Patrick Gleason, PharmD, FCCP, BCPS

Dr GleasonThis summer the first 2 drugs in a new class of cholesterol-lowering medications, called PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors, are expected to be approved by FDA. If approved, they will be the first specialty drugs to treat a common chronic condition, high cholesterol, raising questions about how health plans, employers and members will afford the expected price tag of $7,000 to $12,000 per person treated per year. 

	Here are 5 things you need to know about PCSK9 inhibitors. 

#1. They provide a new treatment option for people with very high cholesterol who can’t be treated with statins

	While statins work by blocking an enzyme in the liver that controls cholesterol production, PCSK9 inhibitors work by preventing the protein PCSK9 from interfering with the liver’s low density lipoprotein (LDL) cholesterol removal process, allowing more LDL to be removed from the blood. Unlike statins, which are taken orally, PCSK9 inhibitors need to be self-injected monthly or twice a month. They could be an important treatment for people with very high cholesterol, such as those with the genetic condition familial hypercholesterolemia who are statin intolerant.

#2. They’re expected to come with a significant price tag that could bust budgets and stretch pocketbooks.

	According to an analysis by pharmacy benefit manager 

 (Prime), if FDA restricts the drugs to people with the genetic disorder that results in dangerously high cholesterol levels for which statins are ineffective, the cost would be $2.1 billion per year. If people with a history of heart disease are included, yearly treatment costs would reach $13.2 billion. And if approved for anyone who hasn’t seen effective results from statins, including the previous two groups, the bill for the drugs rises to $23.3 billion per year. That’s equivalent to $73 for every American annually. Because PCSK9s work best when used in combination with statins, PSCK9 costs will be added to the current costs payers incur for statins.

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#3. We don’t know yet if these drugs are safe long-term or how well they work at preventing cardiovascular events, such as heart attacks.

	PCSK9 inhibitors are, at a minimum, equally effective in reducing LDL cholesterol as the most potent statins, and for some individuals better at lowering LDL than the most potent statins. However, clinical trials that will evaluate how effective the new drugs are at reducing heart attacks and deaths won’t be completed until 2017. Lowering cholesterol also generally requires life-long treatments. Yet, the longest safety data for PCSK9s is 2 years. We don't know how safe PCSK9s are when used to treat someone for a lifetime. Conversely, statins have been proven to improve outcomes, such as preventing death or a cardiovascular event such as a heart attack or need to undergo a coronary artery procedure. And statins have more than 25 years of use and many long-term safety studies. This may lead FDA to limit approval of the drugs to those with hypercholesterolemia and those with a history of cardiovascular disease who are statin intolerant.

#4. Prescribing behavior and adherence to treatment will impact how many people use these drugs.

	PCSK9 inhibitors need to be self-injected, which raises concerns about long-term adherence to the medications. On the other hand, it’s expected that some physicians will be interested in using these drugs for additional people with high cholesterol who do not have a prior history of heart attack. These factors will impact the total number of people who could end up using the drug.

#5. Health plans and pharmacy benefit managers will need to manage the drugs carefully to ensure the right people have access.

	While treating high cholesterol with statins cost just pennies a day, PCSK9 inhibitors could cost $10,000 per year. It’s critical that the right level of utilization management is in place to help ensure these medicines are used for people who have had an adequate trial and failure of more than 1 statin previously. Remember, statins have a long-term safety record and have been shown to reduce cardiovascular events, while PCSK9 inhibitors do not yet have this data.

 is director of health outcomes at Prime Therapeutics, Dr. Gleason leads Prime’s clinical health outcomes assessment team in the development and improvement of pharmacy benefit management programs.

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Articles

This summer, the first two drugs in the new class of cholesterol-lowering medications, known as PCSK9 inhibitors, are expected to be approved by the FDA. If approved, PCSK9 inhibitors will change how chronic conditions are treated.

Patrick Gleason, PharmD, FCCP, BCPS

Articles

5 things to know about cholesterol-lowering PCSK9 inhibitors