Docs say that off-label use of PCSK9s is premature

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Prescribing the newly approved PCSK9 inhibitors off-label in the short and intermediate term is “premature,” Dr. Patrick O'Gara, a senior physician at Brigham and Women's Hospital, said during a panel discussion on Thursday.

Harvard T.H. Chan School of Public Health hosted a panel discussion about the new cholesterol-lowering drugs in collaboration with Reuters and Harvard Health Publications.

Sanofi's and Regeneron's Praluent is approved for use in heterozygous familial hypercholesterolemia (HefH), while Amgen's Repatha is approved for use in HefH and homozygous familial hypercholesterolemia. Both drugs are approved to treat patients with heart disease who require additional lowering of cholesterol beyond what statins can provide.

O'Gara called attention to the off-label use of PCSK9 inhibitors by a broad set of patients who complain that statins give them muscle aches, or myalgia. If the drugs had been approved to treat patients who are statin intolerant, the market would be significantly larger. Still, some payers have expressed concerns that the drugs will be prescribed off-label to this group of patients.  

“It's very difficult to measure,” O'Gara said. “There's not a biomarker [for myaglia]—it's a very inexact science.”

He described the inherent hardship in identifying the level of intolerance and discomfort that some people report about statins. “There's some skepticism that people are not as intolerant as they claim to be,” he said.

He added that conversations about cholesterol between patients and doctors must be individualized.

Pharmacy benefit managers have noted that expanding the use of these cholesterol-lowering drugs to a broad set of patients, such as those who complain that statins cause them muscle aches or myalgia, could push the market of these drugs from an estimated 620,000 patients to above 3 million patients while incurring significant and untenable costs on the health system, according to CVS Health estimates.

O'Gara also expressed concern that there may be adverse events related to the drug that were not seen in clinical trials and that utilizing the drug for patients complaining about “relatively mild” symptoms may not be worth the $14,600 price tag for Sanofi's Praluent, or $14,100 for Amgen's Repatha. 

“Release of these medications in an unfiltered way to a large number of patients is a very worrisome proposition looking ahead and thinking about cost,” he added.

Dr. Paul Ridker, director of the center for cardiovascular disease prevention at Brigham and Women's Hospital, noted that there are other steps for doctors to choose for patients who complain about milder side effects from statins, namely, going to a lower dosage or alternating the days on which they take it. “Many patients who think they can't take one, [actually] they can,” he said.

Ridker also said compliance for these drugs could be an issue, given that they are taken as an injection once or twice a month, saying that many people are “looking for long-acting approaches,” including the possibility of an injection once or twice a year, a suggestion that implies that the drugmakers could improve the convenience of administering the drugs.

This story has been updated to clarify the indications and price of currently marketed PCSK9 inhibitors. 

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