Why hope, hype and headlines should never substitute for clean, clear analysis.
SO-CALLED "RARE DISEASES" are those that affect less than one in 2000 Canadians (.05 percent), and for which companies often sell drugs priced at hundreds of thousands of dollars per patient per year. Parsing the pharmaceutical forecasting literature, I’ve come to understand that the drug market for rare diseases is huge, growing, and seems to be the main area where the industry is putting much of its current research efforts. Why? For the same reason famous bank robber Willie Sutton kept robbing banks: “Because that’s where the money is.”
Money, and lots of it, seems to be at the heart of a narrative that repeats with alarming frequency: along comes a new and expensive drug—characterized in the press as “lifesaving” and essential, yet priced into the stratosphere. It could mean a slight increase in a patient’s quality of life, yet priced at $250,000 per patient per year, how much “quality” are we really buying?
Here in Victoria over the last year, headlines have been filled with heart-wrenching stories featuring drugs like Orkambi for cystic fibrosis, and Ilaris, which treats a rare disease called systemic juvenile idiopathic arthritis (SJIA). Then there was Soliris, which treats atypical hemolytic-uremic syndrome (aHUS), an extremely rare disease. It is among the priciest drugs we’ve ever seen, costing around $700,000 per patient per year. It’s no wonder governments everywhere in the world are reluctant to pay for these medications, and that the stories of patients being denied access make headlines.
Each time a new “breakthrough” drug is either so expensive, so experimental (where there’s little data on the drug’s efficacy and safety)—or both—that the provincial drug plans won’t cover it, there is public outcry. Sometimes the Ministry of Health gives in, and sometimes it doesn’t—leaving one with the distinct impression that political pressure might play a larger role than science when determining whether a new, expensive drug becomes a covered benefit. Would any of us call this an optimal system?
The narrative never seems to change—only the drug names. While some journalists love to advocate for a good cause, what if the cause they are jumping aboard is one where they only know half the story? Maybe instead of providing lifesaving treatment, the new drug actually leads to more people suffering and dying, due to some unknown toxicity. Could that happen? It happens all the time.
The Michener Award is considered one of the most prized honours in journalism, recognizing “meritorious public interest journalism in Canada.” A Michener is like an Olympic Gold Medal for reporters, recognizing the best of the best in what is the highest calling in journalism: the public interest. In 2006, I wrote a letter to the Michener Awards committee complaining about the 2005 winner. I felt that the Globe and Mail writer who won the award did so with a series of articles about a new and expensive breast cancer drug, Herceptin, that was so biased and misleading, it most certainly caused great harm to the psyches of women with breast cancer, as well as wasting huge amounts of money in funding from public drug plans. I felt the articles undermined the independent scientists who were suggesting caution. The committee ignored me.
I based my letter to them on work I and my colleagues did, documenting the reporting of trastuzumab (brand name: Herceptin), a treatment for adjuvant breast cancer, for over a year. Here’s an excerpt of what I wrote: “From our perspective, 2005 was a long summer of frustration as Herceptin generated breathless front page coverage based on a poor explanation of the drug’s absolute benefits, and an improper assessment of risks and uncertainties related to this treatment. We found that the Globe’s news reports of Herceptin routinely broke simple principles of proper pharmaceutical reporting, creating excitement and demand for what are unproven treatments. This has the effect of driving provincial health departments into rapid, yet potentially harmful funding decisions which can have serious adverse effects on patients and the health system; not what you’d call ‘public interest’ journalism.”
Herceptin, used after chemotherapy treatment, was advertised, promoted and lobbied for on the basis of a “50-percent reduction in tumour recurrence for all clinical-trial participants.” The drug was tested in a special population of those with breast cancer: the 30 percent of them who have the HER2 gene, which can affect growth of breast tumours. The women in the trials had been treated for breast cancer, and the drug was prescribed to avoid a recurrent tumour. But that figure of 50 percent is misleading; the Globe (as well as other media outlets) were reporting the relative numbers from the clinical trials. In one of the key randomized trials of the drug, about 10 percent of the women given the placebo developed a new breast tumour over 3 years. Of those who took Herceptin, only 5 percent developed a recurrent breast cancer in that time. Using relative math logic, if you go from a 10-percent risk (placebo patients) down to 5 percent (Herceptin patients), that’s a 50 percent reduction. “Cuts cancer risk in half!” proclaim the headlines.
But whoa! In absolute terms, the drug really only contributes to a five-percent reduction, because 10 minus 5 leaves 5. In other words, the difference in benefit of the drug over the placebo is only 5 out of 100—only one in 20 taking this drug will benefit. Based on those data, doctors would have to give Herceptin to 20 women with this certain type of gene—after being treated for breast cancer—for 3 years, to prevent one recurrent breast tumour.
In the Herceptin stories we examined, another thing was obvious: the harms of the drug were consistently downplayed, or simply not mentioned. We knew (closely parsing the clinical trials) that Herceptin increased risks of congestive heart failure, infection, and vascular disorders. In this case, 3.5 percent more women taking the drug experienced these effects than those who took the placebo. And here’s the kicker: there was no overall improvement in survival in the Herceptin group.
Basically, after a summer of hyped headlines, lobbying, and beating up the Ontario government for not covering Herceptin, it turned out the drug was not a lifesaver. Why was the Ontario government reluctant to pay for it? Maybe because it cost somewhere between $30,000 to $45,000 per patient per year (now about double that), and would “break the bank” of the Ontario drug plan.
The citation for the Globe’s 2005 Michener Award for meritorious public service journalism reads: “One series about the breakthrough breast cancer drug Herceptin prompted provincial government to fast-track the drug approval process and expand use of the drug. It had been restricted to women who were dying of breast cancer.”
Let’s be clear: all the media activity, all the personal heart-wrenching narratives of individuals begging for a drug that they claimed was helping them wage their personal war on cancer, was genuine and heart-felt. But it was also based on a big misunderstanding of the facts. The company made millions on the basis of that one drug, and the media campaign was so powerful, and so overwhelming, even women who didn’t have the HER2 gene started pestering their doctors for prescriptions for Herceptin. (It’s now on BC’s list of approved drugs for treating breast cancer.)
It is possible that certain people may be helped by new and expensive drugs. It is also possible they may be harmed, or die taking the new treatment—at the expense of millions of dollars wasted. Original drug trial reports contain both spin and bias, and it seems most reporters aren’t asking enough tough questions. Tragically, many vulnerable patients get an unnaturally rosy picture of a new medication through these media reports and end up feeling desperate—that without this wonder-drug, they will surely die.
A few years after the Herceptin debacle, I saw a major Canadian study which emerged with this headline: “What we know of breast cancer drugs may be spin & bias.” This examination found that of 164 major cancer drug trials, a third were biased in how they reported the benefits of the treatment, and two-thirds spun the reporting of the toxic effects, downplaying or ignoring them. In other words, what we discovered around the Herceptin story is not an outlier. Spin and bias are all part of the packaging around a new drug.
Noteworthy villains in any media/drug saga include the academic researchers who produce slanted reports—sometimes quite unreliable—of the drugs they study; and the “advocacy” journalists who allow themselves to be unwittingly employed as part of the drug company’s PR strategy. The collateral damage is clear—just ask the terrified patients who get caught up in the corporate profit machine.
Here’s the lesson I learned many years ago, which we must keep relearning: we need clean, clear, health journalism as urgently as we need clean, clear water. Our lives depend on it.
Alan Cassels is taking a break from pharmaco-journalism for the time being. He continues to work as a reviewer with healthnewsreview.org which analyzes and evaluates health reporting, and now works at UBC.