Understanding the effects of interventions is central to scientific progress, with randomized controlled trials (RCTs) regarded as the gold standard for causal inference in many applied fields. However, RCTs are costly, time-consuming, and often constrained by ethical or practical limitations, motivating the need for causal methods able to draw conclusions from observational data. While such data is collected at ever larger scale, making its use for causal inference is often hindered by the fact that not all variables affecting treatment allocation and the outcome are observed - an issue known as unobserved confounding. In this paper, we introduce a new study design called confounder detection via treatment intent. The idea is to query a human expert who makes treatment decisions, and ask them to compare pairs of units proposed by a principled matching strategy, with the goal of eliciting unobserved variables that explain why treatment decisions differ. We provide a theoretical basis for such a procedure, ascertaining conditions under which such a study design may elicit unobserved confounders. Building on this newly established foundations, we study treatment effects of interventions in the intensive care unit (ICU). First, we show empirical evidence strongly indicating that electronic health records (EHRs) collected in ICUs are subject to unobserved confounding. By using clinical text notes as a proxy for physicians' knowledge and leveraging natural language processing, we provide a proof of concept for our methodology in a semi-synthetic environment with a known ground truth.

Usability testing is a fundamental research method that user experience (UX) researchers use to evaluate and iterate their new designs. But what about evaluating and iterating the usability testing study design itself? Recent advances in Large Language Model-simulated Agent (LLM Agent) research inspired us to design UXAgent to support UX researchers in evaluating and iterating their study design before they conduct the real human-subject study. Our system features a Persona Generator module, an LLM Agent module, and a Universal Browser Connector module to automatically generate thousands of simulated users and to interactively test the target website. The system also provides a Result Viewer Interface so that the UX researchers can easily review and analyze the generated qualitative (e.g., agents' post-study surveys) and quantitative data (e.g., agents' interaction logs), or even interview agents directly. Through a heuristic evaluation with 16 UX researchers, participants praised the innovation of our system but also expressed concerns about the future of LLM Agent usage in UX studies.

Sample size calculations for power analysis are critical for clinical research and trial design, yet their complexity and reliance on statistical expertise create barriers for many researchers. We introduce PowerGPT, an AI-powered system integrating large language models (LLMs) with statistical engines to automate test selection and sample size estimation in trial design. In a randomized trial to evaluate its effectiveness, PowerGPT significantly improved task completion rates (99.3% vs. 88.9% for test selection, 99.3% vs. 77.8% for sample size calculation) and accuracy (94.1% vs. 55.4% in sample size estimation, p < 0.001), while reducing average completion time (4.0 vs. 9.3 minutes, p < 0.001). These gains were consistent across various statistical tests and benefited both statisticians and non-statisticians as well as bridging expertise gaps. Already under deployment across multiple institutions, PowerGPT represents a scalable AI-driven approach that enhances accessibility, efficiency, and accuracy in statistical power analysis for clinical research.

Accurately capturing individual differences in semantic networks is fundamental to advancing our mechanistic understanding of semantic memory. Past empirical attempts to construct individual-level semantic networks from behavioral paradigms may be limited by data constraints. To assess these limitations and propose improved designs for the measurement of individual semantic networks, we conducted a recovery simulation investigating the psychometric properties underlying estimates of individual semantic networks obtained from two different behavioral paradigms: free associations and relatedness judgment tasks. Our results show that successful inference of semantic networks is achievable, but they also highlight critical challenges. Estimates of absolute network characteristics are severely biased, such that comparisons between behavioral paradigms and different design configurations are often not meaningful. However, comparisons within a given paradigm and design configuration can be accurate and generalizable when based on designs with moderate numbers of cues, moderate numbers of responses, and cue sets including diverse words. Ultimately, our results provide insights that help evaluate past findings on the structure of semantic networks and design new studies capable of more reliably revealing individual differences in semantic networks.

Empirical software engineering faces a critical gap: the lack of standardized tools for rapid development and execution of Test-Driven Software Experiments (TDSEs) - that is, experiments that involve the execution of software subjects and the observation and analysis of their "de facto" run-time behavior. In this paper we present a general-purpose analysis platform called LASSO that provides a minimal set of domain-specific languages and data structures to conduct TDSEs. By empowering users with an executable scripting language to design and execute TDSEs, LASSO enables efficient evaluation of run-time semantics and execution characteristics in addition to statically determined properties. We present an example TDSE that demonstrates the practical benefits of LASSO's scripting capabilities for assessing the reliability of LLMs for code generation by means of a self-contained, reusable and extensible study script. The LASSO platform is freely available at: https://softwareobservatorium.github.io/, and a demo video is available on YouTube: https://youtu.be/tzY9oNTWXzw

The validity of medical studies based on real-world clinical data, such as observational studies, depends on critical assumptions necessary for drawing causal conclusions about medical interventions. Many published studies are flawed because they violate these assumptions and entail biases such as residual confounding, selection bias, and misalignment between treatment and measurement times. Although researchers are aware of these pitfalls, they continue to occur because anticipating and addressing them in the context of a specific study can be challenging without a large, often unwieldy, interdisciplinary team with extensive expertise. To address this expertise gap, we explore the use of large language models (LLMs) as co-pilot tools to assist researchers in identifying study design flaws that undermine the validity of causal inferences. We propose a conceptual framework for LLMs as causal co-pilots that encode domain knowledge across various fields, engaging with researchers in natural language interactions to provide contextualized assistance in study design. We provide illustrative examples of how LLMs can function as causal co-pilots, propose a structured framework for their grounding in existing causal inference frameworks, and highlight the unique challenges and opportunities in adapting LLMs for reliable use in epidemiological research.

