Money-back guarantees fail to boost cost-effectiveness of PCSK9 inhibitors

Outcomes-based pricing agreements would do little to help the poor cost-effectiveness of PCSK9 inhibitors. The event rates in preventive medicine are simply too low and medication costs too high for such a compromise to make a difference, a group from University of California, San Francisco reported in the Annals of Internal Medicine.

PCSK9 inhibitors are lipid-lowering agents that have been associated with a decreased risk of myocardial infarction (MI) and stroke in individuals with atherosclerosis. But the drugs carry high prices, leading to interest in outcomes-based contracts in which manufacturers offer to refund payers some or all of the cost of treatment if it is deemed to be ineffective.

Dhruv S. Kazi, MD, MSc, and coauthors analyzed whether such an agreement could work for PCSK9 inhibitors using data from the FOURIER trial, which showed a 15 percent reduction in the composite endpoint of MI, stroke and cardiovascular death for a PCSK9 inhibitor plus statin regimen versus a statin-plus-placebo combination. The researchers then compared the incremental cost effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained for two combinations of therapy: statin plus PCSK9 inhibitor and statin plus ezetimibe, a cheaper lipid-lowering agent with a similar indication.

Even if drug manufacturers agreed to refund inpatient hospitalization costs and medication costs for cases of MI, stroke or death for up to five years, the researchers found the ICER of PCSK9 inhibitors would be $314,000 per QALY—well above a reasonable threshold of $100,000 per QALY. That agreement only marginally improved the ICER from no refund at all ($324,000 per QALY), pointing to issues with outcomes-based pricing models for preventive medicine.

“Event rates in prevention are generally low and preventive treatment is lifelong,” Kazi et al. wrote. “The annual ASCVD (atherosclerotic cardiovascular disease) event rate in those with a history of this condition is approximately 3 percent; among 100 eligible patients, 97 would thus incur the full costs each year and refunds would apply to only three patients.”

It would also be difficult to definitively attribute an event to a medication failure, and to track which payers are eligible for reimbursement if their patients frequently switch insurance, the authors noted.

“Although outcomes-based pricing may have a role in other settings, our analysis of its application to PCSK9 inhibitors—effective preventive medications with a high price tag—shows the potential limitation of this approach for high-cost preventive therapies,” Kazi and colleagues concluded.

In an accompanying editorial, Sham Mailankody, MBBS, and Peter B. Bach, MD—both with Memorial Sloan Kettering Cancer Center in New York—said money-back guarantees are gimmicky and impractical for healthcare.

“The approach of ‘if you don't like it, get your money back’ seems more the nonsense of late-night infomercials than a method for serious health policy reform,” they wrote. “However, the idea has been pushed hard by the pharmaceutical industry.”

A better approach for preventive drugs, they wrote, would be lowering the cost of all of the medication instead of wasting time and resources trying to figure out which patients actually benefitted from it. The editorialists maintain that is an impossible exercise anyway.

“Preventive interventions are ever thus: They benefit only a few who receive them, and we nearly never know who those persons are,” Mailankody and Bach wrote. “No scenario exists under which insurers should choose a refund-based approach rather than the simpler and administratively cheaper solution of having the drug corporation charge them the anticipated net price in the first place.”