This article presents business developments related to Zafgen’s beloranib drug in a clear and accessible manner. But there was room for improvement in several areas, including sourcing. The only sources interviewed have links to Zafgen and were involved with the study being reported on. The story also could have done a better job reporting on the quantitative results of the study. For example, it never defines what it means by “significant” when discussing the weight loss achieved by study participants.
Prader-Willi syndrome is a rare but life-threatening cause of obesity for which there is no current treatment. The drug candidate beloranib, if successful, would then be the first treatment for this patient population.
As the drug is still pending approval from the FDA, the exact cost of the drug is unknown. However, it seems close enough to approval that some cost estimate should have been attempted — perhaps via a market analyst or company officials. Should it be approved, the drug will most likely be quite expensive.
The article only states that the data shows beloranib met its two primary endpoints of significantly reducing weight and decreasing excessive eating. No numbers are attached to these statements, however. In particular, how is excessive eating measured? Clearly this is harder to define than weight loss. Presumably, surrogate variables were introduced to quantify the degree of excessive eating. A significant decrease in these surrogate variables can be difficult to interpret in many situations.
The article discloses the possible side effects of beloranib related to blood clotting along with the caveat that Prader-Willi patients are predisposed to clotting issues. Two deaths out of 100 patients is a significant issue, even if they can’t be linked definitively to the drug, so it’s good that this is a focal point. But the story mentions no other adverse effects that were observed in the study, and it should have. We’ll give the benefit of the doubt and hope for improvement next time.
From the presentation of the results, it is not clear exactly how many patients were included in the final analysis. It is said that the six-month study involved 107 patients who showed significant weight loss and reduction in excessive eating. However, the very next paragraph reveals that only 68 patients completed the full study, while 27 patients completed only 20 of the trial’s scheduled 26 weeks. Furthermore, it is reported that a “small number other patients withdrew early.” This small number, by our calculation, is 107 – 68 – 27 = 12, which is not such a small number considering there were only 107 patients to begin with.
The article provides an accurate profile of the Prader-WIlli syndrome emphasizing its life-threatening consequences but also conceding that it is a rare disease affecting roughly 6,000 to 8,000 Americans.
Two sources appeared to have been interviewed for this piece, Dr. Hughes and Dr. Butler. The former is the chief executive of Zafgen while the latter is a medical researcher serving as a principal investigator of the clinical trial at the University of Kansas Medical Center. To meet our standard here, the story would have had to interview a source completely independent of the company and the trial. We rate this Not Satisfactory, but at least the article makes it clear that the studies under consideration are for a commercial drug.
It is stated that there are no current treatments for Prader-Willi syndrome.
It is made clear that the beloranib drug is still under experimentation and pending FDA approval.
Having stated that there are currently no treatments for Prader-Willi, the novelty of the beloranib should be self-evident to the reader.
There’s enough original reporting that we can be sure the story isn’t based entirely on a news release.
Systematic, Criteria-Driven
The tale of two “negative” studies. Or, how spin is applied to make research findings look more impressive. A must read. https://johnmandrola.substack.com/p/the-tale-of-two-negative-studies
The @nytimes chose to interview one of the editors of the @AnnalsofIM (publisher of this paper) who "gushed with extraordinary glee: It’s huge! There are very few things that reduce your mortality by 30%."
Turns out coffee is not one of those few things despite all that glee. https://twitter.com/garyschwitzer/status/1533202075928215558
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