Redefining Expectations for life with hereditary angioedema (HAE)

HAE is a rare genetic condition that causes repeated episodes or ‘attacks’ of severe swelling. HAE attacks can affect any part of the body, but the most commonly affected are the face, limbs and extremities, gut and airway.

Scientific advancements have unlocked significant progress in the treatment of HAE in recent years, with new therapies providing additional options for patients and clinicians, to manage the condition and reduce its impact on daily life.

However, some countries have been slower to adopt these new treatments than others, meaning that considerable variation exists internationally in patients’ access to treatment. Without action to remove barriers to access, there is a risk that progress will not be made on patient outcomes.

Takeda wants to be at the forefront of working with others to address these challenges. We therefore investigated variations in approaches to the management of HAE in Europe and Canada, to identify the extent to which patients in these countries are receiving care in line with international best practice standards

This site sets out some of the findings from our ‘Redefining Expectations’ report, which is aimed at supporting collaboration with patients, clinical experts and policymakers to drive improvements in outcomes for people living with HAE across the world.

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HAE is very rare, occuring in
1 in 50,000 people¹

HAE can affect
Various parts
of the body,
including the hands, feet,
face and airway¹

HAE can be life-threatening, as
attacks that effect the throat can
close the airway and lead to
suffocation¹

HAE can be very hard to
diagnose, with the average
time between the onset
of symptoms and diagnosis
ranging from
13-21 years¹

Diagnosis is vital, as the
risk of death is up to
nine times
higher with undiagnosed
HAE compared to those
who have a diagnosis¹

HAE can be very painful, with
two thirds
of patients rating the pain
of their most recent swelling
attack as 'moderate' or 'severe'¹

HAE can be life-limiting, with one
in three patients experiencing
more than one attack per month
and the average attack lasting
2 - 5 days¹

HAE can have wider
socioeconomic impacts on both
patients and caregivers, with
82% of patients
with severe HAE reporting
work orschool absenteeism
during their lastattack, for an
average of 5.1 days¹

Recommendations for improving the quality of HAE care

In recent years, many countries have made progress in improving HAE outcomes. However, Takeda believes that more can be done to redefine expectations for HAE, across the patient journey.

We have identified four specific areas in which we believe progress can and should be made:

Speeding up
diagnosis

Too often people with HAE have to wait too long to receive a diagnosis. These delays can have a material impact on both their physical and mental wellbeing.

All health systems should set ambitious goals to reduce delays in diagnosis for HAE.

Delivering holistic
patient care

Patients with HAE have a diverse set of needs that require comprehensive support from a range of different health specialties.

There is a need for expert clinical and patient consensus to be forged on service standards and patient pathways, to inform the adoptions of best practice by health services.

Providing access to the right treatments at the right time

There is a high level of international variation in access to treatments, with patients in some countries unable to access treatments recommended in international best practice guidelines.

Health systems should take steps to ensure that patients are able to access the medicines that are recommended by clinical experts and seek to provide rapid access to new treatments that could improve outcomes.

Collaborating internationally to share best practice and data

International collaboration between patient and clinical experts can be a key enabler of improved care. However, participation in international initiatives is not consistent.

National expert groups and networks should work with governments and healthcare systems to encourage greater collaboration in best practice sharing and data collection mechanisms.

Prevalence of HAE

Due to its rarity, the exact prevalence of HAE is still not known. Globally, it is thought to affect 1 in 50,000 people.¹

The rarity of the condition creates significant challenges for people living with HAE, from diagnosis, through to treatment and care.

From the biological perspective, there are three main types of HAE:²

Type 1 HAE, which is estimated to occur in 80% to 85% of patients, which is caused by the decreased production of a protein, C1-INH, that is important to the normal functioning of the immune system
Type 2 HAE, which is estimated to affect 15% to 20% of patients, in which the body produces C1-INH, but only in a damaged form that does not function properly
Type 3 HAE, now typically referred to as HAE with normal functioning C1-INH, which is an extremely rare form of the condition and is less well understood than types 1 and 2

Diagnosis of HAE

Early diagnosis of HAE is vitally important. Undiagnosed HAE is associated with poorer outcomes for patients, including a much higher risk of death.⁴

However, HAE is difficult to diagnose, as the symptoms are similar to those for many more common conditions. This can result in individuals seeing multiple healthcare professionals over an extended period of time before they are eventually diagnosed.

As a result, patients with HAE typically face long waits to receive a diagnosis, compared to those with more common conditions.

However, there are some signs that increased knowledge and understanding among clinicians is starting to drive down the average length of time patients have to wait in some countries, before receiving a diagnosis of HAE.⁵

Median Mean

Outcomes of HAE

Without effective treatment, some people living with HAE experience regular, lengthy and very painful attacks.

As a result, uncontrolled or poorly controlled HAE can have a significant impact on the ability of those affected to live normal lives, due to both the frequency and severity of attacks.

Scientific advancements have unlocked significant progress in the treatment of HAE in recent years, with new therapies providing additional options for patients and clinicians to manage the condition and reduce its impact on daily life.

However, some countries have been slower to adopt these new treatments than others, meaning that considerable variation exists internationally in patients’ access to treatment.¹ Without action to remove barriers to access, there is a risk that progress will not be made on patient outcomes.

Number of attacks Severity of attacks

Total population:

Estimated people with HAE:

HAE patient organisation:

Patient organisation website

More Information

Source of population statistics (2019 data): https://data.worldbank.org/indicator/SP.POP.TOTL

Reference

HAE International, The Current State of Management of HAE in Europe, November 2015. Available at: https://haei.org/wp-content/uploads/2016/03/HAEi-Updated-Eu-SoM-Artwork_ENGLISH_EUROPE_280216_FINAL.pdf
NZEAKO, UC et al. (2001). Hereditary Angioedema: A Broad Review for Clinicians. Arch Intern Med.161(20). 2417–242
Takeda data on file
ZANICHELLI, A et al. (2013). Hereditary angioedema with C1 inhibitor deficiency: delay in diagnosis in Europe. Allergy, Asthma & Clinical Immunology. 9, Article 29
ZANICHELLI, A et al. (2018). Improvement in diagnostic delays over time in patients with hereditary angioedema: findings from 36. the Icatibant Outcome Survey. Clinical and translational allergy. 8(42)
CABALLERO, T et al. (2017). The Icatibant Outcome Survey: 37. experience of hereditary angioedema management from six European countries. Journal of the European Academy of Dermatology and Venereology: JEADV. 31(7). 1214–1222