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

From: Clinical trials in allergen immunotherapy in the age group of children and adolescents: current concepts and future needs

Methodological topic addressed in this reviewCurrent statusFuture needs
The regulation of AIT products and Pediatric Investigational PlanAIT 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 EMASolutions 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 trialsPatient 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 lackingFurther 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 relevanceThe 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 trialsThere 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 improvementPlacebo 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 levelMultiple 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 approachmHealth 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 routineWhile 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