Despite the importance of vaccine efficacy against post-infection outcomes like transmission or severe illness, these estimands are unidentifiable, even under strong assumptions that are rarely satisfied in real-world trials. We develop a novel method to nonparametrically point identify these principal effects while eliminating the assumptions of monotonicity and perfect infection and post-infection measurements, and show that this extends to multiple treatments. The result is applicable outside of vaccine efficacy due to the generality of the results. We show that our method can be applied to a variety of clinical trial settings where vaccine efficacy against infection and a post-infection outcome can be jointly inferred. This can yield new insights from existing vaccine efficacy trial data and will aid researchers in designing new multi-arm clinical trials.
翻译:尽管疫苗对传染或严重疾病等感染后结果的功效十分重要,但这些估计值是无法辨别的,即便在现实世界试验中很少满足的强烈假设下也是如此。我们开发了一种非对称点的新方法,在消除单一性、完美感染和感染后测量假设的同时,确定这些主要效果,并表明这扩大到多种治疗。由于结果的普遍性,结果在疫苗功效之外适用。我们表明,我们的方法可以适用于各种临床试验环境,在这些环境中,疫苗对感染的功效和感染后结果可以共同推断。这可以从现有的疫苗功效试验数据中得出新的见解,并将帮助研究人员设计新的多武器临床试验。