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PLOS BLOGS Speaking of Medicine

Measuring the right outcomes in clinical trials

Astonishingly, for most clinical areas there is no general consensus on what outcomes should be reported in clinical trials. Alhough one speciality, Rheumatology, has gone a very long way towards addressing this issue in the form of the OMERACT initiative, most others have not.

To take this situation to its extreme, a researcher could decide to do a clinical trial on a treatment for a life-threatening disease and decide, say, not measure deaths or indeed any outcomes that matter  to patients. A paper published in 2009 in PLoS ONE by researchers from Liverpool on outcomes in trials of asthma illustrated this problem exactly – the title “Outcomes in Clinical Trials of Inhaled Corticosteroids for Children with Asthma Are Narrowly Focussed on Short Term Disease Activities” says it all.

Another paper from this same group, “A Systematic Review of Studies That Aim to Determine Which Outcomes to Measure in Clinical Trials in Children” published in PLoS Medicine showed that the problem is not confined to asthma – as the authors say: “Very few studies address the appropriate choice of outcomes for clinical research with children, and in most paediatric specialties no research has been undertaken. Among the studies… very few involved parents or children in selecting outcomes that should be measured, and none directly involved children.”

A group, including clinicians, researchers and editors (I attended from PLoS Medicine), recently met in Liverpool as part of the activities of North West Hub for Trial Methodology Research under the COMET initiative to try to begin to stimulate some action around this important aspect of clinical research. Some of the talks from the meeting are now available online.

Discussion
  1. What outcomes are selected as results for a clinical trial needs serious review. Short-term effects, soft markers, and surrogate endpoints all serve to mislead us as to the true medical effect versus what might be an interesting research effect. Head to head comparisons of treatments and interventions as we seek to evaluate changes in the medical care system will also require that we clearly define what really matters.

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