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

Rating

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

The story concentrates 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 youthful people. And while childhood leukemia is uncommon, 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 peak of the iceberg, with an array of other research initiatives aimed at using customized immunotherapy to tackle a multiplicity 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 significant 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 scarcely. 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, eighty three 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 probe? Eighty three percent could be thirteen out of sixteen patients, or it could be 1,328 patients out of 1,600. And how does eighty three 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 eighty three 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 meticulously.

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 slew of unknowns with a fresh cancer treatment, such as lack of data on long-term prognosis.

Does the story seem to take hold of 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 extra 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 truly no disease mongering here, however there is one point that we’d like to single out. The story states that the CAR T-cell treatment “primarily would be available only for the puny number of children and youthfull adults whose leukemia does not react 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, accomplished input.

Does the story compare the fresh treatment with existing alternatives?

Satisfactory

Very 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. 2nd, the story does articulate the difference inbetween this form of immunotherapy (which is customized to the patient) and other immunotherapy mechanisms, 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 flipped 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 treatment?

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 emerge to rely solely or largely on a news release?

Satisfactory

The story goes well beyond any news release.

Total Score: seven of ten Satisfactory

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We Welcome Comments. But please note: We will delete comments left by anyone who doesn’t leave an actual very first and last name and an actual email address.

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We will delete comments that include individual attacks, unfounded allegations, unverified claims, product pitches, profanity or any from anyone who does not list a utter name and a functioning email address. We will also end any thread of repetitive comments. We don”t give medical advice so we won”t react to questions asking for it.

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There has been a latest burst of attention to troubles with many comments left on science and science news/communication websites. Read “ Online science comments: trolls, trash and treasure .”

“Shed light, not just warmth. Facts, challenges, disagreements, corrections — those are all fine. Attacking the person, instead of the idea or the interpretation, is neither acceptable nor helpful.”

We”re also worried about anonymous comments. We ask that all commenters leave their total name and provide an actual email address in case we feel we need to contact them. We may delete any comment left by someone who does not leave their name and a legitimate email address.

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The capability to leave comments expires after a certain period of time. So you may find that you’re incapable to leave a comment on an article that is more than a few months old.

You might also like

Animal studies can expose significant scientific insights and, when appropriately reported, can provide useful news…

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

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

Rating

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

The story concentrates 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 youthfull people. And while childhood leukemia is infrequent, 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 peak of the iceberg, with an array of other research initiatives aimed at using customized immunotherapy to tackle a multiplicity 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 significant 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 hardly. 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, eighty three 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 examine? Eighty three percent could be thirteen out of sixteen patients, or it could be 1,328 patients out of 1,600. And how does eighty three 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 eighty three 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 meticulously.

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 slew of unknowns with a fresh cancer treatment, such as lack of data on long-term prognosis.

Does the story seem to take hold of 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 extra 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 truly no disease mongering here, tho’ there is one point that we’d like to single out. The story states that the CAR T-cell treatment “primarily would be available only for the puny number of children and youthfull adults whose leukemia does not react 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, pro input.

Does the story compare the fresh treatment with existing alternatives?

Satisfactory

Very 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. 2nd, the story does articulate the difference inbetween this form of immunotherapy (which is customized to the patient) and other immunotherapy technics, 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 spinned 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 treatment?

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 show up to rely solely or largely on a news release?

Satisfactory

The story goes well beyond any news release.

Total Score: seven of ten Satisfactory

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Animal studies can expose significant scientific insights and, when appropriately reported, can provide useful news…

This story discusses the state of evidence for neurofeedback for treating depression, ADD, anxiety, and…

This story does a good job putting a fresh explore on chondroitin for knee ache in…

Comments

We Welcome Comments. But please note: We will delete comments left by anyone who doesn’t leave an actual very first and last name and an actual email address.

Our Comments Policy

We welcome comments, which users can leave at the end of any of our systematic story reviews or at the end of any of our blog posts.

But before leaving a comment, please review these notes about our policy.

You are responsible for any comments you leave on this site.

This site is primarily a forum for discussion about the quality (or lack thereof) in journalism or other media messages (advertising, marketing, public relations, medical journals, etc.) It is not intended to be a forum for definitive discussions about medicine or science.

We will delete comments that include individual attacks, unfounded allegations, unverified claims, product pitches, profanity or any from anyone who does not list a total name and a functioning email address. We will also end any thread of repetitive comments. We don”t give medical advice so we won”t react to questions asking for it.

We don”t have sufficient staffing to contact each commenter who left such a message. If you have a question about why your comment was edited or eliminated, you can email us at [email protected] .

There has been a latest burst of attention to troubles with many comments left on science and science news/communication websites. Read “ Online science comments: trolls, trash and treasure .”

“Shed light, not just fever. Facts, challenges, disagreements, corrections — those are all fine. Attacking the person, instead of the idea or the interpretation, is neither acceptable nor helpful.”

We”re also worried about anonymous comments. We ask that all commenters leave their utter name and provide an actual email address in case we feel we need to contact them. We may delete any comment left by someone who does not leave their name and a legitimate email address.

And, as noted, product pitches of any sort – pushing treatments, tests, products, procedures, physicians, medical centers, books, websites – are likely to be deleted. We don”t accept advertising on this site and are not going to give it away free.

The capability to leave comments expires after a certain period of time. So you may find that you’re incapable to leave a comment on an article that is more than a few months old.

You might also like

Animal studies can expose significant scientific insights and, when appropriately reported, can provide useful news…

This story discusses the state of evidence for neurofeedback for treating depression, ADD, anxiety, and…

This story does a good job putting a fresh explore on chondroitin for knee agony in…

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