This story is based on an FDA news release announcing the approval of a drug to treat cancer that springs from an abnormality called neurotrophic receptor tyrosine kinase, or NTRK, gene fusion.
The story accurately reports that Vitrakvi (larotrectinib) is the second drug approved for use based on a tumor biomarker, rather than where the cancer is located in the body.
However, it omits important information like the drug’s hefty price tag ($400,000 a year), its harms, the lack of evidence to show it prolongs life or improves the quality of patients’ lives, and the fact that it applies to a very small number of patients.
It’s also larded with generic statements extolling the virtues of targeted cancer therapies, with no comments from independent sources.
Cancer wonks may get excited about this new “site-agnostic oncology therapy,” but patients and their families aren’t served by mainstream news stories that ignore their basic concerns.
Stories need to refrain “new hope” hype and address questions like: Does it help patients live longer? How does it affect qualify of life? Who can use it? How much does it cost? What’s the evidence that it works?
Surprise — or at least it will be to readers of this Newsweek story: The list price of the drug is nearly $400,000 a year, as a more complete story in Forbes reported. The company marketing the drug said it will help patients pay their share of the cost.
The headline calls this drug “aggressive.” But the story doesn’t report data on the drug’s response rate, which is the percentage of patients whose tumors shrunk after treatment. Response rate was the basis of the FDA’s approval.
According to FDA, there was a 75% overall response rate across different types of solid tumors, with 73% of responses lasting at least six months and 39% lasting a year or more.
Readers are not told that there is no evidence to show patients who get this drug actually live longer or have a better quality of life than those who don’t get it. (More on this under the quality of evidence rating, below.)
There is no discussion of harms. An FDA news release mentioned fatigue, nausea, cough, constipation, diarrhea, dizziness, vomiting, and elevated enzyme levels that might indicate liver damage. The FDA said patients should have regular liver tests and warned against treating women who are pregnant or breastfeeding because of potential harm to their fetus or baby.
Readers are told the FDA used an accelerated approval process to get the drug to patients “before the usual clinical trials are completed,” and that there will be further tests of the drug’s safety and effectiveness.
But we don’t think that fully explains the quality of evidence.
Readers aren’t told that the only human trials involved a total of 55 patients who were not blinded, with no comparison group of patients who didn’t get the drug. The upshot is, we don’t know if this drug performs any better than other treatments or a placebo.
The story doesn’t caution that “breakthrough” drugs like this are often approved on the basis of surrogate endpoints like tumor shrinkage, rather than outcomes that are meaningful to patients, like longer survival. For a deeper dive, read our toolkit, “Surrogate markers may not tell the whole story.”
What’s more, readers are unlikely to understand from this story that continued FDA approval of the drug may depend on the results of future trials. This is an important omission: One analysis of cancer drugs that won similar FDA approval through a “breakthrough” designation concluded that, four years after being approved, only one in seven could show that they actually helped patients live longer.
The story doesn’t explain that this drug is applicable only to a small number of patients — those who have this rare biomarker and who have seen their tumors progress despite conventional treatment or have no alternative treatments.
There are no independent sources mentioned in this story, which also doesn’t state that all of the clinical trials have been sponsored by the company selling the drug.
There is no discussion of how this treatment compares to alternatives. Readers are not told that such comparisons have not yet been studied.
The story reports that the drug has received FDA approval.
The story reports that this is the second drug to receive FDA approval for use based on genetic features of tumors, rather than the organ in which the tumor originated or is growing.
The story appears to be entirely based on an FDA news release.