Survivors of stroke complete a survey at 90 days indicating their willingness to be contacted for studies. Please, please, please agree to be contacted and tell them EXACTLY HOW FUCKING BAD THEY WERE TO NOT GET YOU 100% RECOVERED!
Research Note: Registry-based randomised controlled trials with examples from the Australian Stroke Clinical Registry
Summary
By combining the advantages of clinical quality registries with randomised controlled trials (RCTs), registry-based randomised controlled trials (RRCTs) can facilitate the investigation of clinical research questions using pragmatic and high-quality designs. Advantages of RRCTs include the opportunity to recruit a wider representative sample of the target population, use existing registry infrastructure to reduce the administrative burden, readily compare the trial cohort and registry patients who were not enrolled, and potentially decrease costs. Disadvantages include limited variables. This Research Note presents examples from the Australian Stroke Clinical Registry (AuSCR), established in 2009, to demonstrate how registry infrastructure can advance clinical trial research.
Introduction
Clinical quality registries (CQRs) are data systems used to collect standardised information on patients with particular characteristics, with the primary objective of monitoring the quality of care and benchmarking health services.1 These data systems also offer a ready source of observational data for these patient cohorts.2,3 The infrastructure of CQRs can support clinical trial research in three main ways: as a source of patients who might meet the eligibility criteria (recruitment facilitation); as a source of additional patient-level data for a trial participant (data provisioning); and as infrastructure to conduct all aspects of a clinical trial. That is: enabling an embedded RCT from recruitment, intervention delivery and outcome assessment (ie, RRCT). This Research Note mainly focuses on RRCTs; however, other examples of how CQR infrastructure can support RCTs are also provided.
Background on clinical trials and registry-based research
Randomised controlled trials have been widely used to investigate novel drugs or health service/therapy interventions and are considered the gold standard for evaluating the effectiveness of medical interventions.4 They can vary in complexity and design and often require significant resourcing and infrastructure. For example, investigators must invest in prospective screening for the desired patient cohort, create a bespoke trial database and randomisation schedule, perform data quality assessments and verification processes, ensure blinding procedures and personnel for outcome assessments, and maintain stringent documentation management.5,6 Furthermore, RCTs often have narrower inclusion criteria for their patient samples than a CQR cohort, which represents a more general population. Therefore, if the CQR captures the intended subset of the population for the trial, it can offer a source of patients from which to directly recruit participants. Conversely, if the trial sample is not representative of the intended population for an intervention (eg, due to selection bias arising from the types of centres recruiting the participants), being able to draw on broader population data from the CQR could be an advantage.
More recently, the established infrastructure of a CQR has offered the potential to nest efficacy (treatment versus placebo), effectiveness (treatment versus usual care or no treatment), or comparative effectiveness (treatment A versus treatment B) study designs. Comparative effectiveness research enables real-world studies of alternate options for existing healthcare interventions that can be used to prevent, diagnose, treat or monitor a clinical condition or improve the delivery of care.7 The registry infrastructure is cost-effective for answering clinical questions in a pragmatic way.8 This is because RRCTs aim to combine the methodology of an RCT that leads to high internal validity with the external validity of a clinical registry by enabling the recruitment of real-world patients, and leveraging existing registry platforms for data collection.5 Registry-based RCTs can be used for testing hypotheses involving pharmaceutical interventions, devices and other interventions already available in the real-world clinical setting.9 They can also provide the opportunity to evaluate the effectiveness of allied health interventions, for example: where there is variable implementation or uncertainty regarding optimal treatment combinations, sequencing or duration, or where multiple standard-of-care options are available.9 Table 1 outlines the characteristics of RCTs, CQRs and RRCTs. Investigators should be aware that there may be differences in the quality of reporting of an RCT that used a registry to assess a secondary outcome (a trial supported by registry data) and an RRCT, where the trial is fully planned and embedded within the infrastructure of a registry.
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