Life-saving drug costs four times US price

A drug used to treat a rare, life-threatening condition is prescribed to fewer than 80 patients in the UK and costs the NHS more than £6m a year - at least £75,000 each. But specialists say the enormous cost means a further 40 patients who could benefit from it are being denied treatment and most will die within 18 months.

A drug used to treat a rare, life-threatening condition is prescribed to fewer than 80 patients in the UK and costs the NHS more than £6m a year - at least £75,000 each. But specialists say the enormous cost means a further 40 patients who could benefit from it are being denied treatment and most will die within 18 months.

The drug, prostacyclin, is prescribed for a disease called primary pulmonary hypertension (PPH) which causes the arteries of the lung to become narrowed and blocked. It affects about six people per million in the UK.

The high cost was blamed yesterday on the refusal of the drug's manufacturers, GlaxoWellcome, to reduce the price to its level in the US and the failure of the Department of Health to organise central purchasing of the drug. Professor Tim Higenbottam, a specialist in respiratory medicine at the Royal Hallamshire Hospital, Sheffield, said: "Health authorities think it is too costly but the cost is high because they have no single system of purchasing. It's crazy."

The parents of one young patient who needed large doses costing £150,000 a year contacted their MP because they feared their health authority would be unable to pay. As a result of his intervention they succeeded in importing the British-made drug from the US, to which it is exported, at a quarter of the price that it is sold in the UK.

Nick Ainger, Labour MP for Carmarthen West and South Pembrokeshire, said: "The average cost of [a starting dose of] the drug in the UK is £37,000 a year compared with £9,000 a year in the US ... It is totally unacceptable that British people are dying because of the high price of a British-made drug."

Most patients with PPH are treated in four specialist centres in England and one in Scotland. But although the Scottish centre has official recognition and central funding, the four in England do not. As a result, patients requiring prostacyclin can only get it on an individual basis if their local health authority agrees to pay for it.

Prostacyclin is costly to manufacture because it oxidises quickly and has to be produced and stored under special conditions.

At least six studies in different countries have proved its efficacy in treating PPH, says Professor Higenbottam, but the Government's National Institute for Clinical Excellence has no plans to assess it because PPH is a rare condition.

GlaxoWellcome said the drug was not licensed for the treatment of PPH in the UK and its high price was based on short-term, occasional use in kidney patients, for which it is licensed. The company had applied for a licence for the treatment of PPH and in the meantime was offering the drug to hospitals at a lower cost on a named-patient basis.

In a statement, GlaxoWellcome said: "In the US drugs for the treatment of life-threatening conditions affecting small percentages of the population receive orphan drug status, through which the government provides financial support for the development of these drugs. This arrangement does not exist in the UK."