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Open Access

Infographic

Decision-making in surgical study designs: a proposed decision algorithm to aid in the selection of an appropriate research study design for a given surgical intervention: the PERFECT tool



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Abstract

Cite this article: Bone Joint Res 2023;12(9):598–600.

High-quality clinical research in surgery is characterized by randomized controlled trials (RCTs)1,2 and large registry-based investigations.3-5 Trial design is a nuanced technique and requires careful thought. For example, if the clinician chooses to investigate a newly introduced intervention with a pragmatic RCT to estimate its effectiveness (how it works in the real world), without having first established its efficacy (the effect of the intervention under ideal conditions), then even if the pragmatic trial finds the intervention to be ineffective, this finding cannot be relied upon – we cannot know whether this finding is because the intervention is not effective, or because the delivery is not effective.6-8 It would therefore be more beneficial to identify whether the intervention is efficacious (with an explanatory trial), and to identify which sources of variation may influence outcome (such as learning curve,6,9 case-volume, and case-mix10) prior to embarking on a pragmatic trial. The PRagmatic-Explanatory Continuum Indicator Summary-2 (PRECIS-2) tool is a helpful aid indicating that the domains of a trial can vary between being explanatory or pragmatic in nature.1 The IDEAL framework11 for new techniques aids clinicians in deciding which surgical study design would be most appropriate based on the stage of development of a new surgical device/innovation. However, it does not encompass the comparison of existing surgical techniques, or established surgical and non-surgical treatments. For established techniques, registries or large datasets may exist. The power of existing data should also not be underestimated – appropriate use of existing datasets and pooled analyses (systematic reviews or meta-analyses) can be invaluable in deciding whether a trial is required, and/or what type.10 Registries can also help to determine the type of study that would be suited for investigation of an intervention, based on the source of variation identified: if the variation is at the patient level, the next step may be to carry out a pragmatic trial, however if these datasets show large variation in the surgical care systems involved in procedure administration, then reorganization of the system, using a quality improvement approach, may be preferred.10,12 Surgery should be considered a complex intervention, and quality improvement necessitates meaningful engagement with patients, practitioners, and policy-makers, ensuring that research moves beyond binary questions of effectiveness, to whether interventions can be safely implemented in an acceptable and cost-effective manner, scaling across settings and populations.13 The National Institute for Health and Care Research (NIHR)/Medical Research Council (MRC) complex intervention framework provides guidance that should be more broadly applied in surgical settings.13 Our infographic (PERFECT – Pragmatic, Explanatory, Registry Framework for sElection of Clinical Trial format) provides a proposed decision tool for selecting the appropriate study design for the intervention-based research question for treatments already in current practice.

Fig. WL1


Correspondence should be sent to A. Hamish R. W. Simpson. E-mail:

References

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Author contributions

A. H. R. W. Simpson: Conceptualization, Writing – original draft, Writing – review & editing.

N. S. Makaram: Writing – original draft, Writing – review & editing.

E. Harrison: Writing – review & editing.

J. Norrie: Writing – review & editing.

Funding statement

The authors received no financial or material support for the research, authorship, and/or publication of this article.

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© 2023 Author(s) et al. This is an open-access article distributed under the terms of the Creative Commons Attribution Non-Commercial No Derivatives (CC BY-NC-ND 4.0) licence, which permits the copying and redistribution of the work only, and provided the original author and source are credited. See https://creativecommons.org/licenses/by-nc-nd/4.0/