PCSK9 Inhibitors: A Technology Worth Paying For?

Food and Drug Administration in the United States has approved the (PCSK9) inhibitors alirocumab and evolocumab as an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (FH) or clinical atherosclerotic cardiovascular disease requiring additional lowering of LDL-C. Evolocumab has also been approved for homozygous FH. Long-term outcomes studies are pending. The drugs are expensive, costing over $12,000 a year. There is concern that these drugs may not provide good value. While this can be studied with cost-effectiveness analysis, this will be challenging to do, especially when considered for therapy in young people which may be life-long. While inexpensive preventative therapies are cost-effective in the young, expensive therapies may not meet a societal willingness-to-pay threshold as the costs are high and accrue immediately, while the benefits may be decades in the future.