At the end of September, Amarin Corp. teased some early findings for Vascepa, its preventive medicine for people at risk of heart disease. The claim was astounding: a 25% relative risk reduction for deaths related to heart attacks, strokes, and other conditions. Headlines proclaimed a potential game changer in treating cardiovascular disease. And company shares quickly soared, from $3 a share to about $20.
Vascepa is Amarin’s only product. The company hopes to turn its pill made of purified fish oil into a cash cow, allowing it to staff up both in the United States and abroad so it can sell doctors and millions of consumers on its medical benefits. Although the product has been on the market for more than 5 years, its first TV ad campaign rolled out this summer in anticipation of the study findings.
Except there is one problem. The particulars of the scientific study on which this claim was based remain a mystery.
Amarin’s preliminary announcement came via a news release on Sept. 24. The company plans to release detailed findings in November at the national American Heart Association conference. Then early next year, it plans to seek Food and Drug Administration approval to use the drug as a preventive for a range of heart conditions, beyond its current role targeting high triglyceride levels.
In the interim, a battle is brewing among physicians, cardiovascular experts, and pharma watchers who say Vascepa brings to the foreground troubling trends in the marketing and advertising of new drugs. Companies sometimes promote new products, but withhold the detailed findings until much later. The consequences for both consumers and the health system are vast.
“Until all the data is available for review by the public and medical community, it’s really premature to see some of the cheerleading that’s being done,” said Eric Strong, MD, a hospitalist and clinical assistant professor at Stanford (Calif.) University. “It’s harder to change people’s minds once you have these rosy pictures.”
John Thero, Amarin’s CEO, argued that the imminent release of the drug’s complete picture should alleviate those concerns.
In unveiling topline findings in a news release, he said, the company’s playbook doesn’t diverge from that of other pharmaceutical makers and provides a necessary level of disclosure for shareholders.
But it’s the specifics in the data – for instance, which patients benefited, by how much, their absolute risk reduction and which precise conditions saw improvement – that illustrate whether a product is cost effective, said medical and drug experts.
That’s especially true in the case of Vascepa, whose manufacturer is working hard to convince people the product is clinically superior to ordinary fish oil supplements. Fish oil, which can retail for a few dollars a bottle, has long been promoted as a preventive for heart disease. But the substance has never held up in clinical trials as a way to systematically lower disease risk, said experts.
That’s where Amarin’s product is superior, Mr. Thero said.
The manufacturer has tried to limit competition by seeking to block other fish oil products, arguing to the U.S. International Trade Commission that omega-3 supplements aren’t equivalents, and calling on the FDA to block a chemical component of fish oil, known as EPA or eicosapentaenoic acid, and marketed by a number of supplement companies, from being sold as a dietary supplement. Amarin hasn’t yet prevailed.
Preston Mason, PhD, a biologist and faculty member in the division of cardiology at Brigham and Women’s Hospital, Boston, who consults for Amarin and has advocated on its behalf, argued that ordinary fish oil supplements carry risks because they are not regulated or approved by the FDA, which does oversee prescription drugs like Vascepa.
How Vascepa performs against regular fish oil remains unknown. Amarin’s trial compared the drug against a placebo, not over-the-counter supplements.
Vascepa itself isn’t new. It was approved in 2012 as a remedy for extremely high triglyceride levels, which can put patients at risk for pancreatic problems. But reducing that fat hadn’t been conclusively tied to, say, lowering the risk of heart attacks, or other major cardiac problems.
That link, ostensibly, is what Amarin is trying now to assert. And there’s plenty of money to be made if it succeeds.
As of last December, Vascepa retailed for about $280 for a month-long supply, a list price increase of 43% over 5 years, though the company says its net sale price has stayed the same. (That difference would come if Amarin increased the size of rebates, or discounts it provides, commensurate with price hikes.)
Now, citing the drug’s potentially increased value, Amarin has declined to say whether it will change the price again – though Mr. Thero said he sees greater profit potential if the company increases sales volume rather than price.
This gets at the crux of this debate. If a company makes available the technical details of a product, but only after hyping the findings, and if the details undercut some of that buzz – is it too late?
Khurram Nasir, MD, a cardiologist at Yale University in New Haven, Conn., acknowledged that it’s unclear how effective Vascepa really is, but maintained those ambiguities will be cleared up soon enough.
“As the findings reveal themselves, there will be a lot of discussion around cost effectiveness, and whether this is worth the spend,” Dr. Nasir said.
Dr. Mason, the Amarin scientist, said FDA scrutiny also can alleviate concerns about overhype.
But others worry the perception of Vascepa’s effectiveness is now set.
“People are weighing in with really strong language, without enough information,” said Lisa Schwartz, MD, MS, who codirects the Center for Medicine and Media at Dartmouth Institute in Hanover, N.H., and studies effective scientific communication.
That has both clinical and financial consequences, she added. Doctors are more likely to prescribe a product that’s been heavily promoted, even if subsequent discussion indicates the drug isn’t as powerful as initially implied. And manufacturers can cash in, whether through increased company stock market value or by charging higher list prices.
For Vascepa, the central question is which specific heart conditions saw risk reduction, she and others said. In its news release, Amarin noted a “composite outcome” – that is, the 25% relative improvement encompassed all conditions for which the researchers tested.
“People are saying, Wow, it reduced heart attack, stroke and blah, blah, blah – when it may just reduce the least important one,” said Steven Woloshin, MD, MS, who also codirects the Center for Medicine and Media at Dartmouth.