News
19 August 2021
EU
CARE, - Medicines

The European Commission launched a consultation in May to hear from civil society on how to update current EU Regulations governing the commercialisation of medicines for children and for rare diseases in Europe.

As the European patient voice advocating change for all allergy and airways diseases patients in Europe, EFA participated in this process to inform the Commission about accessibility, inequalities and unmet needs on allergy and asthma treatments for children, and medicines targeting allergy and asthma disease subtypes.

This process is part of the Commission’s new General Pharmaceutical Strategy to establish new legislation responding to patient’s unmet needs, boost medicines research, and ensure the accessibility and availability of drugs throughout the Union.

An EU evaluation of current regulations shows that while they work for many diseases and population types, they are failing to materialise treatment solutions for children and for people living with a rare disease. There are numerous reasons behind their limitations, but a prominent cause is the trend to first approve drugs for adults, which delays specific research trials and authorisation for paediatric needs, while on the other hand there exists a lack of incentives to research into diseases affecting fewer people.

EFA’s response to the consultation outlines the most pressing needs around allergy and asthma that could be addressed by the regulations. Our contribution has been developed with EFA Members, and EFA’s European Allergy and Asthma Youth Parliament, endorsed by the European Academy of Allergy and Clinical Immunology (EAACI), and supported by the European Lung Health Group (ELHG).

Barriers accessing allergy and asthma medicines among children

EFA welcomes the Commission’s objectives and supports its proposed incentives for developing tailored medicine to better treat diseases, and we encourage the EU institutions to truly integrate patients’ needs and voices alongside input from other stakeholders such as academia.

On allergy and asthma, EFA has identified several barriers in accessing medicines, such as underdiagnosis of disease subtypes (which leaves many allergic and respiratory disease patients ill-informed around disease-management), delay and lack of adaption of adult drugs for children, limited access to clinical trials in medicine development, and limited knowledge (such as the lack of understanding of the underlying mechanisms in the development of diseases).

Unmet needs for allergy and asthma patients

Our allergy and asthma patient community stresses the need for personalised medicines to be developed for rare diseases and paediatric drugs which also offer accurate calculation of side effects, reduce allergens in drugs, and provide timely risk assessment information to patients. Additionally, we strongly advocate for the development of drug and device manufacturing legislation to provide patients with safe and easy to use lifesaving tools (e.g. adrenaline auto-injectors).

Lastly, EFA supports research incentives for the pharmaceutical industry with a strong legal framework behind. We also propose the creation of task force around paediatric medicine that does not neglect the need to explore scientific areas of great potential such as the impact of medicinal products during pregnancy or effects on new-borns.

Approaches to improve rare disease medicines legislation

We welcome the initiative of the European Commission to address patients’ unmet needs and to define new mechanisms to better deliver on those needs. While we acknowledge the EU’s main focus to encourage the development of therapeutic options for rare disease patients who lack access, we also recall that there are many chronic patients who are undertreated. We urge the European Commission to avoid working in silos and legislate for all children across disease areas, looking also at needs beyond the currently considered rare diseases.

Next Steps

The Commission plans to adopt the new regulation early 2022. EFA will continue to monitor negotiations on this topic, so that patient unmet needs in the EU respond to what patients identify as their more pressing needs.

The patient perspective is crucial when regulating medicines, especially when addressing rare and low prevalence diseases, as patient participation in trials and feedback throughout the lifecycle of a healthcare product is essential to maximise results and make the current unmet needs, a thing of the past.

You can read EFA's response to the consultation here.

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The EFA Team