British watchdog wants U.S. biotech Alexion to justify cost of drug

LONDON, March 4 (Reuters) - Britain's healthcare costs watchdog has asked U.S. biotech group Alexion Pharmaceuticals (NasdaqGS: ALXN - news) to explain the high price for its Soliris drug before deciding whether the treatment should be paid for by the state health service.

Funding Soliris for the around 200 patients in England it could benefit would cost an estimated 58 million pounds ($97 million) in the first year, rising to 80 million pounds a year in five years time, the National Institute for Health and Care Excellence (NICE (Milan: NICE.MI - news) ) said.

Soliris is designed to treat two extremely rare chronic genetic conditions that can damage vital organs including the kidneys, heart and brain and has a U.S. list price of about $440,000 per patient a year.

"Drugs for very rare conditions that affect just a few people in the country are inevitably more expensive than for more common diseases," said NICE's chief executive Andrew Dillon. "However, before we can make a confident recommendation for routine use (of Soliris) ... we need more information."

He said that while Soliris is recognised as an effective drug, NICE's independent advisory committee wants more information from Alexion "on aspects of the manufacturing, research and development costs" of a rare disease medicine.

The committee had also asked the country's taxpayer-funded National Health Service for more general information about the costs of highly specialised treatments, he said.

Alexion criticised NICE for further delaying a decision, but said it would continue to work with the costs body.

Sales of Soliris are forecast by analysts to reach $3.1 billion a year by 2017.

NICE is evaluating the cost effectiveness of Soliris' use in a condition called atypical Haemolytic Uraemic Syndrome (aHUS) that causes severe inflammation of blood vessels and the formation of blood clots in small blood vessels.

The drug, given intravenously as initial treatment at a dose of 900 milligrams (mg) for four weeks, and then for maintenance at a higher dose, but with treatments only every 12 to 16 weeks, costs 3,150 pounds per 30 millilitre vial in Britain, giving a treatment cost of 340,200 pounds per adult patient in the first year, according to NICE.

The summary product characteristics for the drug also states that "treatment is recommended to continue for a patient's lifetime, unless discontinuation of treatment is clinically indicated".