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NICE recommends first sickle cell disease treatment in two decades

The National Institute for Health and Care Excellence (NICE) has recommended that a new therapy for sickle cell disease is made available on the NHS for the first time in 20 years.

Novartis’ Crizanlizumab (Adakveo) has been recommended as a treatment option for preventing recurrent sickle cell crises in people aged 16 or over. More than 300 people a year are expected to receive the treatment via a Managed Access Agreement (MAA), increasing to more than 450 people in subsequent years.

Current treatments to prevent sickle cell crises include a tablet called hydroxycarbamide and regular blood transfusions, which are not always effective for people with severe diseases. Crizanlizumab is a new intravenous treatment option that can be taken on its own or alongside hydroxycarbamide.

Clinical evidence suggests that people treated with crizanlizumab have significantly fewer sickle cell crises in a year than those receiving other standard treatment options.

Currently, there is high uncertainty about the long-term effectiveness of the treatment and the cost-effectiveness, so crizanlizumab could not be recommended for routine use on the NHS right now. Instead, the committee has recommended the treatment through the MAA with the NHS, which will allow people to access crizanlizumab while additional data is collected through clinical trials to address these uncertainties.

The decision was based on crizanlizumab being cost-effective according to the terms of the MAA. The committee’s decision-making also took into account the high unmet need for treatments for sickle cell disease and NICE’s aim of reducing health inequalities.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: “Sickle cell disease can be a debilitating condition that has a huge impact on patients’ quality of life. Yet treatment for sickle cell disease has been limited for years, and there has been a lack of treatments for patients whose lives are affected by the condition. Crizanlizumab is an innovative treatment that has shown the potential to improve hundreds of lives, and we are delighted to be able to recommend it as the first new treatment for sickle cell disease in two decades. We don’t yet know whether the benefits will translate in longer-term outcomes, and we look forward to seeing what the data collected through this managed access agreement will uncover about its benefits for the future.”