Potential Social Value of PCSK9 Inhibitors, Depending on Price and Efficacy

Enormous social benefit could result from decreasing LDL by 50% in high risk groups with hyperlipidemia despite treatment, and PCSK9 inhibitors may be valuable in achieving these goals, according to a study published online on June 15 2016 in the American Journal of Managed Care.¹

 “Our study confirmed what we already knew: reducing lipid levels significantly in high-risk patients can have a considerable health impact. Our study suggests that depending on the resulting confirmation of current estimates of effectiveness, PCSK9 inhibitors can provide considerable value when appropriately targeted, even at currently negotiated prices,” wrote first author Anupam Jena, MD, PhD, of Harvard Medical School (Boston, MA), and colleagues.

Despite therapy, many patients find it difficult to achieve LDL goals. PCSK9 inhibitors were recently approved for treating familial hypercholesterolemia, as well as hyperlipidemia in patients with atherosclerotic disease who are unable to reach an LDL goal of ≤70 mg/dL despite maximally tolerated lipid lowering therapy. Studies have suggested that PCSK9 inhibitors may reduce LDL by 50-77%, and major cardiovascular events (MACE) by 50%.  However, the high price tag (over $14,000 annually), has raised concern.

In the study, researchers used a simulation model to estimate the social value of decreasing LDL levels by 50% in people with hyperlipidemia in statin benefit group 1 (established atherosclerotic disease) and statin benefit group 2 (LDL ≥190 mg/dL), as defined by the American College of Cardiology (ACC) and the American Heart Association (AHA).² They also looked at potential economic and mortality benefits of PCSK9 inhibitor therapy in these groups. 

They used associations established by the Cholesterol Treatment Trialists’ (CTT) Collaboration meta-analysis to estimate how decreasing LDL by 50% would affect CVD events and mortality. Finally, they used metanalytic techniques to develop efficacy estimates of PCSK9 inhibitor therapy: conservative efficacy (42-50% reduction in MACE risk, 32-45% reduction in CVD mortality risk) and high efficacy estimates (50% reduction in MACEs and CVD mortality).

Estimates for 2015-2035 suggested that decreasing LDL by 50% in individuals with LDL >70 mg/dL would result in:

• $2.9 trillion in savings from averted MACEs and CVD deaths

• 1.6 million deaths averted

PCSK9 inhibitor therapy would result in: 

• $3.4 - $5.1 trillion in savings from averted MACEs and CVD deaths

• 1.9-2.8 million deaths averted
• 17-19 million MACEs averted

• 2-3 million CVD-related deaths averted

The authors noted that PCSK9 inhibitor therapy would result in a net positive social benefit (in excess of drug costs) if the annual price of therapy stays below $18,000 (assuming high efficacy) or $12,000 (assuming conservative efficacy) for patients in statin benefit groups 1 and 2.

They concluded: “Although our estimates suggest that these drugs may generate significant value for society, PCSK9 inhibitors’ net impact will depend on the final costs of these therapies and on pending results of trials evaluating their clinical outcomes. The number of individuals initially prescribed PCSK9 inhibitors is likely to be significantly lower than that suggested by previous estimates.”

Take Home Points

• Modeling study suggests PCSK9 inhibitors could have significant societal value in appropriately targeted patient groups, though the value depends on final costs of treatment and further efficacy data.

• Between 2015-2035, estimates suggest that PCSK9 inhibitor therapy in statin benefit groups 1 and 2 would avert 1.9-2.8 million deaths, and save $3.4 - $5.1 trillion due to averted MACEs and CVD deaths.

• PCSK9 inhibitor therapy would result in a net positive social benefit (in excess of drug costs) if the annual price of therapy stays below $18,000 (assuming high efficacy) or $12,000 (assuming conservative efficacy) for patients in statin benefit groups 1 and 2.

The study was supported by Amgen. Dr Goldman is founder and Ms Chou, Drs Stevens, and Ton are employees at Precision Health Economics (PHE), which received funding from Amgen for this work. Drs Jena, and Blumenthal have received consulting fees from PHE,

References

Jena AB, Blumenthal DM, Stevens W, et al. Value of improved lipid control in patients at high risk for adverse cardiac events. Am J Manag Care. 2016 Jun 1;22(6):e199-207.

Stone NJ, Robinson JG, Lichtenstein AH, et al; American College of Cardiology/American Heart Association Task Force on Practice Guidelines. 2013 ACC/AHA guideline on the treatment of blood cholesterol to reduce atherosclerotic cardiovascular risk in adults: a report of the American College of Cardiology/American Heart Association Task Force on Practice Guidelines. J Am Coll Cardiol. 2014;63(25, pt B):2889-2934. doi: 10.1016/j.jacc.2013.11.002