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New drug ‘big deal’ in cystic fibrosis fight

Rating

2 Star

Categories

New drug ‘big deal’ in cystic fibrosis fight

Our Review Summary

More than 20 years after researchers identified the genetic mutation responsible for cystic fibrosis (CF), a treatment aimed at the cause of the disease, rather than its effects, has shown it can improve lung function and some other measures in patients with a rare form of the disease. While this story accurately reports key drug trial results and provides some context, it falls short in important ways by failing to note that there is not yet evidence the drug is safe for life-long use or can extend lives; and that it is unlikely to cure CF by itself. The story doesn’t tell readers that drug is likely to come with an extremely high price tag. It fails to note that the CF Foundation, whose CEO is prominently quoted touting the drug, has funded the drug’s development and may stand to profit from its sales.

 

Why This Matters

Cystic fibrosis affects only about 70,000 people worldwide, but it is the most common lethal genetic disease in whites, slowly destroying their lungs and other organs. Since the primary CF mutation was identified in 1989, researchers have sought a treatment that would correct the underlying defect. As the story reports, this trial is an important proof of concept; it is a ‘big deal’ in the decades-long effort to find a treatment for the genetic defect underlying CF. Nevertheless, it is important to highlight the long, hard road traveled to reach this point and the years of work ahead that will be needed to determine if this sort of treatment can lengthen the lives of typical CF patients.

Criteria

Does the story adequately discuss the costs of the intervention?

Not Satisfactory

Although the company that wants to sell this drug has yet to announce a price, blogs that follow pharmaceutical companies quote a Wall Street analyst who predicts the drug will cost about $150,000 a year. That massive price tag for a drug that patients likely would have to continue to take for their entire lives is certainly something that readers should be told about.

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

Not Satisfactory

Although this story accurately reports the specific results of this drug trial, including lung function, disease symptoms, weight and sweat chloride (a measure of disease); it misses important aspects of the big picture. As the journal editorial accompanying this research report noted, the trial does not prove that this drug halts the progression of disease or that patients who take the drug will live longer.

The story could also have been clearer about the distinction between the small percentage of patients who have the specific gene mutation that is the target of this drug and the 90 percent of cystic fibrosis patients who have a different mutation that may not respond to this treatment. The editorial notes that the complexities of CF “suggest that no single drug will be entirely suitable as a therapeutic agent.” Readers should have been given this important context.

When the story reported that patients doubled their ability to walk, it should have noted that this apparent benefit was not something that was measured as part of the formal trial.

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

Not Satisfactory

The story does not make any reference to harms. Although the journal article reporting the trial findings says that the patients receiving the active drug generally had fewer problems than those in the placebo group and that the adverse effects were not serious enough to cause people to stop taking the drug for more than a short period, the editorial points out that this trial does not prove that long term use of this drug is safe. The story should have told readers that patients will probably need to take the drug their entire lives and that further tests are needed before long term safety questions can be answered.

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

Satisfactory

The story reports that only 84 patients were randomized to receive the experimental drug. It also reported the length of the trial and the specific endpoints that researchers evaluated. However, there were also speculative comments about which patients the drug could help as well as references to benefits that were not measured as part of this trial. Readers might well be confused about what was actually demonstrated by this trial and what is mere speculation or personal observation.

We could have gone either way with this grade, but will give the story the benefit of the doubt.

Does the story commit disease-mongering?

Satisfactory

The story reports that this drug works on a gene mutation seen in only 4 percent of patients with cystic fibrosis.

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

Not Satisfactory

This story fails to include an independent source and fails to report financial disclosures. What’s worse is that most readers probably believe there is an independent source quoted. While the average reader is likely to assume that the CEO of the Cystic Fibrosis Foundation is speaking independently, the actual fact is that the foundation is a major funder of the drug’s development and may stand to profit from sales of the drug. Readers should have been told of the tight financial relationship between the CF Foundation and Vertex Pharmaceuticals.

The story should have also noted that the researcher quoted in the story works as a consultant to Vertex, and that employees of the company were involved in designing and conducting the trial as well as writing the final report. These disclosures were openly declared in the journal article and accompanying documents and should have been included in the story.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

The story should have noted that advances in managing cystic fibrosis have dramatically increased the life span of people with the disease. Indeed, the editorial accompanying the journal article notes that the health of CF patients declines so gradually now that it will require following many patients for many years to find out if this new drug actually helps them live longer than the treatments already available.

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

Not Satisfactory

Although the story is clear that this drug is not yet available, it includes a claim that it could be on the market next year. The manufacturer recently applied for FDA approval, but the drug has not been reviewed, so any claim about availability is mere speculation.

Does the story establish the true novelty of the approach?

Satisfactory

The story does highlight the fact that this is the first treatment for cystic fibrosis that works on a genetic mutation that is the underlying cause of the disease, rather than just managing the effects of the disease.

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

Satisfactory

The story does not appear to rely on a news release.

Total Score: 4 of 10 Satisfactory

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