Novartis update on sickle cell treatment in England and Wales 

Blood cells

Novartis has announced that eligible patients in England and Wales will soon have routine access to Adakveo (crizanlizumab) under a Managed Access Agreement (MAA), which the company says not only helps with sickle cell disease (SCD), but also demonstrates a commitment to supporting health equity for a community that has been historically underserved, without any new treatment options for more than 20 years, and disproportionately impacted by Covid-19. 

The news comes as the National Institute for Health and Care Excellence (NICE), published the Final Appraisal Determination (FAD) recommending crizanlizumab as an option for preventing recurrent SCD crises (two or more vaso-occlusive crises, VOCs, in a year, managed at home or in hospital) in people aged 16 or older with SCD.2   

John James OBE, Chief Executive, Sickle Cell Society, said: “Sickle cell crises are much more than unpredictable, painful events: recurrence is a constant worry that pervades every aspect of daily life. For the thousands of people living with this complex and life-long disease in this country, this decision offers hope of a life with less disruption and stress from their condition, as well as reducing the associated costs to the NHS.” 

VOCs are one of the main symptoms of SCD.3,4 The sickling of red blood cells can result in the cells sticking together, along with increased adhesion to other blood cells and blood vessel walls. This can cause blockages in smaller blood vessels and trigger episodes of acute pain (crises).4,5 Sickle cell pain crises disrupt patients’ lives physically, socially, and emotionally – and can worsen into acute and long-term complications.6 People living with SCD suffer an increased risk of high blood pressure, eyesight loss, and kidney and urinary problems, along with long-term damage to internal organs, such as kidneys, liver, spleen, heart and lungs.3 In addition, SCD can have a significant impact on an individual’s ability to engage in normal life, limiting career and educational opportunities, along with associated mental health challenges.7 

“Vaso-occlusive crises are acute episodes of pain that often last for days, and have a substantial impact on the quality of life of people living with sickle cell disease. These episodes can also lead to organ damage and significant long-term complications,” said Professor Baba Inusa, Chair of the National Haemoglobinopathy Panel, Professor of Paediatric Haematology at King’s College London and lead consultant for paediatric haemoglobinopathies, Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust. “Today’s decision is an important milestone for the medical community: for the first time in 20 years, there will now be a new treatment regime that we can offer this community, which has gone for so long without additional support in managing their condition.” 

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