As of 2014 there was insufficient evidence to show that eculizumab therapy improves life expectancy for people with paroxysmal nocturnal hemoglobinuria, and the cost-effectiveness was poor.  In 2010 Alexion priced Soliris as the most expensive drug in the world, at approximately US$409,500 a year in the United States (2010), €430,000 per year for ongoing treatment in the UK, and $500,000 a year in Canada (2014).  Alexion started selling Soliris in 2008, making $295 million in 2007 with its stock price rising to 130% in 2010.

In December 2013, New Zealand's government pharmaceutical buyer Pharmac declined a proposal to subsidize the drug after Alexion refused to budge on a NZ$670,000 (US$590,000) per person per year price and Pharmac's economic analysis determined the price would need to be halved before the drug was cost-effective enough to subsidize. Pharmac's decision upset many people with PNH in New Zealand PNH, although Pharmac has not ruled out reviewing the decision at a later date, should the drug be made available at a lower price.

According to a 2014 report, the orphan drug market has become increasingly lucrative for a number of reasons. The cost of clinical trials for orphan drugs is substantially lower than for other diseases —trial sizes are naturally much smaller than for more common diseases that affect more people. Small clinical trials and little competition place these orphan agents at an advantage when they come up for regulatory review. Further reduction to the cost of development is because of the tax incentives in the Orphan Drug Act of 1983. On average the cost per person for orphan drugs is "six times that of non-orphan drugs, a clear indication of their pricing power". Although there are much smaller orphan disease populations, the cost of per-person outlays are the largest and are expected to increase with wider use of public subsidies.

In December 2014, the provincial government of Ontario, Canada negotiated the price with the manufacturer, the only drug approved by Health Canada to treat aHUS. People can apply for it on "compassionate grounds" "on a case-by-case basis for example individuals who have been urgently hospitalized due to an immediate life-, limb-, or organ-threatening complication." It then was already "funded by the Ontario government for the treatment of another rare illness, paroxysmal nocturnal hemoglobinuria (PNH), through a bulk-buy deal reached by the provincial premiers in 2011.

With Regards,
Nancy Ella
Managing Editor
Drug Designing: Open Access