After last week's expert panel meetings, the FDA may put some label limits on PCSK9 cholesterol drugs. But if word from doctor surveys is any indication, those limits won't put a huge damper on sales.
According to InCrowd market research, surveyed doctors are already considering prescribing the meds--Sanofi and Regeneron's Praluent and Amgen's Repatha--to 18% of their patients with high cholesterol levels. And 76% of surveyed cardiologists are enthused about the potential new meds, planning to use them solo for 10% of their patients and as an add-on to statins for another 16%. And only about one-third thought the meds should be restricted to high-risk patients.
"This is groundbreaking therapy--a real game-changer," one doctor told InCrowd.
All great news for Sanofi ($SNY), Regeneron ($REGN) and Amgen ($AMGN), who've been racing their PCSK9 meds to market. Weighed last week at back-to-back FDA advisory panel meetings, the two drugs are injectables that have racked up impressive trial data on their ability to dramatically lower "bad" LDL cholesterol. (In fact, they're so effective that some panelists worry about LDL levels dropping too low.)
The FDA's expert advisers voted to back the meds for patients with a hereditary disease that causes very high LDL levels, familial hypercholesterolemia. They also backed the meds as statin add-ons in patients at high risk of heart problems. The committee looked askance, however, at broader use--such as in patients intolerant of statins--particularly without data from outcomes trials expected to wrap up next year. Praluent is up for agency approval first--July 24--thanks to a priority review voucher Sanofi purchased from another drugmaker. Amgen's Repatha has a decision deadline of August 27.
The committee's reservations may well end up affecting the drugs' official labeling. We say "may" because the FDA doesn't have to take the panel's advice, though it usually does. But though the drugmakers obviously want the broadest label possible--the broadest label means the biggest market, after all--doctors can use the meds however they please, and the InCrowd survey isn't the only one to show that cardiologists and even primary care docs might be willing to go ahead with the PCSK9 meds in statin-intolerant patients, with or without an official FDA approval in that group.
But Praluent and Repatha will face certain obstacles. Among them is price--the drugs are expected to cost upward of $8,000 per year, a not-insignificant amount that could put a sizable share on patients' shoulders. Almost 80% of the InCrowd survey respondents saw patient cost as a barrier to use. Then there's the injectable aspect; as one doctor noted, "I cannot get the patients to take a pill, let alone an injectable," for a condition that doesn't cause them immediate suffering. (Cardiologists were less worried about this than primary care docs were, however.)
Then there's the payer angle. The FDA's label may not hinder doctors, but limits would give payers plenty of excuses to put up hurdles to coverage. Already sparring with the drugmakers on PCSK9 pricing, insurers and pharmacy benefits managers are also publicly lamenting the potential cost to the healthcare system. CVS Health ($CVS) executives went as far as to predict a whopping $150 billion cost burden, were the PCSK9 meds to be adopted as widely as statins. Given that that's not likely to happen anytime soon, a more recent cost projection of $23 billion seems more reasonable.
- see the InCrowd release
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