was living my life in limbo'

By Jane Elliott
Health reporter, BBC News

Ruth Bell
Ruth needed blood transfusions every three months

Three years ago Ruth Bell signed up to a drugs trial which transformed her life.

She has a rare, chronic disorder which means her red blood cells are weak and destroyed more rapidly than normal.

Before the trial she needed blood transfusions every three months to keep her alive and suffered from jaundice, blood in the urine, stomach pains, difficulty swallowing and tiredness.

The drug Eculizumab (Soliris), made by the pharmaceutical company Alexion, relieved the symptoms of her disorder - paroxysmal nocturnal haemoglobinuria (PNH) - as well as ending her need for transfusions.

High drug costs

But at a cost of 245,700 per patient a year the drug, which had to be given every two weeks by intravenous drip, was not considered cost effective by many primary care trusts.

PNH is an extremely rare condition, affecting between 8,000 and 10,000 people in North America and western Europe.

Because it affects so few, it does not reach the threshold for evaluation by the NHS treatments watchdog NICE, the National Institute for health and Clinical Excellence, so it was referred to the Department of Health for a decision.

Ministers have now given their approval and after years of funding uncertainty, Ruth, the other trial patients and others who need the drug have been told that, from next April, central NHS funds will pay for it.

When the trial of the drug ended, last year, most of the 40 patients on it had their treatment funded by the drug company, with only three funded by the NHS.

It is like giving someone the freedom for a better life and then saying back in prison
Ruth Bell

But Ruth had worried this would not continue for ever, and pinned her hopes on the drug receiving public funding.

'A glow about me'

"This is great news. This drug gave me my life back.

"It is not until you have the drug that you realise how much better life is with it.

"We all just assumed it would be funded and thought there was no way they could take it from us. But we had this uncertainty.

"There was no way we wanted to go back to the life we had before."

Ruth Bell
Ruth feared the return of her symptoms

Ruth, aged 48, from London, was diagnosed with PNH at the age of 21 and says she had a lot of distressing symptoms.

"I had a low haemoglobin and, being an active person, I had to constantly monitor when I was going to feel good.

"I used to get jaundice quite a bit and a lot of blood in my urine when I was stressed or had an infection.

"I used to be quite ill and have palpitations and generally feel quite low and depressed when my haemoglobin was low."

But Ruth, who works for a teaching recruitment firm, said: "Since I went on the trial I have felt absolutely different.

"I have had no blood in my urine. I have a very stable haemoglobin level and, when I go to see my mother in Australia she says that I now have a glow about me."

Quality of life

Dr Modupe Elebute, a consultant haematologist at King's College Hospital, London, said stopping the drug would have meant a significant reduction of quality of life for patients on the trial.

"Once treatment is withdrawn the patients immediately start breaking down their red cells at the previous rate and will return to requiring regular blood transfusions for chronic anaemia and fatigue.

"In addition, other symptoms will return resulting in rapid reduction in their quality of life."

Dr Elebute, who was the principal investigator for the drug trials in London, said the results at her centre had been dramatic.

"In our centre all of the eight patients had a very significant reduction in the number of transfusions they need and six have not needed any transfusions at all since starting the drug."

"This decision will make such a difference to patients."

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