2021年5月，美国食品药品监督管理局（FDA）发布了一份长篇简报文件，涉及一种用于治疗罕见自身免疫性血管炎的实验性药物，这种血管炎常损害肾脏和肺部。该机构对是否批准这种疗法犹豫不决，希望由一个独立咨询委员会提供意见。当时的问题之一是：这种药物是否明显优于现有疗法？
In May 2021, the US Food and Drug Administration released a lengthy briefing document on an experimental medication for a rare autoimmune form of vasculitis that often damages the kidneys and lungs. The agency was on the fence about whether to approve the treatment, and it wanted an independent advisory committee to weigh in. Among the questions at hand: Was the drug meaningfully better than existing therapies?
这项研究由加利福尼亚生物技术公司ChemoCentryx开展，研究参与者似乎在一年后症状缓解率略高。但据FDA称，由于研究设计不够理想，结果难以解读。一些咨询委员会成员表达了类似看法。其中一人说，该药物的效果“并未得到这项研究的充分验证”。与此同时，许多患者和一些医生从数据中看到了一种可能有用的药物，它或许能减少患者对类固醇的依赖——类固醇非常有效，但常引起严重副作用。
Participants in the study, which had been conducted by the California biotechnology company ChemoCentryx, did appear to have slightly higher rates of symptom remission after one year. But according to the FDA, it was hard to interpret the results because the study’s design had been less than optimal. Some advisory committee members expressed a similar view. The effect of the drug, said one, “is just not well verified by this study.”At the same time, many patients, and some physicians, saw in the data a potentially helpful medication, one that might lessen patients’ reliance on steroids, which are highly effective but often cause severe side effects.