Alexion Pharmaceuticals Inc. (ALXN) should provide information on research and manufacturing costs for its Soliris rare-disease drug to help justify a price of 340,200 pounds a year ($569,000), the U.K.’s health-cost agency said.
The drug, also known as eculizumab, is an effective treatment for a blood disorder known as atypical hemolytic uremic syndrome, the National Institute for Health and Care Excellence said in an e-mailed statement. Still, the product will cost the National Health Service about 58 million pounds in the first year of treatment, rising to more than 80 million pounds in five years, the institute said.
“Before we can make a confident recommendation for routine use, we need more information,” said Andrew Dillon, NICE’s chief executive. The agency asked for “clarification from the company on aspects of the manufacturing, research and development costs” of Soliris, he said.
The request shows the pressure governments are putting on drugmakers to justify the rising costs of medicines for rare illnesses. The Netherlands last year demanded reduced prices for enzyme-replacement therapies, while Ireland won a reduction in the price of Vertex Pharmaceuticals Inc.’s Kalydeco for cystic fibrosis.
Alexion, whose main product is Soliris, wants information about the cost of the drug to be kept confidential, NICE said. “We’re disappointed about this decision, for which we have not had an adequate explanation,” the agency said.
“Alexion has proactively provided confidential proprietary commercial information to NICE and NICE has had Alexion’s information to evaluate over many months now,” Alexion said in an e-mailed statement. “As is standard industry practice, Alexion has requested that NICE can use, but not disclose, this confidential information.”
Soliris had sales of $1.55 billion last year, according to the Cheshire, Connecticut-based company. Alexion shares have almost doubled in the last 12 months, valuing the company at about $34.1 billion.
NICE conducts cost-benefit analyses and then advises the NHS, the U.K.’s state-run health system, on which medicines it should pay for. The NHS is providing Soliris on an interim basis while awaiting the outcome of NICE’s review.
The drug will cost about 340,200 pounds per adult patient in the first year of treatment, and 327,600 pounds a year thereafter for maintenance treatment, NICE said. The agency’s estimates of the total cost are based on 170 patients treated in the first year. Soliris would typically be taken for life, NICE said.
Most people with atypical hemolytic uremic syndrome develop kidney failure requiring dialysis and transplantation, NICE said. Plasma infusions or exchanges were the main treatment before Soliris, though not all patients responded to plasma therapy, the agency said.
Soliris produced gains in patient quality of life “of a magnitude that is rarely seen for any new drug treatment,” NICE said. The U.K. agency hasn’t sought any additional information about the medicine’s effectiveness, Alexion said.
Alexion, health workers, patient groups and the public have until March 25 to comment on Soliris, the agency said. The agency will issue draft guidance after NICE’s Evaluation Committee considers the information provided by Alexion at its next meeting in April.
“Alexion is concerned that nearly three years after the government commenced its evaluation, and more than one year after eculizumab was referred to NICE, the committee was still unable to provide a formal recommendation on whether eculizumab should be nationally commissioned,” Alexion said.
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