Randomized Controlled Trials of Electronic Health Record Interventions: Design, Conduct, and Reporting Considerations

Area of Science:

• Clinical Informatics
• Health Services Research
• Biostatistics

Background:

• Electronic health record (EHR) systems offer potential for novel interventions influencing clinical decision-making.
• EHR interventions require efficient randomized controlled trials (RCTs) to assess effectiveness, safety, and costs.
• Designing RCTs for EHR interventions presents unique challenges compared to traditional clinical trials.

Purpose of the Study:

• To outline key considerations for designing and conducting RCTs of EHR interventions.
• To provide guidance on methodological choices impacting the validity and efficiency of EHR intervention trials.
• To ensure robust evaluation of EHR-based clinical decision support and its impact on healthcare delivery.

Main Methods:

• Discusses the critical choice of randomization unit (patient, encounter, clinician, clinical unit) and its implications for contamination and confounder control.
• Addresses the timing of computational assessment components (e.g., predictive algorithms) relative to randomization (pre- or post-randomization).
• Highlights the need to consider subgroup heterogeneity when computational components are applied post-randomization.
• Emphasizes institutional review board (IRB) review, informed consent, data and safety monitoring, and CONSORT reporting guidelines.

Main Results:

• Careful selection of randomization units balances contamination risks with confounder control.
• Pre- or post-randomization application of computational components affects trial design and analysis.
• Heterogeneity in subgroup effects necessitates specific analytic approaches and sample size considerations.
• RCTs of EHR interventions may not always require patient-level informed consent under minimal risk or quality improvement conditions.

Conclusions:

• Effective RCT design for EHR interventions hinges on strategic decisions regarding randomization units and computational components.
• Adherence to reporting standards like CONSORT, with extensions for pragmatic and cluster RCTs, is crucial for reproducibility.
• Robust trial conduct, including appropriate monitoring and ethical review, ensures reliable evidence for implementing EHR interventions.