Table 1

Strengths and limitations of registry-based randomised controlled trials

  • Simplify data collection, utilising information collected during routine clinical care and minimising collection of additional (non-critical) elements

  • Require utilisation of objective endpoints

  • Less complex in terms of regulatory documents (eg, ICH-GCP guidelines)

  • Efficient use of existing infrastructure and personnel resources

  • Address problem of allocation bias inherent in observational registries

  • More likely to reflect generalisable population

  • Less restrictive enrolment criteria increase rate of recruitment

  • Permit study of clinical questions unlikely to be funded from other sources (eg, evaluation of therapies that are inexpensive to society, such as generic drugs)

  • Faster implementation of results

  • Less expensive to conduct than typical randomised trial

  • Facilitate pretrial feasibility studies

  • Usually open-label; blinding may reduce efficiencies

  • Endpoints are limited to those less subject to bias (eg, all-cause mortality); blinded adjudication committees are necessary to reduce potential bias

  • Less certain about validity of baseline and outcome data

  • Less practical for trials requiring extensive data collection or involving centres without reliable electronic health records or centralised event-reporting systems; require consistent health informatics systems

  • Multicentre/multiregional studies can be difficult to conduct due to interoperability of data systems, harmonisation of data dictionaries or other data compatibility issues

  • Monitoring and validation still required

  • Concerns about acceptance from funders, reviewers, editors and regulators

  • ICH-GCP, International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use–Good Clinical Practice.