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New York Times is upfront about risk of ‘absolutely crazy’ prices for gene therapy treatment


4 Star


In a First, Gene Therapy Halts a Fatal Brain Disease

Our Review Summary

This story is one of two that we are reviewing (the other is from the Associated Press) that focuses on a recent paper published in the New England Journal of Medicine. At issue is a novel gene therapy used to limit or eliminate symptoms related to the disease adrenoleukodystrophy (ALD).

The New York Times story does a good job of describing everything that led up to the study, does a fair job of describing the study itself (and its results), and highlights the potential price tag of this treatment. However, the story doesn’t make clear how far this treatment is removed from widespread clinical use or the outstanding questions that remain about the treatment — such as the fact that we don’t know if the benefits are temporary.


Why This Matters

Left untreated, ALD often leads to death within 1-10 years of diagnosis. And there are precious few treatment options available. What’s more, the primary treatment, bone marrow transplantation, relies on the availability of a donor — and poses significant health risks of its own. All this makes new treatment options incredibly important for ALD patients and their loved ones. However, the fact that we are talking about the life and well-being of patients — usually children — also makes it incredibly important to place the work in context. Limitations, availability and unanswered questions are always key points worth making, and especially so when many parents are eager for information about something that may affect the health and well-being of their children.


Does the story adequately discuss the costs of the intervention?


The story tackles this issue head on, though what they report may not be comforting for those affected. Here’s how they address the issue: “The results of the new study also give rise to a concern that is becoming a regular feature of gene therapy work and other new biotech therapies: How much will this treatment cost? Bluebird Bio is not saying…[but one scientist involved in the study] expects the price to be similar to the hundreds of thousands of dollars it costs for a bone-marrow transplant.”

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


The story describes the outcomes for all 17 patients who were enrolled in the study, 15 of whom did not have symptoms of ALD after two years.

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

Not Satisfactory

Potential adverse effects were not adequately addressed. The story did do a good job of highlighting concerns about gene therapies in general, pointing to problems that have cropped up in previous, unrelated gene therapy trials. And it mentioned that bone marrow transplants have risks. However, it didn’t address potential harms associated with this study in particular. According to a supplement of the journal article, side effects ranged from fever and seizures to gastroenteritis. The side effects listed in the supplement also included those for one patient (2018), whose ALD symptoms worsened significantly after treatment began. It’s not clear what effect, if any, the treatment had on the relevant patient.

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

Not Satisfactory

The story does an adequate job of describing the study, but it’s missing at least one key point: Namely, the story doesn’t make clear that the study can’t prove ALD is “cured” in these patients — a longer follow-up time is needed to determine what the long-term impact of the gene therapy treatment will be. We also don’t know whether there will be significant adverse effects from the therapy (even if it is curative) in the long run.

Does the story commit disease-mongering?


No disease mongering here.

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


The story includes input from multiple independent sources, which is good. The story also notes that the relevant study was funded (in part) by Bluebird Bio — a company that hopes to market the relevant gene therapy.

However, the story does not note that the principal investigator for the study, David Williams, has a conflict of interest. According to an appendix of the journal article, Bluebird Bio also funds other projects by Williams. The company has also licensed one of Williams’ patents. That doesn’t necessarily mean that anything in the study is shady, but conflicts of interest should be acknowledged.

Does the story compare the new approach with existing alternatives?


The story did a good job of describing other treatment options — including bone marrow and cord blood transplants — as well as the associated risks.

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

Not Satisfactory

The story doesn’t make clear that this treatment is, at the very least, years away from clinical use. The story notes only that Bluebird Bio is “in hopes of marketing gene therapy for ALD.” This line, found more than halfway through a 1,300 word story, isn’t sufficient. This is an area where the Associated Press story fared better, noting that the FDA “requires gene therapy participants to be monitored for 15 years, so these patients will continue to be studied.” The AP story also notes that Bluebird Bio “plans to seek approval of the therapy in the U.S. and Europe.” The AP was stronger on this criterion.

Does the story establish the true novelty of the approach?


This is a strong point for the story, which provides a fairly detailed background on how this treatment technique developed over time, how the treatment works and what sets it apart from other treatment options.

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


The story goes well beyond a news release.

Total Score: 7 of 10 Satisfactory


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