What makes a great clinical trial? Editorial about PEDro’s Top 5 Trials in 2014-2019 provides some insights

Some trials are ground-breaking and grab a great deal of attention. Others fail to excite. What makes them different? A recent editorial published in the British Journal of Sports Medicine featured the PEDro Top 5 Trials in 2014-2019 initiative and offers some insights.

To mark PEDro’s 20th anniversary, we invited physiotherapists from all over the world to nominate randomised clinical trials published between 2014 and 2019 that answered important clinical questions in a robust and novel way. A panel of experts in clinical trials judged the nominations and established the Top 5 Trials: LIPPSMAck POP, SARAH, AVERT, HIHO, and UK FASHIoN.

LIPPSMAck POP demonstrated that a single 30 minutes education and training session delivered by physiotherapists halves the incidence of hospital-acquired pneumonia when compared with an information booklet. SARAH found that adding hand exercises to usual pharmacological care improves hand function in rheumatoid arthritis and is cost-effective. AVERT revealed that very early mobilisation following stoke (within 24 hours) led to more disability than usual care (OR 0.73, 95% CI 0.59 to 0.90). HIHO found that intensive inpatient rehabilitation (two sessions/day for 10 days) was not better than monitored home-exercise for uncomplicated total knee arthroplasty. UK FASHIoN demonstrated that hip arthroscopy improved quality of life more than ‘best’ conservative care (6.8 points; 95% CI 1.7 to 12.0). Short videos summarising each trial are available on the PEDro website.

The PEDro Top 5 Trials attracted widespread attention. AVERT has already been cited over 250 times, HIHO was picked up by 13 news outlets, including the New York Times. SARAH and AVERT have changed guidelines. LIPPSMAck POP, AVERT and UK FASHIoN have informed the development of decision tools for clinicians.

What sets the Top 5 Trials apart from other trials? These five trials had rigorous methods (scoring 8 out of 10 on the PEDro scale), high follow-up rates (89% to 99%), prospective registration, moderate or large sample sizes (range 165 to 2,104) and recruited from many sites (2 to 56). But there are two other crucial characteristics that set these trials apart! First, they all asked important questions that addressed significant gaps in clinical practice for conditions with high disease burden. And second, they all had clinically important endpoints and clear implications for clinicians and health systems. Interestingly, AVERT and HIHO were ‘negative trials’ that highlighted a need to change current practice. Trials do not have to have large positive findings to be influential. Negative findings can also have important implications for patient care and health policy.

Inspection of the Top 5 Trials in physiotherapy tells us that if you want to conduct a landmark trial, you need to have more than top-notch methods and a large sample size. Landmark trials ask important questions, challenge conventional thinking and have immediate implications for clinical practice. Including health economic evaluation makes trial results more impactful because those data can influence policy. In short, ask important questions … and answer them with clinically relevant results!

Amorim AB, et al. What makes a great clinical trial? Exploring the features of five important physiotherapy trials. Br J Sports Med 2020 Aug 7:Epub ahead of print

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