Principles and Guidelines for Randomized Controlled Trials in AI Evaluation
Authors: Christopher Kelly, Angelica Chowdhury, Alexandra Campili, Bimpe Ayoola, Devin Barbour, Thomas Chen Dawson, Ze Shen Chin, Rokas Gipiškis
Abstract
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.
Human uplift studies, or studies that measure the effects of AI access on human performance via randomized controlled trials (RCT) or similar methodologies, increasingly inform frontier AI governance and deployment decisions. While RCT methods are robust in other fields, their interaction with the distinctive properties of frontier AI systems remains underexamined, particularly when results are used to inform high-stakes decisions. We present findings from interviews with 16 expert practitioners with experience conducting human uplift studies in domains including biosecurity, cybersecurity, education, and labor. Across interviews, experts described a recurring tension between the standard causal inference assumptions upon which human uplift studies rely and the object of study itself. Rapidly evolving AI systems, shifting baselines, heterogeneous and changing user proficiency, and porous real-world settings strain assumptions underlying internal, external, and construct validity, complicating the interpretation and appropriate use of uplift evidence. We contribute (1) a synthesis of methodological challenges in human uplift studies, mapped to risks to study validity and classified by their degree of specificity to large language model (LLM) systems, and (2) a mapping from challenges to proposed solutions. By collating expert-identified challenges and solutions, we seek to clarify the interpretive limits and appropriate uses of human uplift evidence, to align evaluation practice with the decisions it informs, and to support more coordinated methodological foundations for AI governance.
Systematic reviews of Randomised Controlled Trials (RCTs) are routinely used as evidence for clinical care guidelines. Such evidence has to meet high research integrity standards to prevent low quality or false research outputs influencing the clinical care. However, assessing research integrity of published RCTs is a complex process requiring manual effort, and potentially resulting in diverse opinions of the human assessors. This paper describes INSPECT-AI, an LLM-based interactive tool that assists human reviewers with research integrity assessments of published RCTs based on the community approved INSPECT-SR framework, and the Research Integrity Provenance and Evidence ontology (RIPE-O) for documenting the provenance of the assessment process. In addition, we present the Research Integrity Provenance and Evidence knowledge graph (RIPE-KG), an initial set of 140 expert research integrity assessments of 95 RCT publications generated by INSPECT-AI and described using RIPE-O.
Milan Markovic, Goutham Indukuri, Somayajulu Sripada +7
AI measurement science has a wide variety of methodologies and measurements for comparing AI systems, resulting in what often appear to be "apples-to-oranges" comparisons across AI evaluations. To move toward "apples-to-apples" comparisons in real-world AI evaluations, this work advocates for methodological transparency in evaluation scenarios, operational grounding, and human-centered design (HCD) principles. We propose a repeatable process for transforming high-level use cases to detailed scenarios by eliciting use cases from subject matter experts (SMEs) via a structured AI Use Case Worksheet with six key elements: use case, sector, user (direct and indirect), intended outcomes, expected impacts (positive and negative), and KPIs and metrics. We demonstrate utility of the worksheet and process in the U.S. financial services sector. This paper reports on example high-level AI use cases identified by financial services sector SMEs: cyber defense enablement, developer productivity, financial crime aggregation, suspicious activity report (SAR) filing, credit memo generation, and internal call center support. These AI use cases provided are illustrative of the process and not exhaustive. Central to our work is a three-stage expansion pipeline combining LLM prompting with human reviews to generate 107 scenarios from those use cases elicited from SMEs. This process integrates iterative human reviews at every juncture to ensure operational grounding: for scenario titles and descriptions; for core scenario elements like users, benefits and risks, and metrics; and for scenario narratives and evaluation objectives. Human checkpoints ensure scenarios remain reflective of real-world usage and human needs. We describe a validation rubric to assess scenario quality. By defining key scenario components, this work supports a more consistent and meaningful paradigm for human-centered AI evaluations.