Patients win drug funding victory

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Patients with a rare but debilitating blood disorder have welcomed a Department of Health decision to fund their expensive treatment, centrally.

Research found the drug Eculizumab (Solaris) was highly effective in treating paroxysmal nocturnal haemoglobinuria (PNH).

But there were fears that local NHS bodies would be unable to foot the bill of £245,700 per patient.

Medics said the decision would greatly improve people's quality of life.

Breathing difficulties

PNH affects between 8,000 and 10,000 people across North America and western Europe.

Caused by a mutation in blood stem cells, it is often characterised by dark red urine. Symptoms include anaemia, fatigue and difficulty breathing and swallowing.

It is not until you have the drug that you realise how much better life is with it Ruth Bell <a class="" href="/1/hi/health/7609962.stm">Patient's delight at decsion</a>

Before she took part in a trial of PNH, Ruth Bell from London needed blood transfusions every three months to keep her condition under control.

She said she was delighted by the department's decision.

"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."

About 40 patients were enrolled on the trial, which started six years ago.

But when it ended in 2007, few primary care trusts were prepared to fund the drug, claiming it was not cost-effective.

Because the condition affects fewer than 20 patients per million it was not deemed suitable for consideration by NICE - the National Institute for Clinical Excellence.

It was then referred to the Department of Health which has now said it will fund the drug centrally for all UK patients.

A spokesman said: "From 1 April 2009, the PNH service will be nationally commissioned for clinically eligible patients."

Dr Modupe Elebute, a consultant haematologist at King's College Hospital, London, said the decision would be a big boost for patients.

'Monitored'

"We are delighted with this decision to provide funding for this novel therapy and to establish a nationally commissioned service for PNH patients at King's College Hospital London and Leeds General Infirmary.

"It will ensure that the drug is prescribed and monitored by specialists in the field."

But Dr Jonathan Howell, a consultant in public health at the West Midlands Specialised Commissioning Team said, although the PCTs would not pay directly, there would still be a knock-on cost, as money would be top-sliced from their budgets so each PCT would ultimately get less.

He added: "PCTs do not see this money, but it is less money than they would otherwise have had in their overall budgets to fund new local developments.

"PNH patients will clearly benefit but at the expense of patients elsewhere, where larger numbers might have had more benefit with better value treatments."