Statistics of Rare Events.

Proschan, Michael A; Kherabi, Yousra · NEJM Evid · 2024

review · Level V

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Abstract

Rare events can sometime arise in clinical development of treatments. For example, CYPIDES was a single-arm study of the CYP11A1 inhibitor ODM-208 to treat metastatic prostate cancer.<sup>1</sup> Preclinical testing of the compound identified elevated thyroid-stimulating hormone (TSH) and bilirubin in rats and dogs. Unusual findings in preclinical testing focus attention and magnify evidence if similar results occur in humans. By analogy, imagine a murder trial in which the only evidence against the defendant arose from a database search of DNA matching the partial profile found at the crime scene. Multiple people could match, so without other evidence, the perpetrator could be any of them.

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