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Washington Post wisely notes that CAR T-cell therapy may cost at least $300,000

Rating

4 Star

Categories

First gene therapy — ‘a true living drug’ — on the cusp of FDA approval

Our Review Summary

The story focuses on a treatment called CAR T-cell therapy, which is under review by the FDA for use in treating children with leukemia who are not responding to standard treatments.

The story does a good job of describing the treatment, how it’s different from similar therapies, and how expensive it is. However, the story could have done a better job of discussing the numbers on both the remission rates and the side effects.

 

Why This Matters

This story looks specifically at using an immunotherapy treatment to tackle treatment-resistant leukemia in young people. And while childhood leukemia is rare, it is the most common form of cancer in children and adolescents. That alone makes the subject matter here of widespread interest.

But the story also makes clear that the CAR T-cell treatment may be only the tip of the iceberg, with an array of other research initiatives aimed at using customized immunotherapy to tackle a variety of cancers. That means that the CAR T-cell treatment is being viewed — fairly or unfairly — as a bellwether for individualized immunotherapy in general.

As a result, the way that the public perceives this type of cancer therapy may be strongly influenced by the way people perceive CAR T-cell treatment for juvenile leukemia cases. That makes it especially important for stories to give readers accurate information, and to place it in a context that helps people understand the associated pros and cons. Overall, we think the story does a pretty good job here.

Criteria

Does the story adequately discuss the costs of the intervention?

Satisfactory

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.”

Does the story adequately quantify the benefits of the treatment/test/product/procedure?

Satisfactory

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.

Does the story adequately explain/quantify the harms of the intervention?

Not Satisfactory

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.

Does the story seem to grasp the quality of the evidence?

Not Satisfactory

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.

Does the story commit disease-mongering?

Satisfactory

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.

Does the story use independent sources and identify conflicts of interest?

Not Satisfactory

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.

Does the story compare the new approach with existing alternatives?

Satisfactory

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.

Does the story establish the availability of the treatment/test/product/procedure?

Satisfactory

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.

Does the story establish the true novelty of the approach?

Satisfactory

The story does a good job here, articulating what makes this treatment novel and how it works relative to other immunotherapy treatments.

Does the story appear to rely solely or largely on a news release?

Satisfactory

The story goes well beyond any news release.

Total Score: 7 of 10 Satisfactory

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