The National Institute for Health and Clinical Excellence (NICE) has published final guidance recommending Roche Products' RoActemra (tocilizumab) for the treatment of systemic juvenile idiopathic arthritis (sJIA).
NICE recommends the drug for use in patients aged two and older who have sJIA, a condition which causes severe pain and other difficulties in children and young people, in whom specific previous treatments have not produced an adequate response - if Roche makes it available with the discount agreed as part of a patient access scheme (PAS).
RoActemra is not recommended in patients aged two years and older whose disease continues to respond to methotrexate, or who have not been treated with methotrexate, NICE adds. Patients currently being treated with RoActemra for sJIA who do not meet the recommendation criteria should have the option to continue treatment until it is considered appropriate to stop, in agreement with their clinicians and parents/carers, the Institute says.
sJIA, which has no known cause, can affect children of any age, and it has "a huge impact on the lives of the children and young people with the disease, causing severe pain, fatigue and disability. This affects the child's family and school life, as well as their physical and emotional well-being," commented Professor Carole Longson, director of the health technology evaluation centre at NICE.
The discount agreed as part of the PAS put forward by Roche, in additional to the further information provided by the firm at the request of NICE's independent appraisal committee, has enabled the recommendation for RoActemra as a possible treatment on the NHS, said Prof Longson, who added that this was good news for children with sJIA and those caring for them.
NICE's final decision was welcomed by Eileen Baildam, consultant paediatric rheumatologist at Alder Hey Children's NHS Foundation Trust Hospital in Liverpool, where RoActemra has been trialled for sJIA.
The condition is "much worse than rheumatoid arthritis in adults," said Dr Baildam, adding: "I am excited that RoActemra is now available in sJIA, as for the first time, clinicians can treat this debilitating disease with an effective, licensed medicine. The fact that it will now be readily available for patients across the UK gives thousands of children hope in restoring normality to their day-to-day lives."