New drugs for ultra high cholesterol cause pricing debate

     The debate about the appropriate price for medicine now includes a new group of drugs designed to treat ultra high cholesterol, called PCSK-9 inhibitors.

     Praluent - made by Regeneron and distributed by its partner Sanofi - is the first of the group to hit the market. It is meant to lower the LDL - "bad" cholesterol - of patients with hypercholesterolemia. Amgen is working on a similar drug that might be approved soon.

     However, what some suggest were vague FDA guidelines included in the approval and relatively old prescribing guidelines from cardiologists, mean that a $15,000 per year drug might become a life-long treatment for millions of people, even if they are outside the original parameters.

     Statins - Pfizer's Lipitor, AstraZeneca's Crestor and now some generic versions of Lipitor - have long been used by millions of people to control unhealthy levels of plaque and other gunk that collects inside arteries of the heart. Too much of the gunk causes heart attacks and, sometimes, death. The costs of regular statins have fallen, especially since Lipitor lost patent protection, so much so that some generic drugmakers don't bother because the profit margin is so low.

     But if 73 million people with high cholesterol were prescribed a $15,000 per year drug for 30 or 40 or 50 years, the cost will be astronomical. Payer groups - including taxpayers, through government programs - are worried about the huge bills.

     "This class also poses a new challenge for health care payers," wrote Dr. William H. Shrank and two other physicians in an article in the online version of the Journal of the American Medical Association. (The link here.) "It is an expensive specialty medication that targets a very common condition; more than 73 million adults (32%) in the United States have elevated LDL-C. As a reference, when sofosbuvir, which is used to treat hepatitis C, was approved and marketed over a year ago, it shocked the health care system because of the high cost and relatively large eligible population, up to 3 million infected individuals. Unlike new therapies for hepatitis C, which represent a cure for most patients, PCSK-9 inhibitors will be used long-term—generally for the remainder of the lives of treated patients. As a result, most payers, both government and commercial, should begin to consider thoughtful ways to rationalize the use of these medications. The best approach will be to promote use of low-cost statin medications rather than PCSK-9 inhibitors, but this approach will be complicated by recent changes in recommendations for treating hyperlipidemia."

Shrank is an employee of CVS Health, which has its own financial interests in the pricing equation. While most people know of CVS as a drug store, a growing piece of business for CVS Health is its pharmacy benefit division. CVS Health is now the second largest PBM after Express Scripts. PBMs are hired by insurance companies and large employers with healthcare plans to manage drug programs. Those clients expect the PBM to negotiate lower prices for drugs, but the results of such negotiations are closely held secrets of the companies involved. Individual patients might not see any savings, even if they paid close attention. Many do not.

Dan Rader of the University of Pennsylvania helped develop a drug -- Juxtapid by Aegerion - to treat a smaller population of people with a rare type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).

Rader was one of the doctors that presented a study of Praluent to the American College of Cardiology’s 64th Annual Scientific Sessions & Expo in San Diego on March 14. Rader received consultant/advisory board fees from Sanofi, according to an SEC filing by Regeneron (link here).