- On the face of it, Amgen Inc.'s offer to insurers of a money-back guarantee for its expensive cholesterol drug Repatha looks to be the type of solution that could help align price with the clinical benefit a patient actually experiences.
- But new research published April 3 in the Annals of Internal Medicine suggests Amgen's outcomes-based payment scheme would only marginally improve the cost-effectiveness of Repatha.
- Repatha, along with Sanofi SA and Regeneron Pharmaceuticals Inc.'s rival PCSK9 inhibitor Praluent, carry list prices of roughly $14,000 a year — sparking criticism and payer resistance. In response, the drugmakers have sought to win broader coverage by extending concessions on drug cost.
When Amgen reported study results last year proving Repatha (evolocumab)'s cardio-protective benefit, the Thousand Oaks, California-based drugmaker said it would refund the drug's cost for eligible patients who still suffered a heart attack or stroke while on treatment.
Data from Amgen's trial showed Repatha reduced the risk of major adverse heart events by 15% in patients with atherosclerotic cardiovascular disease (ASCVD) — a boost to the drug's clinical profile.
At the time, sales of Repatha had widely fell short of expectations. With the new cardiovascular outcomes data in hand, Amgen sought to lower some of barriers to access thrown up by insurers and boost prescriptions.
But the new research pokes some holes in Amgen's seemingly attractive money-back guarantee. Researchers at the University of California, San Francisco and the Institute of Clinical Economic Review (ICER) compared incremental cost-effectiveness ratios for treatment with either a PCSK9 inhibitor or ezetimibe on top of baseline statin therapy.
In the study, a model simulation found a full refund of drug and related inpatient costs in the event of a heart attack or stroke would only improve Repatha's cost-effectiveness ratio by 3.1% — still well above thresholds commonly used by ICER to determine a drug's value.
According to the researchers, the very modest effect of Amgen's refund offer is tied to the preventive context in which the drug would be used. Since the rates of heart attack and stroke across the entire patient population are low, there are relatively few cases in which Amgen would actually have to refund Repatha's cost.
In a theoretical group of 100 patients with an ASCVD event rate of 3% (as proposed by the researchers), 97 patients would receive treatment at full cost each year while adverse heart events in three would trigger a refund.
"Emphasizing the refund — the product being free if you do not like it — works for late-night salespersons because it provides false comfort to the buyer. It distracts from the more essential question of how much the product is worth when it does work," wrote Sham Mailankody and Peter Bach, researchers and physicians at Memorial Sloan Kettering Cancer Center, in a related editorial also published in the Annals of Internal Medicine.
Additionally, the analysis identified two other complicating factors that could make Amgen's refund scheme less effective: deciding what constituted an eligible adverse event might be difficult in practice, as would tracking patients as they changed insurance plans.
Amgen has consistently pushed back on analyses criticizing Repatha's cost, and notes rebates and discounts substantially lower the net price of the drug.
"We believe our outcomes based contracts provide a valuable option to payers interested in evolving from volume based contracts to those focused on the value Repatha brings to their patients, who are at high risk for heart attack and stroke," said a spokesperson for Amgen in an emailed statement.
Over the past several months, Amgen has given further discounts on Repatha's price to those insurers that have lowered coverage restrictions, the spokesperson said.
Meanwhile, Amgen faces renewed competition from Sanofi and Regeneron. Results from the two companies' own cardiovascular outcomes study showed a similar heart benefit to Praluent (alirocumab), and the companies have signaled willingness to reduce the net price of Praluent for certain high-risk patients.
In fact, Sanofi and Regeneron actually worked with ICER, giving the non-profit group access to the study data ahead of time in order to update their cost-effectiveness estimates for the drug.