Evidence to guide healthcare decisions is often limited by a lack of relevant and trustworthy literature as well as difficulty in contextualizing existing research for a specific patient. Large language models (LLMs) could potentially address both challenges by either summarizing published literature or generating new studies based on real-world data (RWD). We evaluated the ability of five LLM-based systems in answering 50 clinical questions and had nine independent physicians review the responses for relevance, reliability, and actionability. As it stands, general-purpose LLMs (ChatGPT-4, Claude 3 Opus, Gemini Pro 1.5) rarely produced answers that were deemed relevant and evidence-based (2% - 10%). In contrast, retrieval augmented generation (RAG)-based and agentic LLM systems produced relevant and evidence-based answers for 24% (OpenEvidence) to 58% (ChatRWD) of questions. Only the agentic ChatRWD was able to answer novel questions compared to other LLMs (65% vs. 0-9%). These results suggest that while general-purpose LLMs should not be used as-is, a purpose-built system for evidence summarization based on RAG and one for generating novel evidence working synergistically would improve availability of pertinent evidence for patient care.

This paper describes a rapid feasibility study of using GPT-4, a large language model (LLM), to (semi)automate data extraction in systematic reviews. Despite the recent surge of interest in LLMs there is still a lack of understanding of how to design LLM-based automation tools and how to robustly evaluate their performance. During the 2023 Evidence Synthesis Hackathon we conducted two feasibility studies. Firstly, to automatically extract study characteristics from human clinical, animal, and social science domain studies. We used two studies from each category for prompt-development; and ten for evaluation. Secondly, we used the LLM to predict Participants, Interventions, Controls and Outcomes (PICOs) labelled within 100 abstracts in the EBM-NLP dataset. Overall, results indicated an accuracy of around 80%, with some variability between domains (82% for human clinical, 80% for animal, and 72% for studies of human social sciences). Causal inference methods and study design were the data extraction items with the most errors. In the PICO study, participants and intervention/control showed high accuracy (>80%), outcomes were more challenging. Evaluation was done manually; scoring methods such as BLEU and ROUGE showed limited value. We observed variability in the LLMs predictions and changes in response quality. This paper presents a template for future evaluations of LLMs in the context of data extraction for systematic review automation. Our results show that there might be value in using LLMs, for example as second or third reviewers. However, caution is advised when integrating models such as GPT-4 into tools. Further research on stability and reliability in practical settings is warranted for each type of data that is processed by the LLM.

HRI research using autonomous robots in real-world settings can produce results with the highest ecological validity of any study modality, but many difficulties limit such studies' feasibility and effectiveness. We propose Vid2Real HRI, a research framework to maximize real-world insights offered by video-based studies. The Vid2Real HRI framework was used to design an online study using first-person videos of robots as real-world encounter surrogates. The online study ($n = 385$) distinguished the within-subjects effects of four robot behavioral conditions on perceived social intelligence and human willingness to help the robot enter an exterior door. A real-world, between-subjects replication ($n = 26$) using two conditions confirmed the validity of the online study's findings and the sufficiency of the participant recruitment target ($22$) based on a power analysis of online study results. The Vid2Real HRI framework offers HRI researchers a principled way to take advantage of the efficiency of video-based study modalities while generating directly transferable knowledge of real-world HRI. Code and data from the study are provided at https://vid2real.github.io/vid2realHRI

Personalized adaptive interventions offer the opportunity to increase patient benefits, however, there are challenges in their planning and implementation. Once implemented, it is an important question whether personalized adaptive interventions are indeed clinically more effective compared to a fixed gold standard intervention. In this paper, we present an innovative N-of-1 trial study design testing whether implementing a personalized intervention by an online reinforcement learning agent is feasible and effective. Throughout, we use a new study on physical exercise recommendations to reduce pain in endometriosis for illustration. We describe the design of a contextual bandit recommendation agent and evaluate the agent in simulation studies. The results show that, first, implementing a personalized intervention by an online reinforcement learning agent is feasible. Second, such adaptive interventions have the potential to improve patients' benefits even if only few observations are available. As one challenge, they add complexity to the design and implementation process. In order to quantify the expected benefit, data from previous interventional studies is required. We expect our approach to be transferable to other interventions and clinical interventions.