This is basically a stock market performance story dressed up as a clinical trial story, one of many such stories where someone on the business desk is asked to write about a "breakthrough" for the purposes of giving investors/readers some insight on where to make their next investment move. Because the reporter is well above average, the story is packed with information about the trial, about the drug, about hepatitis and about the company. But there is quite a bit of information missing that would be helpful for patients who, most likely, aren’t going to see perhaps unfounded hope in phrases such as, "a new era in treating a sometimes fatal disease."
Even in a stock market story, it is important to get the clinical part right. And, especially in a stock market story, it is important to get the cost part right.
If you can estimate that a drug is going to have sales of "$3 billion a year by 2015," then you can estimate how much it is going to cost patients and how much more it is going to cost over existing treatments. This story does neither.
The story talks a lot about the benefits but doesn’t really quantify them. Given that 1,095 patients were in the trial, let’s assume that half were in the drug group and half in the placebo group. Of the drug group, let’s assume half were in the 24 week group and half were in the eight week group. By our math, that means that about 154 people had better outcomes than those in the control group. Also, the story does a pretty good job of walking people through the results, but the story is framed as a "new era" versus an old, torturous treatment. Only when you get to the end of the story do you realize that the new drug only works in combination with the old, torturous treatment. So the torture is not going away, it’s just taking on a new layer of medication, and new costs.
Relapses after completing therapy are common with standard therapy of IFN and ribavirin. In a recently published study in the NEJM involving patients who had previously failed IFN and ribavirin, there was a 76% "cure" at the end of the treatment period, but only a 51% "cure" 24 weeks after the end of treatment. So in the data reported in this article, is the 75% "cure" 24 weeks after the end of treatment or is it at the end of the treatment period? Finally, the information presented doesn’t translate into how many patients need to be treated to have one meaningful cure.
The story makes the standard treatment sound terrible. But it glosses over the side effects of the new treatment, saying only that it causes a "nasty rash." Where is the rash? That can make a world of difference. Does it cause the skin to break and bleed? Does it interfere with basic functions like sight, breathing, eating? It says that 7% of the people in the drug group dropped out, while about half that percentage dropped out of the placebo group. Again, some absolute numbers would be good here, as would some context about what percentage of patients experience side effects with the standard treatment or with the competing drug in the same family of drugs mentioned earlier in the story. And the story could have been more clear that all patients get the standard therapy, IFN and ribavirin. So they will face the side effects of the current regimen, PLUS the side effects of this new agent.
There are a lot of numbers in this story, but there are two crucial numbers missing: the absolute risk reduction and the number needed to treat. It says at the top that "about 75 percent of patients in the trial who got the standard dose of Vertex’s drug in combination with the existing treatment were essentially cured, compared with 44 percent of those who got only the existing therapy." Later it says that "in Vertex’s trial, about 70 percent of those who achieved an effective cure were able to do so in 24 weeks, or about six months." Then it says "Patients got either 12 weeks or eight weeks of telaprevir or a placebo. The standard therapy of interferon and ribavirin was given for either 24 weeks or 48 weeks, depending on how the patients were faring after four weeks and 12 weeks. Of those who got 12 weeks of telaprevir, 75 percent had a sustained viral response, meaning the virus was not detectable in their blood 24 weeks after completing treatment. That is seen as essentially a cure. The rate was 69 percent for those who got eight weeks of telaprevir." More on this later.
Finally, it is not clear where any of this data came from. In searching for the primary source information, it appears to come from the company itself rather than from publication in a medical journal.
The description of the scope of the disease is fairly standard and well sourced. "Some 2.7 million to 3.9 million Americans are estimated to have hepatitis C, and 12,000 die from it each year, according to a recent report by the Institute of Medicine. The disease can cause cirrhosis, which is a scarring of the liver, as well as liver cancer. The virus is transmitted by blood, so most people who have hepatitis C either have injected illegal drugs or received blood transfusions or blood products before the early 1990s." We wish we’d seen a source on the idea that most recent Hep C patients have injected illegal drugs.
We would have liked to have seen independent voices inject more context into the story, but the reporter did at a minimum take these results to another legitimate researcher. The reporter also went to a patient advocacy group that, unfortunately, just spat out the standard "we welcome any cure" spiel.
The comparison could have been more detailed, but, you do get the picture that this new drug will simply be an add on to existing therapies, albeit with the potential for cutting down the treatment time considerably.
The second word in the story is "experimental." It talks about the drug being in three different Phase 3 clinical trials and about the company looking forward to applying for approval from the FDA. What it does not do is make it clear how quickly the drug could be brought to market (or slowly) especially given the problems of side effects also noted in the story.
The storyt makes clear that this is a new treatment that is not yet FDA approved – a novel agent and class.
Not applicable because we can’t be sure of the extent to which a news release impacted this story. But we also can’t figure out what the source is of the information provided unless it’s the company.