NICE gives limited blessing to Effient

Eli Lilly's new blood thinner Effient got the nod from the U.K.'s National Institute for Health and Clinical Excellence. Good, right? Yes, to a point. The cost-effectiveness gatekeeper decides whether the National Health Service will pay for specific treatments, and its approval often sways other governments' buying decisions.

But NICE gave Lilly's anti-clotting med a less-than-sweeping recommendation. The watchdog group said prasugrel, sold as Effient in Europe and Effient in the U.S., should only be used in certain higher-risk patients undergoing percutaneous coronary intervention, a procedure to clear blocked arteries. Namely, in patients who need the procedure on an emergency basis--when there's no time for a loading dose of the current standard-of-treatment drug Plavix--and in those who either have diabetes or have had a stent thrombosis while using Plavix.

NICE's concerns? Partly Effient's risk of excessive bleeding, including fatal bleeding. And partly the fact that the trial submitted in support of the new drug didn't conform to standards of treatment in England and Wales. Namely, in the Triton trial, patients assigned to the Plavix group didn't get a pre-procedure loading dose, and they got half the dose generally used in the U.K., 300 mg as opposed to 600 mg. So, Effient's superiority in the trial could have stemmed at least partly from those treatment differences.

"[I]n most aspects the design of the trial favored prasugrel," NICE's analysis states. "[T]he Committee agreed that there was considerable uncertainty about whether prasugrel was clinically superior to clopidogrel (Plavix) in terms of net clinical benefit." But NICE did see greater benefits from Effient in particular patient groups. Hence the limited recommendation.

- read the statement from NICE (pdf)
- see the Reuters story

ALSO: An FDA official explains the rationale behind agency approval of Effient (prasugrel) in a commentary in the latest New England Journal of Medicine. Article