PCSK9 Inhibitors

PBIRx®

Intelligent Solutions in Pharmacy Benefits

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What are PCSK9 Inhibitors?

PCSK9 inhibitors are a new class of drugs that can greatly reduce LDL (Low Density Lipoprotein) cholesterol levels.  LDL is the “Bad Cholesterol” associated with increased risk of heart disease, including heart attack and stroke.

What does PCSK9 stand for?

PCSK9 stands for Proprotein Convertase Subtilisin Kexsin 9.

How much do they cost?

We do not yet know the specific cost but do know they will initially range about $6-12K per year.

Are these drugs currently available?

Two different brands of this drug Praluent™ (alirocumab) and Repatha™ (evolocumab) will be approved third quarter 2015.

What does the FDA say?

The FDA has not formally approved these drugs for market. It is highly expected they will formally approve this drug in the coming months. An FDA medical advisory committee has recommended approval of these drugs for certain conditions.

Are the PCSK9’s for everyone with “high cholesterol”?

No.  As of today, the FDA medical advisory committee has unanimously recommended use of these drugs in people with HoFH (Homozygous Familial Hypercholesterolemia). This is a rare genetically induced high cholesterol disorder that affects 1 in 2000 people.

Additionally the committee was split for recommendation for use for people with any other indication for these agents.

Other indications include:

All patients with high LDL cholesterol

Patients that are “statin resistant”

Patients that are “statin intolerant”

Why is the FDA advisory committee not unanimous for these other indications?

All the data is not yet available. Studies to date have proven that these drugs will reduce LDL by up to 60%. While in theory this is a good sign, it has yet to be shown that this will result in lower heart disease rates.  Two studies involving 20,000 patients are currently under way but the results will not be avialable until at least 2017.

Why the caution?

Historically other cholesterol reducing agents have been approved based upon their cholesterol improving abilities, but later studies showed limited outcomes benefits. These agents include niacin, fibrates and Zetia™ (ezetimibe). While they have all found a place in the cholesterol lowering arsenal their actual results are much less then the initial studies had shown.

Are there other concerns?

Yes.  As with any other newly approved drug there is an unknown side effect adverse event profile. New drugs are studied in controlled populations. After drugs come to market they are exposed to a much greater cross section of the general population. Invariably unknown issues come to light. What is known to date is that the PCSK9 inhibitors have shown a greater degree of “cognitive dysfunction” vs. placebo.  While not statistically significant in the limited studies to date, it is an issue of concern for the FDA advisory committee.

Additionally theses drugs will initially be available only in a subcutaneous injectable form. Similar to Insulin in administration, they will be administered either once or twice monthly depending on the PCSK9 dispensed.  Historically there is some resistance to injections from a portion of the population. An oral version of this drug is currently under study.

Are PCSK9 drugs a “cure”?

No. These agents will lower LDL cholesterol levels but will not treat the underlying causes. This means that these drugs, and any other cholesterol lowering drugs, must be given for the lifetime of the patient.

What is the current treatment for lowering cholesterol?

The drug class of choice is the “Statins”, for which there are many low cost generics available.  This is a class of drugs with proven effectiveness in lowering LDL. While highly effective there is a subset of patients that are resistant to the positive effects of statins and others who suffer from its side effects, primarily muscle pain.

How much do statins cost?

Generic statins cost around $100 per year. Crestor™(rosuvastatin), a brand statin, can cost around $450 per year.

How many Americans have “high cholesterol”?

70 million Americans have high LDL.

Are there other means of lowering cholesterol?

Yes.  Diet, exercise and stress control are all contributing factors and can be controlled, or mitigated, by lifestyle changes.

What does PBIRx recommend?

At this point in time we recommend restricted use of these medications only for those with HoFH, or patients at high CV risk that are statin intolerant or statin resistant. We work within the guidelines of the American College of Cardiology and American Heart Association as to the definition of these conditions.

Our recommendation is to only allow use of these agents with a Prior Authorization from your PBM, after consultation with the patient’s doctor.

Because of the potential costs involved we believe you, the payer, have a right to know that this drug is being prescribed under the most optimum conditions and being used correctly and appropriately. 

As these are a new class of drugs, with multiple competitors, incoming information is evolving rapidly, and criteria and situations may change. As your pharmacy consultants we strive to be aware of this evolving landscape and will keep our clients informed.