This ABC story undertakes a worthy goal — throwing a dose of reality on a new drug for ALS that’s being billed as the first to be approved in 22 years. This story attempts to ground the glowing headlines by right away focusing on the drug’s high cost, intensiveness of administering, and importantly, to remind readers that this is not a cure.
But, the story didn’t explain that this drug was only shown to work in a very specific small subset of ALS patients. The description of this subset of patients is not given, suggesting to readers that this drug could work for any ALS patient, which is misleading.
Amyotrophic lateral sclerosis (ALS) is a progressive neurologic disease in which motor neurons die and result in people losing the ability to speak, eat, move, and breathe. Some 5,000 people are diagnosed each year in the U.S. and average survival is three years. Clearly there’s a need for better treatments — if not a cure, a way to slow down the progression with more oomph that we currently have. The only other FDA-approved drug for ALS is riluzole, which slows progression and can increase survival by 2-3 months. This new drug comes with many limitations, and patients deserve to know all of them.
We applaud the story for tackling the costs of not just treatment but of the nursing care that would be required. From the article: “the cost is around $1,000 per infusion – and treatment, which involves multiple infusions, may total about $146,000 per year” — and that’s just for the drug, not the in-office visit to get the hour-long infusion, for as many as 14 days in a row.
“This is just for the drug; you also need a nurse and infusion equipment,” McCluskey said. “We don’t know who is going to cover it or what criteria will be used to decide who gets it.”
From the article: “Researchers found that those getting edaravone had a 33 percent reduction in their rate of decline in physical function.”
This sure sounds good, as does the independent expert’s characterization: “Thirty percent is pretty robust.” Still to get a Satisfactory rating here, we’d want to know how many of the patients in the treatment group saw that benefit. All of them, or some of them? Also, the “33%” difference represents differences in the change of a scale used to measure ALS severity (example), and it is unclear if this difference means something clinically important for ALS patients, such as walking, swallowing or difficulty breathing.
The story also should have pointed out that this study wasn’t just a general group of ALS patients. It was limited to a subset of patients–which we address in the evidence quality criterion in this review.
Harms were not discussed. They include bruising, gait problems, headaches, skin rashes, respiratory failure, among others.
The story should have made it very clear that this study was limited to patients who had a specific type of ALS disease process. In a commentary that accompanied the study, we learn that “less than 7% of patients with ALS would be eligible for enrollment.”
The commentary also pointed out that this was a short study “was short, comprising 24 weeks in total, preceded by a 12-week observation period. Most trial guidelines of the European Medicines Agency propose a duration of at least 12–18 months.”
No disease-mongering here. The article provides factual information on how many Americans are diagnosed with ALS each year and that typical survival times are two to five years. These were welcome details. However, if the drug does work, it is only useful in a subset of ALS patients.
An independent expert helps put the findings in context and gives voice to some necessary cautions. However, the story doesn’t explain that this approval was based on studies funded by the drug’s manufacturer, which is a detail readers need to know.
Even a brief mention of how ALS patients are currently treated would help put the promise and pitfalls of the new drug in better context.
The article discusses availability issues — particularly the difficulty of getting to a health care provider day after day for patients with mobility problems. Readers are told that the drug maker is working to facilitate access.
The article makes clear that the drug is newly approved and the first new drug for ALS in over two decades.
The article is not based on a news release.