First New Sickle Cell Disease Treatment in 20 Years to Be Available on NHS

Dawn O'Shea

October 05, 2021

For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the NHS. Crizanlizumab (Adakveo) has been 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 years or over.

Crizanlizumab infusions can be given as add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate.

Over 300 people a year will initially be eligible for the treatment via a managed access agreement (MAA), increasing to more than 450 people in subsequent years. It is also estimated that the drug will reduce the number of times a sickle cell patient needs to go to accident and emergency (A&E) by two fifths.

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, NICE has recommended the treatment through the MAA, which will allow people to access crizanlizumab while additional data are collected through clinical trials to address these uncertainties.

This decision is based on crizanlizumab being cost effective according to the terms of the MAA. NICE has also taken into account the high unmet need for treatments for sickle cell disease and the aim of reducing health inequalities.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: "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 which 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."

The NHS has welcomed the decision. NHS chief executive Amanda Pritchard said: "This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades."

"This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduce trips to A&E by almost half."

Last year, NHS England set up 10 new dedicated centres to treat sickle cell disease across the country, and patients will be able to access the new treatment through their consultant at one of these clinics.

Toks Odesanmi, a sickle cell patient attending Cambridge University Hospitals Trust, said: "Sickle cell disease has defined me, defined my body and made a big dent to my dreams. No matter how hard I fight it continues to defeat me. A new treatment brings hope and might make dreaming possible again."

This article originally appeared on Univadis, part of the Medscape Professional Network.

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