Repatha belongs to a potent new class of drugs known as PCSK9 inhibitors that are extremely effective at lowering “bad” LDL cholesterol.

But the National Institute for Health and Care Excellence (NICE) cost watchdog said on Wednesday the question as to whether this would reduce heart attacks and strokes remained unanswered, given a lack of long-term clinical outcomes studies.

NICE, which decides if treatments should be paid for on the National Health Service (NHS), also said in its draft guidance that analyses presented by Amgen had limitations that “called into question the reliability of the cost-effectiveness results”.

Amgen said it was disappointed by the decision, since the evidence “strongly indicates that high cholesterol is linked to heart attacks and strokes”, and it aimed to provide more data and analyses to demonstrate the cost-effectiveness of Repatha.

The British list price for Repatha is 4,448.60 pounds ($6,769) for a year’s treatment, which is less than half the U.S. list price of more than $14,000. NICE said Amgen had also offered the NHS a further undisclosed discount.

Regeneron Pharmaceuticals and Sanofi have a rival product called Praluent that has not yet been assessed by NICE.

The high price of PCSK9s has already stirred controversy in the United States, where Express Scripts, the largest manager of prescription drug plans for U.S. employers and health plans, recently reached discount deals for the medicines.


(Reporting by Ben Hirschler, editing by Louise Heavens and David Evans)

Source: Reuters Health