Trial Shows Cystic Fibrosis Drug Helped Ease Breathing
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Trial Shows Cystic Fibrosis Drug Helped Ease Breathing
Our Review Summary
Our main critique is the lack of any independent medical voice in this story. With the exception of an analyst, all quoted sources bet on this horse to win. Perhaps the analyst did, as well. When results are only available in a press release, it’s even more critical to get assessment from clearly independent sources. This kind of informal peer review would’ve strengthened the story’s authority substantially.
Why This Matters
This story is important for a couple of reasons. New CF treatments are needed. Desperately. The announcement of positive results for a new mode of therapy is sure to cause great excitement among patients, providers, and investors. This blog on the health care business does a solid job of describing the study from a business perspective, covering the core features of its design, results, and some interesting context, such as the drug’s 20-year journey. It takes a bit of background in research to pick up on the study’s limitations, but they’re here, too. The cystic fibrosis gene was identified in 1989 and with the identification came the hope that a the mutation could in some way be manipulated or new treatments identified. VX-770 is a step in that direction. It remains to be seen if the drug will make it to market.
Criteria
Satisfactory
It discusses the drug’s costs, which are expected to be substantial. Given the relatively small population that the drug might be of benefit to, price considerations are important. The story briefly notes both aspects.
Satisfactory
The primary result is quantified in absolute terms. The blog provides the investment context for the magnitude of benefit. As noted above, we appreciated some background on how the primary endpoint was measured, and its attempt to translate a 10% change into how patients felt. The results for secondary endpoints were also summarized.
Satisfactory
It lists the main side effects reported by the company. Ideally we like quantified harms to balance quantified benefits, but given the limited available data, we think the author’s tack may have been the more balanced one. While the press release does report some numbers for discontinuation rates and severe side effects, it doesn’t quantify the most common harms, and in fact all the numbers portray VX-770 as safer than placebo. Perhaps that’s a larger trend in the data, but it’s hard to say. Thus, given the source and the lack of transparency of the data at the moment, it makes sense to leave out the numbers in the press release to steer clear from any perception of selective reporting.
Satisfactory
Many key morsels are here: the press release source, the lack of peer review, the number of subjects, their ages, the type of disease they had, the length of the study, and the identity of primary and secondary endpoints, as well as some practical definitions. It would’ve been nice to state explicitly whether the study was randomized.
Satisfactory
We liked the early caveat that VX-770 is designed for use in only 4% of people with CF, or 1200 Americans.
Not Satisfactory
The story’s major flaw, in our opinion, lies here. There were no independent sources besides an investment analyst. Even the study’s lead investigator found out the study’s results after being briefed by the company. No matter if, in reality, the study is indeed a gamechanger, those of us who aren’t steeped in CF knowledge can’t judge vested claims. That’s why we rely on independent experts, to separate rational from irrational exuberance. It would have been relatively easy to find an additional expert in the field to make comments.
All conflicts of interest were spelled out.
Satisfactory
It summarizes the alternative treatments for CF. It would’ve been interesting to note what other therapies subjects in this study received.
Satisfactory
It describes the drug’s pre-market status. It also resists projecting too far into the future, concluding that it might be nearing the market, and listing some upcoming milestones.
Satisfactory
The story does a good job of explaining the way in which the drug works and the limited number of patients with the specific genetic mutation.
Satisfactory
While the nut meat of the story, the study information, is from a press release, the author did use several other sources, including an interview. Some of the background information, such as CF prevalence, is from the press release, but perhaps the reporter confirmed these data before repeating them. We would normally chastise this type of reporting but think that in this instance it is reasonable. Cystic fibrosis is a terrible disease that has defied attempts at a cure or even a satisfactory treatment. A drug that shows promise, albeit with the information dissemination coming from a press release, is newsworthy.
Total Score: 9 of 10 Satisfactory
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