Proxy endpoints - bridging clinical trials and real world data.

Objective: Disease severity scores, or endpoints, are routinely measured during Randomized Controlled Trials (RCTs) to closely monitor the effect of treatment. In real-world clinical practice, although a larger set of patients is observed, the specific RCT endpoints are often not captured, which makes it hard to utilize real-world data (RWD) to evaluate drug efficacy in larger populations.

Methods: To overcome this challenge, we developed an ensemble technique which learns proxy models of disease endpoints in RWD.