- A study sponsored by Amgen, Inc. to probe the cost-effectiveness of its PCSK9 inhibitor Repatha found the drug would approach a commonly used benchmark of cost effectiveness when priced at a roughly 30% discount to its current annual list cost of $14,523.
- The analysis, published Wednesday in JAMA Cardiology, comes a day after other research painted a more pessimistic picture of the drug's value at current selling prices. While the Amgen-backed study is on the whole more supportive, the headline findings are dependent on a key assumption that a long-term cardiovascular mortality benefit would emerge after five years of treatment.
- Both studies highlight the challenges posed by the high cost of Repatha as well as its PCSK9 rival Praluent, developed by Sanofi SA and Regeneron Pharmaceuticals, Inc. The two drugs reduce LDL cholesterol dramatically, yet adopting the treatments for a broader group of patients beyond those at highest risk would strap payer budgets for potentially only incremental health gains over standard therapy.
Amgen has staunchly defended Repatha's value in the debate over the drug's cost, offering a money-back guarantee to insurers who lower restrictions on coverage for the drug. The biotech's case was helped by the results of its FOURIER outcomes study, which proved the PCSK9 inhibitor's cardiovascular benefit.
Yet, the data from that trial wasn't a slam dunk, falling short of expectations in the magnitude of risk reduction shown. Results also failed to demonstrate a statistically significant effect on cardiovascular mortality, potentially giving cost-conscious payers a reason to keep coverage barriers in place.
Faced with continued pushback from payers, Amgen has focused on proving the value of Repatha in higher-risk patients, where the drug's value is clearer even at higher costs. The biotech emphasized that argument in a statement put out in conjunction with the release of the JAMA study on Wednesday morning.
"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," said Joshua Ofman, a senior vice president of access and policy at Amgen.
In the study, researchers constructed a model representative of the U.S. patient population with atherosclerotic cardiovascular disease on standard background therapy for high LDL cholesterol.
Results showed adding Repatha (evolocumab) at its current list price would result in an incremental cost-effectiveness ratio (ICER) of $268,637 per quality-adjusted life year (QALY) — a measure used to estimate value by accounting for costs and health benefits. Essentially, an ICER attempts to measure what it would cost to add one life year, adjusted for the quality of health.
Of course, as every drugmaker is quick to point out in debates over drug cost, rebates and discounts negotiated with payers usually lower the price actually paid for a drug. If a 29% discount to Repatha's price is assumed — lowering annual costs to $10,311 — the ICER falls to $165,689 per QALY, nearing a commonly used benchmark of $150,000 per QALY.
To meet that threshold, Repatha would need to be sold at an annual net price of $9,669. What price Amgen actually sells the drug at after rebates and discounts is not public knowledge. But reports have indicated the drugmaker has typically negotiated around a 30% discount.
While that level of discount may put Repatha's net selling price near the value-based price laid out by the study, the authors still highlight the stress placed on health systems by the drug's cost.
"However, with an ICER of $268,637 per QALY with the current list price, there are legitimate concerns regarding the appropriate allocation of health care resources along with challenges in coverage and access to this therapy, despite demonstration of clinical event reduction in FOURIER," the study authors wrote.
Additionally, the study presumes a cardiovascular mortality benefit to Repatha treatment emerges after five years — an assumption not supported by FOURIER.
"The evidence from FOURIER, with a median follow-up of just 2.2 years, cannot settle this question, and the results of the cost-effectiveness models are exquisitely sensitive to the assumptions about long-term effects," wrote three doctors unaffiliated with the study in a corresponding editorial also published Wednesday.
If no mortality benefit emerges, Repatha's ICER would soar to $483,800 per QALY at list price and $290,601 per QALY at a 29% discount, well over accepted cost-effectiveness figures. In that scenario, Repatha would need to be discounted by more than half to be considered a good value.
While FOURIER showed Repatha's cardiovascular benefit increased over time, suggesting such a benefit could be possible, past studies of other LDL-lowering drugs have failed to demonstrate a later mortality benefit.