A Causal Inference Framework for Leveraging External Controls in Hybrid Trials

We consider the challenges associated with causal inference in settings where data from a randomized trial is augmented with control data from an external source to improve efficiency in estimating the average treatment effect (ATE). Through the development of a formal causal inference framework, we outline sufficient causal assumptions about the exchangeability between the internal and external controls to identify the ATE and establish the connection to a novel graphical criteria. We propose estimators, review efficiency bounds, develop an approach for efficient doubly-robust estimation even when unknown nuisance models are estimated with flexible machine learning methods, and demonstrate finite-sample performance through a simulation study. To illustrate the ideas and methods, we apply the framework to a trial investigating the effect of risdisplam on motor function in patients with spinal muscular atrophy for which there exists an external set of control patients from a previous trial.

Acquisition Conditioned Oracle for Nongreedy Active Feature Acquisition

We develop novel methodology for active feature acquisition (AFA), the study of how to sequentially acquire a dynamic (on a per instance basis) subset of features that minimizes acquisition costs whilst still yielding accurate predictions. The AFA framework can be useful in a myriad of domains, including health care applications where the cost of acquiring additional features for a patient (in terms of time, money, risk, etc.) can be weighed against the expected improvement to diagnostic performance. Previous approaches for AFA have employed either: deep learning RL techniques, which have difficulty training policies in the AFA MDP due to sparse rewards and a complicated action space; deep learning surrogate generative models, which require modeling complicated multidimensional conditional distributions; or greedy policies, which fail to account for how joint feature acquisitions can be informative together for better predictions. In this work we show that we can bypass many of these challenges with a novel, nonparametric oracle based approach, which we coin the acquisition conditioned oracle (ACO). Extensive experiments show the superiority of the ACO to state-of-the-art AFA methods when acquiring features for both predictions and general decision-making.