RCTs for Frontier AI Governance: Methodological Challenges and Solutions for Human Uplift Studies

This work establishes a foundational framework for standardizing AI evaluation RCTs (sometimes called human uplift studies). Drawing on established experimental practices from disciplines with established RCT traditions, including software engineering, economics, clinical and health sciences, and psychology, we adopt the (Shadish et al., 2002) four-validity framework and extend it with a fifth principle on transparency, repeatability, and verification adapted from the Transparency and Openness Promotion (TOP) Guidelines (Center for Open Science, 2025). We operationalize all five principles into 33 guidelines adapted for AI evaluation RCT contexts, expressed as requirements with rationales, implementation instructions, and evidence bases. We position the principles and guidelines as serving three key roles for AI evaluation RCTs: a design tool for planning studies, an evaluation rubric for assessing existing work, and a blueprint for standard setting as the field converges on norms. Our framework extends prior work by centering evaluation on human performance rather than model output alone, formalizing causal inference through RCT methodology for AI contexts, integrating heterogeneity analysis and practical significance assessment, implementing a graded transparency and repeatability framework, and addressing AI-specific challenges including model versioning, human-AI interaction dynamics, contamination and spillover effects, and equitable impact assessment.

Safety risks of AI are becoming increasingly evident in human interactions with AI technologies. The prominent approaches to evaluating these risks favor technical methods, such as model benchmarks and LLM simulations, often sidelining empirical research with human subjects. To examine this apparent gap in the acceptance of human research, we conduct an expert survey (n=93) and expert interviews (n=17) with AI Safety & Ethics (AISE) researchers from Technical, Sociotechnical, Governance, and Normative backgrounds. Our findings suggest that although there is a consensus that human research is valuable for generating evidence for AISE, its adoption and acceptance are constrained by perceived validity issues, tangible resource barriers, epistemic and personal preferences in methods, and infrastructural constraints from the broader research community. In particular, Technical researchers tend to value human research less and collaborate across disciplines less, suggesting an epistemic tension towards human methods. We propose recommendations for establishing the epistemic fit of human research within AISE and bridging the prohibitive limitations that researchers face, while avoiding performative 'human-washing'.

Large language models (LLMs) are increasingly used for tasks once reserved for trained researchers, including hypothesis generation, specification choice, and drafting conclusions. We argue that the reliability of AI-assisted research depends not only on model capability, but also on how cognitive labour is structured between humans and machines. We study this problem through Human-in-the-Loop Economic Research (HLER), a decision architecture based on pre-commitment, decision sequencing, accountability, and attention allocation. In a pre-specified 2*4 factorial experiment with 280 complete research runs across four datasets, an unconstrained multi-agent baseline produced critical failures in 72% of runs. Using the same underlying model, the same agent decomposition, and identical prompts for the shared reasoning agents, HLER reduced the failure rate to 16% by imposing three architectural commitments: LLMs reason but do not execute data work, data and estimation are handled deterministically, and three human decision gates bind the workflow. Fisher's exact test rejects equality of failure rates at p<0.001. Reliability gains were largest on the least publicly represented dataset, a Qing-dynasty population register, consistent with a task-based production model with Frechet-distributed output quality. An 80-run ablation suggests that deterministic computation and human gates contribute independently, with exploratory evidence of complementarity. We interpret HLER as a research harness rather than an autonomous AI scientist: it sharply reduces failures, makes residual weaknesses more visible, and prevents unreliable claims from being advanced as publication-ready outputs.