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Readers will be swept in by Newsweek’s story on pending muscular dystrophy drug, but what does the evidence show?

Rating

4 Star

Categories

MUSCULAR DYSTROPHY: HEARTBROKEN FAMILY AWAITS FDA DECISION ON EXPERIMENTAL DRUG

Our Review Summary

This story focuses on a family tragedy—three young boys in a single family with a fatal genetic disorder called Duchenne muscular dystrophy—and the family’s hope that an experimental drug, which the three boys have been taking as part of a clinical trial, will receive FDA approval later this month.

The story is filled with emotional freight, pitting anecdotal evidence (the mother’s reflection on her sons’ vigor, which she attributes to the drug) against systematic evidence that that finds little in the way of robust benefits. While such a story offers readers a compelling look at the sometimes anguished process of clinical research in the face of a lethal disease, it gives too short shrift to the clinical research evidence available here. On the other hand, the story did a great job explaining the costs and insurance hurdles around drugs like this, as well as the possible mechanisms behind how this drug and similar ones work.

 

Why This Matters

Letting the emotional intensity of anecdotal information duke it out with systematic research findings is a proven reader-grabber. But selecting this narrative route requires dedication to a story line that never lets the reader forget that good, systematic evidence is paramount. Given the vividness of anecdotal accounts, the systematic details in a story like this must be a dominant and detailed part of the story. This text would have done a better job of achieving that, and doing so convincingly, with more evidentiary detail.

 

Criteria

Does the story adequately discuss the costs of the intervention?

Satisfactory

Cost plays an important role in this story, as the drug (should it come to market) would be expensive and there is considerable uncertainty about whether insurance would pay for it.

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

Not Satisfactory

Most of the “benefit” information in this story is provided by the mother of the three boys with Duchenne muscular dystrophy, who reflects in detail on a level of activity among her sons that she feels demonstrates the drug’s effectiveness. The reporter reflects briefly on clinical trial results that offered “some slight positive results,” but we never learn what those are.

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

Not Satisfactory

The text briefly mentions side effects associated with the drug, increases in blood pressure and cholesterol levels, an assertion contested by the drug’s manufacturer, PTC Therapeutics. However, it would have been more helpful to quantify and more clearly describe these side effects, and tap independent experts for their viewpoints. We found the NCHR’s testimony about the risks of combining this drug with other drugs these kids commonly take to be important, for example.

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

Not Satisfactory

On the one hand, the story does a good job of making the point that clinical trial results are murky, leading the National Center for Health Research to recommend that the FDA vote “no” on approving the drug. But on the other, the text makes no effort to help readers understand the nature of the evidence on which such a decision would be based.

Does the story commit disease-mongering?

Satisfactory

Duchenne muscular dystrophy is a killer.

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

Satisfactory

Quite a few sources were interviewed for the story. And, all sources are clearly identified, including the CEO of the company that is apparently funding the clinical trials.

However, we do think the story would have been stronger had it tapped the viewpoints of an expert in muscular dystrophy who has no vested interested in the drug receiving approval.

Does the story compare the new approach with existing alternatives?

Satisfactory

There is no known cure for Duchenne muscular dystrophy.  The story does devote some space to another drug, Exondys 51, which has been approved by the FDA, but the text is clear that the type of DMD that afflicts the three boys is not responsive to that drug.

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

Satisfactory

A main point of narrative tension in the story is the family’s wait to see if the FDA will approve the drug. So it is clear that the drug is not yet available.

Does the story establish the true novelty of the approach?

Satisfactory

The story makes it clear that this drug is unique, in that it aims to override the specific genetic mutations causing this type of Duchenne muscular dystrophy.

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

Satisfactory

The story clearly does not rely on a news release.

Total Score: 7 of 10 Satisfactory

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