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Something is moving in European drug research for children, but a more focused effort concerning all therapeutic needs is necessary
  1. C Pandolfini,
  2. M Bonati
  1. Laboratory for Mother and Child Health, “Mario Negri” Pharmacological Research Institute, Milan, Italy
  1. Chiara Pandolfini, Laboratory for Mother and Child Health, “Mario Negri” Pharmacological Research Institute, Via Giuseppe La Masa 19, 20156 Milan, Italy; pandolfini{at}

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There is a well-recognised lack of suitable paediatric therapies. This is caused by the great difficulty in performing paediatric clinical trials. However, a recent assessment1 evaluating and comparing ongoing and published paediatric drug-therapy trials found that significant therapeutic research efforts are ongoing.

The analysis examined data from both clinical trials in the Drug Evaluation in Children (DEC-net) European register of paediatric drug therapy trials2 and published articles, and compared the results with the WHO European Region Top 7 Burden of Disease data for 0–14 year olds (ERBoD) to assess adherence of research to therapeutic needs based on documented disease impact. The main area for which European paediatric research seems to be carried out, neoplasms, is not among the top 7 ERBoD.

The study’s data were analysed further. Data from trials representing ongoing research in Europe and published trials of EU research were compared with data from the ERBoD. Only 5% of the ongoing, and 9% of the published, trials addressed one of the three ERBoD areas concerning conditions treatable with drug therapies. The most commonly addressed, by both published and ongoing research, was asthma: the area of lowest burden in the Top 7 list.

Another recent analysis3 evaluated the published trials more thoroughly and compared the EU trials to the European Medicines Agency (EMEA) priority list for studies into off-patent paediatric medicinal products to compare the European research situation with paediatric drug therapy needs. The trials addressed the infectious and parasitic disease area most frequently (21,4%), and only addressed four of the 25 EMEA priority conditions, the most common of which were malignant diseases (18% of trials) and asthma (3%). This revealed a lack of overlap between therapeutic needs and research.

Two of the EMEA priority areas also appear in the ERBoD: migraine and asthma. However, while several trials (published and ongoing) addressed asthma, only one of the published, and none of the ongoing, addressed migraine. A possible explanation for such prioritisation of asthma research may be market pressure,4 as is true for infectious disease therapy studies.

The data evaluated show an overall lack of response of research to documented paediatric needs. Published research’s main interests involve three conditions (tumours, asthma and infections), which are always characterised by abundant research compared with other recognised therapeutic priorities. Thus, in Europe, children’s need for appropriate, effective medicines often remains neglected. Paediatric Regulation will certainly lead to more, improved, research but close monitoring is fundamental to verify that such research truly reflects children’s needs.



  • Competing interests: None.