Fight Intensifying Over Which Patients Will Get Regeneron, Sanofi's $15,000 a Year Cholesterol Drug
August 11, 2015
By Mark Terry, BioSpace.com Breaking News Staff
As Regeneron Pharmaceuticals, Inc. and Sanofi launch their new cholesterol-lowering drug, physicians with CVS Pharmacy published a letter to The Journal of the American Medical Association (JAMA) proposing guidelines regarding the more expensive medications.
Sanofi and Regeneron’s Praluent (alirocumab) was approved on July 25 by the U.S. Food and Drug Administration (FDA). It is a first-in-class proprotein convertase subtilisin/kexin type 9 (PCSK-9) inhibitor. It appears to be a powerful new drug in the battle against high cholesterol. The catch? It is priced at $15,000 per year, compared to the more typical $50 per month for statins, the current most commonly used treatment.
The authors of the JAMA letter wrote, “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.”
In 2013, the American College of Cardiology and American Heart Association (ACC/AHA) provided cholesterol management guidelines that “abandoned the longstanding principle that clinicians should treat patients to a specific LDL-C target,” the physicians wrote. Instead, patients are stratified based on risk. The three physicians argue that cardiologists need to reevaluate those guidelines and set clear cholesterol levels that their patients should hit in order to help decide whether or not statins are appropriate or whether PCSK-9 inhibitors would be better.
When the FDA approved Praluent, its guidelines were vague regarding who should receive the new drug, indicating only that it could be used by patients with atherosclerotic disease whose cholesterol wasn’t reduced enough by statins. The problem apparently is that “enough” wasn’t specified. The current guidelines shifted away from specific cholesterol targets, instead moving toward an overall cardiovascular disease risk assessment.
“I definitely think they are going to have to relook at the guidelines,” said Steve Miller, chief medical officer of Express Scripts Inc. in a Bloomberg interview. “The question is how do you define failure? There is a lot of ambiguity in the current classification system.”
The story has more weight because Thousand Oaks, Calif.-based Amgen is expected to also have a PCSK-9 inhibitor approved on Aug. 27. In June an FDA panel recommended approval of Amgen’s Repatha (evolocumab), but backpedaled by indicating it should only be used for homozygous familial hypercholesterolemia (HoFH). Even with the limitations, analysts believe Repatha, if approved, could generate $2.5 billion by 2020. Analysts also project Praluent could create $1.9 billion for Sanofi in the same period.
Of course, it makes sense that payers would want to limit access to extremely expensive drugs if more affordable options are available and effective. Having specific cholesterol levels would make it simpler to determine appropriateness of prescriptions for higher-priced drugs. The physicians wrote in JAMA, “As clinicians and payers prepare for the entry of PCSK-9 inhibitors to the market, there is a need to achieve consensus around management strategies for patients with hyperlipidemia. The differences in costs between newer and older therapies are substantial (Praluent is priced at $14,600 a year). The comparative clinical outcomes are not yet clear. Large-scale clinical trials currently under way will provide greater insight into the long-term clinical outcomes and population health effects of different management strategies. The guidelines will likely change.”
On the other hand, the need to determine prescription guidelines for use, at least in terms of setting efficacy guidelines, should probably be made without regards to cost. “The potential of this new class of drugs is exciting,” said Kim Allan Williams, president of the American College of Cardiology (AAC) in a Reuters interview, “and we look forward to data from the clinical trials in progress that could demonstrate whether these new cholesterol-lowering drugs will benefit a wider group of patients. In the meantime … any changes to them (guidelines) must be supported by evidence.”