For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK National Health Service (NHS). Crizanlizumab (Adakveo) by Novartis is recommended by the National Institute for Health and Care Excellence (NICE) 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.
Sickle cell disease is a blood disorder characterised by red blood cells that become sickle-shaped, leading to clumping and blockages in the small blood vessels. These blockages can cause a loss of blood flow and severe pain in episodes known as vaso-occlusive crises (VOCs), or sickle cell crises. The frequency and severity of VOCs are unpredictable, and each episode can take days or weeks to recover, sometimes requiring hospitalisation.
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 disease. 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.
However, 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 at this stage. 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.
This 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.”
Dr Perla Eleftheriou, Consultant Haematologist, University College London Hospitals, and TIF’s Scientific Advisor commented on the decision stating that it constitutes a positive step towards expanding the limited therapeutic options of patients with the disease and addressing their unmet needs.