The story was generally fairly complete. But the cheerleading drowned out the caveats in our view.
It strikes us as a silly and empty projection to state in the story’s 2nd sentence:
If these initial results pan out in larger trials, the drug could prove to be more effective than statins, the widely prescribed drugs which have been very successful in lowering levels of LDL – or bad cholesterol.
These were small, short-term Phase I studies. Let’s wait until more data are in hand before the coronation.
The story noted that two editorial writers “caution that it will remain unclear how important this class of drugs will be. The cost of this drug will also play a role in determining which patients might use it.”
But better cost context could have easily been provided.
The story stated that “the subjects who received REGN727 had a striking reduction of 60 to 65% in LDL cholesterol, according to (the lead researcher who characterized the results of the trial as ‘pretty dramatic.’ ”
The story also quoted an American Heart Association spokesman as calling this “game changing science” – putting that phrase in the headline as well – and “a very important breakthrough.”
To describe results of potential efficacy from a small Phase I study this way without emphasizing to the general public that Phase I studies aren’t designed to focus on efficacy is troublesome.
In fact, MedPage Today ended its story with the reminder: “Given the small number of subjects and the short duration of exposure, our ability to evaluate the safety profile of REGN727 in these trials was limited,” the researchers cautioned.
If a Phase I trial, which is designed to measure safety and not efficacy, is too small and short to fully evaluate safety, then making bold claims about efficacy is even more of a reach at this point. A consumer-targeted story needs to make such points with emphasis. This story did not.
The story stated that the researchers found that the drug was safe and “None of the subjects who received REGN727 discontinued the study because of adverse effects.” While true, it does not give the whole picture. Here’s what the investigators said:
“The proportions of subjects who had at least one treatment-emergent adverse event were similar among subjects who received intravenous REGN727 and those who received placebo. As compared with subjects who received placebo, a higher proportion of subjects who received subcutaneous REGN727 had an adverse event in the single-dose and the multiple-dose studies. Headache was the most common adverse event.”
It didn’t include the caveat that the MedPage Today story did:
“Given the small number of subjects and the short duration of exposure, our ability to evaluate the safety profile of REGN727 in these trials was limited,” the researchers cautioned.
But it did report:
However, without long-term safety data and evidence that PCSK9 inhibitors truly help prevent heart disease, (two editorial writers) caution that it will remain unclear how important this class of drugs will be.
Good excerpts from the story regarding the quality of the evidence:
Weaker points in the story regarding the quality of the evidence:
No disease mongering.
The story did quote the editorial writers and did seek comment from an American Heart Association spokesman.
But the story did not report, as MedPage Today did, that: “Many of the co-authors were employees of Regeneron, one of whom jointly wrote the paper with Stein.”
We’ll give the story the benefit of the doubt.
Superficial comparisons were included:
- If these initial results pan out in larger trials, the drug could prove to be more effective than statins, the widely prescribed drugs which have been very successful in lowering levels of LDL – or bad cholesterol.
- A PCSK9 inhibitor, Stein said, differs from statins “because it’s unlike any other drug. With statins you get toxicity – with these drugs we don’t see any side effects with the antibody.”
Again, we’ll give the story the benefit of the doubt on this but it is too early to even suggest that the antibody won’t have any side effects.
It’s clear from the story that this is an experimental approach.
The story explained that “Other companies including Amgen, Merck & Company, Novartis and Pfizer, are also hot on the trail developing their own PCSK9 inhibitor drugs.”
We don’t think the story relied solely or largely on a news release.