Selecting and measuring optimal outcomes for randomised controlled trials in surgery

Abstract

BackgroundRandomised controlled trials (RCTs) in surgery are complex to design and conduct and face unique challenges compared to trials in other specialties. The appropriate selection, measurement and reporting of outcomes are one aspect that requires attention. Outcomes in surgical RCTs are often ill-defined, inconsistent and at high risk of bias in their assessment and historically, there has been an undue focus on short-term outcomes and adverse events meaning the value of trial results for clinical practice and decision-making is limited.PurposeThis review addresses three key problems with surgical trial outcomes—choosing the right outcomes for the trial design and purpose, selecting relevant outcomes to measure from the range of possible outcomes, and measuring outcomes with minimal risk of bias. Each obstacle is discussed in turn, highlighting some suggested solutions and current initiatives working towards improvements in these areas. Some examples of good practice in this field are also discussed.ConclusionsMany of the historical problems with surgical trial outcomes may be overcome with an increased understanding of the trial design and purpose and recognition that pragmatic trials require assessments of outcomes that are patient-centred in addition to measurement of short-term outcomes. The use of core outcome sets developed for specific surgical interventions and the application of novel methods to blind outcome assessors will also improve outcome measurement and reporting. It is recommended that surgeons work together with trial methodologists to integrate these approaches into RCTs in surgery. This will facilitate the appropriate evaluation of surgical interventions with informative outcomes so that results from trials can be useful for clinical practice.

What outcomes are important in the assessment of Enhanced Recovery After Surgery (ERAS) pathways?

“…9 Outcomes are inherently multidimensional, and there is no single outcome that fully captures the results of care for any condition. 10 Clinical outcomes are endpoints used in clinical practice, such as complications, and are assessed by an observer.…”

“…A core set of outcomes is an agreed set of outcomes (usually less than ten) to be measured for a particular health condition or treatment, prioritized by the relevant stakeholders. 9 These include clinical outcomes and PROs. Measurement of the processes of care, such as adherence to elements of an ERAS pathway, is important for understanding how a system is functioning and where improvements should be made, but it does not replace outcomes measurement and should be considered separately.…”

Investigating the collection and assessment of patient-reported outcome data amongst unplanned surgical hospital admissions: a feasibility study

“…Furthermore, surgical trials often focus on outcomes relevant to surgeons (e.g. technical endpoints such as morbidity), and there is a lack of RCTs in surgery with high-quality patient-reported outcome (PRO) data [5, 6]. …”

A systematic literature review of time to return to work and narcotic use after lumbar spinal fusion using minimal invasive and open surgery techniques

“…This might to some extent reflect the actual situation in surgical research, i.e., retrospective case series are more commonly used, usually with small cohorts, while RCTs are more widely used for pharmacological therapies [54, 55]. TLIF was found to be the most frequently studied technique for lumbar spinal fusion operations regardless if MIS or OS was used.…”