The role of science and research in paediatrics

Why science and research are relevant to all paediatricians

The practice of medicine is described as both ‘art and science’, a helpful phrase that emphasizes that the care a doctor provides encompasses subjective (empathy, sensitivity, understanding, communication) and objective (evidence, factual knowledge, competencies) elements working in harmony. We all need to practise the ‘art’ of medicine when we explain science and research concepts to patients.

The very best paediatricians are also able to critically evaluate what they are taught, synthesize existing evidence, challenge dogma, identify knowledge gaps and understand how medicine advances through patient-centred research. We hope that this chapter will enable you to appreciate why these are professional obligations for paediatricians, and central, not peripheral, to clinical practice. We also hope that you will find that applying scientific principles to diagnostic and therapeutic problems is fun and rewarding .

The word science is derived from the Latin word for knowledge, ‘scientia’. Science is systematic; it builds knowledge incrementally through testing hypotheses. Science is perhaps best defined by the acceptance that there are few absolute truths, only ever diminishing uncertainty with each null hypothesis that is rejected following empirical testing. As we progress through our careers, we have a responsibility not only to test new therapies as they become available, but also to help identify which treatments and clinical practices in current use are harmful or useless, and progressively reduce uncertainties in care.

Try and project yourself thirty years into the future; look back at what you are being taught now; much of this will not have withstood the advance of knowledge and scientific scrutiny. If you think this is an exaggerated argument, consider two salutary lessons from the history of paediatrics ( Box 1.1 ). These examples illustrate two key points: that it is dangerous to assume that an untested practice is harmless and that getting evidence adopted into practice can be problematic. Try and think of examples of treatments or practices that are not evidence based but are widely used. Might these practices be harmful? What studies could be done to resolve these uncertainties? Try also to think of examples where translation of evidence into healthcare policy could be expedited through collaborative advocacy by professional bodies, charities and other third sector organizations.

Why children's research is essential

‘Children are not little adults.’

Children's research is necessary because the biology of disease in children is not necessarily the same as in adults. Human physiology alters with age, so that the actions of medicines may differ in the fetus, in children, and in adults (see also Chapter 36, Pharmacology and therapeutics ). There are some important examples of where this is clearly the case. Aspirin is widely used for pain relief and to reduce fever in adults but is not recommended for use in children because of the risk of a serious condition, Reye's syndrome, which causes liver damage and encephalopathy. Young people with cancer have significantly better survival when treated with protocols developed for children compared with protocols used for adults. The use of treatments designed for adults in children without adequate testing is dangerous and new treatments are not necessarily better than old ( Box 1.2 ). Understanding the science of children's disease can also help develop adult treatments ( Box 1.3 ).