A new drug to treat a rare inherited bone disorder from Alexion Pharmaceuticals ALXN.O that costs more than $550,000 a year for each patient has been deemed too expensive by Britain’s health cost watchdog NICE.

The case highlights the sky-high price of some medicines for treating rare diseases and the dilemma this poses for cash-strapped healthcare providers.

The National Institute for Health and Care Excellence (NICE) does not dispute that Alexion’s Strensiq is effective for the handful of patients, estimated at around seven a year in England, diagnosed with pediatric-onset hypophosphatasia.

But NICE said on Wednesday it was not satisfied that Alexion had provided an adequate justification for the drug’s high price of 366,912 pounds ($552,000) per patient a year, assuming an average weight of 19.3 kg.

Left untreated, hypophosphatasia causes weakening of bones and can lead to chronic debilitating pain, muscle weakness, generalized seizures because of vitamin B6 deficiency, as well as renal and respiratory complications.

The NICE decision is still at the draft stage and is open to a period of consultation.


(Reporting by Ben Hirschler; editing by David Clarke)

Source: Reuters Health