The news release focuses on a phase 3 clinical trial that evaluated how effective the drug regorafenib was at treating patients who had a form of liver cancer called hepatocellular carcinoma (HCC), and whose HCC had gotten worse despite taking sorafenib — which is currently the only available form of targeted drug therapy for treating HCC. The study, which was recently written up in The Lancet, found that regorafenib extended a patient’s life by just under three months, compared to the use of a placebo. The release addresses these findings in a straight-forward way, which is good. However, the release does not adequately address conflicts of interest related to the study, nor does it sufficiently address cost or potential harms associated with the use of regorafenib.
When patients or their loved ones hear about a “successful” drug trial for a disease they are fighting, it is easy for them to jump to the conclusion that “success” means the disease is cured. That’s not the case for a study like this one. It’s important for people to know that “success” in this case means that patients survive for an additional 2.8 months. We’re glad the release states this clearly. But in order for people to truly make informed choices, they need to understand how a treatment could effect their quality of life. For many patients, it’s also important for them to understand whether a treatment option could create a significant financial burden for their families. And when evaluating any drug study’s findings, it’s important to know what sort of financial ties the researchers have with the pharmaceutical manufacturer.
Cost is not addressed at all. This is particularly problematic because regorafenib (brand name Stivarga) has already been approved, and is on the market, for treating other forms of cancer. For example, an online price guide shows that 84 40-milligram tablets of regorafenib cost $15,817. This is a fair estimate of the cost of treatment, given that the initial treatment regime for patients in the clinical trial receiving regorafenib was 84 40-milligram tablets (four 40-milligram tablets per day for 21 days).
The release is pretty clear on this point, noting that patients who received regorafenib had an overall survival of 10.6 months, compared to 7.8 months for patients who received a placebo.
The release states only that “regorafenib was well-tolerated with manageable adverse events” [emphasis added]. This is an example of the disconnect that can occur between language commonly used among researchers and language that people use in everyday life. For example, of the 374 patients who received regorafenib, seven had deaths that, according to the journal article, “were considered by the investigator to be related to study drug.” Few members of the lay public would be likely to automatically assume that death is a manageable adverse event, even when talking about last-ditch medical treatments for advanced cancers. Other side effects that cropped up in the study, and which could significantly affect quality of life for patients, included hypertension, pain and swelling in the hands and feet, diarrhea and severe bleeding. Fatigue was also present as a side effect but the degree of fatigue is not mentioned.
The release gives a concise, accurate, overview of the study. But the study reports a lot of other issues that aren’t mentioned in the news release. It appears that 75% of the patients in both groups had cirrhosis. The effect of this is not likely to be important since the percentage was the same in both groups. An open question on the study is that the authors used a one-sided statistical test, making it more likely that they would find statistical significance, when it would not be so if they had used the more common two-tailed test (which tests for the possibility of a relationship in both directions). How this statistical change would affect the results is not clear.
The release does note that Bayer funded the clinical trial. However, the release doesn’t tell readers that Bayer manufactures regorafenib. We might be able to let this slide, assuming that Bayer’s ties to regorafenib are inferred, but there’s another problem. The journal article makes clear that a number of the researchers involved in the clinical trial have not only received grants from Bayer, but are also paid advisors and/or consultants for Bayer. That sort of potential conflict of interest is important to note.
The release notes that patients in the study had already been treated with sorafenib, and that sorafenib is the “only other drug with proven clinical benefit” in treating HCC. That’s enough to earn it a satisfactory rating here. However, it would have been stronger if it had mentioned other elements of treatment, such as surgery, chemotherapy and radiation therapy. In addition, hospice or palliative care programs for patients who had run out of treatment options might result in improvement in outcomes.
The release does tell readers that “In January, Bayer announced that the U.S. Food and Drug Administration had granted priority review status for Stivarga (regorafenib) as a second-line systemic treatment for patients with hepatocellular carcinoma.” However, the release doesn’t tell readers that regorafenib has already passed FDA review for treatment of other cancers, nor does it explain to readers what “priority review status” means in terms of when the drug might be used for HCC treatment on a widespread basis.
The release makes clear that this is only the second pharmaceutical treatment for HCC proven to affect patient survival, and outlines what the study findings may mean for future research on how regorafenib may be used in treating earlier stages of HCC.
There are two issues here. First, as noted above under “Why This Matters,” writers need to be cautious when throwing around terms like “successful” — because they can mean different things to different people. When a headline trumpets a “successful” cancer drug, many readers may assume that the drug does more than extend life for 2.8 months. The fact that the release makes clear, in the third paragraph, that we’re talking about an extra couple of months (rather than remission) doesn’t forgive the headline. It would have been much better to have a headline that focuses on specifics, such as “extends life by 2.8 months,” rather than vague terms that can be easily misunderstood. Second, while the clinical trial’s findings are certainly worth reporting, we are rarely pleased to see a news release use vague, over-hyped (and overused) terms like “breakthrough,” as was done here.
Systematic, Criteria-Driven
The tale of two “negative” studies. Or, how spin is applied to make research findings look more impressive. A must read. https://johnmandrola.substack.com/p/the-tale-of-two-negative-studies
The @nytimes chose to interview one of the editors of the @AnnalsofIM (publisher of this paper) who "gushed with extraordinary glee: It’s huge! There are very few things that reduce your mortality by 30%."
Turns out coffee is not one of those few things despite all that glee. https://twitter.com/garyschwitzer/status/1533202075928215558
Comments
Please note, comments are no longer published through this website. All previously made comments are still archived and available for viewing through select posts.
Our Comments Policy
But before leaving a comment, please review these notes about our policy.
You are responsible for any comments you leave on this site.
This site is primarily a forum for discussion about the quality (or lack thereof) in journalism or other media messages (advertising, marketing, public relations, medical journals, etc.) It is not intended to be a forum for definitive discussions about medicine or science.
We will delete comments that include personal attacks, unfounded allegations, unverified claims, product pitches, profanity or any from anyone who does not list a full name and a functioning email address. We will also end any thread of repetitive comments. We don”t give medical advice so we won”t respond to questions asking for it.
We don”t have sufficient staffing to contact each commenter who left such a message. If you have a question about why your comment was edited or removed, you can email us at feedback@healthnewsreview.org.
There has been a recent burst of attention to troubles with many comments left on science and science news/communication websites. Read “Online science comments: trolls, trash and treasure.”
The authors of the Retraction Watch comments policy urge commenters:
We”re also concerned about anonymous comments. We ask that all commenters leave their full name and provide an actual email address in case we feel we need to contact them. We may delete any comment left by someone who does not leave their name and a legitimate email address.
And, as noted, product pitches of any sort – pushing treatments, tests, products, procedures, physicians, medical centers, books, websites – are likely to be deleted. We don”t accept advertising on this site and are not going to give it away free.
The ability to leave comments expires after a certain period of time. So you may find that you’re unable to leave a comment on an article that is more than a few months old.
You might also like