- New research published online Tuesday found Amgen, Inc.'s Repatha would need significant price reductions in order to meet a measure of cost-effectiveness for certain patients — even when taking into account the proven cardiovascular benefit of the PCSK9 inhibitor shown in the company's FOURIER outcomes trial.
- In the study's simulation, adding Repatha to statin therapy for all eligible U.S. adults would prevent nearly 2.9 million more major cardiovascular events over a lifetime horizon than pairing the commonly used ezetimibe with statins. Yet Repatha's annual list cost would need to come down by 71% to meet the cost-effectiveness threshold used by the study's authors.
- Amgen has faced pushback from payers on Repatha's relatively high annual cost. Both Repatha as well as a rival PCSK9 inhibitor from Sanofi SA and Regeneron Pharmaceuticals, Inc. have struggled to gain traction on the market despite superior efficacy in lowering LDL cholesterol.
Correction: A previous version of this article cited an inaccurate conversion from euros to U.S. dollars for sales of Praluent.
The slow starts for both PCSK9 inhibitors reflects both the tightening pricing environment in the U.S. as well as the state of the current cholesterol-lowering market. Statins — previously blockbuster branded products — are now generic and cheap, making it more difficult for the PCSK9 drugs to stand out on their LDL-lowering abilities alone.
FOURIER, a 27,564-patient cardiovascular outcomes trial, was designed to prove Repatha's (evolocumab) heart benefit in hopes of improving the drug's clinical profile and boosting uptake.
While the study succeeded, reducing the risk of major cardiovascular adverse events, the effect was smaller than some had hoped would be shown. Additionally, treatment with the drug failed to show a statistically significant effect on cardiovascular death.
After release of the results, analysts speculated whether cost-conscious payers might continue to restrict use of Repatha to only those patients at highest risk, given the drug's high cost of roughly $14,000 a year.
Amgen believes Repatha can exceed current expectations and has offered to cut outcomes-based deals with payers who drop barriers to coverage. An agreement announced in May with the insurer Harvard Pilgrim would fully refund Repatha's cost if a patient is hospitalized with either a heart attack or stroke.
Yet cost analyses continue to highlight the cost of Repatha and Praluent (alirocumab) as a potential hurdle.
Tuesday's study, published as a research letter in JAMA, updated a previous 2016 analysis to reflect the impact of the FOURIER results on the cost-effectiveness of Repatha and the PCSK9 class more broadly.
Looking only at patients with atherosclerotic cardiovascular disease who are already taking statins, the current average annual list price for PCSK9 inhibitors exceeded the study's targeted threshold of $100,000 per quality-adjusted life year (QALY) — a commonly used, if controversial, benchmark.
"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, citing a March article from Reuters which reported Amgen sells Repatha at a discount of about 30% to its annual list price.
Amgen disputes the finding, highlighting that list prices don't reflect the average net cost payers end up paying.
"Several published analyses have shown value-based price ranges that support our current average net selling price, which has been incorporated in several value-based arrangements with payers," Amgen said in an emailed statement. "Key differences that need to be considered are real world event rates and value for patient life years in the U.S."
Sales of Repatha totaled $83 million in the second quarter, $60 million of which was earned in the U.S. market. The Food and Drug Administration is expected to decide by early December on whether to update Repatha's label to include the FOURIER results — a potential competitive advantage that could boost sales. An expanded label would allow Amgen to market the drug's cardiovascular benefit directly to physicians and patients.
By comparison, Sanofi and Regeneron's rival drug Praluent pulled in only €29 million ($32 million) in U.S. sales last quarter, hampered by "significant payer utilization management restrictions."
Data cited by Salim Syed, an analyst at Mizuho Securities USA LLC, shows Repatha has secured a roughly 60% share of total weekly PCSK9 prescriptions in the U.S.
Amgen has conducted its own cost-effectiveness study in partnership with cardiologists and expects the results to be published imminently.