The story tackles this issue head on, as here: “Novartis has not disclosed the price for its therapy, but analysts are predicting $300,000 to $600,000 for a one-time infusion.” This is satisfactory, but barely. Here’s how the story addresses benefits for the CAR T-cell treatment: “In the pivotal trial testing the therapy in almost a dozen countries, 83 percent of patients went into remission. A year later, two-thirds remained so.” That information would be much more useful if it placed those numbers in context. For example, how many patients were enrolled in the study? 83 percent could be 13 out of 16 patients, or it could be 1,328 patients out of 1,600. And how does 83 percent compare to standard treatment? Similarly, is the story telling us that two-thirds of all patients were still in remission a year later? Or did it mean that two-thirds of the 83 percent were still in remission a year later? Contextual information would have provided some much-needed clarity here. The story notes: “The revved-up immune system [that results from the CAR T-cell treatment] becomes a potent cancer-fighting agent but also a dangerous threat to the patient. Serious side effects abound, raising concerns about broad use.” The story then goes on to discuss those potential harms more thoroughly. Ordinarily, this would be sufficient for a Satisfactory rating. But given the severity and frequency of serious, life-threatening events, the story needed numbers: What percentage of patients in the trial experienced a serious immune reaction? How many died? There are also plenty of unknowns with a new cancer treatment, such as lack of data on long-term prognosis. The story refers to a “pivotal trial testing the therapy in almost a dozen countries,” but offers little additional information. While the story offers an in-depth history of the research that led up to this treatment, readers are given very little information about the trials that provide insight into how well the treatment works and its associated risks. There’s really no disease mongering here, though there is one point that we’d like to single out. The story states that the CAR T-cell treatment “initially would be available only for the small number of children and young adults whose leukemia does not respond to standard care. Those patients typically have a grim prognosis” [emphasis added]. What does it mean to “typically have a grim prognosis”? It’s a fine turn of phrase, but it doesn’t actually convey much information — and it’s scary. In a case like this, it would likely be better to let the numbers speak for themselves. Or, if a story does include this sort of language, to back it up with some numbers. All of the sources in the story are patients, parents of patients, are associated with the pharmaceutical industry, or were involved with the relevant research being discussed. That said, the sources — and their conflicts of interest — were all clearly identified. Still, the story would have benefited from some independent, expert input. First of all, the story makes clear that CAR T-cell treatment — if approved — would be available only to those for whom conventional treatments have not worked. That’s a key point. Second, the story does articulate the difference between this form of immunotherapy (which is customized to the patient) and other immunotherapy techniques, which are so-called “checkpoint inhibitors” and are not individualized. As mentioned earlier, we would have liked to have seen more data on the success rates of standard treatment vs these treatments. The story clearly lays out where this treatment is in the FDA review process, how that process may unfold, and what this may mean for future availability. In addition, the story explains that CAR T-cell treatment would not be rolled out universally, but — if approved — would be available only through designated treatment facilities that have the relevant expertise. The story does a good job here, articulating what makes this treatment novel and how it works relative to other immunotherapy treatments. The story goes well beyond any news release.
First gene therapy — ‘a true living drug’ — on the cusp of FDA approval