WASHINGTON -- When Dickens wrote, "It was the best of times, it was the worst of times, it was the age of wisdom, it was the age of foolishness, it was the epoch of belief, it was the epoch of incredulity ... " he was talking about the French Revolution, but for today's healthcare -- and maybe especially for the pharmaceutical industry -- the description is equally apt.
Case in point: PCSK9 inhibitors. This class of compounds pretty much skipped through the clinical trial phase, with one study after another piling on the extraordinary news about the potency of these lipid-lowering compounds that could drive LDL concentrations down to previously unimagined lows: less than 50 mg/dL and even less than 30 mg/dL.
Nirvana: a way to tame the high-risk profiles of persons with familial hypercholesterolemia and a treatment that could be used in statin-intolerant patients, a group not as easily defined as FH.
The FDA agreed, moving rapidly to approve first alirocumab (Praluent) in July 2015 and, a month later, evolocumab (Repatha). In both cases the drugs were approved based on surrogate endpoints (e.g., LDL effects) with a caveat that outcomes trials needed to be done.
But neither drug is even close to achieving the blockbuster status that some predicted as both face considerable barriers to use, the first being an arduous pre-approval process required by payers and the second being price: most statins are off patent so treatment costs are minimal, as is also the case with ezetimibe (Zetia). Alirocumab and evolocumab, on the other hand, are priced in the $14,000-$15,000 range.
Outcomes data, the experts said, could tip the balance in favor of the PCSK9s -- if the drugs not only cut LDL by 60% or so, but also prevented heart attacks, strokes and, most importantly, could save lives that could be the crucial difference.
FOURIER, reported March 17 at the American College of Cardiology meeting and simultaneously published online by The New England Journal of Medicine, found a benefit for both stroke and heart attacks, but no mortality benefit. And although there were impressive reductions in relative risk for those events, there was only about 2% absolute benefit.
The data were based on about 2 years of follow-up and at that point, the number needed treat to prevent one event was 74. That means treating 74 patients for 2 years -- back of the envelope calculation is roughly $1.5 million spent to prevent that event.
That made it a little difficult to keep the band playing the victory song.
What now?
A money back guarantee, that's what.
Just hours after the FOURIER results were reported, Amgen, which markets the drug, said it was so convinced of the value of its drug that it would not only continue patient assistance programs but also offer to "refund ... the cost of Repatha for all of their eligible patients who have a heart attack or stroke."
MedPage Today reached out to Amgen for more details on this offer: Who gets refund? How much do they get? Who determines that a refund is warranted?
Amgen spokesperson Kristen Neese supplied answers, but cautioned, "Some of the specific details you asked about are going to vary based on the contract with the payer." Pretty much all of them, in fact:
1. To whom would refunds be made? Third-party payers only? Or would patients get co-pays also refunded?
"For any compliant Repatha patient who had a heart attack or stroke after taking Repatha for at least six months, payers would receive a refund in the form of an additional rebate. Amgen does not have access to patient information. The plans do however have access therefore, the option we offer is to provide a refund to the payers, but as part of our negotiations we will ask the plans to pass the savings to the patient. This would be the subject of negotiation with each payer."
2. Would the refund cover amounts paid from initiation of treatment or for some specified period, e.g. for 30, 60, or 90 days treatment.
"The precise details will be the subject of a negotiated contract with each payer."
3. Who determines eligibility for refund?
"The precise details will be the subject of a negotiated contract with each payer.To underscore the Company's conviction around these outcomes results, Amgen will offer additional contracting options in the U.S. to payers willing to remove access barriers. These options include one that offers a refund of the cost of Repatha for all of their eligible patients who have a heart attack or stroke. In addition, Amgen will continue to offer innovative contracts that provide reasonable budget predictability to help address budget impact concerns raised by payers."