The first new treatment sickle cell disease in 20 years has been made available on the NHS, it was announced last week.
The antibody-based drug crizanlizumab works by binding to a protein, P-selectin, to prevent the sticking of blood cells to the walls of blood vessels, which contributes to a loss of blood flow and severe pain known as vaso-occlusive crises.
Although clinical evidence has suggested it can reduce sickle cell crises, there is uncertainty about its long-term effectiveness, according to regulators at the National Institute for Health and Care Excellence (NICE).
NICE said it cannot approve the drug for routine use yet, but will approve it through a Managed Access Agreement, which will allow people to access the drug on the NHS while further studies take place.
NICE said there is a high unmet demand for treatments for sickle cell disease. NHS England say that up to 5,000 people could receive the treatment over the next three years.
Meindert Boysen, deputy chief executive of 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.”
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.
“The NHS has agreed on a deal for this drug, so we are able to provide the latest and best possible treatments for patients at a price that is affordable for taxpayers.”
Source: NICE/NHS England