New antihyperlipidemic drug, Juxtapid, is the first in its class and was approved just before the end of the year 2012 by the FDA. Another drug in the same unique class of drugs approved for the rare genetic diagnosis of homozygous familial hypercholesterolemia (HFH) was approved this past week.
The approval comes with a box warning about the risk of hepatotoxicity and a Risk Evaluation and Mitigation Strategy (REMS) Program which will require certification of health care providers and pharmacies before the drug can be prescribed and dispensed.
Juxtapid is given orally while Kynamro has to be injected, making it a less likely used product. People HFH tend to develop significant atherosclerosis earlier in life and have serious and significant cardiac events even in their 40s.
The novel drug, which is a microsomal triglyceride transfer protein inhibitor is indicated, according to the label, “as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH)” The label notes that the safety and effectiveness of the drug has not been established in hypercholesterolemia patients who do not have homozygous FH and that the effect of the drug on cardiovascular morbidity and mortality has not been determined.
One small hang up on these treatments for HFH, which affects about 1 in one million Americans, is the price tag: estimates are it will cost patients $200 to $300,000 annually, making it fairly certainly the drugs’ utilization will be closely managed by pharmacy benefit plans via tier placement, prior authorization and, in some cases, total exclusion from benefit coverage.
ESH understands why that is so necessary so these modalities for rare diseases are used appropriately for people with the disease and not just willy nilly for run of the mill high cholesterol patients. Two or 3 people on such a drug for one employer or group offering drug benefits could be cripled with just 2 or 3 patients on these drugs in their population.
Situations such as these show how the down side of Obamacare will ration such health care to patients who truly don’t have other options. With some benefit maximums at $3000 under Obamacare, some patients will never get or see needed therapy.
As the Kenyan kommie would say: “just take a blue pill…”