This is a story about the early promise of gene therapy failing to translate into an array of new clinical treatments. In a way, the story mirrors the problems with gene therapy that it describes. It promises to be a powerful antidote to all the hype surrounding gene therapy. It provides some targeted information relevant to a subset of patients, but it ultimately does not deliver a very well rounded picture of the drug it describes, skipping over specific details about benefits, harms, availability and true novelty. We give NPR a lot of credit for taking on the topic at all and admire the economy of storytelling for such a complicated topic. At the same time, the story may have covered more ground in the same amount of time by including some independent voices and providing a few hard numbers to back up claims that the drug was “remarkable” and “stunning.”
Gene therapy continues to garner hyperbolic claims. We wish more reporters would follow the lead of this story and showcase the “long, hard slog,” as this story describes it. One of the best aspects of the story is how it takes on the notion of “a cure.” It quotes a patient who is taking the drug as saying, “Everyone talks about curing a disease — cure CF, cure these other diseases. [But] Kalydeco controls CF at the basic defect, so I’m OK with the other ‘c’ word, control, because I’m living it and I’ve never felt better in my life.” While we wish there were more evidence provided about how well the drug controls the disease, we applaud the way the story shifts the focus away from finding silver bullets toward finding treatments that can improve patients’ lives, even if they still suffer from the underlying disease. Sometimes control is a more achievable goal.
It doesn’t come until the last sentence in the story, but it also leaves listeners and readers with a pretty sobering reality: “The time from gene discovery to successful drug may be shortening, but there are only a handful of successful drugs so far, and for a while at least, the appearance of new ones will be slow. They’re also likely to be expensive. Kalydeco costs in the neighborhood of $300,000 per year.”
We kept reading this piece and listening to the audio for any sort of data to back up some of the claims made. We understand that numbers don’t always translate well on the radio, but even a supplementary graphic illustrating how effective this drug is would have provided some much needed context. The story does say, “It only works on a small subset of people with CF. They have to have a particular mutation in the CF gene, or the drug is of little use.” Then it says, though, “But for people who do have that mutation, the drug works remarkably well.” How small is that subset? And how well does it work?
There was no mention of harms in the story. We think that it’s especially important to include information about harms when you feature a patient who describes the benefits of the drug by saying “something huge had happened. It was just something I had never felt in my life before.”
The prescribing information for the drug states: “Overall, the most common adverse reactions in 353 patients with CF were headache (17%), upper respiratory tract infection (16%), nasal congestion (16%), nausea (10%), rash (10%), rhinitis (6%), dizziness (5%), arthralgia (5%), and bacteria in sputum (5%).”
The story doesn’t get into the evidence in depth, but we give the story high marks for the way it frames the gene treatment discovery process. We have seen very few stories tackle the topic in this fashion, and it is a service to the public to cut through the hype and provide a more clear-eyed picture. For example, the story points out that the drug company “tested over 600,000 chemicals in cells with the defective protein that causes cystic fibrosis” and that, ultimately, the company “knew there was a problem with Kalydeco: It only works on a small subset of people with CF. They have to have a particular mutation in the CF gene, or the drug is of little use.”
There is no disease mongering here. The story does a nice job describing the disease in a short space.
The reporter clearly did a lot of digging to craft this very well told piece, but the only two people ultimately quoted are Fred Van Goor at Vertex Pharmaceuticals, which developed the drug in question, Kalydeco, and Emily Schaller, who was in a clinical trial for the drug and apparently saw a big benefit. No truly independent expert source was quoted.
There’s no mention of what people typically do to treat cystic fibrosis and what advantage, if any, this drug brings. The mention of the clinical trial says that a patient was given a placebo and then the drug. But have tests been done comparing existing treatments to this new treatment?
A Wall Street Journal story on the drug last February at least mentioned that until now patients took “medicines that only tackled the symptoms of the disease, such as antibiotics to treat the infections. Patients typically spent a few hours each day wearing vibrating vests to dislodge mucus build-up.”
Readers can infer that this drug is available from the final sentence that establishes cost. It would have been easy for the story to specify clearly that the drug is now available by prescription, with some estimate of how widespread is the use. Nonetheless, we’ll give the story the benefit of the doubt.
We’re torn about how to apply this criterion to this story. The story doesn’t make any overt claims of novelty – nothing about being “first” or “only.” We’ve already dinged the story on the “Compare With Alternatives” criterion, so we’ll rule this one Not Applicable.
The story does not rely on a press release.
Systematic, Criteria-Driven
Monday at #ADA2021, an infectious disease epidemiologist, a journalist, and a researcher shared perspectives on communication of risk in the era of COVID-19: http://ow.ly/ROfR30rMeE7 @AstleyCM @garyschwitzer @berthahidalgo @ADA_DiabetesPro
Shoutouts to @susan_bewley @drkevinknopf @MichaelBaum11 in this call for more than fawning coverage from press releases for these kinds of stories.
Also, linked history inside goes back 5 years just on Grail apparently trying to become Holy Grail. https://twitter.com/garyschwitzer/status/1408849696416841731
Comments (3)
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Joe Palca
January 7, 2013 at 10:29 amThanks for the thoughtful comments. They are all valid. I wonder, however, if you’ve ever considered the possibility that independent experts were consulted, just not quoted in the piece? I can assure that was the case for this story. Does a reporter have to prove to his audience that he has done due diligence in covering the story, or can the audience trust his professionalism?
You guys provide a valuable service.. Keep up the good work.
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