A budget impact analysis of Alirocumab was performed for a hypothetical health plan.

1. A Budget Impact Analysis of Alirocumab in Heterozygous
Familial Hypercholesterolemia Treatment
Kemper May, Ryan Tercho, PharmD Candidates 2016
Duquesne University, Mylan School of Pharmacy, Pittsburgh, PA.
BACKGROUND
Heterozygous Familial Hypercholesterolemia (HeFH)
is an autosomal dominant disease characterized by a
single mutant allele within the gene that regulates low-
density lipoprotein receptor formation. Uncontrolled
HeFH patients may experience premature
atherosclerotic cardiovascular disease as a result of
an inability to effectively remove LDL from the blood.
While current estimates demonstrate the disease’s
prevalence in the US to be 1 in 500, only 20% of
diagnosed cases achieve satisfactory LDL levels
according to ATP III guidelines. Historically, the
standard of care for this disease state is largely
comprised of high intensity statins. However, a novel
drug class in the form of PCSK9 inhibitors has
demonstrated sufficient efficacy and safety to warrant
FDA approval. On July 24th of 2015, Sanofi and
Regeneron Pharmaceuticals’ Praluent (Alirocumab)
was granted such approval making it the first PCSK9
inhibitor to reach the market. This injectable
monoclonal antibody binds and inhibits PCSK9, a
protein responsible for LDL receptor destruction, and
has demonstrated significant reductions in LDL.
METHODS
A budget impact model was developed using Microsoft
Excel© using a hypothetical commercial health plan
consisting of 1 million members aged 18-64. Data
from the Centers for Disease Control and Prevention
estimates the disease prevalence of HeFH at 1:500 or
0.20%. Assuming an 80% treatment rate for those
patients yielded a target population of 1,600 out of the
1,000,000 original constituents. The Red Book was
referenced for the Wholesale Acquisition Costs (WAC)
of Vytorin, Zetia, and statins. The cost of Praluent was
estimated to be $10,000 per year. Efficacy data
obtained from the literature estimate the pre-
introduction market shares of Vytorin at 27% and
statins alone at 68%; it was assumed that Zetia alone
occupied the remaining 5%. Post-introductory market
shares were estimated at 70%, 20%, 1% and 9% for
Praluent, statins, Zetia, and Vytorin respectively.
These three inputs – target population, market share,
and WAC costs – comprised the budget impact model.
Three outputs were generated: total overall cost, per
member per month (PMPM) cost, and per treated
member per month cost (PTMPM). All costs were
adjusted to 2015 US dollars. In order to better
illustrate statistical uncertainty associated with our
estimates, a one-way sensitivity analysis was also
performed by applying a 5% variance to the following
parameters: cost of Praluent, the percentage of
patients with HeFH, percentage of treated patients with
HeFH, cost of Vytorin, cost of Zetia, and cost of statins.
OBJECTIVES
Because of the high costs associated with monoclonal
antibody medications and the lack of outcomes data
for Praluent, a budget impact analysis may facilitate
formulary decision-making within a commercial health
plan. The purpose of this study is therefore to quantify
the budget impact of Praluent for the treatment of
HeFH over a one year time frame in order to assist in
such formulary decisions.
REFERENCES
DISCUSSION
• Pijlman, A.H. et al. “Evaluation of cholesterol lowering treatment of patients with familial hypercholesterolemia: a large
cross-sectional study in The Netherlands Atherosclerosis , Volume 209 , Issue 1 , 189 – 194
• Red Book Online, Micromedex Solutions. Accessed June, 2015
• Kastelein et al. "Efficacy and Safety of Alirocumab in Patients with Heterozygous Familial Hypercholesterolemia Not
Adequately Controlled with Current Lipid-Lowering Therapy: Design and Rationale of the ODYSSEY FH Studies."
Cardiovascular Drugs and Therapy. Springer US, 20 May 2014. Web. 01 July 2015.
• FDA advisory committee briefing document PraluentTM (alirocumab). Endocrinologic and Metabolic Drugs Advisory
Committee.
Although clinical trials have demonstrated significant
improvement in intermediate biomarkers (LDL, total
cholesterol) in the HeFH patient population, long-term data
have yet to confirm reductions in disease state endpoints.
Because of this, it may be difficult for health plans to make
informed decisions regarding Alirocumab’s role in their
formularies based on cost data alone. However, as
evidenced by the poor long-term efficacy rates for historic
HeFH treatment options, Alirocumab’s relatively high cost
may be offset by its superior efficacy and potentially
superior long-term data. Recently, Sanofi/Regeneron
announced the price of $14,600 per year which is
significantly higher than our estimated $10,000 price tag. A
second budget impact or a cost-effectiveness analysis may
be warranted once the price stabilizes and more outcomes
data is available in order to assist health plans in drafting
policies surrounding Alirocumab.
RESULTS
The pre-introduction Total Budget Impact for HeFH
was estimated at $1,652,256 or $0.138 per member
per month. Based on a calculated 1,120 members
treated with Praluent, the total drug spend for the
hypothetical health plan would be $11,712,160 over a
1 year time horizon. This cost spread out over
1,000,000 members on a month-by-month basis
yielded a cost of $0.976 per member per month. On
an exclusively treated member basis, the cost of
Praluent per month would be $7,320. The one-way
sensitivity analysis revealed that our model outputs
were most sensitive to the WAC of Praluent with a
calculated delta of $117,000 based on a 5% variance
applied in both directions to the estimated $10,000
cost. The calculated delta for both “Percentage of
Patients with HF” and “Percentage of Patients Treated”
were $116,298. The model was relatively insensitive
to the costs of Vytorin, Zetia, and statins with
calculated deltas of $468, $234, and $0 respectively.
CONCLUSIONS
Multiple double blind randomized controlled trials have
shown Alirocumab to both safe and effective at improving
intermediate outcomes; 94% of patients treated achieved
LDL levels less than 100. Because of this, it could
potentially become the primary treatment option for HeFH
patients if long-term outcomes data proves its worthiness
within formularies. Of course, the significant price tag of
Alirocumab will be need to be weighed heavily against
outcomes data for this decision.
$1,652,256
$11,712,160
$0
$2,000,000
$4,000,000
$6,000,000
$8,000,000
$10,000,000
$12,000,000
$14,000,000
Current New
Total Budget Impact
$0.138
$0.976
$0.000
$0.200
$0.400
$0.600
$0.800
$1.000
$1.200
Current New
Per Member per Month (PMPM)
$1,104,480
$1,104,831
$1,104,831
$1,163,214
$1,163,097
$1,221,480
$1,221,129
$1,221,129
$1,162,746
$1,162,863
$1,162,980
$1,040,000 $1,060,000 $1,080,000 $1,100,000 $1,120,000 $1,140,000 $1,160,000 $1,180,000 $1,200,000 $1,220,000 $1,240,000
Cost of Drug Praluent
Percentage of patients with HF
Percentage of patients treated
cost of vytorin
cost of zetia
cost of statin
One-Way Sensitivity Analysis
Low High
RESULTS
ACKNOWLEDGMENTS
We thank Duquesne University Mylan School of Pharmacy
for the financial support they provided for this presentation.
We thank Khalid Kamal who provided mentorship and
guidance from this project’s conception through its
completion.