Amgen's Repatha Gets Early Thumbs Down by Britain's NICE

The cholesterol drug evolocumab, being marketed by Amgen as Repatha, has been turned down by the British health service, which cited the lack of long-term cardiovascular data but also “called into question the reliability of cost-effectiveness results.”

The National Institute for Health and Care Excellence (NICE), which acts as cost watchdog, did not echo the broad approval given by European regulators, who acted ahead of the FDA. But NICE has a different mandate—it doesn’t decide on the efficacy and safety of drugs; it decides if the National Health Service should pay for them.

NICE’s ruling is preliminary; doctors and Amgen can comment until December 8, 2015, and a final ruling will come in early 2016. But it underscores the intense debate on both sides of the Atlantic over the cost of the new class of cholesterol drugs, called PCSK9 inhibitors. Sanofi and Regeneron’s entrant, alirocumab, being marketed as Praluent, and Repatha were both priced above $14,000 wholesale when approved in the United States; while cheaper in Europe, they are still very expensive.

The British list price for Repatha is 4448.60 pounds, or $6769 a year. Right now, both drugs are given in biweekly injections, although Repatha is expected to introduce a once-a-month version.

According to Bloomberg, NICE reported that Amgen “used an unrealistically high factor” to estimate the risk of cardiovascular events in the British population. Clinical trial results presented in the United States in 2014 showed that the drugs reduced low-density lipoprotein (LDL) or “bad” cholesterol by as much as 60%, far more than other available therapies.

The drug’s backers also saw it as an alternative to statins for those who cannot tolerate high doses of these drugs, but FDA did not approve either PCSK9 inhibitor for this purpose.

Some cardiologists have greeted the arrival of the drug class with enthusiasm for patients who have been unable to get LDL cholesterol under control. But health plans have been bracing for their arrival; some fear that if they replaced low-cost statins they could be the most expensive drug class in history.