The challenge of recruiting patients into a placebo-controlled surgical trial

Abstract

BackgroundRandomized placebo-controlled trials represent the gold standard in evaluating healthcare interventions but are rarely performed within orthopedics. Ethical concerns or well-known challenges in recruiting patients for surgical trials in general have been expressed and adding a placebo component only adds to this complexity. The purpose of this study was to report the challenges of recruiting patients into an orthopedic placebo-controlled surgical trial, to determine the number of patients needed to be screened and allocated in order to include one participant into the trial, and to identify reasons associated with participation in a placebo-controlled randomized surgical trial.MethodsData were extracted from an ongoing placebo-controlled randomized controlled trial (RCT) on meniscectomy versus placebo surgery. We calculated the number of patients needed to be screened in order to include the required number of participants into the RCT. Participating patients were asked about their rationale for joining the study and which type of information was most useful for deciding upon participation.ResultsA total of 476 patients entered the screening group, of which 190 patients fulfilled the inclusion and exclusion criteria. 102 patients declined to participate in the study due to various reasons and 46 were later excluded (no meniscus lesion on the magnetic resonance imaging scan or withdrawn consent). A total of 40 patients were finally included in the RCT. To include one patient into the RCT, 11.9 individuals needed to be screened. A total of 69% of participating patients considered the oral information to be the most important and the most common reason for participating was the contribution to research (90%).ConclusionsPatients are willing to participate in an orthopedic placebo-controlled surgical trial. Oral information given by the surgeon to the patient and the contribution to research are important aspects to enhance patient recruitment.Trial registrationClinicalTrials.gov NCT01264991, registered 21 December 2010.

Pain, placebo, and test of treatment efficacy: a narrative review

“…106 Also, not all patients may agree to take part in a surgical trial, especially if one of the arms is a sham procedure. 107 Second, in order to ensure blinding it is important that the placebo control is indistinguishable from a real surgery, so an imitation procedure has to be performed. This imitation procedure has to involve an incision and analgesia both of which are associated with risks.…”

“…111 As for patients, they are also opposed to deception but they find placebo treatment acceptable and are willing to take part in placebo-controlled surgical trials. 107,112,113 Still, many trials under-recruited or had to be terminated prematurely because they could not recruit more than one patient a month. This is because of patient preference and the fact that surgical trials have many exclusion criteria and often use additional inclusion criteria such as presence or absence of certain pathologies on MRI scans.…”

“…This is because of patient preference and the fact that surgical trials have many exclusion criteria and often use additional inclusion criteria such as presence or absence of certain pathologies on MRI scans. 103,107 With respect to the ethical issues, the counter-argument to Macklin's 110 opinion that surgery is unethical unless it can be used for therapeutic purposes, is that many nonpharmacological therapies never had their efficacy confirmed in an RCT, 114,115 and it is unethical to consent patients into a potentially risky treatment of unproven efficacy. A placebo-controlled trial can be ethically justified when outcomes are subjective and only this type of trial can provide an unbiased proof of treatment efficacy.…”

Femoroacetabular impingement surgery: are we moving too fast and too far beyond the evidence?

“…We appreciate the argument that it is difficult to convince patients to participate in these types of studies,135 136 where they fear to miss out on the already orthopaedically accepted ‘gold standard’ treatment 137. The problem of this argument, though, is that it also creates the very selection bias eventually criticised by orthopaedists, leaving the progress of orthopaedic and sports medicine practice in a real paradox, if such arguments are not challenged.…”

Randomised placebo-controlled trials of surgery: ethical analysis and guidelines

“…However, experience from the CSAW trial suggests that a survey among potential patients may be sufficient 14. Patients are willing to take part in SPTs42 43 and acceptability of placebo depends on how risky patients perceive it 44. There has been very little research on how patients conceptualise placebos and what is their attitude towards placebo-controlled trials 45.…”