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