On 13 December 2018, the EMA published a “Concept paper on a Guideline for allergen products development in moderate to low-sized study populations”. Four EMA guidelines concerning medicinal products containing allergens are currently applicable. They provide guidance for the clinical development and quality control of medicinal products containing allergens. All of these guidelines start from the premise that the population of the clinical trial is large enough to gather adequate data.

The goal of the concept paper is to create a scientific guideline for the development and manufacturing of medicinal products for the treatment of rare allergic diseases. The consequence of the smaller population is that there is not enough adequate data to develop treatment for these diseases.

The concept paper concerns medicinal products containing allergens for the purposes of in vivo diagnosis as well as for treatment of allergic disease. The paper explains that there is an unmet medical need for effective diagnosis and disease modifying treatment[1] for patients suffering from these rare allergic diseases. The Guideline is intended to provide guidance on how to gather adequate data despite the smaller size of the population.

Focus points

The EMA Rheumatology and Immunology Working Party[2] has recommended that the new guidance address general aspects on the development of medicinal products containing allergens for small populations. It is to provide guidance on how to select patients, how to assess the efficacy of a medicinal product, how to design therapeutic studies as well as guidance concerning safety aspects and quality considerations.

The Working Party has recommended focussing in particular on:

  • Identifying manufacturing and quality control aspects that should apply to medicinal products containing allergens to treat rare allergic diseases;
  • Defining classes of prevalence and feasibility of allergen sources to conduct clinical trials;
  • Defining Strategies for adequate dose selection considering aspects of feasibility and necessity.


The members of the Working Party will develop the draft guideline. The Working Party has proposed 07/2020 as a target release date for the draft guideline.

Stakeholders have the opportunity to submit comments until 30 January 2019.