Paediatric Allergen-Specific Immunotherapy Studies Required by the European Medicines Agency: Is It Time for a Reassessment?

Abstract

Allergen-specific immunotherapy (AIT) works well both in children and adults. An often-alleged gap between the level of evidence of AIT efficacy in adults versus children is based upon the flawed ‘children-are-not-small-adults’ and ‘children-are-therapeutic-orphans’ mantras, both of which emerged in the 1960s. These mantras led to paediatric legislation in the USA in 1997 and the European Union (EU) paediatric regulation 10 years later. Although preterm newborns and newborns are vulnerable, during the first year of life their organs mature. Young children are no longer physiologically newborns; their immune system can overreact and cause allergic reactions, and AIT works for them just like it does in adults. Young patients need dosing recommendations and safety observations, rather than repetition of proof of efficacy. Placebo-controlled efficacy studies withhold effective treatment, increase the risk of asthma in the placebo group and are, therefore, in the authors’ opinion, unethical as well as in breach of the declaration of Helsinki. Individuals under the age of 18 years are not offered AIT treatments that are available to adults that are 18 years or older, but AIT treatment would be a suitable option. Since 2007, there were >100 EMA paediatric investigation plans that demanded ‘paediatric’ AIT studies involving tens of thousands of minors. Almost none were successfully undertaken and those that were done were unnecessary. It is time for the specialty of allergy to face this challenge.