The headline seemed too good to be true: Amarin fish oil capsule shows dramatic benefit for cardiovascular patients.
"In a stunning clinical trial result that upends years of skepticism about the long-term heart benefits of products containing omega-3 fatty acids, Amarin's Vascepa significantly reduced the risk of deaths, heart attacks, strokes, and other serious cardiovascular events compared to placebo," wrote Adam Feuerstein for medical news site STAT. Matthew Budoff, a cardiologist at UCLA and a Vascepa study investigator, told STAT, "This is absolutely the most significant study in the field of cardiovascular risk reduction."
This story referred to a new study, called Reduce-It, that purported to show a 25 percent relative risk reduction for cardiovascular patients taking a fish oil supplement. But, like many cardiologists, I was surprised to see this news, because the results of the trial are not due until November, when they are scheduled for release at the American Heart Association meetings.
I have been looking forward to these study results. Full disclosure: I have been a long-term believer in the possible beneficial effects of omega-3 fatty acids (aka fish oil), only recently concluding that overwhelming scientific information suggests that fish oil supplements may be worthless for most people. (The one exception is if blood fats called triglycerides are elevated.) I am also a co-investigator in an omega-3 trial called Strength, which looks at a different omega-3 product, similar to Vascepa, whose results have not yet been released.
So, it seemed important to take a deeper dive into what sounded like a fishy story. In fact, the results of the study have not yet been released, but instead were what the pharmaceutical industry calls a "topline result" — a news release put out by a company stating that an upcoming trial either has met or outperformed its goals. Topline results are considered a way for drug companies to partially leak the results of trials to excite their investors.
The supposed purpose, according to the Wall Street Journal, is to comply with securities requirements of timely disclosures of material information. But, the company cannot give out too much information or it risks losing the medical cachet of presenting results to doctors and scientists at a major meeting, such as that AHA meeting in November. These releases can also be very self-serving for the pharmaceutical company. Not coincidentally, the price of Amarin stock went up more than 200 percent in the hours after this news release.
So, what exactly is Vascepa, and will it finally show that fish oil supplements are helpful? It is a prescription brand of omega-3 fatty acids. Most fish oil is comprised of two kinds of fatty acids, called DHA and EPA. Vascepa, which costs about $2,400 a year, is pure EPA, and was approved by the Food and Drug Administration in 2012 for people with very high triglyceride levels. Up to now, there has been no proof that treating high triglyceride levels with fish oil can decrease the chance of having a heart problem.
If the trial does show (when full results are released in November) that Vascepa decreases this risk, that is important news. But the news release did not include a couple of important facts. First, the people studied in the trial (and in the upcoming Strength trial) are only people who have known heart disease or many risk factors; are already treated with statins to lower their cholesterol; and who have elevated triglycerides. The results would not be applicable to others.
Second, other trials have not lived up to the hype of topline results. For example, Amgen announced positive topline findings for the cholesterol drug Repatha in 2017; there was disappointment when the actual study was presented several months later.
Third, in some cases, these sneak peaks at trials have led to cases of insider trading. The Wall Street Journal wrote, "The window between topline report and full disclosure is a risky time for drug-company insiders to trade shares."
Finally, none of these incomplete tidbits can be vetted by independent scientists and physicians until all of the results are released.
The bottom line: The study results, when released, may show that people will benefit from Vascepa, and potentially help some people live longer. But, the process of releasing partial results through a "topline" report is inherently flawed and self-serving, can cause misleading headlines, and is ripe for abuse.