As part of the marketing authorisation process for allergen products, clinical data must be submitted in accordance with current regulatory guidelines. However, this can lead to considerable challenges if only a small number of patients are affected, such as in the case of allergies to rarer allergen sources, and may hinder market access for products for the diagnosis and treatment of the corresponding allergies. The Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA) has therefore commissioned the Rheumatology and Immunology Working Party (RIWP) to draw up a specific guideline. This guideline deals with the question how sufficient scientific evidence can be provided for the authorisation of test and therapeutic allergens as part of product development if the number of patients available for the necessary clinical studies is too small and therefore adequate data cannot be obtained in accordance with the existing regulatory guidelines.
In a first step, a concept paper [1] for allergen products for whose development only medium to small study populations are available was drawn up in collaboration with the Paul-Ehrlich-Institut. The new scientific guideline (EMA/CHMP/72790/2024) has now been developed on the basis of this concept paper. It covers numerous aspects of test allergen products for the detection of type I and type IV allergies as well as aspects of therapeutic allergen products for whose development only a limited number of patients are available, such as patient selection, assessment of efficacy, study design as well as safety and quality issues.
The draft of this scientific guideline [2] was published by the EMA in February 2024 and can be commented on until 31 May 2024.
Updated: 09.04.2024
