Outcomes-Based Pricing for PCSK9 Inhibitors Does Not Reduce Costs

Recent research in the Annals of Internal Medicine found that outcomes-based contracting for expensive PCSK9 inhibitors did not reduce drug costs or improve cost-effectiveness of the drug.

“Outcomes-based pricing has been proposed as an alternative to cost-control tools, such as prior authorization and step therapy for new, high-priced drugs,” Dhruv S Kazi, MD, MSc, MS, of the University of California, San Francisco, and colleagues wrote. “Shared financial risk between manufacturer and payer is believed to improve access for patients who respond to therapy while discouraging uptake among nonresponders, thus improving overall cost-effectiveness.”

Dr Kazi explained that this approach recently worked to reduce drug costs among multiple myeloma patients, by refunding costs among patients who did not respond to the drug. He also explained that this approach was recently proposed as a solution to the high cost of PCSK9 inhibitors.

“Despite clinical effectiveness, large reductions in the price of PCSK9 inhibitors (which is currently $14,500 per patient per year) would be needed to achieve cost-effectiveness at conventional willingness-to-pay thresholds prompting interest in this novel pricing model,” the researchers wrote.

The researcher used the Cardiovascular Disease Policy Model to estimate the cost-effectiveness of PCSK9 inhibitors. They calculated the cost-effectiveness among

8.9 million adult high-risk patients aged 40 to 84 years. Patients were considered to meet the treatment criteria if they had atherosclerotic cardiovascular disease and low-density lipoprotein cholesterol levels despite statin therapy. PCSK9 inhibitors were compared to results from baseline statin therapy with ezetimibe.

The researchers found that use of value-based pricing for PCSK9 inhibitors did not meaningfully reduce cost outcomes or improve cost-effectiveness. Results showed that ICER frameworks for a PCSK9 inhibitor without refund were $324,000 per QALY added. When the researchers applied 3 different pricing schemes, there were slight reductions in the ICER results; however, cost-effectiveness remained significantly higher than $100,000 per QALY.

The researchers noted that value-based pricing does not work with PCSK9 inhibitors because of the long-term use of the drug

“Event rates in prevention are generally low and preventive treatment is lifelong,” Dr Kazi and colleagues wrote. “The annual event rate in those with a history of this condition is approximately 3% among 100 eligible patients, 97 would thus incur the full costs each year and refunds would apply to only 3 patients.”

They also explained that failure of preventative treatment would be hard to determine for reimbursement purposes.

“Attributing an event to the failure of a preventive medication is complicated. In practice, events eligible for reimbursement would depend on clinical context (for example, whether stroke in the setting of poorly controlled blood pressure would be considered a failure of a PCSK9 inhibitor),” they wrote.

The researchers concluded by noting that while value-based pricing contracts have a place in certain treatment areas, PCSK9 inhibitors do not see to benefit from this design.

“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,” Dr Kazi and colleagues concluded.

—David Costill