Championing change for rare disease patients in Europe: the case of hereditary angioedema

By Lutz Bonacker

Lutz Bonacker is SVP & General Manager of Commercial Operations in Europe, CSL Behring.

16 May 2024

HAE Day, celebrated globally on May 16, is a chance to recognise progresses made in the care of people living with hereditary angioedema (HAE), while shining a light on remaining challenges.

 “HAE places extraordinary strain on patients, often restricting their ability to lead normal lives. Untreated HAE patients can lose 100 to 150 workdays per year, if not more. Global awareness initiatives such as hae day :-) are necessary in order to help people with HAE receive a faster diagnosis and appropriate medical care” explains HAE International (HAEi), the global non-profit network of HAE patient associations.

This year carries a specific sense of opportunity for European patients, with the next European Parliament and Commission mandates bringing new hope for patient care and access to innovation across the European Union.

About HAE

HAE is a debilitating genetic disorder characterised by episodic and unpredictable swelling in the hands, feet, face and airways. It is estimated to affect 1 in 50 000 individuals. HAE has a major impact on patients’ quality of life, as frequent, painful attacks affect everyday life and increase feelings of anxiety and depression.

Approximately 50% of patients will experience at least one potentially fatal airway attack during their lifetime, with undiagnosed patients at a greater risk of asphyxiation and facing a higher mortality rate. Yet, diagnosis remains a significant challenge with the lack of disease-awareness making late diagnosis or misdiagnosis a reality for many patients whose symptoms are often misconstrued as allergies.

Understanding the incremental value of innovation for rare disease

The burden of the disease remains high for HAE patients, despite the availability of effective preventative treatments. Current options may not be well-tolerated or can require a high frequency of administration, with attacks still likely to occur. Other patients continue to face issues with self-administration or treatment eligibility. 

It is essential to monitor unmet needs, including the impact of persistent attacks on all aspects of patients’ quality of life, but also the impact between attacks due to their unpredictability, which can cause stress and anxiety.

Amplifying the patient voice across the medicine lifecycle is crucial to inform the development of high-quality, well-tolerated and convenient therapies and one day make attack-free lives a reality for all patients.

Collaboratively shaping a comprehensive EU Action Plan on Rare Diseases 

While the past decades have proven European frameworks’ role in harmonising care standards and pathways, the development of a comprehensive EU Rare Disease Action Plan should be a priority of the next Commission to ensure treatment access in practice.

In order to adequately answer patient unmet needs, the Plan should strive to holistically address the challenges faced by the rare disease community in accessing care by formalising cooperation and leveraging good practices to address knowledge gaps, building on previous European achievements and centers of excellence.

Today more than ever, CSL Behring advocates for multi-stakeholder dialogue and joins HAEi’s call for further collaboration among national governments, regulatory authorities and industry to enable an EU-wide access to existing and next-generation HAE therapies.

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DISCLAIMER: This article was initiated by CSL Behring and builds on the contribution of patients from HAEi to mark HAE Day - a global, patient-led disease awareness day celebrated on May 16. For more information on this day, please visit: