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The guidance document titled “Allergen Products Development for Immunotherapy and Allergy Diagnosis in Moderate to Low Sized Study Populations,” published by the European Medicines Agency (EMA) and set to take effect on January 1, 2026, is specifically designed for allergy types where the number of patients is limited. We will discuss the importance, scope, and details of this guideline in our blog post.
What innovations does this guideline—which offers flexible regulatory frameworks to researchers and manufacturers during the development of diagnostic and immunotherapy products—bring? Here are the key highlights…
The difficulty of conducting clinical trials for certain types of allergies is the primary reason for this guideline. The challenges in finding enough patients for studies on rare insect venom allergies or specific food allergies are the main drivers behind the guideline. The EMA acknowledges that alternative approaches may be possible in such cases.
Allergen products covered by the guideline:
• Affected organ system (e.g., upper and lower respiratory tract, eyes, skin, multi-organ involvement (systemic reaction)),
• Allergen source (e.g., pollen, dust mites, animal dander, molds, insect venoms, foods, chemicals),
• Allergen product (e.g., extracts, purified allergens, modified allergens, adsorbed allergens)
• Regardless of the route of administration (e.g., subcutaneous, sublingual, oral, percutaneous), allergen products for the allergen immunotherapy of Type I allergies and the diagnosis of Type I and Type IV allergies
When evaluated on its own, this guidance is not considered sufficient. Evaluating it in conjunction with other EMA, CHMP, and ICH guidelines facilitates achieving results. A key point for manufacturers and researchers is to clarify the development strategy by engaging with regulatory authorities early in the planning of studies.
The relevant guidelines in question:
• Guideline on clinical trials in small populations - CHMP/EWP/83561/2005
• Guidance on missing data in confirmatory clinical trials - EMA/CPMP/EWP/1776/99 Rev. 1
• Guidance on adjustment of baseline covariates in clinical trials - EMA/CHMP/295050/2013
• Guidance Note on Statistical Principles for Clinical Trials - CPMP/ICH/363/96
• ICH E9 (R1) Addendum on Estimates and Sensitivity Analysis in Clinical Trials to the Guidance on Statistical Principles for Clinical Trials - EMA/CHMP/ICH/436221/2017
• Guidance Note on the Selection of the Control Group in Clinical Trials - CPMP/ICH/364/96
• Guidance on the Clinical Evaluation of Diagnostic Agents - CPMP/EWP/1119/98/Rev. 1
• Guidance on the clinical development of specific immunotherapy products for the treatment of allergic diseases - CHMP/EWP/18504/2006
• Guidance on Allergen Products: Manufacturing and Quality Issues - EMEA/CHMP/BWP/304831/2007
• Guidance on Process Validation for Finished Products - Information and Data to be Provided in Regulatory Applications - EMA/CHMP/CVMP/QWP/BWP/70278/2012-Rev1, Corr.1
• Recommendations on Common Regulatory Approaches for Allergen Products - CMDh/399/2019
The allergen categories covered by the guideline are as follows.
• Allergens for diagnostic purposes in in vivo tests: Type I (prick test, provocation test, intradermal/intrakutaneous test) and Type IV (epicutaneous patch test)
• Allergen immunotherapies - AIT (inhaled allergens, insect venom allergens, food allergens).
It is noted that in cases where standard laboratory tests or comprehensive non-clinical studies cannot be conducted, alternative approaches such as literature reviews and expert opinions may be accepted. It is believed that this approach will help accelerate the development process in small populations.
In allergic studies with a small number of patients, the guideline emphasizes that evidence such as retrospective data, case reports, and “named patient” product use can be utilized as supporting data, and that alternative metrics like PR (positivity rate) and RI (reaction index) also have a role in assessing diagnostic accuracy.
With this EMA guideline, a new regulatory framework is introduced for allergen product studies that can be conducted with a small number of patients. Thanks to flexible data sources, alternative clinical designs, and adaptations in quality processes, the development of diagnostic and treatment options for rare types of allergies is becoming easier.
You can find detailed information by reviewing the guideline: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-allergen-products-development-immunotherapy-allergy-diagnosis-moderate-lowsized-study-populations_en.pdf
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