Determining Who Can't Tolerate Statins

For years now, a class of drugs called statins have been the treatment of choice for lowering LDL cholesterol levels. They're cheap (pennies a day), effective, and safe. The problem is that 5-20% of patients say they can't tolerate the statins because of muscle-related side effects, including muscle pain and weakness.

A new class of class cholesterol-lowering drugs, called PCSK-9 inhibitors, avoids the muscle-related side effects. However, the big downside to the PCSK-9 inhibitors is that they are very expensive - approximately $14,000 a year. For that reason, insurance companies would like the PCSK-9 inhibitors to be used only by the few people who are truly intolerant of the statins. But how is intolerance to the statins to be defined, other than by the patients and their physicians?

A recent study showed how big a problem this is. Researchers wanted to explore the effectiveness of several newer LDL cholesterol-lowering drugs in statin-intolerant patients with very high cholesterol levels. To find such patients, they recruited more than 500 patients who had tried two or more statins and claimed that they could not tolerate them because of muscle pain or weakness. The patients were randomly assigned to take a statin (atorvastatin) or a placebo for ten weeks. Then the drugs were switched for an additional 10 weeks. It turned out that only 43% of the patients could be defined as statin-intolerant; that is, they reported muscle-related symptoms while they were on the statins but not while they were on the placebo. 26% of the patients reported muscle pain only while on the placebo, 17% never experienced muscle-related symptoms on either the statin or the placebo, and 10% complained of pain on both the statins and the placebo.

The bottom line is that fewer than half of all patients who say they are statin-intolerant actually are statin-intolerant. Because the PCSK-9 inhibitors are so expensive, some insurance companies are suggesting that patients who request them should be tested for statin intolerance (as the patients in the above study were) before insurance will pay for the new drugs.

What do you think? Should patients be required to submit to several months of testing before their (and your) insurance company agrees to pay for a $14,000-a-year drug for them?