A potentially amazing discovery in cardiology

This is a story about a new kind of drug and a potentially amazing discovery in cardiology. In July, a new cholesterol lowering medication, called Praluent, was approved by the FDA. A second, similar medication, called Repatha, was just approved by the FDA yesterday.  Look out Lipitor and Crestor, there are new drugs in town!

Let's start at the beginning…

Twelve years ago, scientists found two families in France with very high LDL cholesterol, the so-called "bad cholesterol." These families had a genetic mutation that allowed the bad LDL to stay in the bloodstream, and caused them to develop premature heart disease.

By studying this unfortunate situation, scientists developed Praluent and Repatha.  They work in a completely different way than the current commonly used statins. To begin with, they are not pills.  They are administered once or twice a month by a small injection just under the skin.  The injection causes cholesterol levels to drop to previously unseen levels. Perhaps one of the most exciting aspects of this recent pharmacologic breakthrough is the reduction in unpleasant side effects. Gone are the muscle aches, fuzzy thinking, liver abnormalities, and a host of other side effects that oral anti-cholesterol meds that have been known to cause.

It should come as no surprise that since this discovery, several pharmaceutical companies (all competing to bring similar products to market) have been spending hundreds of millions of dollars doing research to develop similar products.

On the technical side, Praluent and Repatha are the latest developments in the field of biotechnology, but here's the rub: These medications are super expensive. Biotech drugs were responsible for a 12 percent increase in medication costs in this country last year, and are being aggressively marketed.

There is no doubt that Praluent and Repatha will lower cholesterol, especially in someone who cannot get their cholesterol levels low enough by taking statins. But this is not good enough. A medication like this is only helpful if it also saves lives. Cardiologists have learned painfully from their experience over the past decade that prescribing other medications like Niaspan and Zetia to lower cholesterol will not necessarily lead to preventing heart attacks, or prevent cardiac deaths.  We do not yet know if these two new medications will prevent heart attacks, and this information will not be available for at least two more years.

So, why did the FDA approve these drugs now?  Why not wait until more information is available?

Only the FDA knows the answer, and their decision to release both Praluent and Repatha is a gamble. Like any new medication, more side effects may show up when more people begin taking it.

It is always exciting when something new comes to town. There are millions of people with high cholesterol who could become eligible for these medications, but with a price tag of $15,000 per year, who will be willing to pay for it?

As Praluent and Repatha strut into the world of cardiology, the basics of heart health must not be lost. Those who will benefit from these new medications must be identified through careful clinical evaluation.  It is never the right decision to promote expensive medications in lieu of a healthy lifestyle.

I hope these new medications will turn out to be an amazing discovery — combining the best of ingenuity, the rapid race between pharma companies to get a product to market, with a touch of good old fashioned capitalism. I just hope it will not break the financial back of the health care system at the same time.