PCSK9 heart drugs have proven to be one of the biggest commercial disappointments in recent biopharma times. Held back by payers who don’t see the value in providing the drugs even at a hefty 30% or so discount on the list price, sales of Amgen’s Repatha and Praluent from Regeneron/Sanofi have languished — further hampered by cost analysis studies that ascribe a relatively small value to the drugs.
So now, in a widely anticipated study of its own, Amgen is laying out the economic argument to justify its $14,523 list price of Repatha (evolocumab).
In what is bound to be quickly labeled as a self-serving analysis, Amgen insists that if most payers are refusing to provide Repatha at less than $10,000 a year with the discount, many of their members may well be losing out.
To get to that conclusion, the prominent investigators enlisted for this study used real-world numbers on high-risk patients, pegging it to quality adjusted life years (QALY), a controversial, variable and commonly used measure of assessment. They also noted that sticking with the patient population recruited for Amgen’s big FOURIER study, the discount off the list price would need to be more than 50%.
That FOURIER study was supposed to be Amgen’s ticket to the blockbuster sales that the leaders in this field feel they are due drugs that can significantly reduce the risk of major cardio events. Instead, the lack of a mortality benefit — failing to significantly reduce the risk of death — helped prompt payers to hold fast with treatment guidelines that continue to deny coverage to the vast majority of patients in this huge market.
FOURIER is now used to demonstrate why Amgen’s price should be far lower than it is. In a study published in the Journal of the American Medical Association earlier this week, researchers concluded that drug is worth no more than $4,536 a year, less than a third of its current list price.
Amgen — which has been working out money-back guarantees in the event of a heart attack — disputes those numbers. Given payers’ trenchant attitudes, and the range of cost-justification reports now available, their chance of success appears limited.
That payer kickback is a key concern for everyone in drug development, where the opaque drug pricing system in the US — inflated list prices and uncertain net amounts tend to create more confusion than clarity — is bedeviling the future of many drugs now in development. Getting the data needed for an approval is no guarantee of commercial success, and that is a key issue that will bear increasing influence in Big Pharma’s decisions about what it wants in the pipeline.
Amgen’s assessment includes this bottom line assessment of the value of a drug that slashes LDL by about 60% on average.
Several clinical scenarios were explored to determine whether Repatha is worth the investment, including event rates seen in real world clinical practice settings, as well as event rates seen in the actual outcomes trial, and in patients who have higher baseline LDL levels. The researchers determined that to remain below generally accepted cost-effectiveness thresholds in a typical U.S. ASCVD population ($150,000 per quality adjusted life year (QALY)), the annual net price for Repatha would need to be at or below $9,669. Targeting the more severe clinical practice population with baseline LDL levels ≥100 mg/dL despite maximal intensity statin therapy reveals that an annual net price of $13,225 is cost-effective. In a population with event rates seen in the Repatha outcomes study, $6,780 is cost-effective.
“This is the first cost-effectiveness assessment of evolocumab using a model based on a high- quality outcomes trial, combined with U.S. clinical practice data. The analysis identifies the types of high-risk patients for whom this therapy is both clinically beneficial and cost-effective,” said Gregg Fonarow, professor of medicine at UCLA David Geffen School of Medicine and the study’s principal author. “This study provides a critical input to the overall cost-effectiveness debate that has surrounded PCSK9 inhibitors.”
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