NICE Recommends Soliris for Rare Kidney Disease

The UK’s health cost watchdog has recommended Alexion’s Soliris for the treatment of patients with atypical hemolytic uremic syndrome, a life-threatening disease that causes abnormal blood clots to form in small vessels in the kidneys.

The final guidance is the first produced under the National Institute for Health and Care Excellence’s highly specialized technologies program, which looks at treatments for very rare diseases. Atypical hemolytic uremic syndrome affects only about 200 patients in the UK.

According to NICE’s assessment, Soliris (eculizumab) has the potential to add up to 24 years to patients’ life expectancy.  But its cost — about $560,000 per patient per year — gave the agency pause, because it is far higher than many other drugs treating very small patient populations.

NICE expects Soliris to cost the National Health Service up to $95 million in the first year alone.

To allay these concerns, NICE is asking Alexion to provide it with the following:

• Any clinical or safety requirements during Soliris’ development that might justify the higher cost compared with other treatments for small populations;
• Postmarket research plans, and their costs, for the treatment of atypical hemolytic uremic syndrome and for other indications, such as paroxysmal nocturnal hemoglubinuria;
• An explanation of the relationship between Soliris’ development costs and the price being proposed for the NHS;
• Any additional information that will help the evaluators reach a conclusion on whether the incremental therapeutic improvement over standard therapy justifies the proposed cost of Soliris.

Alexion did not return a request for comment by press time.

View the guidance at www.nice.org.uk/guidance/hst1/chapter/1-guidance. — Lena Freund