Register or electronic health records enriched randomized pragmatic trials: The future of clinical effectiveness and cost-effectiveness trials?

  • Joakim Ramsberg Department of Learning, Informatics, Management and Ethics, Medical Management Center, Karolinska Institutet, Stockholm, Sweden
  • Martin Neovius Clinical Epidemiology Unit, Department of Medicine, Karolinska Institutet, Stockholm, Sweden
Keywords: clinical trials, pragmatic trials, disease registries, electronic health records, costs


For many interventions in health care, there is limited information on efficacy, safety and cost-effectiveness even long after they have been implemented. To decide between treatments, the randomized controlled trial (RCT) provides the strongest evidence. This creates a problem because RCTs are very expensive, logistically challenging and generally cumbersome. Observational studies are inexpensive, but create weaker evidence. The pragmatic randomized trial, enriched with routinely collected register or electronic health record  (EHR) data may be a solution to this dilemma since they are much less costly than traditional RCTs but create much stronger evidence than observational studies. Pragmatic randomized trials mean that patients in routine care are randomly allocated to alternative treatments. The outcome of the treatment is then followed up in existing registers with patient data. This means that it is possible to 1) follow patients in the normal care situation - unlike the often artificial situation in the traditional RCT, 2) that the costs are low, even for large studies and 3) that a broad spectrum of outcomes, including both health and economic outcomes, can be collected. Pragmatic randomized trials using register or EHR data in principle lend themselves well to health economic evaluations. We have identified a number of such trials in the literature. Very few, however, include economic outcomes.