This story is about the long journey to approval of a new drug called Xiaflex used to treat Dupuytren’s contracture–a condition in which cords of collagen cause fingers to clench involuntarily. While the main story focuses mostly on the financial maneuverings related to the drug’s development, this sidebar provides a very informative overview of the drug’s benefits and risks for patients, costs, and effectiveness compared with other treatments. Overall, an excellent effort – markedly different than an earlier story on this drug by ABC News.com.
A drug’s approval by the FDA is often accompanied by excessive hype regarding the new therapy’s benefits but little scrutiny of potential downsides. It’s nice to see an approval story that balances excitement about benefits with sober analysis of costs and possible harms.
Dupuytren’s contractures can be a debilitating disease that until recently has required surgical interventions. The availability of a new drug treatment is a welcomed addition to the treatment options available to patients with the disorder. The role of Xiaflex as compared to surgery is unclear at the present time.
The main story estimates a course of treatment will cost about $5400, and the sidebar helpfully compared this amount to some of the other treatment options including surgery and needle aponeurotomy.
The sidebar describes the outcomes of the main clinical trial of Xiaflex in absolute terms. It reports that 64% of treated joints straightened out, compared with 7% of control group joints. The story relied a bit heavily on a positive patient anecdote in the sidebar lead-in, but it also managed to find a second patient who offered a unique perspective on the treatment. This second patient had successful Xiaflex injections on three fingers and open surgery on a fourth finger which didn’t respond to the drug. In this patient’s view, the drug was preferable to surgery.
The sidebar mentions that Xiaflex can rupture tendons "in rare circumstances" and that the injections "often" cause temporary pain and bruising and "sometimes" cause allergic reactions. We wish that the story had been a bit more precise in its quantification of these adverse effects, but we feel the descriptions provided are enough to paint a reasonably accurate portrait of the drug’s potential harms.
The sidebar provided the essential details on the main clinical trial used to support approval of Xiaflex. It noted that the study compared Xiaflex to placebo in 306 patients with Dupuytren’s. It also pointed out limitations in the currently available data, including the lack of long-term follow-up on these patients and the lack of data showing whether Xiaflex is better than the standard surgical treatment.
There was no exaggeration of the prevalence or consequences of Dupuytren’s contracture.
Most of the sources in this story are affiliated in some way with organizations that will profit from the sale of Xiaflex. These conflicts are disclosed, however, and the story does offer some clinical perspective from a hand specialist who does not appear to be connected to Xiaflex.
The story compares costs and outcomes for Xiaflex with open surgery and needle aponeurotomy.
The story states that Xiaflex was approved last month and will go on sale later this month.
The story chronicles Xiaflex’s 50-year journey from discovery to FDA approval. It does not attempt to portray the drug as "new."
The depth and sourcing of this story precludes the possibility that it was based on a press release.