Why the unmet needs of Sickle Cell Disease communities must be recognised

DISCLAIMER: All opinions in this column reflect the views of the author(s), not of EURACTIV Media network.

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Sickle Cell Disease needs focus and investment to provide patients access to appropriate care across the EU and policy makers need to act urgently to put an end to patient misery

Under the United Nation Sustainable Development Goals, European countries have committed to reduce premature mortality from non-communicable diseases by one third, and to promote mental health and well-being, by 2030. Moreover, under the European Commission’s EU Pharma Strategy, addressing unmet need in rare disease is a priority. Yet, despite these commitments, Sickle Cell Disease (SCD) patients in Europe are still falling through the gaps.

Currently, there is no unified approach at the EU level for increasing awareness and understanding of SCD among healthcare professionals. The lack of education about the disease and its complexity is a barrier to patients in need of a timely diagnosis and care.

The COVID-19 pandemic has further exacerbated issues in access to care for SCD patients across the EU and it is clear that more needs to be done to address these unmet needs, not only by industry but also by policy makers, clinicians and other stakeholders.

What is SCD?

SCD is an inherited blood condition present at birth. The red blood cells in people with the Sickle Cell gene cannot properly distribute oxygen to the body’s cells. When oxygen is released in the cells, the cells contract to become sickle or crescent shaped. When this happens, the cells become blocked in the blood vessels causing immense pain and resulting in what is known as a sickle cell crisis.

Recognising the scale of the issue in Europe

As much as 5% of the global population has the genetic trait for SCD.i The disease is more common in people of African descent as the disease originated in populations in the West African region. The sickle cell trait is also found in Hispanic, South Asian, Middle Eastern and Southern European populations.[i] Currently, SCD affects approximately 1 in 10,000 people across Europe, equivalent to 52,000 European citizens.[iv]

SCD is particularly common in France, in the UK but also in various other EU countries due to factors such as migration and the arrival of refugees from populations with a high prevalence of hemoglobinopathies.

Yet, the perception that SCD is not significantly prevalent in the EU means there has been little effort to track the disease population. This leaves a severe lack of epidemiological data to help understand the scale of the issue in the EU, and a lack of political will to use available policy levers to address disparities in SCD treatment across Member States.

Few options for care

SCD is the first monogenetic disorder for which the underlying cause has been identified. There are limited treatments available for managing the severe symptoms patients experience such as sickle cell crises (vaso-occlusive crises or VOCs), pain, anaemia, fatigue, and increased risk of serious infections. The current standard of curative treatment is for patients to undergo a bone marrow transplant, which is an invasive and risky procedure with no guarantee of curing the patient.

Some people with SCD can also experience strokes and complications affecting various parts of the body including lungs, eyes, kidneys, bones, and the heart and circulatory system.

The lack of clinical knowledge of the disease among healthcare professionals, and the unfair stigma associated with it, have lowered patients’ confidence in national health systems’ ability to provide care to them. Research from the UK suggests that most SCD patients do not use their primary care doctor to help manage their disease.[ii]

Beyond the physical effects, depression is diagnosed in 35.2% of adults with SCD because of living with intense pain episodes and overall decreased quality of life.Children with SCD may grow up with a bleak view of their future, as people with SCD have just 33 years of quality-adjusted life expectancy.[iii]

Addressing the unmet need in Sickle Cell Disease

SCD is recognised by WHO as a global health problem and yet there is not a co-ordinated approach to tackling the disease at the EU level. In view of the political discussions and the development of the EU Pharma Strategy, it is the right time to further improve patient access to diagnosis, information, and care for SCD.

Commitment from Novartis

Novartis works to raise the profile of diseases with high unmet medical need with few  or no treatment options. As part of this, Novartis is at a Global level involved in a medicines discovery and development programme to address the need of patients with SCD worldwide with the aspiration to provide curative solutions to patients in the near future.

Health equality is a fundamental right that needs to be more strongly protected . As we approach World Sickle Cell Disease Day on 19 June, we urge policy makers to focus on the unmet needs of SCD patients, including supporting research to improve treatment options and access to care to improve the lives of SCD patients in the EU.

[i] Piel, F.B. (2016) The present and future global burden of the inherited disorders of hemoglobin. Hematology/oncology Clinics of North America, 30, 327–341.

[ii] Juburi, G.A. et al (2012) Views of Patients about Sickle Cell Disease Management in Primary Care: A Questionnaire-Based Pilot Study. JRSM Short Rep., 11, 78.

[iii] Adam, S.S. et al (2017) Depression, quality of life, and medical resource utilization in sickle cell disease. Blood Adv., 23, 1983-1992.

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