Table 1 Current status and future needs of current concepts in AIT

The regulation of AIT products and Pediatric Investigational Plan

AIT products are regulated by the European Medicines Agency (EMA) in Europe and the Food and Drug Administration (FDA) in the US following similar principles regarding the clinical documentation in principle. Marketing authorization for AIT products in children have to be in line with the “Pediatric Investigational Plan” of the EMA

Solutions regarding the feasibility of clinical trials in children should be elaborated by all relevant stakeholders in the field of AIT

Patient reported outcomes and clinical endpoints in AIT trials

Patient Reported Outcomes and clinical endpoints in AIT trials should be harmonized and further evaluated re. their composition and clinical relevance. Though academy has proposed further recommendations for adult patients, definition of ideal endpoints in the pediatric population is still lacking

Further efforts should be put into the development and standardization of clinical endpoints suitable for children in pediatric AIT trials. Symptom-medication scores need to be defined with a specific emphasis on pre-school children

Minimal clinically important difference in AIT trials and clinical relevance

The aim of AIT trials is to demonstrate clinical efficacy and safety of investigated products. However, the minimal magnitude of the treatment effect size which is clinically relevant to the patient (minimal clinically important difference (MCID)) is not thoroughly validated and therefore justified for almost all endpoints in AIT clinical trials

There is a clear need for further exploration of MCID in AIT trials for multiple endpoints and this is in particular important in pediatric AIT trials to improve quality and feasibility of trials in this age group

Placebo effects in AIT trials and considerations for trial design improvement

Placebo mechanisms play an inevitable role in the field of AIT. These effects should be used within the whole treatment concept in clinical routine on one hand, but may also have some bias on conclusions drawn from clinical trials in AIT where they should be reduced to the most minimal level

Multiple effects of “the” placebo mechanisms in AIT treatment should be better evaluated and understood. These effects may play an important role especially in AIT of pediatric patients

mHealth in the AIT precision medicine approach

mHealth continuously plays an important role in daily healthcare delivery in general. Mobile used Apps may become a very effective tool in clinical trials in AIT (e.g., selection of patients, readouts of efficacy measures and others) and also for monitoring patients in daily life routine

While Apps and other of mHealth tools are steadily improving (‘machine learning’), easy and smart applications especially for monitoring of children and adolescents are needed. Even more this technique would allow a more precise evaluation of any kind of medical intervention such as e.g., AIT in pediatrics