Budget Impact Analysis of PCSK9 Inhibitors for the Management of Adult Patients with Heterozygous Familial Hypercholesterolemia or Clinical Atherosclerotic Cardiovascular Disease

Abstract

ObjectiveThe aim of this study was to assess the budget impact of introducing the proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) alirocumab and evolocumab to market for the treatment of adults with heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular (CV) disease requiring additional lowering of low-density lipoprotein cholesterol (LDL-C).MethodsA 3-year model estimated the costs of lipid-modifying therapy (LMT) and CV events to a hypothetical US health plan of 1 million members, comparing two scenarios—with and without the availability of PCSK9i as add-on therapy to statins. Proportions of patients with uncontrolled LDL-C despite receiving statins, and at risk of CV events, were estimated from real-world data. Total undiscounted annual LMT costs (2017 prices, including PCSK9i costs of $14,563.50), dispensing and healthcare costs, including the costs of CV events, were estimated for all prevalent patients in the target population, based on baseline risk factors. Maximum PCSK9i utilization of 1–5% over 3 years according to risk group (following the same pattern as current ezetimibe use), and 5–10% as a secondary scenario, were assumed.ResultsTotal healthcare budget impacts per target patient (and per member) per month for years 1, 2 and 3 were $3.62($0.10), $7.22($0.20) and $10.79($0.30), respectively, assuming 1–5% maximum PCSK9i utilization, and $15.81($0.44), $31.52($0.88) and $47.12($1.31), respectively, assuming 5–10% utilization. Results were sensitive to changes in model timeframe, years to maximum PCSK9i utilization and PCSK9i costs.ConclusionsThe budget impact of PCSK9i as add-on therapy to statins for patients with hypercholesterolemia is relatively low compared with published estimates for other specialty biologics. Drug cost rebates and discounts are likely to further reduce budget impact.Electronic supplementary materialThe online version of this article (10.1007/s40273-017-0590-5) contains supplementary material, which is available to authorized users.

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“…Of course, alternative courses of action are possible, for example stopping statins altogether and treating the patient with alternative drugs. It should be remembered, although, that one such alternative, PCSK9 inhibitors, is very expensive, shows low treatment adherence by the patients and may decrease glucose tolerance …”

“…Of course, alternative courses of action are possible, for example stopping statins altogether and treating the patient with alternative drugs. It should be remembered, although, that one such alternative, PCSK9 inhibitors, is very expensive, 70 shows low treatment adherence by the patients 71 and may decrease glucose tolerance. 72 Further-Furthermore, lowering low-density lipoprotein (LDL)-cholesterol may not, by itself, be effective in preventing cardiovascular events, as shown by a recent study where Evacetrapib, an inhibitor of cholesteryl ester transfer protein was not able to prevent cardiovascular events despite a reduction in LDL-cholesterol levels.…”

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“…The use of MEAs has been implemented in several countries, including the United Kingdom (where MEAs are defined as “patient access schemes”), Italy, Belgium, and Australia. For example, in Italy the modulation of price and reimbursement schemes are planned by using two categories of MEAs, which include the health outcomes-based agreements (payment by result, risk sharing, and success fee) and the finance-based agreements (cost-sharing agreements and capping) (Mallya et al, 2017; Scavone et al, 2018).…”

PCSK9 inhibition, atherosclerotic cardiovascular disease, and health economics: Challenges at the crossroads

“…37 Indeed, some have suggested that despite their established clinical value, perceived costs and budgetary concerns relating to the use of PCSK9 inhibitors in the target patient groups have led to lower than expected uptake of these treatments. 38 This is a simplistic approach, however, which does not integrate possible rebates and discounts that may be offered by the manufacturers (It is noteworthy that, in the US, both manufacturers of PCSK9 inhibitors have lowered their price recently by 60%); indeed, recent developments suggest that the budget impact of such treatments is lower than anticipated, 39 especially when weighed against the burden of cardiovascular complications.…”