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Towards evidence-based medicine for paediatricians
  1. Bob Phillips
  1. Centre for Reviews and Dissemination, University of York Alcuin College, York, UK
  1. Correspondence to Dr Bob Phillips, Centre for Reviews and Dissemination, University of York Alcuin College, York, UK; bob.phillips{at}doctors.org.uk

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Back to the future

I have been struck recently by a wave of ‘repurposing’ washing around, and the challenges which emerge from thinking through the problems and promise of new-old methods. Where the idea of ‘repurposing’, that is, taking (usually) drugs for one indication and using in another, is said to gain is the wealth of experience with the drugs and their often lower costs.

How do we use these old safety data? The key things to ask are ‘How might the ‘old’ population differ to the new one, in ways that would affect how the drug adverse events appear?’ and ‘How much can we trust reports from Z years ago?’

These are not new concepts. The latter asks you to consider—with your clinical expertise and knowledge—what the methodologies were and what biases we can see in them. We are learning more and more about the inadequacy of published trial reports for adverse events, and not just in cases of active suppression.1 The former asks you to consider not just what is different—differences are easy to spot—but which of those differences might actually matter. (For example, it might be the data for a diabetes therapy come from people aged 50–60 years old with serious cardiovascular risk factors, and you are considering it being repurposed in the treatment of leukaemia in children. Age directly would not be an issue, but we might reduce our belief in cardiac events and be more concerned about hypoglycaemia in a developing brain.)

In children’s health, we already know that relying on multiple large, direct, randomised trials is a pretty pointless way to practice. We are often left in the wash of adult evidence and have developed a skill for appreciating the indirect. Just repurpose these skills on the older drug data.

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Footnotes

  • Twitter @drbobphillips

  • Competing interests None declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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