No need for 60%-plus discounts on Repatha, Amgen says, rolling out real-world data as backup

Amgen has rolled out a new economic analysis of Repatha’s outcomes study, Fourier, which initially failed to wow the market when unveiled earlier this year. The gist? The PCSK9 drug is cost-effective at $9,669 per year or less, almost $5,000 off its list price.

That’s a reduction of 31%—and it’s in line with typical discounts, Amgen says—but it’s still too pricey under previous assessments, including one published earlier this week in the Journal of the American Medical Association, which favored a price cut of 71%.

But this new study is different, Amgen says, because it includes real-world data, including rates of cardiovascular events outside of clinical trials. One of the criticisms of Fourier results was the number of patients treated to prevent one heart attack or stroke—and the cost of treating all those patients compared with the cost of statins. Amgen has argued that, in patients at high risk, that number is considerably lower.

"With statins, there’s often less than a 1% annualized risk of an event. The risk of people who get Repatha is 11%, 12%, 13%, an order of magnitude higher," Martin Zagari, Amgen’s VP of global health economics, said in an interview earlier this year. "In the Repatha patient versus the statin patient, the same relative risk reduction prevents 10 times more events. That’s the story we are trying to get people to understand."

Today’s data seeks to illustrate that difference. After running several clinical scenarios—using a combination of real-world and Fourier data—the researchers determined that in a "typical" U.S. patient with atherosclerosis, Repatha is worth the price at $9,669.

But in patients with an LDL level of 100 or more, despite aggressive statin therapy, the cost-effective price is $13,225.

"This study affirms the clinical benefits and economic value of delivering Repatha to the right high-risk patients, specifically patients who have had a heart attack or stroke with high LDL levels despite maximally-tolerated statin therapy," Joshua Ofman, SVP of global value, access and policy, said in a statement. "The actual net prices for payers in the market today after discounts and rebates are quite aligned to the value-based price range identified in this study."

Here’s how the calculation using real-world data stacks up against an analysis with just the Fourier results. Using that trial data, the researchers found a cost-effective price of $6,780.

That’s higher than the price with a 60%-or-greater discount  ($5,720) recommended in 2015 by the Institute for Clinical and Economic Review (ICER), an unofficial cost-effectiveness watchdog in the U.S. And it’s still higher than the $4,147 price with the 71% discount recommended in JAMA Tuesday.

"The 71% price reduction required to make PCSK9 inhibitor therapy cost-effective is greater than the 25% to 30% discounts typically offered by manufacturers," the study authors wrote.

But, obviously, it’s lower than the real-world analyses. That’s something new that Amgen might be able to parlay into a better reception from payers, which have put up hurdles to PCSK9 prescriptions and thus limited the drug’s uptake. By identifying patients most likely to benefit, it limits the eligible population while also highlighting its effectiveness.

"The analysis identifies the types of high-risk patients for whom this therapy is both clinically beneficial and cost-effective," according to lead author Gregg Fonarow, M.C., who’s a professor at the UCLA David Geffen School of Medicine. "This study provides a critical input to the overall cost-effectiveness debate that has surrounded PCSK9 inhibitors."

Since Fourier, Repatha script levels have grown, but better numbers might move the needle more. The key word is "might," of course. And there’s another set of data that could make a difference, and it’s expected later this year or early next. That’s Sanofi and Regeneron’s outcomes study of Repatha’s head-to-head rival Praluent. The companies are locked in a patent fight that could render that data moot. But if not—and if that outcomes data proves more promising than Repatha’s—it could give Praluent a competitive boost.

Then again, ICER figures CV benefits are a classwide effect. So Repatha might benefit from that data, too.