This CNN story covers the FDA’s third-ever approval of a gene-editing treatment — Luxturna, which treats a rare eye condition that can lead to blindness — from the angle of its exorbitant cost. Treatment costs aren’t covered as often as they should be in the popular press, even though they can drive insurance premium hikes, high deductibles, and other healthcare costs, so this story is a refreshing outlier.
However, the article inadequately explains how the treatment is done, how well it works, and for how long — all crucial information when deciding whether an effectively $850,000 procedure is worth it. (Note: Many stories, including CNN’s, used “$425,000 per eye”–but treatment appears to be recommended for both eyes.)
This story matters for two reasons: 1) the extreme price tag of the therapy, and 2) the novelty of the treatment, being only the third gene-editing technology ever approved by the FDA for use in humans. The cost especially makes it important for news stories to explore how effective the therapy is, and the unknowns–such as the long-term performance. This story provided lots of important details, but not quite enough to help a person answer: Is it worth nearly a million dollars?
The price tag of this new treatment is the focus of this article. The story also discusses the models of treatment proposed by the manufacturer. Well done.
Two things, though: It wasn’t clear from the story if the patients in the trial had both eyes treated, which would make the more accurate price tag $850,000 vs the talking point of “$425,000 per eye.”
And does this cost include everything–surgical care costs (operating room, anesthesia, etc), physician payments, and lab costs?
The story stated that 27 of 29 phase 3 clinical trial patients experienced “a gain in functional vision as assessed by a mobility test performed in a maze.” That’s not enough to give readers a sense of scope of the benefits. How did that compare to the control group? And what was everyone’s vision like before? Given the extreme cost of the therapy, it would be useful to provide plenty of details to explain if the cost is worth the results.
For example, it would have been helpful to point out that the way the researchers measured success–completing a maze–is not the gold standard for measuring vision improvement. Instead, measuring visual acuity, such as the ability to read different-sized letters, is more common. Why was it different for this study?
The story mentioned potential harms of the therapy. We do think the story should have mentioned that we don’t know much about the long-term effects and safety of using this technology, however.
The story didn’t provide sufficient discussion of the evidence–what are the limitations to the evidence conducted so far?
The story made it clear that this is a very rare genetic disease that leads to blindness.
The story squeaked by on this criterion, because it quoted a news release by the Patients for Affordable Drugs group that provides important counterbalance to the drug company’s claims. The story would have been stronger if it had contained more input from an independent medical expert who could comment on the veracity of the evidence itself.
[Editor’s note: This criterion was updated on 1/11/18 to reflect the correct rating. It was previously rated “Not Satisfactory.” This change does not affect the overall rating.]
Effective alternative treatments for RPE65 don’t exist yet, and — while this may seem obvious to savvy readers who understand gene-editing technologies — the story should have clearly stated this for the vast majority of people who aren’t familiar with the basics.
The story didn’t discuss where one can have access to this treatment. It likely will be very limited. And will it be available to anyone with this condition, or will some people be ruled out?
The story noted how Luxturna is only the third gene-editing treatment ever approved by the FDA. It also gave readers important context by briefly noting and comparing the new drug to another gene-editing treatment and its cost and payment methods, i.e. Kymriah for leukemia cancer. Good job.
This story contained information not available in the Spark Therapeutics news release.